Rare Disease Trial Updates: June 2026

In June 2026, Trial Friend tracked 748 rare disease clinical trial updates across 111 conditions, including 9 FDA-related actions and 123 trial status changes, alongside enrollment updates and new trial site openings sourced from ClinicalTrials.gov and FDA announcements.

This is a permanent monthly archive. For the latest updates, see the live current events feed.

Major Updates: FDA Actions, Status Changes & Results

FDA ApprovalChronic Graft-versus-Host DiseaseJun 30
FDA Approves New Treatment That Uses Donor Immune Cells to Prevent Serious Complications in Blood Cancer Patients

The U.S. Food and Drug Administration today approved TREGZI, the first regulatory T (Treg) cell-based immunotherapy for improving chronic graft-versus-host disease (GVHD)-free survival in adult patients with blood cancers undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Status ChangeSpinocerebellar AtaxiaJun 30Arrowhead PharmaceuticalsNCT06672445
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJun 29
FDA Selects Seven Participants for PreCheck Pilot Program to Advance U.S. Drug Manufacturing

FDA announces the selection of seven companies to participate in the FDA PreCheck Pilot Program, a new initiative designed to boost American global competitiveness, strengthen domestic drug manufacturing, and improve the resilience of the U.S. drug supply chain.

Status ChangeRetinitis PigmentosaJun 29Beacon TherapeuticsNCT07174726
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJun 29Liquidia Technologies, Inc.NCT06129240
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeNeuromyelitis OpticaJun 28Alexion Pharmaceuticals, Inc.NCT05346354
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisJun 28Novartis PharmaceuticalsNCT02386800
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyasthenia GravisJun 28UCB Biopharma SRLNCT06149559
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyJun 28BioMarin PharmaceuticalNCT06280209
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 28Icahn School of Medicine at Mount SinaiNCT05982925
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeMesotheliomaJun 28Hospices Civils de LyonNCT06790706
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyJun 27Travere Therapeutics, Inc.NCT07219121
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyJun 27Alexion Pharmaceuticals, Inc.NCT06291376
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseJun 27University of WashingtonNCT05596760
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 27Linio Biotech OyNCT07368764
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 27Acepodia Biotech, Inc.NCT05653271
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 27University Health Network, TorontoNCT04566887
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 27Washington University School of MedicineNCT05412290
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal Segmental GlomerulosclerosisJun 26Travere Therapeutics, Inc.NCT07219121
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaJun 26Merck Sharp & Dohme LLCNCT06079879
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyJun 25BioMarin PharmaceuticalNCT06280209
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 25University of EdinburghNCT06753370
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 25Novartis PharmaceuticalsNCT02386800
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJun 25Tel-Aviv Sourasky Medical CenterNCT06723795
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 25AstraZenecaNCT06706817
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJun 25University Health Network, TorontoNCT03991832
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCentral Diabetes InsipidusJun 25Otsuka Pharmaceutical Co., Ltd.NCT04790175
No longer enrolling new participants

No longer enrolling new participants

Status ChangeC3 GlomerulopathyJun 24Novartis PharmaceuticalsNCT07156149
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeJun 24MavriX Bio, LLCNCT07181837
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJun 23Novartis PharmaceuticalsNCT02386800
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePulmonary Arterial HypertensionJun 22V-Wave LtdNCT03838445
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraJun 22Novartis PharmaceuticalsNCT02386800
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeRET Fusion CancerJun 22Shouyao Holdings (Beijing) Co. LTDNCT06031558
No longer enrolling new participants

No longer enrolling new participants

Status ChangeProgressive Supranuclear PalsyJun 22Non-profit organization for scientific research in Parkinson's disease and related disordersNCT06501469
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeuromyelitis OpticaJun 21Icahn School of Medicine at Mount SinaiNCT05982925
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeMultiple System AtrophyJun 21Non-profit organization for scientific research in Parkinson's disease and related disordersNCT06501469
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 21Novartis PharmaceuticalsNCT06551519
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMembranous NephropathyJun 21Novartis PharmaceuticalsNCT06004661
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHypersensitivity PneumonitisJun 20Avalyn Pharma Inc.NCT06329401
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 20AmgenNCT07134088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 20City of Hope Medical CenterNCT03011814
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHodgkin LymphomaJun 20University of AarhusNCT06362148
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 20Memorial Sloan Kettering Cancer CenterNCT04468841
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 20AstraZenecaNCT04594642
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic ParaplegiaJun 20University of MiamiNCT02327845
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGastrointestinal Stromal TumorJun 19Cogent Biosciences, Inc.NCT05208047
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJun 18University of California, San DiegoNCT06646731
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCongenital Adrenal HyperplasiaJun 18Neurocrine BiosciencesNCT07187375
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 18Syndax PharmaceuticalsNCT04710576
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeChronic Graft-versus-Host DiseaseJun 18REVA Medical, Inc.NCT05406622
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 18SanofiNCT06834360
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 18AstraZenecaNCT06744335
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 18Wake Forest University Health SciencesNCT03615235
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 18The University of Tennessee, KnoxvilleNCT06840028
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCharcot-Marie-Tooth DiseaseJun 18Radboud University Medical CenterNCT06573866
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCharcot-Marie-Tooth DiseaseJun 18University of MiamiNCT02327845
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCerebral AdrenoleukodystrophyJun 18Pujol, Aurora, M.D.NCT06513533
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalJun 17
FDA Approves First Single-Dose Generic Treatment for Influenza

The U.S. Food and Drug Administration today approved the first generic of Xofluza (baloxavir marboxil) tablets, the first single-dose treatment for acute uncomplicated influenza and prophylaxis in patients 5 years of age and older.

Status ChangeAmyotrophic Lateral SclerosisJun 17University of MiamiNCT02327845
No longer enrolling new participants

No longer enrolling new participants

Status ChangeStargardt DiseaseJun 16AAVantgarde Bio SrlNCT06591806
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalJun 16
FDA Broadens Access to Over-the-Counter Naloxone Nasal Spray for Opioid Overdose

The U.S. Food and Drug Administration (FDA) today approved another over-the-counter (OTC) intranasal naloxone product, Rextovy, a 4 milligram (mg) naloxone hydrochloride nasal spray for the emergency treatment of opioid overdose.

Status ChangeVon Willebrand DiseaseJun 16Hoffmann-La RocheNCT06883240
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJun 16Lantu BiopharmaNCT06308159
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyJun 16Lantu BiopharmaNCT06288230
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyJun 16University of MiamiNCT02327845
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic Paraplegia Type 4Jun 16Radboud University Medical CenterNCT06573866
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic Paraplegia Type 4Jun 16University of MiamiNCT02327845
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJun 16University of PennsylvaniaNCT05730205
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalJun 15
FDA Approves New Indication for Tzield (teplizumab) for Certain Pediatric Patients with Recently Diagnosed Stage 3 Type 1 Diabetes

On June 12, 2026, the U.S. Food and Drug Administration granted accelerated approval to Tzield (teplizumab) for a new indication, to delay the decline of insulin production in pediatric patients ages 8 through 17 years who have been recently diagnosed with Stage 3 type 1 diabetes (T1D).

Status ChangeRett SyndromeJun 15Neurogene Inc.NCT05898620
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Langerhans Cell HistiocytosisJun 15Novartis PharmaceuticalsNCT03975829
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeurofibromatosis Type 1Jun 14Novartis PharmaceuticalsNCT03975829
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJun 14M.D. Anderson Cancer CenterNCT06113302
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJun 14Molecular Partners AGNCT05673057
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisJun 14COUR Pharmaceutical Development Company, Inc.NCT06106672
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 14AmgenNCT06700343
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaJun 14AstraZenecaNCT06097728
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaJun 14Hyogo Medical UniversityNCT06318286
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLimb-Girdle Muscular DystrophyJun 13Virginia Commonwealth UniversityNCT05618080
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 13M.D. Anderson Cancer CenterNCT04054167
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 13University of VirginiaNCT05404945
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 13The First Affiliated Hospital of Soochow UniversityNCT05470777
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 13Dana-Farber Cancer InstituteNCT05734495
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic ParaplegiaJun 13Radboud University Medical CenterNCT06573866
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJun 12
FDA Clears First Over-the-Counter Continuous Glucose Monitor for Children

The U.S. Food and Drug Administration today cleared for marketing the first over-the-counter (OTC) continuous glucose monitor (CGM) for children, Dexcom Inc.’s Stelo Glucose Biosensor System, an integrated CGM (iCGM) indicated for people two years of age and older who do not use insulin.

Status ChangeGiant Cell ArteritisJun 12University Hospital, BrestNCT04664465
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDermatomyositisJun 11Novartis PharmaceuticalsNCT06665256
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT04557462
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 11Oregon Health and Science UniversityNCT06091202
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJun 11
FDA Issues Emergency Use Authorization for Generic Over-the-Counter Drug to Treat New World Screwworm in Dogs and Cats

FDA today issued an Emergency Use Authorization (EUA) for generic Nitenpyram Tablets (nitenpyram) for the treatment of New World screwworm (NWS) infestations (myiasis) in dogs, puppies, cats, and kittens that weigh at least two pounds and are at least four weeks old.

Status ChangeChronic Graft-versus-Host DiseaseJun 11Regeneron PharmaceuticalsNCT05092347
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 11Indiana UniversityNCT07033117
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeChronic Graft-versus-Host DiseaseJun 11Indiana UniversityNCT05603702
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT06799000
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBullous PemphigoidJun 10University Hospital, RouenNCT03295383
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Jun 10University of Texas Southwestern Medical CenterNCT05489523
No longer enrolling new participants

No longer enrolling new participants

FDA NewsJun 9
FDA Expands Sunscreen Options for the First Time in 20 Years

Today, the U.S. Food and Drug Administration added bemotrizinol to the list of permitted sunscreen active ingredients, marking a significant milestone in the agency's efforts to advance sunscreen innovation.

Status ChangeTuberous SclerosisJun 9Jazz PharmaceuticalsNCT05485831
No longer enrolling new participants

No longer enrolling new participants

Status ChangeStargardt DiseaseJun 9National Eye Institute (NEI)NCT04545736
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJun 8Merck Sharp & Dohme LLCNCT06814145
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeuromyelitis OpticaJun 7Tianjin Medical University General HospitalNCT07184840
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJun 7M.D. Anderson Cancer CenterNCT04128748
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 7BiogenNCT05688436
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 7Jazz PharmaceuticalsNCT05485831
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 7University of Illinois at ChicagoNCT04518657
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaJun 7Udai KammulaNCT03935893
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaJun 7Udai KammulaNCT03935893
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLennox-Gastaut SyndromeJun 6Jazz PharmaceuticalsNCT05485831
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyJun 6Novartis PharmaceuticalsNCT04557462
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyJun 6University of LeicesterNCT04663204
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeIdiopathic Pulmonary FibrosisJun 6First Affiliated Hospital of Wenzhou Medical UniversityNCT05828953
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 6AstraZenecaNCT07154901
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJun 6University of WashingtonNCT05453500
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary AngioedemaJun 6Astria Therapeutics, Inc.NCT06842823
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeChronic Graft-versus-Host DiseaseJun 4University of Erlangen-Nürnberg Medical SchoolNCT06051812
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 4University of Illinois at ChicagoNCT06555172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDravet SyndromeJun 4Jazz PharmaceuticalsNCT05485831
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 4Centre Hospitalier Universitaire de NiceNCT06133907
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJun 4Ausper Biopharma Co., Ltd.NCT07069569
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeCholangiocarcinomaJun 4Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityNCT02757859
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJun 4M.D. Anderson Cancer CenterNCT02379377
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJun 4Udai KammulaNCT03801083
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAplastic AnemiaJun 3Children's Hospital of PhiladelphiaNCT02928991
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAplastic AnemiaJun 3National Heart, Lung, and Blood Institute (NHLBI)NCT05998408
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAplastic AnemiaJun 3National Heart, Lung, and Blood Institute (NHLBI)NCT03312400
No longer enrolling new participants

No longer enrolling new participants

FDA GuidanceJun 2
FDA Issues Draft Guidance to Help Accelerate Cell and Gene Therapies for Patients

The U.S. Food and Drug Administration (FDA) today issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge.

Status ChangeWaldenström MacroglobulinemiaJun 2Dana-Farber Cancer InstituteNCT05734495
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUrea Cycle DisordersJun 2Sequitur Health Corp.NCT06953505
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeRett SyndromeJun 1Guangzhou Women and Children's Medical CenterNCT06856759
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaJun 1PYC TherapeuticsNCT06852963
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJun 1Massachusetts General HospitalNCT07312227
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeOsteogenesis ImperfectaJun 1AmgenNCT05972551
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedWaldenström MacroglobulinemiaJun 30National Cancer Institute (NCI)NCT04840602
Added 1 new site: Maywood, Illinois

Added 1 new site: Maywood, Illinois

sites_addedVon Willebrand DiseaseJun 30Hemab ApSNCT06754852
Added 21 new sites: Phoenix, Arizona, Little Rock, Arkansas, Los Angeles, California and 18 more

Added 21 new sites: Phoenix, Arizona, Little Rock, Arkansas, Los Angeles, California and 18 more

eligibility_changeVon Willebrand DiseaseJun 30Hemab ApSNCT06754852
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseJun 30Novartis PharmaceuticalsNCT06546670
Added 1 new site: Greenville, North Carolina

Added 1 new site: Greenville, North Carolina

sites_addedWild-Type ATTR Cardiac AmyloidosisJun 30BayerNCT07298044
Added 13 new sites: Feldkirch, Austria, Brussels, Belgium, Bruges, Belgium and 10 more

Added 13 new sites: Feldkirch, Austria, Brussels, Belgium, Bruges, Belgium and 10 more

sites_addedWaldenström MacroglobulinemiaJun 30National Cancer Institute (NCI)NCT04840602
Added 1 new site: Coon Rapids, Minnesota

Added 1 new site: Coon Rapids, Minnesota

sites_addedSystemic MastocytosisJun 30Blueprint Medicines CorporationNCT04910685
Added 3 new sites: São Paulo, Brazil, Cork, Ireland, Mersin, Turkey (Türkiye)

Added 3 new sites: São Paulo, Brazil, Cork, Ireland, Mersin, Turkey (Türkiye)

sites_addedStargardt DiseaseJun 30Astellas Pharma Global Development, Inc.NCT07425574
Added 2 new sites: Chicago, Illinois, Salt Lake City, Utah

Added 2 new sites: Chicago, Illinois, Salt Lake City, Utah

eligibility_changeSpinal Muscular AtrophyJun 30Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT04708431
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSickle Cell DiseaseJun 30National Heart, Lung, and Blood Institute (NHLBI)NCT07566494
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSickle Cell DiseaseJun 30St. Jude Children's Research HospitalNCT06477289
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJun 29Liquidia Technologies, Inc.NCT07285655
Added 10 new sites: Scottsdale, Arizona, Chicago, Illinois, Linz, Austria and 7 more

Added 10 new sites: Scottsdale, Arizona, Chicago, Illinois, Linz, Austria and 7 more

eligibility_changePulmonary Arterial HypertensionJun 29Liquidia Technologies, Inc.NCT07285655
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPompe DiseaseJun 29Denali Therapeutics Inc.NCT07354724
Added 2 new sites: Irvine, California, Durham, North Carolina

Added 2 new sites: Irvine, California, Durham, North Carolina

eligibility_changePheochromocytomaJun 29National Cancer Institute (NCI)NCT06427798
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedParoxysmal Nocturnal HemoglobinuriaJun 29AstraZenecaNCT07457151
Reduced from 5 to 2 sites

Reduced from 5 to 2 sites

eligibility_changeParagangliomaJun 29National Cancer Institute (NCI)NCT06427798
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJun 29Liquidia Technologies, Inc.NCT06129240
Added 6 new sites: La Jolla, California, Sacremento, California, Hartford, Connecticut and 3 more

Added 6 new sites: La Jolla, California, Sacremento, California, Hartford, Connecticut and 3 more

sites_addedPulmonary Arterial HypertensionJun 29Merck Sharp & Dohme LLCNCT07218029
Added 6 new sites: Tucson, Arizona, Sacramento, California, Boston, Massachusetts and 3 more

Added 6 new sites: Tucson, Arizona, Sacramento, California, Boston, Massachusetts and 3 more

sites_addedProgressive Supranuclear PalsyJun 29Novartis PharmaceuticalsNCT07498426
Added 5 new sites: Caen, France, Paris, France, Roma, Italy and 2 more

