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Rare Disease Trial Updates: August 2026

In August 2026, Trial Friend tracked 575 rare disease clinical trial updates across 104 conditions, including 5 FDA-related actions and 84 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

Status ChangeRett SyndromeAug 31stRett Syndrome Research TrustNCT06338267
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrimary Biliary CholangitisAug 31stUmecrine Cognition ABNCT07304843
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrimary Biliary CholangitisAug 31stMirum Pharmaceuticals, Inc.NCT05050136
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisAug 30thGeron CorporationNCT05371964
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyAug 30thFondazione I.R.C.C.S. Istituto Neurologico Carlo BestaNCT06846658
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaAug 30thM.D. Anderson Cancer CenterNCT03007030
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLimb-Girdle Muscular DystrophyAug 29thAskBio IncNCT05230459
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseAug 29thHoffmann-La RocheNCT07246941
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

FDA ApprovalPolycythemia VeraAug 28th
FDA Approves First Drug of Its Kind for Polycythemia Vera, a Rare Blood Disorder

The U.S. Food and Drug Administration today approved Mimrylo (rusfertide), a new treatment for adults with polycythemia vera, a rare blood disorder that causes the body to make too many red blood cells.

FDA ApprovalDermatomyositisAug 27th
FDA Approves First Oral Drug Indicated to Treat Dermatomyositis in Adults

The U.S. Food and Drug Administration has approved Lisraya (brepocitinib) tablets for the treatment of dermatomyositis in adults.

Status ChangeCystic FibrosisAug 27thMassachusetts General HospitalNCT06810167
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Granulomatous DiseaseAug 27thEnsomaNCT06876363
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaAug 27thiOMEDICO AGNCT04510740
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeAug 26thIonis Pharmaceuticals, Inc.NCT05127226
No longer enrolling new participants

No longer enrolling new participants

FDA DesignationStiff Person SyndromeAug 25th
FDA grants orphan drug designation to immune globulin intravenous (human) for Stiff Person Syndrome

The FDA granted orphan drug designation to immune globulin intravenous (human), developed by Kedrion S.p.A, on 2026-08-25 for the treatment of stiff person syndrome. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.

Status ChangeSickle Cell DiseaseAug 25thWashington University School of MedicineNCT04406818
No longer enrolling new participants

No longer enrolling new participants

FDA DesignationGaucher DiseaseAug 24th
FDA grants orphan drug designation to ambroxol for Gaucher Disease

The FDA granted orphan drug designation to ambroxol, developed by LILA Holding AG, on 2026-08-24 for the treatment of Gaucher disease. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.

Status ChangeShort Bowel SyndromeAug 24thJessica SosioNCT06326645
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSarcoidosisAug 24thXentria, Inc.NCT06169397
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRett SyndromeAug 24thTaysha Gene Therapies, Inc.NCT07480564
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyotonic DystrophyAug 23rdVertex Pharmaceuticals IncorporatedNCT06185764
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesAug 23rdFranziska WachterNCT05796570
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyAug 23rdDasher Neuroscience Inc.NCT06848231
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisAug 22ndUniversity of California, Los AngelesNCT05365802
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHypersensitivity PneumonitisAug 22ndUniversity of California, Los AngelesNCT05365802
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGeographic AtrophyAug 21stPerceive Biotherapeutics, Inc.NCT06087458
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGaucher DiseaseAug 21stPrevail TherapeuticsNCT05487599
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFabry DiseaseAug 21stSangamo TherapeuticsNCT05039866
No longer enrolling new participants

No longer enrolling new participants

Status ChangeInherited ErythromelalgiaAug 21stSt. Olavs HospitalNCT04039633
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisAug 20thReCode TherapeuticsNCT06237335
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCushing DiseaseAug 20thCrinetics Pharmaceuticals Inc.NCT05804669
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Aug 19thYale UniversityNCT07398950
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeAmyotrophic Lateral SclerosisAug 19thBiogenNCT07223723
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseAug 18thUniversity of CincinnatiNCT06725550
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeSickle Cell DiseaseAug 18thChildren's Hospital Medical Center, CincinnatiNCT03948867
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionAug 17thGustavo A Heresi, MD, MSNCT06992440
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeMyelofibrosisAug 16thChengdu Zenitar Biomedical Technology Co., LtdNCT06457425
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesAug 16thBarbara Ann Karmanos Cancer InstituteNCT00935090
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisAug 16thCabaletta BioNCT06359041
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyasthenia GravisAug 16thNovartis PharmaceuticalsNCT06517758
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisAug 16thTerry L. WahlsNCT05057676
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisAug 16thHoffmann-La RocheNCT07667322
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMembranous NephropathyAug 16thShoichi Maruyama MD PhDNCT05914155
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLimb-Girdle Muscular DystrophyAug 15thRigshospitalet, DenmarkNCT05102799
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyAug 15thHoffmann-La RocheNCT05797610
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHypoparathyroidismAug 15thAmorphical Ltd.NCT06547151
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomozygous Familial HypercholesterolemiaAug 15thArrowhead PharmaceuticalsNCT07037771
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomocystinuriaAug 15thTravere Therapeutics, Inc.NCT03406611
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHodgkin LymphomaAug 15thUniversity of RochesterNCT03739502
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaAug 15thDivaya BhutaniNCT05346809
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaAug 13thApollo Therapeutics LtdNCT06399757
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisAug 13thRegeneron PharmaceuticalsNCT06693531
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDermatomyositisAug 13thNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NCT00059748
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Inflammatory Demyelinating PolyneuropathyAug 13thSanofiNCT06290128
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseAug 13thNational Cancer Institute (NCI)NCT04044365
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Aug 12thAstraZenecaNCT07338942
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWaldenström MacroglobulinemiaAug 11thBeiGeneNCT07169331
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionAug 10thSheffield Teaching Hospitals NHS Foundation TrustNCT05825417
No longer enrolling new participants