Added 5 new sites: Caen, France, Paris, France, Roma, Italy and 2 more

sites_addedPrimary Biliary CholangitisJun 29Gilead SciencesNCT06051617
Added 4 new sites: Rochester, Minnesota, Ramat Gan, Israel, Ramat Gan, Israel and 1 more

Added 4 new sites: Rochester, Minnesota, Ramat Gan, Israel, Ramat Gan, Israel and 1 more

sites_addedPolycythemia VeraJun 29Novartis PharmaceuticalsNCT07357727
Added 6 new sites: Berkeley Heights, New Jersey, Columbus, Ohio, Nantong, China and 3 more

Added 6 new sites: Berkeley Heights, New Jersey, Columbus, Ohio, Nantong, China and 3 more

sites_addedPhenylketonuriaJun 29Agios Pharmaceuticals, Inc.NCT07241234
Added 2 new sites: Indianapolis, Indiana, Pittsburgh, Pennsylvania

Added 2 new sites: Indianapolis, Indiana, Pittsburgh, Pennsylvania

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 29Novartis PharmaceuticalsNCT07229235
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

sites_addedNoonan SyndromeJun 28Ascendis Pharma A/SNCT07221851
Added 2 new sites: Toulouse, France, Zaragoza, Spain

Added 2 new sites: Toulouse, France, Zaragoza, Spain

eligibility_changeNiemann-Pick Disease Type CJun 28Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT00344331
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeNiemann-Pick DiseaseJun 28Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT00344331
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedNeuromyelitis OpticaJun 28Alexion Pharmaceuticals, Inc.NCT05346354
Reduced from 21 to 16 sites

Reduced from 21 to 16 sites

sites_addedMyelofibrosisJun 28iOncturaNCT06887803
Added 1 new site: Meldola, Italy

Added 1 new site: Meldola, Italy

sites_addedMyelofibrosisJun 28Novartis PharmaceuticalsNCT07357727
Added 6 new sites: Berkeley Heights, New Jersey, Columbus, Ohio, Nantong, China and 3 more

Added 6 new sites: Berkeley Heights, New Jersey, Columbus, Ohio, Nantong, China and 3 more

sites_addedMyelofibrosisJun 28Aspera Biomedicines, Inc.NCT07250646
Added 1 new site: Duarte, California

Added 1 new site: Duarte, California

sites_addedMyelofibrosisJun 28Novartis PharmaceuticalsNCT02386800
Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

Enrollment UpdateMyelodysplastic SyndromesJun 28Fred Hutchinson Cancer CenterNCT06492707
Target enrollment increased from 20 to 40

Target enrollment increased from 20 to 40

eligibility_changeMyelodysplastic SyndromesJun 28Fred Hutchinson Cancer CenterNCT06492707
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJun 28Blueprint Medicines CorporationNCT07143669
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedMyelodysplastic SyndromesJun 28Vanderbilt-Ingram Cancer CenterNCT06399640
Added 1 new site: Memphis, Tennessee

Added 1 new site: Memphis, Tennessee

sites_addedMyelodysplastic SyndromesJun 28Novartis PharmaceuticalsNCT07229235
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

eligibility_changeMyelodysplastic SyndromesJun 28Ellipses PharmaNCT04581512
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJun 28Novartis PharmaceuticalsNCT06704269
Added 2 new sites: Philadelphia, Pennsylvania, Chiba, Japan

Added 2 new sites: Philadelphia, Pennsylvania, Chiba, Japan

sites_addedMyasthenia GravisJun 28Novartis PharmaceuticalsNCT06517758
Added 3 new sites: Córdoba, Argentina, Bahia, Brazil, Chiba, Japan

Added 3 new sites: Córdoba, Argentina, Bahia, Brazil, Chiba, Japan

sites_addedMyasthenia GravisJun 28Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 6 new sites: South Brisbane, Australia, Burnaby, Canada, Ulm, Germany and 3 more

Added 6 new sites: South Brisbane, Australia, Burnaby, Canada, Ulm, Germany and 3 more

sites_addedMultiple System AtrophyJun 28Precision BioSciences, Inc.NCT07429240
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedMultiple SclerosisJun 28Novartis PharmaceuticalsNCT07225504
Added 18 new sites: Phoenix, Arizona, Tampa, Florida, Worchester, Massachusetts and 15 more

Added 18 new sites: Phoenix, Arizona, Tampa, Florida, Worchester, Massachusetts and 15 more

sites_addedMultiple SclerosisJun 28Hoffmann-La RocheNCT04544449
Added 2 new sites: Capital Federal, Argentina, Braga, Portugal

Added 2 new sites: Capital Federal, Argentina, Braga, Portugal

sites_addedMultiple SclerosisJun 28Zenas BioPharma (USA), LLCNCT07067463
Added 5 new sites: Columbus, Ohio, Choceň, Czechia, Erbach im Odenwald, Germany and 2 more

Added 5 new sites: Columbus, Ohio, Choceň, Czechia, Erbach im Odenwald, Germany and 2 more

sites_removedMultiple SclerosisJun 28Hoffmann-La RocheNCT04586023
Reduced from 108 to 106 sites

Reduced from 108 to 106 sites

sites_addedMultiple SclerosisJun 28SanofiNCT07325292
Added 2 new sites: Columbus, Ohio, Beijing, China

Added 2 new sites: Columbus, Ohio, Beijing, China

sites_addedMultiple SclerosisJun 28National Institute of Allergy and Infectious Diseases (NIAID)NCT05285891
Added 1 new site: Livingston, New Jersey

Added 1 new site: Livingston, New Jersey

eligibility_changeMultiple SclerosisJun 28University of PatrasNCT07630311
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJun 28Novartis PharmaceuticalsNCT06846281
Added 14 new sites: Richmond, Virginia, Milwaukee, Wisconsin, Porto Alegre, Brazil and 11 more

Added 14 new sites: Richmond, Virginia, Milwaukee, Wisconsin, Porto Alegre, Brazil and 11 more

sites_addedMesotheliomaJun 28IDEAYA BiosciencesNCT07277413
Added 2 new sites: Santa Rosa, California, East Brunswick, New Jersey

Added 2 new sites: Santa Rosa, California, East Brunswick, New Jersey

Enrollment UpdateMesotheliomaJun 28Hospices Civils de LyonNCT06790706
Target enrollment decreased from 154 to 27

Target enrollment decreased from 154 to 27

eligibility_changeMesotheliomaJun 28Hospices Civils de LyonNCT06790706
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaJun 28Novartis PharmaceuticalsNCT04104776
Added 69 new sites: Tampa, Florida, Atlanta, Georgia, Chicago, Illinois and 66 more

Added 69 new sites: Tampa, Florida, Atlanta, Georgia, Chicago, Illinois and 66 more

eligibility_changeMesotheliomaJun 28Novartis PharmaceuticalsNCT04104776
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaJun 28Ohio State University Comprehensive Cancer CenterNCT07215988
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyJun 28Centre Hospitalier Universitaire de NiceNCT06341205
Added 1 new site: Paris, France

Added 1 new site: Paris, France

Enrollment UpdateMembranous NephropathyJun 28Vera Therapeutics, Inc.NCT06983028
Target enrollment increased from 200 to 250

Target enrollment increased from 200 to 250

eligibility_changeMembranous NephropathyJun 28Vera Therapeutics, Inc.NCT06983028
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMedullary Thyroid CancerJun 28National Cancer Institute (NCI)NCT03866382
Added 1 new site: Covington, Louisiana

Added 1 new site: Covington, Louisiana

sites_addedMastocytosisJun 28Blueprint Medicines CorporationNCT04910685
Added 3 new sites: São Paulo, Brazil, Cork, Ireland, Mersin, Turkey (Türkiye)

Added 3 new sites: São Paulo, Brazil, Cork, Ireland, Mersin, Turkey (Türkiye)

sites_addedMastocytosisJun 28Blueprint Medicines CorporationNCT07143669
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedLight Chain AmyloidosisJun 27Regeneron PharmaceuticalsNCT06292780
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedLeber Congenital AmaurosisJun 27Laboratoires TheaNCT06891443
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedIgA NephropathyJun 27Novartis PharmaceuticalsNCT07146906
Added 17 new sites: Iowa City, Iowa, Serra, Brazil, Botucatu, Brazil and 14 more

Added 17 new sites: Iowa City, Iowa, Serra, Brazil, Botucatu, Brazil and 14 more

Enrollment UpdateIgA NephropathyJun 27Vera Therapeutics, Inc.NCT06674577
Target enrollment increased from 476 to 750

Target enrollment increased from 476 to 750

sites_addedIgA NephropathyJun 27BiogenNCT06935357
Added 2 new sites: Bogotá, Colombia, London, United Kingdom

Added 2 new sites: Bogotá, Colombia, London, United Kingdom

sites_removedIgA NephropathyJun 27Alexion Pharmaceuticals, Inc.NCT06291376
Reduced from 267 to 246 sites

Reduced from 267 to 246 sites

Enrollment UpdateIgA NephropathyJun 27Alexion Pharmaceuticals, Inc.NCT06291376
Target enrollment increased from 510 to 579

Target enrollment increased from 510 to 579

eligibility_changeIgA NephropathyJun 27Alexion Pharmaceuticals, Inc.NCT06291376
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateIgA NephropathyJun 27Vera Therapeutics, Inc.NCT06983028
Target enrollment increased from 200 to 250

Target enrollment increased from 200 to 250

eligibility_changeIgA NephropathyJun 27Vera Therapeutics, Inc.NCT06983028
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyJun 27TakedaNCT06963827
Added 1 new site: Sheffield, United Kingdom

Added 1 new site: Sheffield, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisJun 27Contineum TherapeuticsNCT07284459
Added 13 new sites: Buenos Aires, Argentina, Buenos Aires, Argentina, Chermside, Australia and 10 more

Added 13 new sites: Buenos Aires, Argentina, Buenos Aires, Argentina, Chermside, Australia and 10 more

sites_addedIdiopathic Pulmonary FibrosisJun 27Rein TherapeuticsNCT06968845
Added 3 new sites: Los Angeles, California, Westmead, Australia, London, United Kingdom

Added 3 new sites: Los Angeles, California, Westmead, Australia, London, United Kingdom

sites_addedHuntington DiseaseJun 27Novartis PharmaceuticalsNCT07326709
Added 8 new sites: La Jolla, California, Chicago, Illinois, Fairway, Kansas and 5 more

Added 8 new sites: La Jolla, California, Chicago, Illinois, Fairway, Kansas and 5 more

sites_addedHuntington DiseaseJun 27Skyhawk Therapeutics, Inc.NCT07378644
Added 2 new sites: CABA, Argentina, Santiago, Chile

Added 2 new sites: CABA, Argentina, Santiago, Chile

sites_addedHodgkin LymphomaJun 27Novartis PharmaceuticalsNCT04094311
Added 2 new sites: Yokohama, Japan, Kumamoto, Japan

Added 2 new sites: Yokohama, Japan, Kumamoto, Japan

eligibility_changeHodgkin LymphomaJun 27The Lymphoma Academic Research OrganisationNCT06006117
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJun 27C. Babis AndreadisNCT04545762
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 27Nuvation Bio Inc.NCT05303519
Added 10 new sites: La Jolla, California, Chicago, Illinois, Billings, Montana and 7 more

Added 10 new sites: La Jolla, California, Chicago, Illinois, Billings, Montana and 7 more

sites_addedHodgkin LymphomaJun 27University of FloridaNCT04573140
Added 1 new site: Washington D.C., District of Columbia

Added 1 new site: Washington D.C., District of Columbia

eligibility_changeHodgkin LymphomaJun 27University Health Network, TorontoNCT04510636
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 27National Cancer Institute (NCI)NCT06124157
Added 2 new sites: Louisville, Kentucky, Albuquerque, New Mexico

Added 2 new sites: Louisville, Kentucky, Albuquerque, New Mexico

Enrollment UpdateHodgkin LymphomaJun 27Washington University School of MedicineNCT05412290
Target enrollment decreased from 15 to 9

Target enrollment decreased from 15 to 9

eligibility_changeHodgkin LymphomaJun 27MSD R&D (China) Co., Ltd.NCT06189391
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 27AstraZenecaNCT06549595
Added 21 new sites: Albany, New York, Austin, Texas, Milwaukee, Wisconsin and 18 more

Added 21 new sites: Albany, New York, Austin, Texas, Milwaukee, Wisconsin and 18 more

sites_addedHereditary AngioedemaJun 27Pharvaris Netherlands B.V.NCT07266805
Added 17 new sites: San Diego, California, Hershey, Pennsylvania, Clayton, Australia and 14 more

Added 17 new sites: San Diego, California, Hershey, Pennsylvania, Clayton, Australia and 14 more

sites_removedHemophilia AJun 26Swedish Orphan BiovitrumNCT06716814
Reduced from 24 to 21 sites

Reduced from 24 to 21 sites

sites_addedGraves’ DiseaseJun 26AmgenNCT07438405
Added 1 new site: Kissimmee, Florida

Added 1 new site: Kissimmee, Florida

sites_addedGeographic AtrophyJun 26Novartis PharmaceuticalsNCT07441642
Added 3 new sites: Ludwigshafen, Germany, Arecibo, Puerto Rico, Santiago Compostela, Spain

Added 3 new sites: Ludwigshafen, Germany, Arecibo, Puerto Rico, Santiago Compostela, Spain

sites_addedEssential ThrombocythemiaJun 26Novartis PharmaceuticalsNCT07357727
Added 3 new sites: Columbus, Ohio, Wenzhou, China, Fuzhou, China

Added 3 new sites: Columbus, Ohio, Wenzhou, China, Fuzhou, China

phase_changeHemophilia BJun 26Genzyme, a Sanofi CompanyNCT03974113
Moved from Phase 3 to Phase 2, Phase 3

Moved from Phase 3 to Phase 2, Phase 3

sites_addedHemophilia BJun 26SanofiNCT07285460
Added 3 new sites: Dallas, Texas, Hamilton, Canada, Beijing, China

Added 3 new sites: Dallas, Texas, Hamilton, Canada, Beijing, China

phase_changeHemophilia AJun 26Genzyme, a Sanofi CompanyNCT03974113
Moved from Phase 3 to Phase 2, Phase 3

Moved from Phase 3 to Phase 2, Phase 3

sites_addedHemophilia AJun 26SanofiNCT07285460
Added 3 new sites: Dallas, Texas, Hamilton, Canada, Beijing, China

Added 3 new sites: Dallas, Texas, Hamilton, Canada, Beijing, China

sites_addedGraves’ DiseaseJun 26Immunovant Sciences GmbHNCT07286006
Added 3 new sites: Varna, Bulgaria, Varna, Bulgaria, Prague, Czechia

Added 3 new sites: Varna, Bulgaria, Varna, Bulgaria, Prague, Czechia

sites_addedGraves’ DiseaseJun 26Immunovant Sciences GmbHNCT07018323
Added 4 new sites: Cuneo, Italy, Shibuya City, Japan, Riga, Latvia and 1 more

Added 4 new sites: Cuneo, Italy, Shibuya City, Japan, Riga, Latvia and 1 more

sites_removedGranulomatosis with PolyangiitisJun 26GlaxoSmithKlineNCT05263934
Reduced from 76 to 63 sites

Reduced from 76 to 63 sites

sites_removedGranulomatosis with PolyangiitisJun 26Novartis PharmaceuticalsNCT06388941
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedGiant Cell ArteritisJun 26SanofiNCT07286214
Added 5 new sites: Miami, Florida, Santa Fe, New Mexico, Duncansville, Pennsylvania and 2 more

Added 5 new sites: Miami, Florida, Santa Fe, New Mexico, Duncansville, Pennsylvania and 2 more

sites_addedGeographic AtrophyJun 26Complement TherapeuticsNCT07392255
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedGeographic AtrophyJun 26Apellis Pharmaceuticals, Inc.NCT07215390
Added 10 new sites: Scottsdale, Arizona, Campbell, California, Torrance, California and 7 more

Added 10 new sites: Scottsdale, Arizona, Campbell, California, Torrance, California and 7 more

sites_addedGeographic AtrophyJun 26Novartis PharmaceuticalsNCT07441642
Added 3 new sites: Dijon, France, Debrecen, Hungary, Budapest, Hungary

Added 3 new sites: Dijon, France, Debrecen, Hungary, Budapest, Hungary

sites_removedGaucher DiseaseJun 26Shenzhen Geno-Immune Medical InstituteNCT04637503
Reduced from 3 to 1 sites

Reduced from 3 to 1 sites

sites_addedFocal Segmental GlomerulosclerosisJun 26Boehringer IngelheimNCT07220083
Added 12 new sites: Créteil, France, Nice, France, Paris, France and 9 more

Added 12 new sites: Créteil, France, Nice, France, Paris, France and 9 more

sites_addedFocal EpilepsyJun 26Rapport Therapeutics Inc.NCT07594119
Added 1 new site: Colorado Springs, Colorado

Added 1 new site: Colorado Springs, Colorado

sites_addedFabry DiseaseJun 26Genzyme, a Sanofi CompanyNCT00196742
Added 13 new sites: Los Angeles, California, Coral Gables, Florida, Coral Springs, Florida and 10 more

Added 13 new sites: Los Angeles, California, Coral Gables, Florida, Coral Springs, Florida and 10 more

sites_addedEssential ThrombocythemiaJun 26Novartis PharmaceuticalsNCT07357727
Added 3 new sites: Berkeley Heights, New Jersey, Nantong, China, Beijing, China

Added 3 new sites: Berkeley Heights, New Jersey, Nantong, China, Beijing, China

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 25University of EdinburghNCT06753370
Target enrollment increased from 20 to 25

Target enrollment increased from 20 to 25

sites_addedChronic Graft-versus-Host DiseaseJun 25Acotec Scientific Co., LtdNCT06330493
Added 9 new sites: Concord, California, Fremont, California, New Haven, Connecticut and 6 more

Added 9 new sites: Concord, California, Fremont, California, New Haven, Connecticut and 6 more

sites_addedChronic Graft-versus-Host DiseaseJun 25University Hospital, Basel, SwitzerlandNCT05890677
Added 5 new sites: Sankt Pölten, Austria, Vienna, Austria, Basel, Switzerland and 2 more

Added 5 new sites: Sankt Pölten, Austria, Vienna, Austria, Basel, Switzerland and 2 more

eligibility_changeChronic Graft-versus-Host DiseaseJun 25National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT05398783
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 25National Cancer Institute (NCI)NCT04269902
Added 3 new sites: Chico, California, Oxford, Mississippi, Collierville, Tennessee

Added 3 new sites: Chico, California, Oxford, Mississippi, Collierville, Tennessee

eligibility_changeChronic Graft-versus-Host DiseaseJun 25Huahui HealthNCT07499544
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedChronic Graft-versus-Host DiseaseJun 25BeOne MedicinesNCT05006716
Reduced from 130 to 127 sites

Reduced from 130 to 127 sites

sites_addedChronic Graft-versus-Host DiseaseJun 25Connect Biopharm LLCNCT06940154
Added 2 new sites: Campbelltown, Australia, Bendigo, Australia

Added 2 new sites: Campbelltown, Australia, Bendigo, Australia

eligibility_changeChronic Graft-versus-Host DiseaseJun 25Connect Biopharm LLCNCT06940154
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 25Merck Sharp & Dohme LLCNCT05947851
Added 2 new sites: Greenfield Park, Canada, Sherbrooke, Canada

Added 2 new sites: Greenfield Park, Canada, Sherbrooke, Canada

sites_addedChronic Graft-versus-Host DiseaseJun 25Novartis PharmaceuticalsNCT02386800
Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

sites_addedChronic Graft-versus-Host DiseaseJun 25Novartis PharmaceuticalsNCT06632600
Added 1 new site: Caba, Argentina

Added 1 new site: Caba, Argentina

sites_addedCholangiocarcinomaJun 25iOMEDICO AGNCT06607302
Added 6 new sites: Dresden, Germany, Dresden, Germany, Essen, Germany and 3 more

Added 6 new sites: Dresden, Germany, Dresden, Germany, Essen, Germany and 3 more

sites_removedEosinophilic Granulomatosis with PolyangiitisJun 25GlaxoSmithKlineNCT05263934
Reduced from 76 to 63 sites

Reduced from 76 to 63 sites

eligibility_changeDuchenne Muscular DystrophyJun 25Sarepta Therapeutics, Inc.NCT04626674
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDuchenne Muscular DystrophyJun 25Precision BioSciences, Inc.NCT07429240
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedDermatomyositisJun 25Novartis PharmaceuticalsNCT06665256
Added 1 new site: Augusta, Georgia

Added 1 new site: Augusta, Georgia

eligibility_changeCystic FibrosisJun 25University of Alabama at BirminghamNCT07102043
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 25Grifols Therapeutics LLCNCT07540221
Added 1 new site: Cordova, Tennessee

Added 1 new site: Cordova, Tennessee

sites_addedChronic Graft-versus-Host DiseaseJun 25Eli Lilly and CompanyNCT04844606
Added 2 new sites: Philadelphia, Pennsylvania, Beersheba, Israel

Added 2 new sites: Philadelphia, Pennsylvania, Beersheba, Israel

sites_addedChronic Graft-versus-Host DiseaseJun 25Duke UniversityNCT06906627
Added 1 new site: Houston, North Carolina

Added 1 new site: Houston, North Carolina

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 25University Hospital, AngersNCT06533956
Target enrollment increased from 10 to 13

Target enrollment increased from 10 to 13

sites_removedChronic Graft-versus-Host DiseaseJun 25AstraZenecaNCT06706817
Reduced from 45 to 41 sites

Reduced from 45 to 41 sites

sites_addedCholangiocarcinomaJun 25Astellas Pharma Global Development, Inc.NCT07488676
Added 3 new sites: Kashiwa-shi, Japan, Chuo-ku, Japan, Koto-ku, Japan

Added 3 new sites: Kashiwa-shi, Japan, Chuo-ku, Japan, Koto-ku, Japan

sites_addedCholangiocarcinomaJun 25Insel Gruppe AG, University Hospital BernNCT06801899
Added 3 new sites: Münster, Germany, Ulm, Germany, Aarau, Switzerland

Added 3 new sites: Münster, Germany, Ulm, Germany, Aarau, Switzerland

eligibility_changeBecker Muscular DystrophyJun 24Sarepta Therapeutics, Inc.NCT04626674
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJun 24PrileniaNCT07322003
Added 4 new sites: Jerusalem, Israel, Tel Aviv, Israel, Santiago de Compostela, Spain and 1 more

Added 4 new sites: Jerusalem, Israel, Tel Aviv, Israel, Santiago de Compostela, Spain and 1 more

Enrollment UpdateC3 GlomerulopathyJun 24Novartis PharmaceuticalsNCT07156149
Target enrollment decreased from 50 to 32

Target enrollment decreased from 50 to 32

sites_addedBecker Muscular DystrophyJun 24Precision BioSciences, Inc.NCT07429240
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jun 24AstraZenecaNCT06465810
Added 7 new sites: Wuhan, China, Nanjing, China, Suzhou, China and 4 more

Added 7 new sites: Wuhan, China, Nanjing, China, Suzhou, China and 4 more

sites_removedATTR Amyloidosis (Transthyretin Amyloidosis)Jun 24Idoven 1903 S.L.NCT06978660
Reduced from 10 to 8 sites

Reduced from 10 to 8 sites

Enrollment UpdateATTR Amyloidosis (Transthyretin Amyloidosis)Jun 24Idoven 1903 S.L.NCT06978660
Target enrollment increased from 2000 to 2200

Target enrollment increased from 2000 to 2200

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Jun 24Novo Nordisk A/SNCT07207811
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAngelman SyndromeJun 24Ionis Pharmaceuticals, Inc.NCT06914609
Added 2 new sites: New York, New York, Cincinnati, Ohio

Added 2 new sites: New York, New York, Cincinnati, Ohio

sites_addedAmyotrophic Lateral SclerosisJun 24Regeneron PharmaceuticalsNCT06351592
Added 1 new site: Umeå, Sweden

Added 1 new site: Umeå, Sweden

Enrollment UpdateAmyotrophic Lateral SclerosisJun 24Regeneron PharmaceuticalsNCT06351592
Target enrollment increased from 42 to 54

Target enrollment increased from 42 to 54

sites_addedAmyotrophic Lateral SclerosisJun 24Nura BioNCT07369076
Added 2 new sites: Caulfield South, Australia, Nedlands, Australia

Added 2 new sites: Caulfield South, Australia, Nedlands, Australia

eligibility_changeAmyotrophic Lateral SclerosisJun 24Nura BioNCT07369076
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJun 24PrileniaNCT07322003
Added 8 new sites: San Francisco, California, Chicago, Illinois, St Louis, Missouri and 5 more

Added 8 new sites: San Francisco, California, Chicago, Illinois, St Louis, Missouri and 5 more

sites_addedAcromegalyJun 24Debiopharm International SANCT06930625
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedWild-Type ATTR Cardiac AmyloidosisJun 23AstraZenecaNCT06465810
Added 7 new sites: Wuhan, China, Nanjing, China, Suzhou, China and 4 more

Added 7 new sites: Wuhan, China, Nanjing, China, Suzhou, China and 4 more

sites_addedTurner SyndromeJun 23Ascendis Pharma A/SNCT07221851
Added 2 new sites: Toulouse, France, Zaragoza, Spain

Added 2 new sites: Toulouse, France, Zaragoza, Spain

sites_addedStargardt DiseaseJun 23AAVantgarde Bio SrlNCT07161544
Added 1 new site: Salt Lake City, Utah

Added 1 new site: Salt Lake City, Utah

sites_removedWaldenström MacroglobulinemiaJun 23BeOne MedicinesNCT05006716
Reduced from 130 to 127 sites

Reduced from 130 to 127 sites

sites_addedUsher SyndromeJun 23Laboratoires TheaNCT06627179
Added 1 new site: Belo Horizonte, Brazil

Added 1 new site: Belo Horizonte, Brazil

sites_addedTurner SyndromeJun 23Ascendis Pharma A/SNCT07221851
Added 5 new sites: Palo Alto, California, St. Petersburg, Florida, Minneapolis, Minnesota and 2 more

Added 5 new sites: Palo Alto, California, St. Petersburg, Florida, Minneapolis, Minnesota and 2 more

sites_addedThalassemiaJun 23Novartis PharmaceuticalsNCT02386800
Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

sites_addedTay-Sachs DiseaseJun 23Azafaros B.V.NCT07082725
Added 1 new site: Lahore, Pakistan

Added 1 new site: Lahore, Pakistan

sites_addedTay-Sachs DiseaseJun 23Azafaros B.V.NCT07054515
Added 1 new site: Lahore, Pakistan

Added 1 new site: Lahore, Pakistan

sites_addedSickle Cell DiseaseJun 23Novo Nordisk A/SNCT06612268
Added 4 new sites: Cali, Colombia, Al Qaţīf, Saudi Arabia, Riyadh, Saudi Arabia and 1 more

Added 4 new sites: Cali, Colombia, Al Qaţīf, Saudi Arabia, Riyadh, Saudi Arabia and 1 more

sites_addedPulmonary Arterial HypertensionJun 22Regeneron PharmaceuticalsNCT07318597
Added 5 new sites: Hobart, Australia, Le Kremlin-Bicêtre, France, Seoul, South Korea and 2 more

Added 5 new sites: Hobart, Australia, Le Kremlin-Bicêtre, France, Seoul, South Korea and 2 more

sites_addedPulmonary Arterial HypertensionJun 22Merck Sharp & Dohme LLCNCT07218029
Added 1 new site: Porto Alegre, Brazil

Added 1 new site: Porto Alegre, Brazil

sites_removedPulmonary Arterial HypertensionJun 22V-Wave LtdNCT03838445
Reduced from 5 to 3 sites

Reduced from 5 to 3 sites

Enrollment UpdatePulmonary Arterial HypertensionJun 22V-Wave LtdNCT03838445
Target enrollment decreased from 20 to 9

Target enrollment decreased from 20 to 9

sites_addedPrimary Biliary CholangitisJun 22GlaxoSmithKlineNCT04167358
Added 5 new sites: Rehovot, Israel, Milan, Italy, Modena, Italy and 2 more

Added 5 new sites: Rehovot, Israel, Milan, Italy, Modena, Italy and 2 more

sites_addedPolycythemia VeraJun 22Novartis PharmaceuticalsNCT02386800
Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

Added 2 new sites: Copenhagen, Denmark, Bratislava, Slovakia

sites_addedRetinitis PigmentosaJun 22Laboratoires TheaNCT06627179
Added 1 new site: Belo Horizonte, Brazil

Added 1 new site: Belo Horizonte, Brazil

sites_addedPulmonary Langerhans Cell HistiocytosisJun 22National Cancer Institute (NCI)NCT05828069
Added 2 new sites: Long Beach, California, Houston, Texas

Added 2 new sites: Long Beach, California, Houston, Texas

sites_addedPulmonary Arterial HypertensionJun 22Eko Devices, Inc.NCT07087613
Added 1 new site: Norfolk, Virginia

Added 1 new site: Norfolk, Virginia

eligibility_changePulmonary Arterial HypertensionJun 22Eko Devices, Inc.NCT07087613
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedProgressive Supranuclear PalsyJun 22Novartis PharmaceuticalsNCT07498426
Added 14 new sites: San Francisco, California, Jacksonville, Florida, Melbourne, Australia and 11 more

Added 14 new sites: San Francisco, California, Jacksonville, Florida, Melbourne, Australia and 11 more

sites_addedPolycystic Kidney DiseaseJun 22University of CologneNCT07280585
Added 3 new sites: Stuttgart, Germany, Nuremberg, Germany, Freiburg im Breisgau, Germany

Added 3 new sites: Stuttgart, Germany, Nuremberg, Germany, Freiburg im Breisgau, Germany

sites_addedPolycystic Kidney DiseaseJun 22Vertex Pharmaceuticals IncorporatedNCT07161037
Added 1 new site: Tours, France

Added 1 new site: Tours, France

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 22Regeneron PharmaceuticalsNCT07154745
Added 3 new sites: Rome, Italy, Seoul, South Korea, Izmir, Turkey (Türkiye)

Added 3 new sites: Rome, Italy, Seoul, South Korea, Izmir, Turkey (Türkiye)

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 22Regeneron PharmaceuticalsNCT07187401
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 22Regeneron PharmaceuticalsNCT05744921
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedNoonan SyndromeJun 21Ascendis Pharma A/SNCT07221851
Added 5 new sites: Palo Alto, California, St. Petersburg, Florida, Minneapolis, Minnesota and 2 more

Added 5 new sites: Palo Alto, California, St. Petersburg, Florida, Minneapolis, Minnesota and 2 more

sites_addedNiemann-Pick Disease Type CJun 21Azafaros B.V.NCT07054515
Added 1 new site: Lahore, Pakistan

Added 1 new site: Lahore, Pakistan

sites_addedNiemann-Pick Disease Type CJun 21Azafaros B.V.NCT07082725
Added 1 new site: Lahore, Pakistan

Added 1 new site: Lahore, Pakistan

sites_addedNiemann-Pick DiseaseJun 21Azafaros B.V.NCT07054515
Added 1 new site: Lahore, Pakistan

Added 1 new site: Lahore, Pakistan

sites_addedNiemann-Pick DiseaseJun 21Azafaros B.V.NCT07082725
Added 1 new site: Lahore, Pakistan

Added 1 new site: Lahore, Pakistan

sites_removedMyelofibrosisJun 21Geron CorporationNCT04576156
Reduced from 174 to 144 sites

Reduced from 174 to 144 sites

sites_addedMyelodysplastic SyndromesJun 21Regeneron PharmaceuticalsNCT07187401
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedMyelodysplastic SyndromesJun 21Regeneron PharmaceuticalsNCT07154745
Added 3 new sites: Rome, Italy, Seoul, South Korea, Izmir, Turkey (Türkiye)

Added 3 new sites: Rome, Italy, Seoul, South Korea, Izmir, Turkey (Türkiye)

eligibility_changeMyelodysplastic SyndromesJun 21Dana-Farber Cancer InstituteNCT06138587
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJun 21TakedaNCT07422480
Added 43 new sites: Gilbert, Arizona, Duarte, California, Santa Rosa, California and 40 more

Added 43 new sites: Gilbert, Arizona, Duarte, California, Santa Rosa, California and 40 more

sites_addedMyelodysplastic SyndromesJun 21Regeneron PharmaceuticalsNCT05744921
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

eligibility_changeMyelodysplastic SyndromesJun 21Fred Hutchinson Cancer CenterNCT03670966
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJun 21Vor BiopharmaNCT06456580
Added 6 new sites: Scottsdale, Arizona, O'Fallon, Illinois, Columbia, Missouri and 3 more

Added 6 new sites: Scottsdale, Arizona, O'Fallon, Illinois, Columbia, Missouri and 3 more

sites_addedMyasthenia GravisJun 21Immunovant Sciences GmbHNCT07039916
Added 1 new site: Schaumburg, Illinois