No longer enrolling new participants

Status ChangePhenylketonuriaAug 10thThe First Affiliated Hospital of Bengbu Medical UniversityNCT06061614
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeuromyelitis OpticaAug 9thTianjin Medical University General HospitalNCT05792462
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeurofibromatosis Type 1Aug 9thDavid MillerNCT06515860
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesAug 9thM.D. Anderson Cancer CenterNCT02392572
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisAug 9thJuno Therapeutics, Inc., a Bristol-Myers Squibb CompanyNCT06220201
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisAug 9thHoffmann-La RocheNCT07074886
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisAug 9thABLE Human Motion S.L.NCT07550699
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisAug 9thJuno Therapeutics, Inc., a Bristol-Myers Squibb CompanyNCT06220201
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseAug 8thHoffmann-La RocheNCT07246941
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaAug 8thMacroGenicsNCT05362773
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal EpilepsyAug 7thPraxis Precision MedicinesNCT07505004
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisAug 6thUniversity of Alabama at BirminghamNCT07102043
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeCholangiocarcinomaAug 6thServier Affaires MédicalesNCT05876754
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaAug 6thTyra Biosciences, IncNCT06160752
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalNarcolepsy Type 1Aug 5th
FDA Approves First Drug to Treat the Full Range of Narcolepsy Type 1 Symptoms

The FDA approved Orzeyful (oveporexton) tablets for the treatment of narcolepsy type 1 in adults.

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Aug 5thIntellia TherapeuticsNCT05697861
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeWaldenström MacroglobulinemiaAug 4thAbbVieNCT07387471
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseAug 4thCity of Hope Medical CenterNCT05384756
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseAug 4thUniversity of CincinnatiNCT06725550
No longer enrolling new participants

No longer enrolling new participants

Status ChangeShort Bowel SyndromeAug 3rdZealand PharmaNCT07228403
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaAug 3rdjCyte, IncNCT06912633
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionAug 3rdGossamer Bio USA, Inc.NCT06274801
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangePrimary Biliary CholangitisAug 3rdXijing Hospital of Digestive DiseasesNCT07104201
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraAug 3rdDisc Medicine, IncNCT06985147
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycystic Kidney DiseaseAug 3rdVertex Pharmaceuticals IncorporatedNCT07161037
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyotonic DystrophyAug 2ndHanns LochmullerNCT06809049
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesAug 2ndMacroGenicsNCT05362773
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMastocytosisAug 2ndMacroGenicsNCT05362773
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisAug 1stPeking Union Medical College HospitalNCT06629818
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaAug 1stOssium Health, Inc.NCT05589896
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaAug 1stUniversity of Southern CaliforniaNCT07259304
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedPulmonary Arterial HypertensionAug 31stMerck Sharp & Dohme LLCNCT07218029
Added 1 new site: Hamilton, Canada

Added 1 new site: Hamilton, Canada

sites_addedProgressive Familial Intrahepatic CholestasisAug 31stIpsenNCT07185919
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedProgressive Familial Intrahepatic CholestasisAug 31stIpsenNCT07588880
Added 1 new site: Chongqing, China

Added 1 new site: Chongqing, China

sites_addedPrimary Sclerosing CholangitisAug 31stIpsenNCT07387549
Added 7 new sites: Coronado, California, Englewood, Colorado, Littleton, Colorado and 4 more

Added 7 new sites: Coronado, California, Englewood, Colorado, Littleton, Colorado and 4 more

sites_addedPolycythemia VeraAug 31stNovartis PharmaceuticalsNCT07357727
Added 3 new sites: Jupiter, Florida, Houston, Texas, Kocaeli, Turkey (Türkiye)

Added 3 new sites: Jupiter, Florida, Houston, Texas, Kocaeli, Turkey (Türkiye)

sites_removedPrimary Biliary CholangitisAug 31stUmecrine Cognition ABNCT07304843
Reduced from 40 to 38 sites

Reduced from 40 to 38 sites

Enrollment UpdatePrimary Biliary CholangitisAug 31stMirum Pharmaceuticals, Inc.NCT05050136
Target enrollment increased from 260 to 333

Target enrollment increased from 260 to 333

sites_addedPrader-Willi SyndromeAug 31stHarmony Biosciences Management, Inc.NCT06366464
Added 3 new sites: Baltimore, Maryland, Morrisville, North Carolina, Florence, Italy

Added 3 new sites: Baltimore, Maryland, Morrisville, North Carolina, Florence, Italy

sites_addedPolycythemia VeraAug 31stNovartis PharmaceuticalsNCT07357727
Added 4 new sites: Tainan, Taiwan, Ankara, Turkey (Türkiye), Ankara, Turkey (Türkiye) and 1 more

Added 4 new sites: Tainan, Taiwan, Ankara, Turkey (Türkiye), Ankara, Turkey (Türkiye) and 1 more

sites_addedPolycystic Kidney DiseaseAug 31stUniversity Medical Center GroningenNCT05373264
Added 3 new sites: Graz, Austria, Innsbruck, Austria, Leipzig, Germany

Added 3 new sites: Graz, Austria, Innsbruck, Austria, Leipzig, Germany

sites_addedPheochromocytomaAug 31stChildren's Oncology GroupNCT02402244
Added 4 new sites: Peoria, Illinois, Norton Shores, Michigan, Tulsa, Oklahoma and 1 more

Added 4 new sites: Peoria, Illinois, Norton Shores, Michigan, Tulsa, Oklahoma and 1 more

sites_addedPhelan-McDermid SyndromeAug 31stNeuren Pharmaceuticals LimitedNCT07281079
Added 1 new site: Charlottesville, Virginia

Added 1 new site: Charlottesville, Virginia

sites_addedNoonan SyndromeAug 30thBioMarin PharmaceuticalNCT06668805
Added 5 new sites: Milwaukee, Wisconsin, Toronto, Canada, Montreal, Canada and 2 more

Added 5 new sites: Milwaukee, Wisconsin, Toronto, Canada, Montreal, Canada and 2 more

eligibility_changeNoonan SyndromeAug 30thBioMarin PharmaceuticalNCT06668805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyotonic DystrophyAug 30thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Liège, Belgium

Added 1 new site: Liège, Belgium

sites_addedMyelofibrosisAug 30thNovartis PharmaceuticalsNCT07357727
Added 4 new sites: Tainan, Taiwan, Ankara, Turkey (Türkiye), Ankara, Turkey (Türkiye) and 1 more

Added 4 new sites: Tainan, Taiwan, Ankara, Turkey (Türkiye), Ankara, Turkey (Türkiye) and 1 more

sites_addedMyelofibrosisAug 30thTakedaNCT07623161
Added 2 new sites: Gilbert, Arizona, Durham, North Carolina

Added 2 new sites: Gilbert, Arizona, Durham, North Carolina

sites_removedMyelofibrosisAug 30thGeron CorporationNCT05371964
Reduced from 8 to 6 sites

Reduced from 8 to 6 sites

sites_addedMyelodysplastic SyndromesAug 30thNational Cancer Institute (NCI)NCT05554406
Added 1 new site: Ottawa, Canada

Added 1 new site: Ottawa, Canada

sites_addedMyelodysplastic SyndromesAug 30thPedAL BCU, LLCNCT04726241
Added 3 new sites: Peoria, Illinois, Houston, Texas, San Juan, Puerto Rico

Added 3 new sites: Peoria, Illinois, Houston, Texas, San Juan, Puerto Rico

sites_addedMyelodysplastic SyndromesAug 30thJanssen Research & Development, LLCNCT06788509
Added 2 new sites: Copenhagen, Denmark, Odense C, Denmark

Added 2 new sites: Copenhagen, Denmark, Odense C, Denmark

sites_addedMyasthenia GravisAug 30thDianthus TherapeuticsNCT07647510
Added 2 new sites: Boca Raton, Florida, Houston, Texas

Added 2 new sites: Boca Raton, Florida, Houston, Texas

sites_addedMyasthenia GravisAug 30thMiriam FreimerNCT07215949
Added 2 new sites: Tampa, Florida, Kansas City, Kansas

Added 2 new sites: Tampa, Florida, Kansas City, Kansas

sites_addedMyasthenia GravisAug 30thUCB Biopharma SRLNCT07463521
Added 5 new sites: Scottsdale, Arizona, O'Fallon, Illinois, Greenfield, Wisconsin and 2 more

Added 5 new sites: Scottsdale, Arizona, O'Fallon, Illinois, Greenfield, Wisconsin and 2 more

sites_addedMultiple SclerosisAug 30thNovartis PharmaceuticalsNCT06846281
Added 1 new site: Joinville, Brazil

Added 1 new site: Joinville, Brazil

eligibility_changeMultiple SclerosisAug 30thNovartis PharmaceuticalsNCT06846281
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 30thZenas BioPharma (USA), LLCNCT07299019
Added 3 new sites: Cordova, Tennessee, Tallinn, Estonia, Poznan, Poland

Added 3 new sites: Cordova, Tennessee, Tallinn, Estonia, Poznan, Poland

eligibility_changeMultiple SclerosisAug 30thShirley Ryan AbilityLabNCT07132775
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 30thZenas BioPharma (USA), LLCNCT07067463
Added 6 new sites: Sofia, Bulgaria, Tbilisi, Georgia, Niš, Serbia and 3 more

Added 6 new sites: Sofia, Bulgaria, Tbilisi, Georgia, Niš, Serbia and 3 more

Enrollment UpdateMesotheliomaAug 30thM.D. Anderson Cancer CenterNCT03007030
Target enrollment decreased from 55 to 25

Target enrollment decreased from 55 to 25

sites_addedMembranous NephropathyAug 30thBiogenNCT06962800
Added 1 new site: Shanghai, China

Added 1 new site: Shanghai, China

eligibility_changeLymphangioleiomyomatosisAug 29thNational Heart, Lung, and Blood Institute (NHLBI)NCT00001465
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisAug 29thPureTechNCT07284602
Added 1 new site: Reno, Nevada

Added 1 new site: Reno, Nevada

sites_addedIdiopathic Pulmonary FibrosisAug 29thContineum TherapeuticsNCT07284459
Added 1 new site: Songpa-dong, South Korea

Added 1 new site: Songpa-dong, South Korea

sites_addedIdiopathic Pulmonary FibrosisAug 29thAbbVieNCT07230288
Added 43 new sites: Birmingham, Alabama, Kissimmee, Florida, Ann Arbor, Michigan and 40 more

Added 43 new sites: Birmingham, Alabama, Kissimmee, Florida, Ann Arbor, Michigan and 40 more

sites_addedIdiopathic Pulmonary FibrosisAug 29thAvalyn Pharma Inc.NCT06951217
Added 4 new sites: Seattle, Washington, Buenos Aires, Argentina, Buenos Aires, Argentina and 1 more

Added 4 new sites: Seattle, Washington, Buenos Aires, Argentina, Buenos Aires, Argentina and 1 more

sites_addedHypoparathyroidismAug 29thMBX BiosciencesNCT07699471
Added 8 new sites: Los Angeles, California, Las Vegas, Nevada, Reno, Nevada and 5 more

Added 8 new sites: Los Angeles, California, Las Vegas, Nevada, Reno, Nevada and 5 more

sites_addedHypoparathyroidismAug 29thAscendis Pharma Bone Diseases A/SNCT07081997
Added 4 new sites: Chicago, Illinois, Minneota, Minnesota, Greenville, North Carolina and 1 more

Added 4 new sites: Chicago, Illinois, Minneota, Minnesota, Greenville, North Carolina and 1 more

sites_addedGranulomatosis with PolyangiitisAug 28thUniversity of PennsylvaniaNCT00315380
Added 2 new sites: Cambridge, United Kingdom, London, United Kingdom