Added 1 new site: Schaumburg, Illinois

sites_addedMultiple System AtrophyJun 21Teva Branded Pharmaceutical Products R&D LLCNCT06568237
Added 4 new sites: Paris, France, Beelitz, Germany, Elche, Spain and 1 more

Added 4 new sites: Paris, France, Beelitz, Germany, Elche, Spain and 1 more

sites_addedMultiple System AtrophyJun 21Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 1 new site: Sendai, Japan

Added 1 new site: Sendai, Japan

sites_removedMultiple SclerosisJun 21Novartis PharmaceuticalsNCT06551519
Reduced from 109 to 89 sites

Reduced from 109 to 89 sites

Enrollment UpdateMultiple SclerosisJun 21Novartis PharmaceuticalsNCT06551519
Target enrollment decreased from 700 to 489

Target enrollment decreased from 700 to 489

sites_addedMultiple SclerosisJun 21Topadur Pharma AGNCT06954597
Added 1 new site: Zurich, Switzerland

Added 1 new site: Zurich, Switzerland

eligibility_changeMultiple SclerosisJun 21Topadur Pharma AGNCT06954597
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJun 21TG Therapeutics, Inc.NCT06433752
Added 2 new sites: Roeland Park, Kansas, Salem, Virginia

Added 2 new sites: Roeland Park, Kansas, Salem, Virginia

sites_addedMixed Connective Tissue DiseaseJun 21Milton S. Hershey Medical CenterNCT06465199
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedMembranous NephropathyJun 21BiogenNCT06962800
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedMedullary Thyroid CancerJun 21National Cancer Institute (NCI)NCT03866382
Added 3 new sites: Baton Rouge, Louisiana, Metairie, Louisiana, Peabody, Massachusetts

Added 3 new sites: Baton Rouge, Louisiana, Metairie, Louisiana, Peabody, Massachusetts

sites_addedLight Chain AmyloidosisJun 20Columbia UniversityNCT05451771
Added 2 new sites: Birmingham, Alabama, St Louis, Missouri

Added 2 new sites: Birmingham, Alabama, St Louis, Missouri

sites_addedLambert-Eaton Myasthenic SyndromeJun 20argenxNCT06078553
Added 1 new site: Warsaw, Poland

Added 1 new site: Warsaw, Poland

eligibility_changeIgA NephropathyJun 20Hoffmann-La RocheNCT05797610
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyJun 20BiogenNCT06935357
Added 4 new sites: St Leonards, Australia, Guangzhou, China, Wuhan, China and 1 more

Added 4 new sites: St Leonards, Australia, Guangzhou, China, Wuhan, China and 1 more

eligibility_changeIdiopathic Pulmonary FibrosisJun 20University of California, Los AngelesNCT06162884
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJun 20Contineum TherapeuticsNCT07284459
Added 6 new sites: Hadera, Israel, Epsom, New Zealand, Papatoetoe, New Zealand and 3 more

Added 6 new sites: Hadera, Israel, Epsom, New Zealand, Papatoetoe, New Zealand and 3 more

sites_addedHodgkin LymphomaJun 20AmgenNCT07134088
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

sites_addedHodgkin LymphomaJun 20Eli Lilly and CompanyNCT07226843
Added 1 new site: Okayama, Japan

Added 1 new site: Okayama, Japan

sites_addedHodgkin LymphomaJun 20Merck Sharp & Dohme LLCNCT06395103
Added 1 new site: Valencia, Spain

Added 1 new site: Valencia, Spain

Enrollment UpdateHodgkin LymphomaJun 20Loxo Oncology, Inc.NCT05254743
Target enrollment increased from 662 to 737

Target enrollment increased from 662 to 737

sites_addedHodgkin LymphomaJun 20Alliance for Clinical Trials in OncologyNCT05976763
Added 1 new site: Kearney, Nebraska

Added 1 new site: Kearney, Nebraska

Enrollment UpdateHodgkin LymphomaJun 20City of Hope Medical CenterNCT03011814
Target enrollment increased from 38 to 78

Target enrollment increased from 38 to 78

Enrollment UpdateHodgkin LymphomaJun 20Memorial Sloan Kettering Cancer CenterNCT04468841
Target enrollment decreased from 100 to 77

Target enrollment decreased from 100 to 77

sites_addedHodgkin LymphomaJun 20Novartis PharmaceuticalsNCT07422610
Added 3 new sites: Bordeaux, France, Madrid, Spain, Madrid, Spain

Added 3 new sites: Bordeaux, France, Madrid, Spain, Madrid, Spain

sites_removedHodgkin LymphomaJun 20AstraZenecaNCT04594642
Reduced from 27 to 25 sites

Reduced from 27 to 25 sites

sites_addedHodgkin LymphomaJun 20Lyell Immunopharma, Inc.NCT05826535
Added 2 new sites: Washington D.C., District of Columbia, Perth, Australia

Added 2 new sites: Washington D.C., District of Columbia, Perth, Australia

sites_addedHodgkin LymphomaJun 20Janssen Research & Development, LLCNCT06788509
Added 2 new sites: Nedlands, Australia, Barcelona, Spain

Added 2 new sites: Nedlands, Australia, Barcelona, Spain

sites_addedHodgkin LymphomaJun 20CelgeneNCT04884035
Added 7 new sites: Birmingham, Alabama, Duarte, California, Marietta, Georgia and 4 more

Added 7 new sites: Birmingham, Alabama, Duarte, California, Marietta, Georgia and 4 more

eligibility_changeHereditary AngioedemaJun 20Pharvaris Netherlands B.V.NCT05396105
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BJun 19CSL BehringNCT06003387
Added 1 new site: Pittsburgh, Pennsylvania

Added 1 new site: Pittsburgh, Pennsylvania

eligibility_changeHemophilia BJun 19CSL BehringNCT06003387
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BJun 19Novo Nordisk A/SNCT06285071
Added 6 new sites: Aomori, Japan, Kagoshima, Japan, Kitakyusyu-shi, Fukuoka, Japan and 3 more

Added 6 new sites: Aomori, Japan, Kagoshima, Japan, Kitakyusyu-shi, Fukuoka, Japan and 3 more

sites_addedHemophilia BJun 19Novo Nordisk A/SNCT06831734
Added 15 new sites: Fukui, Japan, Fukushima, Japan, Hyōgo, Japan and 12 more

Added 15 new sites: Fukui, Japan, Fukushima, Japan, Hyōgo, Japan and 12 more

sites_addedHemophilia AJun 19Novo Nordisk A/SNCT06285071
Added 6 new sites: Aomori, Japan, Kagoshima, Japan, Kitakyusyu-shi, Fukuoka, Japan and 3 more

Added 6 new sites: Aomori, Japan, Kagoshima, Japan, Kitakyusyu-shi, Fukuoka, Japan and 3 more

sites_addedHemophilia AJun 19Novo Nordisk A/SNCT06831734
Added 15 new sites: Fukui, Japan, Fukushima, Japan, Hyōgo, Japan and 12 more

Added 15 new sites: Fukui, Japan, Fukushima, Japan, Hyōgo, Japan and 12 more

sites_addedGraves’ DiseaseJun 19AmgenNCT07438405
Added 4 new sites: Warsaw, Poland, Seville, Spain, Valencia, Spain and 1 more

Added 4 new sites: Warsaw, Poland, Seville, Spain, Valencia, Spain and 1 more

sites_addedGeographic AtrophyJun 19Regeneron PharmaceuticalsNCT06541704
Added 3 new sites: Metairie, Louisiana, Worcester, Massachusetts, Ottawa, Canada

Added 3 new sites: Metairie, Louisiana, Worcester, Massachusetts, Ottawa, Canada

eligibility_changeGeographic AtrophyJun 19National Eye Institute (NEI)NCT04339764
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyJun 19Regeneron PharmaceuticalsNCT07230834
Added 1 new site: Tbilisi, Georgia

Added 1 new site: Tbilisi, Georgia

sites_addedGeographic AtrophyJun 19Novartis PharmaceuticalsNCT07441642
Added 6 new sites: New York, New York, New York, New York, Lyon, France and 3 more

Added 6 new sites: New York, New York, New York, New York, Lyon, France and 3 more

sites_addedGeographic AtrophyJun 19ADARx Pharmaceuticals, Inc.NCT06990269
Added 3 new sites: Bakersfield, California, Bellaire, Texas, Vancouver, Canada

Added 3 new sites: Bakersfield, California, Bellaire, Texas, Vancouver, Canada

sites_addedGaucher DiseaseJun 19Spur TherapeuticsNCT07223944
Added 30 new sites: Los Angeles, California, Los Angeles, California, San Francisco, California and 27 more

Added 30 new sites: Los Angeles, California, Los Angeles, California, San Francisco, California and 27 more

sites_addedGastrointestinal Stromal TumorJun 19Cogent Biosciences, Inc.NCT05208047
Added 16 new sites: Birmingham, Alabama, Orange City, Florida, Toledo, Ohio and 13 more

Added 16 new sites: Birmingham, Alabama, Orange City, Florida, Toledo, Ohio and 13 more

eligibility_changeGastrointestinal Stromal TumorJun 19Cogent Biosciences, Inc.NCT05208047
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyJun 19Rapport Therapeutics Inc.NCT07563881
Added 1 new site: Bethesda, Maryland

Added 1 new site: Bethesda, Maryland

sites_addedEosinophilic EsophagitisJun 18Regeneron PharmaceuticalsNCT07112378
Added 1 new site: San Francisco, California

Added 1 new site: San Francisco, California

sites_addedEosinophilic EsophagitisJun 18Regeneron PharmaceuticalsNCT06693531
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

sites_addedDermatomyositisJun 18Allogene TherapeuticsNCT07085104
Added 1 new site: Loma Linda, California

Added 1 new site: Loma Linda, California

sites_addedCystic FibrosisJun 18SanofiNCT07547436
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

eligibility_changeChronic Graft-versus-Host DiseaseJun 18AbbVieNCT05753501
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJun 18Brigham and Women's HospitalNCT07599293
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 18Heartfelt TechnologiesNCT06222099
Added 1 new site: Little Rock, Arkansas

Added 1 new site: Little Rock, Arkansas

eligibility_changeChronic Graft-versus-Host DiseaseJun 18Cerenovus, Part of DePuy Synthes Products, Inc.NCT06735833
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJun 18National Cancer Institute (NCI)NCT04269902
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJun 18Kedrion S.p.A.NCT07059000
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 18Annexon, Inc.NCT07020819
Added 4 new sites: Phoenix, Arizona, Carlsbad, California, Jacksonville, Florida and 1 more

Added 4 new sites: Phoenix, Arizona, Carlsbad, California, Jacksonville, Florida and 1 more

sites_addedChronic Graft-versus-Host DiseaseJun 18AstraZenecaNCT07399665
Added 1 new site: Kemerovo, Russia

Added 1 new site: Kemerovo, Russia

eligibility_changeChronic Graft-versus-Host DiseaseJun 18Novartis PharmaceuticalsNCT05456191
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 18X4 PharmaceuticalsNCT06056297
Added 2 new sites: Leuven, Belgium, Floridablanca, Colombia

Added 2 new sites: Leuven, Belgium, Floridablanca, Colombia

sites_addedChronic Graft-versus-Host DiseaseJun 18TakedaNCT06722235
Added 4 new sites: Novara, Italy, Bergen, Norway, Warsaw, Poland and 1 more

Added 4 new sites: Novara, Italy, Bergen, Norway, Warsaw, Poland and 1 more

sites_addedChronic Graft-versus-Host DiseaseJun 18University Medical Center GroningenNCT05374291
Added 13 new sites: Antwerp, Belgium, Hasselt, Belgium, Singapore, Singapore and 10 more

Added 13 new sites: Antwerp, Belgium, Hasselt, Belgium, Singapore, Singapore and 10 more

sites_addedChronic Graft-versus-Host DiseaseJun 18Novartis PharmaceuticalsNCT06868212
Added 15 new sites: Fayetteville, Arkansas, Rolling Hills Estates, California, Santa Monica, California and 12 more

Added 15 new sites: Fayetteville, Arkansas, Rolling Hills Estates, California, Santa Monica, California and 12 more

sites_addedChronic Graft-versus-Host DiseaseJun 18argenxNCT06920004
Added 29 new sites: Tampa, Florida, Rosario, Argentina, Vienna, Austria and 26 more

Added 29 new sites: Tampa, Florida, Rosario, Argentina, Vienna, Austria and 26 more

sites_addedCholangiocarcinomaJun 18AstraZenecaNCT07221253
Added 6 new sites: Palo Alto, California, Indianapolis, Indiana, Pierre, South Dakota and 3 more

Added 6 new sites: Palo Alto, California, Indianapolis, Indiana, Pierre, South Dakota and 3 more

sites_addedDravet SyndromeJun 18Longboard PharmaceuticalsNCT06660394
Added 5 new sites: Antwerp, Belgium, Porto Alegre, Brazil, Roma, Italy and 2 more

Added 5 new sites: Antwerp, Belgium, Porto Alegre, Brazil, Roma, Italy and 2 more

sites_addedDilated CardiomyopathyJun 18Alexion Pharmaceuticals, Inc.NCT07218887
Added 3 new sites: Philadelphia, Pennsylvania, Massa, Italy, London, United Kingdom

Added 3 new sites: Philadelphia, Pennsylvania, Massa, Italy, London, United Kingdom

sites_addedDermatomyositisJun 18argenxNCT05979441
Added 2 new sites: Guangzhou, China, Taichung, Taiwan

Added 2 new sites: Guangzhou, China, Taichung, Taiwan

sites_addedCushing DiseaseJun 18Sparrow PharmaceuticalsNCT07296484
Added 11 new sites: San Diego, California, Atlanta, Georgia, Boise, Idaho and 8 more

Added 11 new sites: San Diego, California, Atlanta, Georgia, Boise, Idaho and 8 more

sites_addedCushing DiseaseJun 18H. Lundbeck A/SNCT06471829
Added 2 new sites: Messina, Italy, Krakow, Poland

Added 2 new sites: Messina, Italy, Krakow, Poland

sites_addedCongenital Adrenal HyperplasiaJun 18Crinetics Pharmaceuticals Inc.NCT07159841
Added 2 new sites: Nedlands, Australia, Brussels, Belgium

Added 2 new sites: Nedlands, Australia, Brussels, Belgium

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18SanofiNCT06859099
Added 2 new sites: Bunkyo-Ku, Japan, Konya, Turkey (Türkiye)

Added 2 new sites: Bunkyo-Ku, Japan, Konya, Turkey (Türkiye)

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18SanofiNCT06290141
Added 5 new sites: Centennial, Colorado, Miami Lakes, Florida, Lille, France and 2 more

Added 5 new sites: Centennial, Colorado, Miami Lakes, Florida, Lille, France and 2 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18argenxNCT06920004
Added 2 new sites: Paris, France, Salford, United Kingdom

Added 2 new sites: Paris, France, Salford, United Kingdom

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18SanofiNCT06290128
Added 4 new sites: Miami Lakes, Florida, Belo Horizonte, Brazil, Bunkyo-Ku, Japan and 1 more

Added 4 new sites: Miami Lakes, Florida, Belo Horizonte, Brazil, Bunkyo-Ku, Japan and 1 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18argenxNCT07091630
Added 5 new sites: Birmingham, Alabama, Rosario, Argentina, Lille, France and 2 more

Added 5 new sites: Birmingham, Alabama, Rosario, Argentina, Lille, France and 2 more

sites_addedChronic Graft-versus-Host DiseaseJun 18nChroma BioNCT07200193
Added 2 new sites: Lyon, France, London, United Kingdom

Added 2 new sites: Lyon, France, London, United Kingdom

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 18Syndax PharmaceuticalsNCT04710576
Target enrollment increased from 241 to 296

Target enrollment increased from 241 to 296

sites_addedChronic Graft-versus-Host DiseaseJun 18Novartis PharmaceuticalsNCT07354074
Added 1 new site: Montreal, Canada

Added 1 new site: Montreal, Canada

sites_addedChronic Graft-versus-Host DiseaseJun 18REVA Medical, Inc.NCT05406622
Added 10 new sites: Darien, Connecticut, Miami, Florida, Miami Beach, Florida and 7 more

Added 10 new sites: Darien, Connecticut, Miami, Florida, Miami Beach, Florida and 7 more

sites_addedChronic Graft-versus-Host DiseaseJun 18Janssen Research & Development, LLCNCT05327114
Added 2 new sites: Rosario, Argentina, Orbassano, Italy