Added 2 new sites: Cambridge, United Kingdom, London, United Kingdom

sites_addedHemophilia BAug 28thCSL BehringNCT06003387
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedGraves’ DiseaseAug 28thMerida BiosciencesNCT07305818
Added 6 new sites: Torrance, California, Morehead City, North Carolina, DeSoto, Texas and 3 more

Added 6 new sites: Torrance, California, Morehead City, North Carolina, DeSoto, Texas and 3 more

sites_addedGraves’ DiseaseAug 28thImmunovant Sciences GmbHNCT07018323
Added 3 new sites: Bergamo, Italy, Rome, Italy, Madrid, Spain

Added 3 new sites: Bergamo, Italy, Rome, Italy, Madrid, Spain

sites_addedGeographic AtrophyAug 28thONL TherapeuticsNCT06659445
Added 60 new sites: Linz, Austria, Vienna, Austria, Vienna, Austria and 57 more

Added 60 new sites: Linz, Austria, Vienna, Austria, Vienna, Austria and 57 more

eligibility_changeGeographic AtrophyAug 28thWashington University School of MedicineNCT07174687
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyAug 28thi-Lumen Scientific AUS PTY LTDNCT06662162
Added 1 new site: Beachwood, Ohio

Added 1 new site: Beachwood, Ohio

eligibility_changeFocal Segmental GlomerulosclerosisAug 28thNational Human Genome Research Institute (NHGRI)NCT06664814
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeFamilial Chylomicronemia SyndromeAug 28thIonis Pharmaceuticals, Inc.NCT07727538
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaAug 28thNovartis PharmaceuticalsNCT07357727
Added 4 new sites: Tainan, Taiwan, Ankara, Turkey (Türkiye), Ankara, Turkey (Türkiye) and 1 more

Added 4 new sites: Tainan, Taiwan, Ankara, Turkey (Türkiye), Ankara, Turkey (Türkiye) and 1 more

eligibility_changeChronic Graft-versus-Host DiseaseAug 27thFred Hutchinson Cancer CenterNCT07011810
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDuchenne Muscular DystrophyAug 27thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Liège, Belgium

Added 1 new site: Liège, Belgium

sites_addedDermatomyositisAug 27thPfizerNCT06698796
Added 3 new sites: Frisco, Texas, Mendoza, Argentina, Gurugram, India

Added 3 new sites: Frisco, Texas, Mendoza, Argentina, Gurugram, India

sites_addedCushing DiseaseAug 27thH. Lundbeck A/SNCT06471829
Added 16 new sites: Ann Arbor, Michigan, St Louis, Missouri, Bron, France and 13 more

Added 16 new sites: Ann Arbor, Michigan, St Louis, Missouri, Bron, France and 13 more

sites_removedCushing DiseaseAug 27thCorcept TherapeuticsNCT03604198
Reduced from 48 to 44 sites

Reduced from 48 to 44 sites

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 27thargenxNCT07091630
Added 6 new sites: Philadelphia, Pennsylvania, Brasília, Brazil, Porto Alegre, Brazil and 3 more

Added 6 new sites: Philadelphia, Pennsylvania, Brasília, Brazil, Porto Alegre, Brazil and 3 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 27thSanofiNCT06290141
Added 6 new sites: New York, New York, Beaumont, Texas, Schwäbisch Hall, Germany and 3 more

Added 6 new sites: New York, New York, Beaumont, Texas, Schwäbisch Hall, Germany and 3 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 27thargenxNCT06920004
Added 7 new sites: Philadelphia, Pennsylvania, Philadelphia, Pennsylvania, São Paulo, Brazil and 4 more

Added 7 new sites: Philadelphia, Pennsylvania, Philadelphia, Pennsylvania, São Paulo, Brazil and 4 more

eligibility_changeChronic Granulomatous DiseaseAug 27thEnsomaNCT06876363
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateChronic Graft-versus-Host DiseaseAug 27thChia Tai Tianqing Pharmaceutical Group Co., Ltd.NCT06300320
Target enrollment increased from 52 to 65

Target enrollment increased from 52 to 65

sites_removedCholangiocarcinomaAug 27thHoffmann-La RocheNCT02568267
Reduced from 130 to 120 sites

Reduced from 130 to 120 sites

sites_addedCentral Diabetes InsipidusAug 27thChildren's Oncology GroupNCT06368817
Added 1 new site: Knoxville, Tennessee

Added 1 new site: Knoxville, Tennessee

eligibility_changeAmyotrophic Lateral SclerosisAug 26thAC Immune SANCT06891716
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedBecker Muscular DystrophyAug 26thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Liège, Belgium

Added 1 new site: Liège, Belgium

sites_addedAlpha-1 Antitrypsin DeficiencyAug 26thAIRNA CorporationNCT07431112
Added 2 new sites: Matosinhos Municipality, Portugal, Barcelona, Spain

Added 2 new sites: Matosinhos Municipality, Portugal, Barcelona, Spain

sites_addedAlpha-1 Antitrypsin DeficiencyAug 26thTessera Therapeutics, Inc.NCT07227207
Added 1 new site: Nottingham, United Kingdom

Added 1 new site: Nottingham, United Kingdom

sites_addedAcromegalyAug 26thDebiopharm International SANCT06930625
Added 1 new site: Krakow, Poland

Added 1 new site: Krakow, Poland

sites_addedAchondroplasiaAug 26thBioMarin PharmaceuticalNCT07441876
Added 6 new sites: Hokkaido, Japan, Tokyo, Japan, Glasgow, United Kingdom and 3 more

Added 6 new sites: Hokkaido, Japan, Tokyo, Japan, Glasgow, United Kingdom and 3 more

sites_addedStargardt DiseaseAug 25thAstellas Pharma Global Development, Inc.NCT07425574
Added 2 new sites: Miami, Florida, Cincinnati, Ohio

Added 2 new sites: Miami, Florida, Cincinnati, Ohio

sites_addedWild-Type ATTR Cardiac AmyloidosisAug 25thAstraZenecaNCT06465810
Added 4 new sites: Baltimore, Maryland, Houston, Texas, Montreal, Canada and 1 more

Added 4 new sites: Baltimore, Maryland, Houston, Texas, Montreal, Canada and 1 more

sites_addedWaldenström MacroglobulinemiaAug 25thBeOne MedicinesNCT05006716
Added 1 new site: Norwich, United Kingdom

Added 1 new site: Norwich, United Kingdom

sites_removedWaldenström MacroglobulinemiaAug 25thFondazione Italiana Linfomi - ETSNCT06441214
Reduced from 28 to 23 sites

Reduced from 28 to 23 sites

sites_addedWaldenström MacroglobulinemiaAug 25thEli Lilly and CompanyNCT07101328
Added 1 new site: Tokyo, Japan

Added 1 new site: Tokyo, Japan

sites_addedTurner SyndromeAug 25thAscendis Pharma A/SNCT07221851
Added 4 new sites: Montpellier, France, Düsseldorf, Germany, Trieste, Italy and 1 more

Added 4 new sites: Montpellier, France, Düsseldorf, Germany, Trieste, Italy and 1 more

eligibility_changeTuberous SclerosisAug 25thNational Heart, Lung, and Blood Institute (NHLBI)NCT00001465
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedThalassemiaAug 25thBristol-Myers SquibbNCT07215975
Added 12 new sites: Al Jahra, Kuwait, Andalusia, Kuwait, Kuwait City, Kuwait and 9 more

Added 12 new sites: Al Jahra, Kuwait, Andalusia, Kuwait, Kuwait City, Kuwait and 9 more

sites_addedSystemic Juvenile Idiopathic ArthritisAug 25thNovartis PharmaceuticalsNCT07203001
Added 1 new site: Salt Lake City, Utah

Added 1 new site: Salt Lake City, Utah

Enrollment UpdateSpinal Muscular AtrophyAug 25thBiogenNCT06555419
Target enrollment increased from 58 to 78

Target enrollment increased from 58 to 78

eligibility_changeSpinal Muscular AtrophyAug 25thBiogenNCT06555419
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSpinal Muscular AtrophyAug 25thAlcyone Therapeutics, IncNCT05866419
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSjögren's SyndromeAug 25thNovartis PharmaceuticalsNCT05985915
Added 10 new sites: The Woodlands, Texas, Burgas, Bulgaria, Angers, France and 7 more

Added 10 new sites: The Woodlands, Texas, Burgas, Bulgaria, Angers, France and 7 more

sites_removedSickle Cell DiseaseAug 25thAgios Pharmaceuticals, Inc.NCT06924970
Reduced from 30 to 20 sites

Reduced from 30 to 20 sites

eligibility_changeSickle Cell DiseaseAug 25thNational Heart, Lung, and Blood Institute (NHLBI)NCT05213572
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseAug 25thNovartis PharmaceuticalsNCT06439082
Added 2 new sites: Chicago, Illinois, Philadelphia, Pennsylvania

Added 2 new sites: Chicago, Illinois, Philadelphia, Pennsylvania

Enrollment UpdateSarcoidosisAug 24thXentria, Inc.NCT06169397
Target enrollment decreased from 94 to 31

Target enrollment decreased from 94 to 31

phase_changeSarcoidosisAug 24thPriovant Therapeutics, Inc.NCT06978725
Moved from Phase 2 to Phase 2, Phase 3

Moved from Phase 2 to Phase 2, Phase 3

Enrollment UpdateSarcoidosisAug 24thPriovant Therapeutics, Inc.NCT06978725
Target enrollment increased from 28 to 168

Target enrollment increased from 28 to 168

eligibility_changeSarcoidosisAug 24thPriovant Therapeutics, Inc.NCT06978725
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedRett SyndromeAug 24thTaysha Gene Therapies, Inc.NCT07480564
Added 2 new sites: Birmingham, Alabama, Chicago, Illinois

Added 2 new sites: Birmingham, Alabama, Chicago, Illinois

Enrollment UpdateRett SyndromeAug 24thTaysha Gene Therapies, Inc.NCT07480564
Target enrollment increased from 3 to 4

Target enrollment increased from 3 to 4

sites_addedPulmonary Arterial HypertensionAug 24thDuke UniversityNCT04278404
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedPulmonary Arterial HypertensionAug 24thMerck Sharp & Dohme LLCNCT07218029
Added 3 new sites: São Paulo, Brazil, Munich, Germany, Kfar Saba, Israel

Added 3 new sites: São Paulo, Brazil, Munich, Germany, Kfar Saba, Israel

sites_addedPulmonary Arterial HypertensionAug 24thRegeneron PharmaceuticalsNCT07318597
Added 2 new sites: Boston, Massachusetts, Toledo, Spain

Added 2 new sites: Boston, Massachusetts, Toledo, Spain

sites_addedPulmonary Arterial HypertensionAug 24thInsmed IncorporatedNCT07481981
Added 1 new site: Okayama, Japan

Added 1 new site: Okayama, Japan

sites_addedProgressive Supranuclear PalsyAug 24thAdam BoxerNCT07173803
Added 7 new sites: Palo Alto, California, San Diego, California, Boston, Massachusetts and 4 more

Added 7 new sites: Palo Alto, California, San Diego, California, Boston, Massachusetts and 4 more

sites_addedPrimary Biliary CholangitisAug 24thHan YingNCT06174402
Added 2 new sites: Shenyang, China, Tianjin, China

Added 2 new sites: Shenyang, China, Tianjin, China

eligibility_changePrimary Biliary CholangitisAug 24thHan YingNCT06174402
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPrader-Willi SyndromeAug 24thSoleno Therapeutics, Inc.NCT07450664
Added 1 new site: Salt Lake City, Utah