Added 2 new sites: Rosario, Argentina, Orbassano, Italy

sites_addedChronic Graft-versus-Host DiseaseJun 18Nurix Therapeutics, Inc.NCT07221500
Added 6 new sites: Coral Gables, Florida, Fort Wayne, Indiana, Indianapolis, Indiana and 3 more

Added 6 new sites: Coral Gables, Florida, Fort Wayne, Indiana, Indianapolis, Indiana and 3 more

sites_addedChronic Graft-versus-Host DiseaseJun 18SanofiNCT05263206
Added 1 new site: Gyula, Hungary

Added 1 new site: Gyula, Hungary

sites_addedCholangiocarcinomaJun 18Kestrel Therapeutics, Inc.NCT07458347
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Jun 17University of Texas Southwestern Medical CenterNCT05489549
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jun 17Alnylam PharmaceuticalsNCT07223203
Added 12 new sites: Aurora, Colorado, Chicago, Illinois, Rochester, Minnesota and 9 more

Added 12 new sites: Aurora, Colorado, Chicago, Illinois, Rochester, Minnesota and 9 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jun 17Alnylam PharmaceuticalsNCT07052903
Added 9 new sites: Liverpool, Australia, Rio de Janeiro, Brazil, Vancouver, Canada and 6 more

Added 9 new sites: Liverpool, Australia, Rio de Janeiro, Brazil, Vancouver, Canada and 6 more

eligibility_changeAmyotrophic Lateral SclerosisJun 17Corcept TherapeuticsNCT05407324
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAdrenocortical CarcinomaJun 17National Cancer Institute (NCI)NCT06900595
Added 2 new sites: Oklahoma City, Oklahoma, Austin, Texas

Added 2 new sites: Oklahoma City, Oklahoma, Austin, Texas

sites_addedStargardt DiseaseJun 16Astellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Ann Arbor, Michigan

Added 1 new site: Ann Arbor, Michigan

sites_addedSickle Cell DiseaseJun 16Bristol-Myers SquibbNCT06481306
Added 15 new sites: Tampa, Florida, Baltimore, Maryland, St Louis, Missouri and 12 more

Added 15 new sites: Tampa, Florida, Baltimore, Maryland, St Louis, Missouri and 12 more

sites_addedWild-Type ATTR Cardiac AmyloidosisJun 16Alnylam PharmaceuticalsNCT07052903
Added 9 new sites: Liverpool, Australia, Rio de Janeiro, Brazil, Vancouver, Canada and 6 more

Added 9 new sites: Liverpool, Australia, Rio de Janeiro, Brazil, Vancouver, Canada and 6 more

sites_addedWaldenström MacroglobulinemiaJun 16National Cancer Institute (NCI)NCT04840602
Added 3 new sites: Minneapolis, Minnesota, Saint Paul, Minnesota, Monroe, North Carolina

Added 3 new sites: Minneapolis, Minnesota, Saint Paul, Minnesota, Monroe, North Carolina

sites_addedThalassemiaJun 16Novo Nordisk A/SNCT06609226
Added 2 new sites: Mersin, Turkey (Türkiye), London, United Kingdom

Added 2 new sites: Mersin, Turkey (Türkiye), London, United Kingdom

sites_addedStargardt DiseaseJun 16Astellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

eligibility_changeSickle Cell DiseaseJun 16University of PennsylvaniaNCT05730205
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseJun 16Novo Nordisk A/SNCT06609226
Added 2 new sites: Mersin, Turkey (Türkiye), London, United Kingdom

Added 2 new sites: Mersin, Turkey (Türkiye), London, United Kingdom

eligibility_changeShort Bowel SyndromeJun 15Napo Therapeutics, S.p.A.NCT06904872
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJun 15Merck Sharp & Dohme LLCNCT07218029
Added 3 new sites: Tampa, Florida, Regensburg, Germany, Otwock, Poland

Added 3 new sites: Tampa, Florida, Regensburg, Germany, Otwock, Poland

sites_addedPulmonary Arterial HypertensionJun 15Insmed IncorporatedNCT07481981
Added 1 new site: Santa Barbara, California

Added 1 new site: Santa Barbara, California

sites_addedPulmonary Arterial HypertensionJun 15Abbott Medical DevicesNCT06526195
Added 5 new sites: Chicago, Illinois, Portland, Maine, Albuquerque, New Mexico and 2 more

Added 5 new sites: Chicago, Illinois, Portland, Maine, Albuquerque, New Mexico and 2 more

eligibility_changePulmonary Arterial HypertensionJun 15National University Hospital, SingaporeNCT05473520
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJun 15United TherapeuticsNCT06388421
Added 5 new sites: Los Angeles, California, Neptune City, New Jersey, Rochester, New York and 2 more

Added 5 new sites: Los Angeles, California, Neptune City, New Jersey, Rochester, New York and 2 more

eligibility_changePulmonary Arterial HypertensionJun 15Lifetech Scientific (Shenzhen) Co., Ltd.NCT06099015
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedPolycythemia VeraJun 15Novartis PharmaceuticalsNCT05548062
Reduced from 35 to 33 sites

Reduced from 35 to 33 sites

sites_addedPolycythemia VeraJun 15Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Capital Federal, Argentina, Pune, India

Added 2 new sites: Capital Federal, Argentina, Pune, India

sites_addedPolycythemia VeraJun 15Prelude Therapeutics IncorporatedNCT07469891
Added 2 new sites: Denver, Colorado, Philadelphia, Pennsylvania

Added 2 new sites: Denver, Colorado, Philadelphia, Pennsylvania

sites_addedPhelan-McDermid SyndromeJun 15Neuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Glendale, California, San Diego, California

Added 2 new sites: Glendale, California, San Diego, California

sites_addedNTRK Fusion CancerJun 15St. Jude Children's Research HospitalNCT06239272
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

sites_addedNarcolepsy Type 1Jun 14Alkermes, Inc.NCT07540897
Added 4 new sites: Macon, Georgia, Dallas, Texas, San Antonio, Texas and 1 more

Added 4 new sites: Macon, Georgia, Dallas, Texas, San Antonio, Texas and 1 more

sites_addedNarcolepsy Type 1Jun 14Centessa Pharmaceuticals (UK) LimitedNCT07598708
Added 1 new site: Huntersville, North Carolina

Added 1 new site: Huntersville, North Carolina

sites_addedMyelofibrosisJun 14Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Capital Federal, Argentina, Pune, India

Added 2 new sites: Capital Federal, Argentina, Pune, India

sites_addedMyelofibrosisJun 14Prelude Therapeutics IncorporatedNCT07469891
Added 2 new sites: Denver, Colorado, Philadelphia, Pennsylvania

Added 2 new sites: Denver, Colorado, Philadelphia, Pennsylvania

eligibility_changeMyelodysplastic SyndromesJun 14Mayo ClinicNCT03017820
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJun 14National Cancer Institute (NCI)NCT06484062
Added 1 new site: Winston-Salem, North Carolina

Added 1 new site: Winston-Salem, North Carolina

Enrollment UpdateMyelodysplastic SyndromesJun 14Fujian Medical University Union HospitalNCT07588360
Target enrollment decreased from 180 to 82

Target enrollment decreased from 180 to 82

Enrollment UpdateMyelodysplastic SyndromesJun 14M.D. Anderson Cancer CenterNCT06113302
Target enrollment decreased from 40 to 10

Target enrollment decreased from 40 to 10

sites_addedMyasthenia GravisJun 14argenxNCT07294170
Added 3 new sites: Detroit, Michigan, Durham, North Carolina, Milan, Italy

Added 3 new sites: Detroit, Michigan, Durham, North Carolina, Milan, Italy

sites_addedMyasthenia GravisJun 14Vertex Pharmaceuticals IncorporatedNCT07501702
Added 2 new sites: O'Fallon, Illinois, Sherman, Texas

Added 2 new sites: O'Fallon, Illinois, Sherman, Texas

sites_addedMyasthenia GravisJun 14argenxNCT07284420
Added 2 new sites: Detroit, Michigan, Durham, North Carolina

Added 2 new sites: Detroit, Michigan, Durham, North Carolina

sites_addedMyasthenia GravisJun 14Kite, A Gilead CompanyNCT07304154
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

eligibility_changeMyasthenia GravisJun 14COUR Pharmaceutical Development Company, Inc.NCT06106672
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple System AtrophyJun 14Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 1 new site: Kassel, Germany

Added 1 new site: Kassel, Germany

sites_addedMultiple System AtrophyJun 14Teva Branded Pharmaceutical Products R&D LLCNCT06568237
Added 3 new sites: Nashville, Tennessee, Caen, France, Barakaldo, Spain

Added 3 new sites: Nashville, Tennessee, Caen, France, Barakaldo, Spain

sites_addedMultiple System AtrophyJun 14BiogenNCT07221669
Added 2 new sites: Chicago, Illinois, Shanghai, China

Added 2 new sites: Chicago, Illinois, Shanghai, China

sites_addedMultiple SclerosisJun 14Novartis PharmaceuticalsNCT06486779
Added 2 new sites: Athens, Greece, Athens, Greece

Added 2 new sites: Athens, Greece, Athens, Greece

Enrollment UpdateMultiple SclerosisJun 14AmgenNCT06700343
Target enrollment decreased from 444 to 152

Target enrollment decreased from 444 to 152

sites_addedMultiple SclerosisJun 14PhenoNet, Inc.NCT07142291
Added 2 new sites: Alicante, Spain, Barcelona, Spain

Added 2 new sites: Alicante, Spain, Barcelona, Spain

sites_addedMultiple SclerosisJun 14Novartis PharmaceuticalsNCT05344469
Expanded from 127 to 129 sites

Expanded from 127 to 129 sites

eligibility_changeMultiple SclerosisJun 14Johns Hopkins UniversityNCT06170970
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMixed Connective Tissue DiseaseJun 14St. Jude Children's Research HospitalNCT06239272
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

eligibility_changeMixed Connective Tissue DiseaseJun 14Milton S. Hershey Medical CenterNCT06465199
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMesotheliomaJun 14National Cancer Institute (NCI)NCT03830229
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMesotheliomaJun 14AstraZenecaNCT06097728
Reduced from 178 to 151 sites

Reduced from 178 to 151 sites

sites_addedMesotheliomaJun 14IDEAYA BiosciencesNCT07277413
Added 2 new sites: West Valley City, Utah, Seattle, Washington

Added 2 new sites: West Valley City, Utah, Seattle, Washington

eligibility_changeMesotheliomaJun 14IDEAYA BiosciencesNCT07277413
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaJun 14AstraZenecaNCT07115043
Expanded from 11 to 13 sites

Expanded from 11 to 13 sites

sites_addedMerkel Cell CarcinomaJun 14Merck Sharp & Dohme LLCNCT07302347
Added 1 new site: Kyoto, Japan

Added 1 new site: Kyoto, Japan

sites_addedMembranous NephropathyJun 14BiogenNCT06962800
Added 5 new sites: Cleveland, Ohio, London, Canada, Xi'an, China and 2 more

Added 5 new sites: Cleveland, Ohio, London, Canada, Xi'an, China and 2 more

sites_removedLimb-Girdle Muscular DystrophyJun 13Virginia Commonwealth UniversityNCT05618080
Reduced from 12 to 9 sites

Reduced from 12 to 9 sites

sites_addedLennox-Gastaut SyndromeJun 13Longboard PharmaceuticalsNCT06719141
Added 6 new sites: Chicago, Illinois, Campinas, Brazil, Toronto, Canada and 3 more

Added 6 new sites: Chicago, Illinois, Campinas, Brazil, Toronto, Canada and 3 more

sites_addedIgA NephropathyJun 13TakedaNCT06963827
Added 3 new sites: London, Canada, Torino, Italy, Preston, United Kingdom

Added 3 new sites: London, Canada, Torino, Italy, Preston, United Kingdom

sites_addedIgA NephropathyJun 13BiogenNCT06935357
Added 1 new site: Herston, Australia

Added 1 new site: Herston, Australia

sites_addedIgA NephropathyJun 13ADARx Pharmaceuticals, Inc.NCT06989359
Added 4 new sites: Liverpool, Australia, Wŏnju, South Korea, Barcelona, Spain and 1 more

Added 4 new sites: Liverpool, Australia, Wŏnju, South Korea, Barcelona, Spain and 1 more

sites_addedIdiopathic Pulmonary FibrosisJun 13Contineum TherapeuticsNCT07284459
Added 4 new sites: Melbourne, Australia, Be’er Ya‘aqov, Israel, Petah Tikva, Israel and 1 more

Added 4 new sites: Melbourne, Australia, Be’er Ya‘aqov, Israel, Petah Tikva, Israel and 1 more

eligibility_changeIdiopathic Pulmonary FibrosisJun 13Mediar TherapeuticsNCT06967805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseJun 13Novartis PharmaceuticalsNCT07326709
Added 9 new sites: Cleveland, Ohio, Tel Aviv, Israel, Seoul, South Korea and 6 more

Added 9 new sites: Cleveland, Ohio, Tel Aviv, Israel, Seoul, South Korea and 6 more

sites_addedHunter SyndromeJun 13Denali Therapeutics Inc.NCT06075537
Added 2 new sites: Chicago, Illinois, Hackensack, New Jersey

Added 2 new sites: Chicago, Illinois, Hackensack, New Jersey

sites_addedHomozygous Familial HypercholesterolemiaJun 13Arrowhead PharmaceuticalsNCT07037771
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedHodgkin LymphomaJun 13Janssen Research & Development, LLCNCT07308132
Added 1 new site: Clayton, Australia

Added 1 new site: Clayton, Australia

sites_addedHodgkin LymphomaJun 13National Cancer Institute (NCI)NCT04546399
Added 15 new sites: Long Beach, California, Los Angeles, California, Gainesville, Florida and 12 more

Added 15 new sites: Long Beach, California, Los Angeles, California, Gainesville, Florida and 12 more

eligibility_changeHodgkin LymphomaJun 13National Cancer Institute (NCI)NCT04546399
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJun 13CelgeneNCT06911502
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJun 13The Lymphoma Academic Research OrganisationNCT06271057
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedHodgkin LymphomaJun 13Novartis PharmaceuticalsNCT03876769
Reduced from 45 to 43 sites

Reduced from 45 to 43 sites

Enrollment UpdateHodgkin LymphomaJun 13M.D. Anderson Cancer CenterNCT04054167
Target enrollment decreased from 80 to 51

Target enrollment decreased from 80 to 51

sites_addedHodgkin LymphomaJun 13PfizerNCT03460977
Expanded from 81 to 82 sites

Expanded from 81 to 82 sites

Enrollment UpdateHodgkin LymphomaJun 13University of VirginiaNCT05404945
Target enrollment decreased from 44 to 24

Target enrollment decreased from 44 to 24

sites_addedHodgkin LymphomaJun 13Merck Sharp & Dohme LLCNCT02332668
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedHodgkin LymphomaJun 13Memorial Sloan Kettering Cancer CenterNCT07231952
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedHodgkin LymphomaJun 13Mayo ClinicNCT04996706
Added 2 new sites: Atlanta, Georgia, Atlanta, Georgia

Added 2 new sites: Atlanta, Georgia, Atlanta, Georgia

eligibility_changeHodgkin LymphomaJun 13AbbVieNCT05753501
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 13University College, LondonNCT04685616
Added 1 new site: Dublin, Ireland

Added 1 new site: Dublin, Ireland

sites_addedHodgkin LymphomaJun 13AstraZenecaNCT06542250
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_removedHodgkin LymphomaJun 13Christine RyanNCT01661881
Reduced from 3 to 1 sites

Reduced from 3 to 1 sites

sites_addedHodgkin LymphomaJun 13Memorial Sloan Kettering Cancer CenterNCT06765317
Added 1 new site: Hackensack, New Jersey

Added 1 new site: Hackensack, New Jersey

eligibility_changeHodgkin LymphomaJun 13Mayo ClinicNCT06508463
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 13BeOne MedicinesNCT06742996
Added 2 new sites: Fresno, California, New Haven, Connecticut

Added 2 new sites: Fresno, California, New Haven, Connecticut

sites_addedHodgkin LymphomaJun 13Hoffmann-La RocheNCT07200375
Added 1 new site: Hong Kong, Hong Kong

Added 1 new site: Hong Kong, Hong Kong

sites_addedHodgkin LymphomaJun 13Fondazione Italiana Linfomi - ETSNCT05929222
Added 1 new site: Sassuolo, Italy

Added 1 new site: Sassuolo, Italy

sites_addedHodgkin LymphomaJun 13National Cancer Institute (NCI)NCT04840602
Added 5 new sites: Minneapolis, Minnesota, Saint Louis Park, Minnesota, Saint Paul, Minnesota and 2 more

Added 5 new sites: Minneapolis, Minnesota, Saint Louis Park, Minnesota, Saint Paul, Minnesota and 2 more

eligibility_changeHodgkin LymphomaJun 13M.D. Anderson Cancer CenterNCT05355051
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BJun 12Novo Nordisk A/SNCT03745924
Added 5 new sites: Zagreb, Croatia, Brno, Czechia, Helsinki, Finland and 2 more

Added 5 new sites: Zagreb, Croatia, Brno, Czechia, Helsinki, Finland and 2 more

sites_addedHemophilia BJun 12Novo Nordisk A/SNCT04082429
Added 3 new sites: Torino, Italy, Porto, Portugal, Malmö, Sweden

Added 3 new sites: Torino, Italy, Porto, Portugal, Malmö, Sweden

sites_addedHemophilia AJun 12Novo Nordisk A/SNCT03745924
Added 5 new sites: Zagreb, Croatia, Brno, Czechia, Helsinki, Finland and 2 more

Added 5 new sites: Zagreb, Croatia, Brno, Czechia, Helsinki, Finland and 2 more

sites_addedHemophilia AJun 12Novo Nordisk A/SNCT04082429
Added 3 new sites: Torino, Italy, Porto, Portugal, Malmö, Sweden

Added 3 new sites: Torino, Italy, Porto, Portugal, Malmö, Sweden

sites_addedHemophilia AJun 12Novo Nordisk A/SNCT05685238
Added 4 new sites: Lille, France, Coimbra, Portugal, Lisbon, Portugal and 1 more

Added 4 new sites: Lille, France, Coimbra, Portugal, Lisbon, Portugal and 1 more

sites_addedGraves’ DiseaseJun 12AmgenNCT06401044
Added 4 new sites: Montreal, Canada, Freiburg im Breisgau, Germany, Athens, Greece and 1 more

Added 4 new sites: Montreal, Canada, Freiburg im Breisgau, Germany, Athens, Greece and 1 more

sites_addedGiant Cell ArteritisJun 12University Hospital, BrestNCT04664465
Added 2 new sites: Nantes, France, Nantes, France

Added 2 new sites: Nantes, France, Nantes, France

sites_addedGastrointestinal Stromal TumorJun 12GlaxoSmithKlineNCT07218926
Added 4 new sites: Pittsburgh, Pennsylvania, Toulouse, France, Pécs, Hungary and 1 more

Added 4 new sites: Pittsburgh, Pennsylvania, Toulouse, France, Pécs, Hungary and 1 more

eligibility_changeGastrointestinal Stromal TumorJun 12GlaxoSmithKlineNCT07218926
Eligibility criteria have been updated

Eligibility criteria have been updated

phase_changeEssential ThrombocythemiaJun 12Step Pharma, SASNCT06786234
Moved from Phase 1 to Phase 2

Moved from Phase 1 to Phase 2

sites_addedEssential ThrombocythemiaJun 12Step Pharma, SASNCT06786234
Added 1 new site: Poitiers, France

Added 1 new site: Poitiers, France

Enrollment UpdateEssential ThrombocythemiaJun 12Step Pharma, SASNCT06786234
Target enrollment increased from 20 to 50

Target enrollment increased from 20 to 50

sites_addedHemophilia AJun 12Hoffmann-La RocheNCT07416604
Added 2 new sites: Ramat Gan, Israel, Nagoya, Japan

Added 2 new sites: Ramat Gan, Israel, Nagoya, Japan

sites_addedGraves’ DiseaseJun 12Immunovant Sciences GmbHNCT07018323
Added 9 new sites: Belo Horizonte, Brazil, São Paulo, Brazil, São Paulo, Brazil and 6 more

Added 9 new sites: Belo Horizonte, Brazil, São Paulo, Brazil, São Paulo, Brazil and 6 more

sites_addedGraves’ DiseaseJun 12Immunovant Sciences GmbHNCT07286006
Added 1 new site: Southport, Australia

Added 1 new site: Southport, Australia

sites_addedGiant Cell ArteritisJun 12SanofiNCT07286214
Added 4 new sites: Plantation, Florida, Willowbrook, Illinois, Wyomissing, Pennsylvania and 1 more

Added 4 new sites: Plantation, Florida, Willowbrook, Illinois, Wyomissing, Pennsylvania and 1 more

sites_addedGeographic AtrophyJun 12Complement TherapeuticsNCT07144137
Added 1 new site: Sunderland, United Kingdom

Added 1 new site: Sunderland, United Kingdom

Enrollment UpdateGeographic AtrophyJun 12Astellas Pharma Global Development, Inc.NCT06779773
Target enrollment decreased from 1000 to 500

Target enrollment decreased from 1000 to 500

eligibility_changeGeographic AtrophyJun 12Astellas Pharma Global Development, Inc.NCT06779773
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeGastrointestinal Stromal TumorJun 12Merck Sharp & Dohme LLCNCT04924075
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFriedreich AtaxiaJun 12Lexeo TherapeuticsNCT06865482
Added 5 new sites: Portland, Oregon, Fortaleza, Brazil, Belo Horizonte, Brazil and 2 more

Added 5 new sites: Portland, Oregon, Fortaleza, Brazil, Belo Horizonte, Brazil and 2 more

sites_addedFocal Segmental GlomerulosclerosisJun 12Boehringer IngelheimNCT07220083
Added 7 new sites: Redding, California, Orlando, Florida, Riverview, Florida and 4 more

Added 7 new sites: Redding, California, Orlando, Florida, Riverview, Florida and 4 more

sites_addedFocal Segmental GlomerulosclerosisJun 12SanofiNCT06500702
Added 2 new sites: Atlanta, Georgia, São Paulo, Brazil

Added 2 new sites: Atlanta, Georgia, São Paulo, Brazil

sites_addedFocal EpilepsyJun 12Neurona TherapeuticsNCT05135091
Added 2 new sites: Birmingham, Alabama, St Louis, Missouri

Added 2 new sites: Birmingham, Alabama, St Louis, Missouri

sites_addedFocal EpilepsyJun 12Rapport Therapeutics Inc.NCT07594119
Added 1 new site: Bentonville, Arkansas

Added 1 new site: Bentonville, Arkansas

sites_addedEssential ThrombocythemiaJun 12Prelude Therapeutics IncorporatedNCT07469891
Added 2 new sites: Denver, Colorado, Philadelphia, Pennsylvania

Added 2 new sites: Denver, Colorado, Philadelphia, Pennsylvania

sites_addedEssential ThrombocythemiaJun 12Merck Sharp & Dohme LLCNCT06456346
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedEssential ThrombocythemiaJun 12Novartis PharmaceuticalsNCT07357727
Added 2 new sites: Capital Federal, Argentina, Pune, India

Added 2 new sites: Capital Federal, Argentina, Pune, India

sites_addedDesmoplastic Small Round Cell TumorJun 11Italian Sarcoma GroupNCT07328425
Added 5 new sites: Candiolo, Italy, Milan, Italy, Padova, Italy and 2 more

Added 5 new sites: Candiolo, Italy, Milan, Italy, Padova, Italy and 2 more

eligibility_changeDermatomyositisJun 11National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NCT05738824
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateDermatomyositisJun 11Novartis PharmaceuticalsNCT06665256
Target enrollment decreased from 123 to 21

Target enrollment decreased from 123 to 21

sites_addedChronic Graft-versus-Host DiseaseJun 11BeOne MedicinesNCT05006716
Added 3 new sites: Louisville, Kentucky, ClermontFerrand, France, Newmarket, United Kingdom

Added 3 new sites: Louisville, Kentucky, ClermontFerrand, France, Newmarket, United Kingdom

eligibility_changeChronic Graft-versus-Host DiseaseJun 11BeOne MedicinesNCT05006716
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 11Gilead SciencesNCT07096193
Added 4 new sites: Louisville, Kentucky, Milan, Italy, Chisinau, Moldova and 1 more

Added 4 new sites: Louisville, Kentucky, Milan, Italy, Chisinau, Moldova and 1 more

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 11Gilead SciencesNCT07096193
Target enrollment increased from 107 to 200

Target enrollment increased from 107 to 200

eligibility_changeChronic Graft-versus-Host DiseaseJun 11Gilead SciencesNCT07096193
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJun 11TakedaNCT06722235
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJun 11Ankara City Hospital BilkentNCT07488429
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 11Verona Pharma, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USANCT07016412
Added 2 new sites: Plymouth, Minnesota, Cincinnati, Ohio

Added 2 new sites: Plymouth, Minnesota, Cincinnati, Ohio

sites_addedChronic Graft-versus-Host DiseaseJun 11Wim JanssensNCT05008081
Added 4 new sites: Vienna, Austria, Kiel, Germany, Golnik, Slovenia and 1 more

Added 4 new sites: Vienna, Austria, Kiel, Germany, Golnik, Slovenia and 1 more

sites_removedChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT04557462
Reduced from 161 to 155 sites

Reduced from 161 to 155 sites

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT04557462
Target enrollment decreased from 540 to 448

Target enrollment decreased from 540 to 448

sites_addedChronic Graft-versus-Host DiseaseJun 11Dianthus TherapeuticsNCT06858579
Added 2 new sites: Jilin City, China, Manchester, United Kingdom

Added 2 new sites: Jilin City, China, Manchester, United Kingdom

sites_addedChronic Graft-versus-Host DiseaseJun 11AstraZenecaNCT06542250
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 11Kite, A Gilead CompanyNCT07304154
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 11Janssen Research & Development, LLCNCT05327114
Added 2 new sites: Rosario, Argentina, Orbassano, Italy

Added 2 new sites: Rosario, Argentina, Orbassano, Italy

sites_addedChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT06824103
Added 3 new sites: Xuzhou, China, Xian, China, Chongqing, China

Added 3 new sites: Xuzhou, China, Xian, China, Chongqing, China

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 11Regeneron PharmaceuticalsNCT05092347
Target enrollment decreased from 56 to 43

Target enrollment decreased from 56 to 43

sites_addedChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT05677451
Expanded from 65 to 66 sites

Expanded from 65 to 66 sites

sites_addedChronic Graft-versus-Host DiseaseJun 11Merck Sharp & Dohme LLCNCT06136559
Added 3 new sites: Albany, New York, Williamsville, New York, Hefei, China

Added 3 new sites: Albany, New York, Williamsville, New York, Hefei, China

sites_removedChronic Graft-versus-Host DiseaseJun 11Novartis PharmaceuticalsNCT06799000
Reduced from 145 to 138 sites

Reduced from 145 to 138 sites

Enrollment UpdateBullous PemphigoidJun 10University Hospital, RouenNCT03295383
Target enrollment decreased from 130 to 92

Target enrollment decreased from 130 to 92

sites_addedAmyotrophic Lateral SclerosisJun 10Washington University School of MedicineNCT07294144
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

sites_addedBarth SyndromeJun 10X4 PharmaceuticalsNCT06056297
Added 2 new sites: Leuven, Belgium, Floridablanca, Colombia

Added 2 new sites: Leuven, Belgium, Floridablanca, Colombia

sites_addedAtypical Hemolytic Uremic SyndromeJun 10Novartis PharmaceuticalsNCT05795140
Added 1 new site: Ankara, Turkey (Türkiye)

Added 1 new site: Ankara, Turkey (Türkiye)

sites_addedAmyotrophic Lateral SclerosisJun 10PrileniaNCT07322003
Added 11 new sites: Florida City, Florida, Atlanta, Georgia, Philadelphia, Pennsylvania and 8 more

Added 11 new sites: Florida City, Florida, Atlanta, Georgia, Philadelphia, Pennsylvania and 8 more

sites_addedAmyotrophic Lateral SclerosisJun 10Merit E. Cudkowicz, MDNCT04297683
Added 3 new sites: La Jolla, California, Burlington, Massachusetts, Houston, Texas

Added 3 new sites: La Jolla, California, Burlington, Massachusetts, Houston, Texas

sites_addedAmyotrophic Lateral SclerosisJun 10PhenoNet, Inc.NCT07142291
Added 2 new sites: Alicante, Spain, Barcelona, Spain

Added 2 new sites: Alicante, Spain, Barcelona, Spain

sites_addedAchondroplasiaJun 10Tyra Biosciences, IncNCT06842355
Added 2 new sites: Edmonton, Canada, Ottawa, Canada

Added 2 new sites: Edmonton, Canada, Ottawa, Canada

sites_addedSpinal Muscular AtrophyJun 9BiogenNCT07221669
Added 2 new sites: Chicago, Illinois, Shanghai, China

Added 2 new sites: Chicago, Illinois, Shanghai, China

sites_addedSpinal Muscular AtrophyJun 9Novartis PharmaceuticalsNCT07190300
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

sites_addedWaldenström MacroglobulinemiaJun 9National Cancer Institute (NCI)NCT04840602
Added 2 new sites: Minneapolis, Minnesota, Saint Paul, Minnesota

Added 2 new sites: Minneapolis, Minnesota, Saint Paul, Minnesota

sites_addedWaldenström MacroglobulinemiaJun 9Memorial Sloan Kettering Cancer CenterNCT07231952
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedWaldenström MacroglobulinemiaJun 9BeOne MedicinesNCT05006716
Added 3 new sites: Louisville, Kentucky, ClermontFerrand, France, Newmarket, United Kingdom

Added 3 new sites: Louisville, Kentucky, ClermontFerrand, France, Newmarket, United Kingdom

eligibility_changeWaldenström MacroglobulinemiaJun 9BeOne MedicinesNCT05006716
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedStargardt DiseaseJun 9Alkeus Pharmaceuticals, Inc.NCT07419334
Added 1 new site: Dallas, Texas

Added 1 new site: Dallas, Texas

sites_addedSpinocerebellar AtaxiaJun 9Lexeo TherapeuticsNCT06865482
Added 5 new sites: Portland, Oregon, Fortaleza, Brazil, Belo Horizonte, Brazil and 2 more

Added 5 new sites: Portland, Oregon, Fortaleza, Brazil, Belo Horizonte, Brazil and 2 more

sites_addedSpinal Muscular AtrophyJun 9Novartis PharmaceuticalsNCT07190300
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedSickle Cell DiseaseJun 9Disc Medicine, IncNCT07187973
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

sites_addedSickle Cell DiseaseJun 9St. Jude Children's Research HospitalNCT06442761
Added 1 new site: Bethesda, Maryland

Added 1 new site: Bethesda, Maryland

eligibility_changeSickle Cell DiseaseJun 9Masonic Cancer Center, University of MinnesotaNCT06872333
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseJun 9Novartis PharmaceuticalsNCT04657822
Added 1 new site: Barranquilla, Colombia

Added 1 new site: Barranquilla, Colombia

sites_addedPulmonary Arterial HypertensionJun 8Merck Sharp & Dohme LLCNCT07218029
Added 1 new site: Dresden, Germany

Added 1 new site: Dresden, Germany

eligibility_changePrimary Sclerosing CholangitisJun 8IpsenNCT05627362
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePrimary Biliary CholangitisJun 8IpsenNCT05627362
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdatePolycystic Kidney DiseaseJun 8PYC TherapeuticsNCT06714006
Target enrollment increased from 116 to 166

Target enrollment increased from 116 to 166

eligibility_changePolycystic Kidney DiseaseJun 8PYC TherapeuticsNCT06714006
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePheochromocytomaJun 8Merck Sharp & Dohme LLCNCT04924075
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeParagangliomaJun 8Merck Sharp & Dohme LLCNCT04924075
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePulmonary Arterial HypertensionJun 8National Institutes of Health Clinical Center (CC)NCT01730092
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPrimary Biliary CholangitisJun 8IpsenNCT06016842
Added 3 new sites: Lodz, Poland, Hat Yai, Thailand, Khon Kaen, Thailand

Added 3 new sites: Lodz, Poland, Hat Yai, Thailand, Khon Kaen, Thailand

eligibility_changePrimary Biliary CholangitisJun 8IpsenNCT06016842
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraJun 8Novartis PharmaceuticalsNCT07340138
Added 1 new site: Kurashiki, Japan

Added 1 new site: Kurashiki, Japan

sites_addedPolycythemia VeraJun 8Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 8Novartis PharmaceuticalsNCT06931691
Added 3 new sites: Hefei, China, Nantong, China, Jinan, China

Added 3 new sites: Hefei, China, Nantong, China, Jinan, China

eligibility_changeNeurofibromatosis Type 1Jun 7National Cancer Institute (NCI)NCT06507748
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateNarcolepsy Type 1Jun 7Hospices Civils de LyonNCT06484348
Target enrollment increased from 60 to 76