Added 1 new site: Salt Lake City, Utah

sites_addedPolycythemia VeraAug 24thNovartis PharmaceuticalsNCT07357727
Added 18 new sites: New Haven, Connecticut, Atlanta, Georgia, CABA, Argentina and 15 more

Added 18 new sites: New Haven, Connecticut, Atlanta, Georgia, CABA, Argentina and 15 more

sites_addedPolycythemia VeraAug 24thOno Pharmaceutical Co., Ltd.NCT07429266
Added 2 new sites: Grand Rapids, Michigan, West Perth, Australia

Added 2 new sites: Grand Rapids, Michigan, West Perth, Australia

sites_addedPhenylketonuriaAug 24thMaze TherapeuticsNCT07694440
Added 4 new sites: Oakland, California, Palo Alto, California, Lexington, Kentucky and 1 more

Added 4 new sites: Oakland, California, Palo Alto, California, Lexington, Kentucky and 1 more

sites_addedParoxysmal Nocturnal HemoglobinuriaAug 24thHoffmann-La RocheNCT04434092
Added 2 new sites: George Town, Malaysia, Kuantan, Malaysia

Added 2 new sites: George Town, Malaysia, Kuantan, Malaysia

sites_addedNoonan SyndromeAug 23rdAscendis Pharma A/SNCT07221851
Added 4 new sites: Montpellier, France, Düsseldorf, Germany, Trieste, Italy and 1 more

Added 4 new sites: Montpellier, France, Düsseldorf, Germany, Trieste, Italy and 1 more

sites_addedNarcolepsy Type 1Aug 23rdTakedaNCT07646678
Added 12 new sites: Brandon, Florida, Miami, Florida, Atlanta, Georgia and 9 more

Added 12 new sites: Brandon, Florida, Miami, Florida, Atlanta, Georgia and 9 more

sites_addedMyotonic DystrophyAug 23rdDyne TherapeuticsNCT07486934
Added 1 new site: Nijmegen, Netherlands

Added 1 new site: Nijmegen, Netherlands

sites_addedMyotonic DystrophyAug 23rdAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Leuven, Belgium

Added 1 new site: Leuven, Belgium

sites_addedMyelofibrosisAug 23rdNovartis PharmaceuticalsNCT07357727
Added 18 new sites: New Haven, Connecticut, Atlanta, Georgia, CABA, Argentina and 15 more

Added 18 new sites: New Haven, Connecticut, Atlanta, Georgia, CABA, Argentina and 15 more

Enrollment UpdateMyelodysplastic SyndromesAug 23rdFranziska WachterNCT05796570
Target enrollment decreased from 37 to 29

Target enrollment decreased from 37 to 29

sites_addedMyelodysplastic SyndromesAug 23rdTakedaNCT04419649
Added 6 new sites: Hot Springs, Arkansas, Bethesda, Maryland, Hackensack, New Jersey and 3 more

Added 6 new sites: Hot Springs, Arkansas, Bethesda, Maryland, Hackensack, New Jersey and 3 more

eligibility_changeMyelodysplastic SyndromesAug 23rdVanderbilt-Ingram Cancer CenterNCT06399640
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesAug 23rdBeijing InnoCare Pharma Tech Co., Ltd.NCT06656494
Added 4 new sites: Orange, California, Tampa, Florida, Chapel Hill, North Carolina and 1 more

Added 4 new sites: Orange, California, Tampa, Florida, Chapel Hill, North Carolina and 1 more

sites_addedMyelodysplastic SyndromesAug 23rdNational Cancer Institute (NCI)NCT06577441
Added 1 new site: Lebanon, New Hampshire

Added 1 new site: Lebanon, New Hampshire

sites_removedMyelodysplastic SyndromesAug 23rdTakedaNCT06499285
Reduced from 179 to 177 sites

Reduced from 179 to 177 sites

sites_addedMyasthenia GravisAug 23rdargenxNCT07294170
Added 3 new sites: Göttingen, Germany, Würzburg, Germany, Hanamaki, Japan

Added 3 new sites: Göttingen, Germany, Würzburg, Germany, Hanamaki, Japan

sites_addedMyasthenia GravisAug 23rdargenxNCT07673627
Added 1 new site: Amherst, New York

Added 1 new site: Amherst, New York

sites_addedMyasthenia GravisAug 23rdTG Therapeutics, Inc.NCT07673744
Added 2 new sites: Farmington Hills, Michigan, New Bern, North Carolina

Added 2 new sites: Farmington Hills, Michigan, New Bern, North Carolina

sites_addedMyasthenia GravisAug 23rdargenxNCT07284420
Added 3 new sites: Göttingen, Germany, Würzburg, Germany, Hanamaki, Japan

Added 3 new sites: Göttingen, Germany, Würzburg, Germany, Hanamaki, Japan

eligibility_changeMultiple SclerosisAug 23rdLuzerner KantonsspitalNCT07256704
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 23rdNovartis PharmaceuticalsNCT05147220
Added 6 new sites: Santiago del Estero, Argentina, Zagreb, Croatia, Dublin, Ireland and 3 more

Added 6 new sites: Santiago del Estero, Argentina, Zagreb, Croatia, Dublin, Ireland and 3 more

sites_removedMultiple SclerosisAug 23rdGenentech, Inc.NCT07609719
Reduced from 11 to 1 sites

Reduced from 11 to 1 sites

eligibility_changeMultiple SclerosisAug 23rdGenentech, Inc.NCT07609719
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 23rdHoffmann-La RocheNCT07282574
Added 5 new sites: Camperdown, Australia, Mannheim, Germany, Essen, Germany and 2 more

Added 5 new sites: Camperdown, Australia, Mannheim, Germany, Essen, Germany and 2 more

sites_addedMultiple SclerosisAug 23rdZenas BioPharma (USA), LLCNCT07299019
Added 4 new sites: Zagreb, Croatia, Bydgoszcz, Poland, Poznan, Poland and 1 more

Added 4 new sites: Zagreb, Croatia, Bydgoszcz, Poland, Poznan, Poland and 1 more

sites_addedMultiple SclerosisAug 23rdZenas BioPharma (USA), LLCNCT07067463
Added 1 new site: Bydgoszcz, Poland

Added 1 new site: Bydgoszcz, Poland

sites_addedMultiple SclerosisAug 23rdCelgeneNCT06782490
Added 2 new sites: Philadelphia, Pennsylvania, Lewisville, Texas

Added 2 new sites: Philadelphia, Pennsylvania, Lewisville, Texas

sites_addedMultiple SclerosisAug 23rdNovartis PharmaceuticalsNCT05156281
Added 8 new sites: Zagreb, Croatia, Vukovar, Croatia, Chiba, Japan and 5 more

Added 8 new sites: Zagreb, Croatia, Vukovar, Croatia, Chiba, Japan and 5 more

sites_addedMultiple SclerosisAug 23rdTG Therapeutics, Inc.NCT05877963
Added 3 new sites: Atlanta, Georgia, Atlanta, Georgia, Cincinnati, Ohio

Added 3 new sites: Atlanta, Georgia, Atlanta, Georgia, Cincinnati, Ohio

eligibility_changeMerkel Cell CarcinomaAug 23rdM.D. Anderson Cancer CenterNCT02479698
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMerkel Cell CarcinomaAug 23rdAstraZenecaNCT07115043
Target enrollment increased from 60 to 120

Target enrollment increased from 60 to 120

eligibility_changeMerkel Cell CarcinomaAug 23rdMayo ClinicNCT05269381
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyAug 23rdBiogenNCT06962800
Added 7 new sites: São Paulo, Brazil, Vancouver, Canada, Shenzhen, China and 4 more

Added 7 new sites: São Paulo, Brazil, Vancouver, Canada, Shenzhen, China and 4 more

eligibility_changeLymphangioleiomyomatosisAug 22ndNational Heart, Lung, and Blood Institute (NHLBI)NCT00001465
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLight Chain AmyloidosisAug 22ndRegeneron PharmaceuticalsNCT06292780
Added 1 new site: Taipei, Taiwan

Added 1 new site: Taipei, Taiwan

sites_addedLight Chain AmyloidosisAug 22ndDana-Farber Cancer InstituteNCT06569147
Added 4 new sites: New Haven, Connecticut, Boston, Massachusetts, Boston, Massachusetts and 1 more

Added 4 new sites: New Haven, Connecticut, Boston, Massachusetts, Boston, Massachusetts and 1 more

sites_addedLennox-Gastaut SyndromeAug 22ndEpygenixNCT05066217
Added 33 new sites: Orlando, Florida, Beijing, China, Beijing, China and 30 more

Added 33 new sites: Orlando, Florida, Beijing, China, Beijing, China and 30 more

sites_addedKawasaki DiseaseAug 22ndDuke UniversityNCT04278404
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedIgA NephropathyAug 22ndJade Biosciences, Inc.NCT07541287
Added 5 new sites: Concord, Australia, Anyang-si, South Korea, Seongnam-si, South Korea and 2 more

Added 5 new sites: Concord, Australia, Anyang-si, South Korea, Seongnam-si, South Korea and 2 more

sites_addedIgA NephropathyAug 22ndBiogenNCT06935357
Added 2 new sites: Hong Kong, China, Hong Kong, China

Added 2 new sites: Hong Kong, China, Hong Kong, China

sites_addedIgA NephropathyAug 22ndNovartis PharmaceuticalsNCT06858319
Added 5 new sites: Iowa City, Iowa, Gosford, Australia, Monterrey, Mexico and 2 more

Added 5 new sites: Iowa City, Iowa, Gosford, Australia, Monterrey, Mexico and 2 more

sites_removedIdiopathic Pulmonary FibrosisAug 22ndVicore Pharma ABNCT06588686
Reduced from 111 to 104 sites

Reduced from 111 to 104 sites

sites_addedIdiopathic Pulmonary FibrosisAug 22ndContineum TherapeuticsNCT07284459
Added 4 new sites: Mar del Plata, Argentina, Quilmes, Argentina, Québec, Canada and 1 more

Added 4 new sites: Mar del Plata, Argentina, Quilmes, Argentina, Québec, Canada and 1 more

sites_addedHuntington DiseaseAug 22ndSkyhawk Therapeutics, Inc.NCT07378644
Added 4 new sites: Englewood, Colorado, São Paulo, Brazil, Santiago, Chile and 1 more

Added 4 new sites: Englewood, Colorado, São Paulo, Brazil, Santiago, Chile and 1 more

sites_addedHodgkin LymphomaAug 22ndUniversity of WashingtonNCT06745076
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

eligibility_changeHodgkin LymphomaAug 22ndNational Cancer Institute (NCI)NCT01896999
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary Hemorrhagic TelangiectasiaAug 22ndAtavistik Bio, IncNCT07601425
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedGraves’ DiseaseAug 21stargenxNCT07596849
Added 5 new sites: Surrey, Canada, Kawasaki, Japan, Kitakyushu, Japan and 2 more

Added 5 new sites: Surrey, Canada, Kawasaki, Japan, Kitakyushu, Japan and 2 more

sites_addedGraves’ DiseaseAug 21stBiogenNCT07722546
Added 2 new sites: Los Angeles, California, Asheville, North Carolina

Added 2 new sites: Los Angeles, California, Asheville, North Carolina

sites_addedEssential ThrombocythemiaAug 21stNovartis PharmaceuticalsNCT07357727
Added 2 new sites: Shanghai, China, Kuching, Malaysia

Added 2 new sites: Shanghai, China, Kuching, Malaysia

sites_addedHemophilia BAug 21stAmerican Thrombosis and Hemostasis NetworkNCT04398628
Added 5 new sites: Miami, Florida, Tampa, Florida, Columbus, Ohio and 2 more

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

sites_addedHemophilia AAug 21stAmerican Thrombosis and Hemostasis NetworkNCT04398628
Added 5 new sites: Miami, Florida, Tampa, Florida, Columbus, Ohio and 2 more