Target enrollment increased from 60 to 76

sites_addedNarcolepsy Type 1Jun 7Alkermes, Inc.NCT07455383
Added 8 new sites: Los Angeles, California, Boulder, Colorado, Miami, Florida and 5 more

Added 8 new sites: Los Angeles, California, Boulder, Colorado, Miami, Florida and 5 more

eligibility_changeMyotonic DystrophyJun 7SanofiNCT06844214
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMyotonic DystrophyJun 7Huashan HospitalNCT06101940
Target enrollment increased from 500 to 1000

Target enrollment increased from 500 to 1000

eligibility_changeMyotonic DystrophyJun 7Huashan HospitalNCT06101940
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyotonic DystrophyJun 7Dyne TherapeuticsNCT07486934
Added 1 new site: Columbia, Missouri

Added 1 new site: Columbia, Missouri

sites_addedMyotonic DystrophyJun 7Vertex Pharmaceuticals IncorporatedNCT06926621
Added 1 new site: Maastricht, Netherlands

Added 1 new site: Maastricht, Netherlands

eligibility_changeMyelofibrosisJun 7University of UtahNCT05535764
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisJun 7Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

sites_addedMyelofibrosisJun 7Novartis PharmaceuticalsNCT07340138
Added 1 new site: Kurashiki, Japan

Added 1 new site: Kurashiki, Japan

sites_addedMyelodysplastic SyndromesJun 7Apollo Therapeutics LtdNCT06372717
Added 2 new sites: Cardiff, United Kingdom, Glasgow, United Kingdom

Added 2 new sites: Cardiff, United Kingdom, Glasgow, United Kingdom

sites_addedMyelodysplastic SyndromesJun 7Janssen Research & Development, LLCNCT06788509
Added 2 new sites: Nedlands, Australia, Barcelona, Spain

Added 2 new sites: Nedlands, Australia, Barcelona, Spain

sites_addedMyelodysplastic SyndromesJun 7Novartis PharmaceuticalsNCT06931691
Added 3 new sites: Hefei, China, Nantong, China, Jinan, China

Added 3 new sites: Hefei, China, Nantong, China, Jinan, China

Enrollment UpdateMyelodysplastic SyndromesJun 7Orca Biosystems, Inc.NCT04013685
Target enrollment decreased from 255 to 155

Target enrollment decreased from 255 to 155

eligibility_changeMyelodysplastic SyndromesJun 7Orca Biosystems, Inc.NCT04013685
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJun 7Orca Biosystems, Inc.NCT03802695
Added 2 new sites: New York, New York, Seattle, Washington

Added 2 new sites: New York, New York, Seattle, Washington

sites_addedMyelodysplastic SyndromesJun 7Regeneron PharmaceuticalsNCT07154745
Added 2 new sites: Ribeirão Preto, Brazil, Istanbul, Turkey (Türkiye)

Added 2 new sites: Ribeirão Preto, Brazil, Istanbul, Turkey (Türkiye)

eligibility_changeMyelodysplastic SyndromesJun 7GlaxoSmithKlineNCT06847867
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJun 7Janssen Research & Development, LLCNCT07217587
Added 2 new sites: Fullerton, California, Indianapolis, Indiana

Added 2 new sites: Fullerton, California, Indianapolis, Indiana

sites_addedMyasthenia GravisJun 7argenxNCT07284420
Added 3 new sites: Tampa, Florida, Columbus, Ohio, San Antonio, Texas

Added 3 new sites: Tampa, Florida, Columbus, Ohio, San Antonio, Texas

sites_addedMyasthenia GravisJun 7Novartis PharmaceuticalsNCT06744920
Added 27 new sites: Sacramento, California, Washington D.C., District of Columbia, Maitland, Florida and 24 more

Added 27 new sites: Sacramento, California, Washington D.C., District of Columbia, Maitland, Florida and 24 more

sites_addedMyasthenia GravisJun 7argenxNCT07294170
Added 5 new sites: Tampa, Florida, Columbus, Ohio, Chattanooga, Tennessee and 2 more

Added 5 new sites: Tampa, Florida, Columbus, Ohio, Chattanooga, Tennessee and 2 more

sites_addedMultiple System AtrophyJun 7Teva Branded Pharmaceutical Products R&D LLCNCT06568237
Added 1 new site: Madrid, Spain

Added 1 new site: Madrid, Spain

sites_addedMultiple System AtrophyJun 7Teva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 3 new sites: Marburg, Germany, Sagamihara, Japan, Pamplona, Spain

Added 3 new sites: Marburg, Germany, Sagamihara, Japan, Pamplona, Spain

sites_addedMultiple SclerosisJun 7Reunion Neuroscience IncNCT07002034
Added 2 new sites: Buffalo, New York, Madison, Wisconsin

Added 2 new sites: Buffalo, New York, Madison, Wisconsin

eligibility_changeMultiple SclerosisJun 7UniQure Biopharma B.V.NCT06063850
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMultiple SclerosisJun 7BiogenNCT05688436
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJun 7Zenas BioPharma (USA), LLCNCT07299019
Added 4 new sites: Phoenix, Arizona, Fullerton, California, West Hollywood, California and 1 more

Added 4 new sites: Phoenix, Arizona, Fullerton, California, West Hollywood, California and 1 more

sites_addedMultiple SclerosisJun 7Novartis PharmaceuticalsNCT06846281
Added 2 new sites: Brno, Czechia, Ulm, Germany

Added 2 new sites: Brno, Czechia, Ulm, Germany

sites_addedMultiple SclerosisJun 7Novartis PharmaceuticalsNCT07225504
Added 7 new sites: Seattle, Washington, Leuven, Belgium, Bonheiden, Belgium and 4 more

Added 7 new sites: Seattle, Washington, Leuven, Belgium, Bonheiden, Belgium and 4 more

sites_addedMultiple SclerosisJun 7PhenoNet, Inc.NCT07142291
Added 1 new site: Seville, Spain

Added 1 new site: Seville, Spain

sites_addedMesotheliomaJun 7Perspective TherapeuticsNCT06710756
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

Enrollment UpdateMesotheliomaJun 7Udai KammulaNCT03935893
Target enrollment decreased from 240 to 20

Target enrollment decreased from 240 to 20

sites_addedMesotheliomaJun 7Context Therapeutics Inc.NCT06756035
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

sites_addedMesotheliomaJun 7Alliance for Clinical Trials in OncologyNCT05647265
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

eligibility_changeMesotheliomaJun 7National Cancer Institute (NCI)NCT01950572
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMerkel Cell CarcinomaJun 7Udai KammulaNCT03935893
Target enrollment decreased from 240 to 20

Target enrollment decreased from 240 to 20

sites_addedMerkel Cell CarcinomaJun 7Merck Sharp & Dohme LLCNCT07302347
Added 2 new sites: Nagoya, Japan, Kobe, Japan

Added 2 new sites: Nagoya, Japan, Kobe, Japan

sites_addedMembranous NephropathyJun 7Vertex Pharmaceuticals IncorporatedNCT07204275
Added 5 new sites: Recife, Brazil, Shijiazhuang, China, Dublin, Ireland and 2 more

Added 5 new sites: Recife, Brazil, Shijiazhuang, China, Dublin, Ireland and 2 more

sites_addedLeber Congenital AmaurosisJun 6Laboratoires TheaNCT06891443
Added 2 new sites: Belo Horizonte, Brazil, São Paulo, Brazil

Added 2 new sites: Belo Horizonte, Brazil, São Paulo, Brazil

sites_addedLambert-Eaton Myasthenic SyndromeJun 6argenxNCT06078553
Added 7 new sites: Brussels, Belgium, São Paulo, Brazil, Ottawa, Canada and 4 more

Added 7 new sites: Brussels, Belgium, São Paulo, Brazil, Ottawa, Canada and 4 more

Enrollment UpdateKawasaki DiseaseJun 6Hawaii Pacific HealthNCT01917721
Target enrollment decreased from 50 to 26

Target enrollment decreased from 50 to 26

sites_addedIgG4-Related DiseaseJun 6SanofiNCT07190196
Added 2 new sites: Boston, Massachusetts, Ulm, Germany

Added 2 new sites: Boston, Massachusetts, Ulm, Germany

sites_addedIgA NephropathyJun 6Biohaven Therapeutics Ltd.NCT07054684
Added 3 new sites: Shelby, Michigan, Houston, Texas, Salt Lake City, Utah

Added 3 new sites: Shelby, Michigan, Houston, Texas, Salt Lake City, Utah

sites_removedIgA NephropathyJun 6Novartis PharmaceuticalsNCT04557462
Reduced from 161 to 155 sites

Reduced from 161 to 155 sites

Enrollment UpdateIgA NephropathyJun 6Novartis PharmaceuticalsNCT04557462
Target enrollment decreased from 540 to 448

Target enrollment decreased from 540 to 448

sites_addedIgA NephropathyJun 6Novartis PharmaceuticalsNCT06994845
Added 2 new sites: Toyoake, Japan, Riyadh, Saudi Arabia

Added 2 new sites: Toyoake, Japan, Riyadh, Saudi Arabia

sites_addedIgA NephropathyJun 6TakedaNCT06963827
Added 3 new sites: East Point, Georgia, Saint Joseph, Michigan, Albuquerque, New Mexico

Added 3 new sites: East Point, Georgia, Saint Joseph, Michigan, Albuquerque, New Mexico

sites_addedIgA NephropathyJun 6University of LeicesterNCT04663204
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

Enrollment UpdateIgA NephropathyJun 6University of LeicesterNCT04663204
Target enrollment increased from 12 to 24

Target enrollment increased from 12 to 24

eligibility_changeIgA NephropathyJun 6University of LeicesterNCT04663204
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyJun 6Novartis PharmaceuticalsNCT06858319
Added 3 new sites: Caba, Argentina, Kogarah, Australia, Kuantan, Malaysia

Added 3 new sites: Caba, Argentina, Kogarah, Australia, Kuantan, Malaysia

eligibility_changeIgA NephropathyJun 6Novartis PharmaceuticalsNCT04573478
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJun 6Reunion Neuroscience IncNCT07002034
Added 2 new sites: Buffalo, New York, Madison, Wisconsin

Added 2 new sites: Buffalo, New York, Madison, Wisconsin

sites_addedIdiopathic Pulmonary FibrosisJun 6Contineum TherapeuticsNCT07284459
Added 7 new sites: Westmead, Australia, Vancouver, Canada, Ramat Gan, Israel and 4 more

Added 7 new sites: Westmead, Australia, Vancouver, Canada, Ramat Gan, Israel and 4 more

sites_addedIdiopathic Pulmonary FibrosisJun 6Rein TherapeuticsNCT06968845
Added 5 new sites: Denver, Colorado, Weston, Florida, Chapel Hill, North Carolina and 2 more

Added 5 new sites: Denver, Colorado, Weston, Florida, Chapel Hill, North Carolina and 2 more

phase_changeIdiopathic Pulmonary FibrosisJun 6First Affiliated Hospital of Wenzhou Medical UniversityNCT05828953
Moved from Phase 2, Phase 3 to Phase 2

Moved from Phase 2, Phase 3 to Phase 2

eligibility_changeIdiopathic Pulmonary FibrosisJun 6First Affiliated Hospital of Wenzhou Medical UniversityNCT05828953
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseJun 6Skyhawk Therapeutics, Inc.NCT07378644
Added 5 new sites: CABA, Argentina, CABA, Argentina, CABA, Argentina and 2 more

Added 5 new sites: CABA, Argentina, CABA, Argentina, CABA, Argentina and 2 more

sites_addedHunter SyndromeJun 6JCR Pharmaceuticals Co., Ltd.NCT04573023
Added 2 new sites: Lyon, France, Montpellier, France

Added 2 new sites: Lyon, France, Montpellier, France

eligibility_changeHodgkin LymphomaJun 6Grit BiotechnologyNCT07205315
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 6Hoffmann-La RocheNCT05533775
Added 1 new site: Chengdu, China

Added 1 new site: Chengdu, China

eligibility_changeHodgkin LymphomaJun 6Merck Sharp & Dohme LLCNCT07570173
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 6National Cancer Institute (NCI)NCT06124157
Added 8 new sites: Downey, California, Sacramento, California, Peoria, Illinois and 5 more

Added 8 new sites: Downey, California, Sacramento, California, Peoria, Illinois and 5 more

sites_addedHodgkin LymphomaJun 6National Cancer Institute (NCI)NCT03739814
Added 6 new sites: Nampa, Idaho, Menomonee Falls, Wisconsin, New Berlin, Wisconsin and 3 more

Added 6 new sites: Nampa, Idaho, Menomonee Falls, Wisconsin, New Berlin, Wisconsin and 3 more

sites_addedHodgkin LymphomaJun 6Merck Sharp & Dohme LLCNCT06395103
Added 1 new site: Toronto, Canada

Added 1 new site: Toronto, Canada

eligibility_changeHodgkin LymphomaJun 6Children's Cancer Group, ChinaNCT07168980
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaJun 6National Cancer Institute (NCI)NCT07003295
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

Enrollment UpdateHodgkin LymphomaJun 6AstraZenecaNCT07154901
Target enrollment decreased from 50 to 34

Target enrollment decreased from 50 to 34

sites_addedHodgkin LymphomaJun 6Novartis PharmaceuticalsNCT07422610
Added 1 new site: Candiolo, Italy

Added 1 new site: Candiolo, Italy

Enrollment UpdateHodgkin LymphomaJun 6AstraZenecaNCT06137118
Target enrollment increased from 163 to 236

Target enrollment increased from 163 to 236

Enrollment UpdateHodgkin LymphomaJun 6Stephan Grupp MD PhDNCT05480449
Target enrollment increased from 89 to 115

Target enrollment increased from 89 to 115

eligibility_changeHodgkin LymphomaJun 6Stephan Grupp MD PhDNCT05480449
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaJun 6National Cancer Institute (NCI)NCT05442515
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary AngioedemaJun 6Astria Therapeutics, Inc.NCT07204938
Added 18 new sites: Colorado Springs, Colorado, Wheaton, Maryland, Santo André, Brazil and 15 more

Added 18 new sites: Colorado Springs, Colorado, Wheaton, Maryland, Santo André, Brazil and 15 more

sites_addedHereditary AngioedemaJun 6CSL BehringNCT07001280
Added 4 new sites: Miami, Florida, Louisville, Kentucky, Linz, Austria and 1 more

Added 4 new sites: Miami, Florida, Louisville, Kentucky, Linz, Austria and 1 more

sites_removedGiant Cell ArteritisJun 5Novartis PharmaceuticalsNCT06331312
Reduced from 99 to 83 sites

Reduced from 99 to 83 sites

sites_addedGeographic AtrophyJun 5Novartis PharmaceuticalsNCT07441642
Added 6 new sites: Sofia, Bulgaria, Bonn, Germany, Milan, Italy and 3 more

Added 6 new sites: Sofia, Bulgaria, Bonn, Germany, Milan, Italy and 3 more

sites_addedFocal Segmental GlomerulosclerosisJun 5Akebia TherapeuticsNCT07268638
Added 4 new sites: Los Angeles, California, San Dimas, California, Arvada, Colorado and 1 more

Added 4 new sites: Los Angeles, California, San Dimas, California, Arvada, Colorado and 1 more

sites_addedHemophilia BJun 5PfizerNCT03861273
Added 1 new site: Hamilton, Pennsylvania

Added 1 new site: Hamilton, Pennsylvania

eligibility_changeHemophilia BJun 5CSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia AJun 5OctapharmaNCT03695978
Added 4 new sites: St Louis, Missouri, San Antonio, Texas, Chambéry, France and 1 more

Added 4 new sites: St Louis, Missouri, San Antonio, Texas, Chambéry, France and 1 more

sites_addedHemophilia AJun 5PfizerNCT06703606
Added 3 new sites: Greenville, North Carolina, Greenville, North Carolina, Fatih, Turkey (Türkiye)

Added 3 new sites: Greenville, North Carolina, Greenville, North Carolina, Fatih, Turkey (Türkiye)

sites_addedHemophilia AJun 5PfizerNCT03861273
Added 1 new site: Hamilton, Pennsylvania

Added 1 new site: Hamilton, Pennsylvania

sites_addedGeographic AtrophyJun 5Novartis PharmaceuticalsNCT07441642
Added 3 new sites: Santa Barbara, California, Prague, Czechia, Créteil, France

Added 3 new sites: Santa Barbara, California, Prague, Czechia, Créteil, France

sites_addedGeographic AtrophyJun 5Apellis Pharmaceuticals, Inc.NCT07215390
Added 25 new sites: Gilbert, Arizona, Huntington Beach, California, Oxnard, California and 22 more

Added 25 new sites: Gilbert, Arizona, Huntington Beach, California, Oxnard, California and 22 more

sites_addedGastrointestinal Stromal TumorJun 5Deciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

eligibility_changeFriedreich AtaxiaJun 5BiogenNCT06953583
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeFocal EpilepsyJun 5Supernus Pharmaceuticals, Inc.NCT06798896
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaJun 5Novartis PharmaceuticalsNCT07340138
Added 1 new site: Kurashiki, Japan

Added 1 new site: Kurashiki, Japan

sites_addedEssential ThrombocythemiaJun 5Jubilant Therapeutics Inc.NCT07612280
Added 6 new sites: Sydney, Australia, Sydney, Australia, Adelaide, Australia and 3 more

Added 6 new sites: Sydney, Australia, Sydney, Australia, Adelaide, Australia and 3 more

sites_addedEssential ThrombocythemiaJun 5Novartis PharmaceuticalsNCT07357727
Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

phase_changeCystic FibrosisJun 4Genentech, Inc.NCT06603246
Moved from Phase 1, Phase 2 to Phase 1

Moved from Phase 1, Phase 2 to Phase 1

sites_addedCongenital Adrenal HyperplasiaJun 4Crinetics Pharmaceuticals Inc.NCT07144163
Added 8 new sites: Los Angeles, California, Minneapolis, Minnesota, Milwaukee, Wisconsin and 5 more

Added 8 new sites: Los Angeles, California, Minneapolis, Minnesota, Milwaukee, Wisconsin and 5 more

sites_addedChronic Graft-versus-Host DiseaseJun 4AstraZenecaNCT07082738
Added 5 new sites: Ciudad Autónoma de Bs As, Argentina, Nedlands, Australia, Delhi, India and 2 more

Added 5 new sites: Ciudad Autónoma de Bs As, Argentina, Nedlands, Australia, Delhi, India and 2 more

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 4AstraZenecaNCT07082738
Target enrollment decreased from 1160 to 987

Target enrollment decreased from 1160 to 987

sites_addedChronic Graft-versus-Host DiseaseJun 4BeOne MedicinesNCT07277231
Added 20 new sites: Seattle, Washington, Beijing, China, Guiyang, China and 17 more

Added 20 new sites: Seattle, Washington, Beijing, China, Guiyang, China and 17 more

eligibility_changeChronic Graft-versus-Host DiseaseJun 4Fred Hutchinson Cancer CenterNCT06928662
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 4Wes ElyNCT06631287
Added 1 new site: Brooklyn, New York

Added 1 new site: Brooklyn, New York

eligibility_changeChronic Graft-versus-Host DiseaseJun 4Assistance Publique - Hôpitaux de ParisNCT05685628
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseJun 4Duke UniversityNCT06765642
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseJun 4Incyte CorporationNCT06585774
Added 3 new sites: Chicago, Illinois, Bari, Italy, Catania, Italy

Added 3 new sites: Chicago, Illinois, Bari, Italy, Catania, Italy

sites_addedCholangiocarcinomaJun 4Eli Lilly and CompanyNCT07213791
Added 1 new site: Shanghai, China

Added 1 new site: Shanghai, China

sites_addedCholangiocarcinomaJun 4Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus NordwestNCT07260175
Added 4 new sites: Essen, Germany, Giessen, Germany, Mainz, Germany and 1 more

Added 4 new sites: Essen, Germany, Giessen, Germany, Mainz, Germany and 1 more

sites_addedCholangiocarcinomaJun 4Astellas Pharma Global Development, Inc.NCT07488676
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedCholangiocarcinomaJun 4Context Therapeutics Inc.NCT06756035
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

sites_addedDuchenne Muscular DystrophyJun 4Solid Biosciences Inc.NCT07160634
Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Enrollment UpdateDilated CardiomyopathyJun 4Peerbridge Health, IncNCT06699056
Target enrollment increased from 1500 to 2000

Target enrollment increased from 1500 to 2000

sites_addedDilated CardiomyopathyJun 4Kardigan, Inc.NCT07210723
Added 10 new sites: San Diego, California, Jacksonville, Florida, Chicago, Illinois and 7 more

Added 10 new sites: San Diego, California, Jacksonville, Florida, Chicago, Illinois and 7 more

sites_addedDermatomyositisJun 4Novartis PharmaceuticalsNCT06665256
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedCystic FibrosisJun 4SanofiNCT07547436
Added 2 new sites: Plantation, Florida, DuBois, Pennsylvania

Added 2 new sites: Plantation, Florida, DuBois, Pennsylvania

sites_addedCystic FibrosisJun 4Arkansas Children's Hospital Research InstituteNCT07148739
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

eligibility_changeCystic FibrosisJun 4George SolomonNCT03161808
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCongenital Adrenal HyperplasiaJun 4Crinetics Pharmaceuticals Inc.NCT07159841
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

eligibility_changeCongenital Adrenal HyperplasiaJun 4Crinetics Pharmaceuticals Inc.NCT07159841
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 4Dianthus TherapeuticsNCT06858579
Added 1 new site: Manchester, United Kingdom

Added 1 new site: Manchester, United Kingdom

sites_addedChronic Graft-versus-Host DiseaseJun 4SanofiNCT06082037
Added 3 new sites: Boston, Massachusetts, Paris, France, Tokyo, Japan

Added 3 new sites: Boston, Massachusetts, Paris, France, Tokyo, Japan

sites_addedChronic Graft-versus-Host DiseaseJun 4Novartis PharmaceuticalsNCT06858319
Added 3 new sites: Caba, Argentina, Kogarah, Australia, Kuantan, Malaysia

Added 3 new sites: Caba, Argentina, Kogarah, Australia, Kuantan, Malaysia

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 4Eli Lilly and CompanyNCT06143956
Target enrollment increased from 1217 to 1481

Target enrollment increased from 1217 to 1481

Enrollment UpdateChronic Graft-versus-Host DiseaseJun 4Ausper Biopharma Co., Ltd.NCT07069569
Target enrollment increased from 127 to 200

Target enrollment increased from 127 to 200

sites_addedChronic Graft-versus-Host DiseaseJun 4AstraZenecaNCT06862206
Added 16 new sites: Anning, China, Changzhou, China, Fenyang, China and 13 more

Added 16 new sites: Anning, China, Changzhou, China, Fenyang, China and 13 more

sites_addedChronic Graft-versus-Host DiseaseJun 4Biohaven Therapeutics Ltd.NCT07054684
Added 3 new sites: Shelby, Michigan, Houston, Texas, Salt Lake City, Utah

Added 3 new sites: Shelby, Michigan, Houston, Texas, Salt Lake City, Utah

sites_addedChronic Graft-versus-Host DiseaseJun 4Celldex TherapeuticsNCT07266402
Added 19 new sites: Augsburg, Germany, Berlin, Germany, Buxtehude, Germany and 16 more

Added 19 new sites: Augsburg, Germany, Berlin, Germany, Buxtehude, Germany and 16 more

eligibility_changeCholangiocarcinomaJun 4Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityNCT02757859
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaJun 4Udai KammulaNCT03801083
Target enrollment decreased from 59 to 4

Target enrollment decreased from 59 to 4

sites_removedCharcot-Marie-Tooth DiseaseJun 4Novartis PharmaceuticalsNCT05156281
Reduced from 225 to 223 sites

Reduced from 225 to 223 sites

sites_addedAmyotrophic Lateral SclerosisJun 3Reunion Neuroscience IncNCT07002034
Added 2 new sites: Buffalo, New York, Madison, Wisconsin

Added 2 new sites: Buffalo, New York, Madison, Wisconsin

sites_addedAlagille SyndromeJun 3Mirum Pharmaceuticals, Inc.NCT07290257
Added 2 new sites: Athens, Greece, Coimbra, Portugal

Added 2 new sites: Athens, Greece, Coimbra, Portugal

Enrollment UpdateAlagille SyndromeJun 3Mirum Pharmaceuticals, Inc.NCT07290257
Target enrollment increased from 100 to 230

Target enrollment increased from 100 to 230

eligibility_changeAlagille SyndromeJun 3Mirum Pharmaceuticals, Inc.NCT07290257
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedC3 GlomerulopathyJun 3Novartis PharmaceuticalsNCT07156149
Added 5 new sites: Fukuoka, Japan, Yokohama, Japan, Tosa-gun, Japan and 2 more

Added 5 new sites: Fukuoka, Japan, Yokohama, Japan, Tosa-gun, Japan and 2 more

sites_addedBecker Muscular DystrophyJun 3Solid Biosciences Inc.NCT07160634
Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Enrollment UpdateATTR Amyloidosis (Transthyretin Amyloidosis)Jun 3Steen Hvitfeldt PoulsenNCT06318260
Target enrollment decreased from 35 to 15

Target enrollment decreased from 35 to 15

Enrollment UpdateAplastic AnemiaJun 3National Heart, Lung, and Blood Institute (NHLBI)NCT05998408
Target enrollment decreased from 145 to 13

Target enrollment decreased from 145 to 13

Enrollment UpdateAplastic AnemiaJun 3National Heart, Lung, and Blood Institute (NHLBI)NCT03312400
Target enrollment decreased from 40 to 18

Target enrollment decreased from 40 to 18

sites_addedAmyotrophic Lateral SclerosisJun 3PhenoNet, Inc.NCT07142291
Added 1 new site: Seville, Spain

Added 1 new site: Seville, Spain

sites_addedAmyotrophic Lateral SclerosisJun 3Leigh R. Hochberg, MD, PhD.NCT00912041
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedAlagille SyndromeJun 3IpsenNCT06850038
Added 6 new sites: Los Angeles, California, San Francisco, California, St Louis, Missouri and 3 more

Added 6 new sites: Los Angeles, California, San Francisco, California, St Louis, Missouri and 3 more

sites_addedAchondroplasiaJun 3BioMarin PharmaceuticalNCT07441876
Added 7 new sites: Chicago, Illinois, Baltimore, Maryland, San Antonio, Texas and 4 more

Added 7 new sites: Chicago, Illinois, Baltimore, Maryland, San Antonio, Texas and 4 more

eligibility_changeWaldenström MacroglobulinemiaJun 2AbbVieNCT05753501
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSpinocerebellar AtaxiaJun 2BiogenNCT06953583
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSpinal Muscular AtrophyJun 2Novartis PharmaceuticalsNCT07190300
Added 4 new sites: Westwood, Kansas, Charleston, South Carolina, Porto Alegre, Brazil and 1 more

Added 4 new sites: Westwood, Kansas, Charleston, South Carolina, Porto Alegre, Brazil and 1 more

sites_addedSickle Cell DiseaseJun 2Novartis PharmaceuticalsNCT06546670
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

Enrollment UpdateWild-Type ATTR Cardiac AmyloidosisJun 2Steen Hvitfeldt PoulsenNCT06318260
Target enrollment decreased from 35 to 15

Target enrollment decreased from 35 to 15

sites_addedWaldenström MacroglobulinemiaJun 2AbbVieNCT07387471
Added 2 new sites: Hiroshima, Japan, Nagano, Japan

Added 2 new sites: Hiroshima, Japan, Nagano, Japan

sites_addedVon Willebrand DiseaseJun 2TakedaNCT05582993
Added 7 new sites: East Lansing, Michigan, Las Vegas, Nevada, Narita, Japan and 4 more

Added 7 new sites: East Lansing, Michigan, Las Vegas, Nevada, Narita, Japan and 4 more

sites_addedTurner SyndromeJun 2Ascendis Pharma A/SNCT07221851
Added 1 new site: Sejong, South Korea

Added 1 new site: Sejong, South Korea

sites_addedStargardt DiseaseJun 2AAVantgarde Bio SrlNCT07161544
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedStargardt DiseaseJun 2Astellas Pharma Global Development, Inc.NCT07425574
Added 2 new sites: Sacramento, California, Portland, Oregon

Added 2 new sites: Sacramento, California, Portland, Oregon

sites_addedSpinal Muscular AtrophyJun 2Leigh R. Hochberg, MD, PhD.NCT00912041
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedPulmonary Arterial HypertensionJun 1Liquidia Technologies, Inc.NCT07285655
Added 5 new sites: Tampa, Florida, Indianapolis, Indiana, Giessen, Germany and 2 more

Added 5 new sites: Tampa, Florida, Indianapolis, Indiana, Giessen, Germany and 2 more

sites_addedPulmonary Arterial HypertensionJun 1Massachusetts General HospitalNCT07312227
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

Enrollment UpdatePulmonary Arterial HypertensionJun 1Massachusetts General HospitalNCT07312227
Target enrollment increased from 22 to 27

Target enrollment increased from 22 to 27

eligibility_changePulmonary Arterial HypertensionJun 1Massachusetts General HospitalNCT07312227
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJun 1AllRock Bio, Inc.NCT07175038
Added 2 new sites: Auchenflower, Australia, Pavia, Italy

Added 2 new sites: Auchenflower, Australia, Pavia, Italy

sites_addedPulmonary Arterial HypertensionJun 1Merck Sharp & Dohme LLCNCT07218029
Added 3 new sites: Scottsdale, Arizona, Edmonton, Canada, Giessen, Germany

Added 3 new sites: Scottsdale, Arizona, Edmonton, Canada, Giessen, Germany

sites_addedPulmonary Arterial HypertensionJun 1Insmed IncorporatedNCT07179380
Added 13 new sites: St. Petersburg, Florida, Kansas City, Kansas, Córdoba, Argentina and 10 more

Added 13 new sites: St. Petersburg, Florida, Kansas City, Kansas, Córdoba, Argentina and 10 more

sites_addedPulmonary Arterial HypertensionJun 1Merck Sharp & Dohme LLCNCT06814145
Added 1 new site: La Jolla, California

Added 1 new site: La Jolla, California

sites_addedPulmonary Arterial HypertensionJun 1Regeneron PharmaceuticalsNCT07318597
Added 3 new sites: Riga, Latvia, Namdong-gu, South Korea, Seoul, South Korea

Added 3 new sites: Riga, Latvia, Namdong-gu, South Korea, Seoul, South Korea

sites_addedProgressive Supranuclear PalsyJun 1Novartis PharmaceuticalsNCT07498426
Added 3 new sites: Leipzig, Germany, Berlin, Germany, Stadtroda, Germany

Added 3 new sites: Leipzig, Germany, Berlin, Germany, Stadtroda, Germany

sites_addedPrimary Sclerosing CholangitisJun 1IpsenNCT07387549
Added 1 new site: Chandler, Arizona

Added 1 new site: Chandler, Arizona

sites_addedPrimary Biliary CholangitisJun 1IpsenNCT07387549
Added 1 new site: Chandler, Arizona

Added 1 new site: Chandler, Arizona

sites_addedPrimary Biliary CholangitisJun 1Gilead SciencesNCT06051617
Added 3 new sites: Birmingham, Alabama, Padova, Italy, Chiayi City, Taiwan

Added 3 new sites: Birmingham, Alabama, Padova, Italy, Chiayi City, Taiwan

sites_addedPrimary Biliary CholangitisJun 1IpsenNCT06447168
Added 3 new sites: Fitzroy, Australia, Linz, Austria, Montreal, Canada

Added 3 new sites: Fitzroy, Australia, Linz, Austria, Montreal, Canada

sites_addedPolycythemia VeraJun 1Disc Medicine, IncNCT06985147
Added 1 new site: Cleveland, Ohio

Added 1 new site: Cleveland, Ohio

sites_addedPolycythemia VeraJun 1Prelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedPolycythemia VeraJun 1Incyte CorporationNCT07441694
Added 3 new sites: Montreal, Canada, Kashiwa Chiba, Japan, Toon, Ehime, Japan

Added 3 new sites: Montreal, Canada, Kashiwa Chiba, Japan, Toon, Ehime, Japan

sites_addedPhenylketonuriaJun 1Agios Pharmaceuticals, Inc.NCT07241234
Added 1 new site: Szczecin, Poland

Added 1 new site: Szczecin, Poland

sites_addedPhelan-McDermid SyndromeJun 1Neuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Chicago, Illinois, Lexington, Massachusetts

Added 2 new sites: Chicago, Illinois, Lexington, Massachusetts

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 1Regeneron PharmaceuticalsNCT07154745
Added 2 new sites: Ribeirão Preto, Brazil, Istanbul, Turkey (Türkiye)

Added 2 new sites: Ribeirão Preto, Brazil, Istanbul, Turkey (Türkiye)