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

sites_addedAug 21stDenali Therapeutics Inc.NCT07354724
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedGiant Cell ArteritisAug 21stUniversity of PennsylvaniaNCT04474847
Added 1 new site: Kirchheim unter Teck, Germany

Added 1 new site: Kirchheim unter Teck, Germany

eligibility_changeGeographic AtrophyAug 21stNovartis PharmaceuticalsNCT07441642
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyAug 21stPerceive Biotherapeutics, Inc.NCT06087458
Added 8 new sites: Poway, California, Boston, Massachusetts, St Louis, Missouri and 5 more

Added 8 new sites: Poway, California, Boston, Massachusetts, St Louis, Missouri and 5 more

sites_removedGaucher DiseaseAug 21stPrevail TherapeuticsNCT05487599
Reduced from 9 to 5 sites

Reduced from 9 to 5 sites

Enrollment UpdateGaucher DiseaseAug 21stPrevail TherapeuticsNCT05487599
Target enrollment decreased from 15 to 8

Target enrollment decreased from 15 to 8

sites_addedGaucher DiseaseAug 21stPrevail TherapeuticsNCT04411654
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedFocal Segmental GlomerulosclerosisAug 21stBoehringer IngelheimNCT07220083
Added 5 new sites: Olympia Fields, Illinois, Dallas, Texas, San Miguel de Tucumán, Argentina and 2 more

Added 5 new sites: Olympia Fields, Illinois, Dallas, Texas, San Miguel de Tucumán, Argentina and 2 more

sites_addedFamilial Chylomicronemia SyndromeAug 21stIonis Pharmaceuticals, Inc.NCT07727538
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

Enrollment UpdateFabry DiseaseAug 21stSangamo TherapeuticsNCT05039866
Target enrollment decreased from 48 to 32

Target enrollment decreased from 48 to 32

sites_addedEssential ThrombocythemiaAug 21stNovartis PharmaceuticalsNCT07357727
Added 16 new sites: New Haven, Connecticut, Atlanta, Georgia, CABA, Argentina and 13 more

Added 16 new sites: New Haven, Connecticut, Atlanta, Georgia, CABA, Argentina and 13 more

Enrollment UpdateInherited ErythromelalgiaAug 21stSt. Olavs HospitalNCT04039633
Target enrollment decreased from 24 to 6

Target enrollment decreased from 24 to 6

sites_addedEosinophilic EsophagitisAug 20thRegeneron PharmaceuticalsNCT07112378
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

eligibility_changeDilated CardiomyopathyAug 20thAffinia TherapeuticsNCT07426419
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDuchenne Muscular DystrophyAug 20thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Leuven, Belgium

Added 1 new site: Leuven, Belgium

sites_addedDuchenne Muscular DystrophyAug 20thUniversity of RochesterNCT07092540
Added 3 new sites: Stony Brook, New York, Cincinnati, Ohio, Columbus, Ohio

Added 3 new sites: Stony Brook, New York, Cincinnati, Ohio, Columbus, Ohio

sites_addedDilated CardiomyopathyAug 20thAlexion Pharmaceuticals, Inc.NCT07218887
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

eligibility_changeDilated CardiomyopathyAug 20thAlexion Pharmaceuticals, Inc.NCT07218887
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateDermatomyositisAug 20thNational Institute of Environmental Health Sciences (NIEHS)NCT07111065
Target enrollment increased from 300 to 3000

Target enrollment increased from 300 to 3000

sites_addedCystic FibrosisAug 20thUniversity of PennsylvaniaNCT06950892
Added 1 new site: Spokane, Washington

Added 1 new site: Spokane, Washington

Enrollment UpdateCystic FibrosisAug 20thReCode TherapeuticsNCT06237335
Target enrollment decreased from 192 to 128

Target enrollment decreased from 192 to 128

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 20thDianthus TherapeuticsNCT06858579
Added 7 new sites: Buenos Aires, Argentina, Monza, Italy, Salerno, Italy and 4 more

Added 7 new sites: Buenos Aires, Argentina, Monza, Italy, Salerno, Italy and 4 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 20thKite, A Gilead CompanyNCT07304154
Added 2 new sites: Newport Beach, California, Montreal, Canada

Added 2 new sites: Newport Beach, California, Montreal, Canada

eligibility_changeChronic Granulomatous DiseaseAug 20thNational Institute of Allergy and Infectious Diseases (NIAID)NCT07113743
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseAug 20thIncyte CorporationNCT06585774
Added 1 new site: Duarte, California

Added 1 new site: Duarte, California

eligibility_changeCholangiocarcinomaAug 20thM.D. Anderson Cancer CenterNCT06439485
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedBecker Muscular DystrophyAug 19thUniversity of RochesterNCT07092540
Added 3 new sites: Stony Brook, New York, Cincinnati, Ohio, Columbus, Ohio

Added 3 new sites: Stony Brook, New York, Cincinnati, Ohio, Columbus, Ohio

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Aug 19thAstraZenecaNCT06465810
Added 4 new sites: Baltimore, Maryland, Houston, Texas, Montreal, Canada and 1 more

Added 4 new sites: Baltimore, Maryland, Houston, Texas, Montreal, Canada and 1 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Aug 19thNovo Nordisk A/SNCT07207811
Added 4 new sites: Baltimore, Maryland, Guiyang, China, Jinan, China and 1 more

Added 4 new sites: Baltimore, Maryland, Guiyang, China, Jinan, China and 1 more

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Aug 19thNovo Nordisk A/SNCT07207811
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateAlpha-1 Antitrypsin DeficiencyAug 19thAlpha-1 FoundationNCT04157049
Target enrollment increased from 4000 to 5000

Target enrollment increased from 4000 to 5000

sites_addedBecker Muscular DystrophyAug 19thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Leuven, Belgium

Added 1 new site: Leuven, Belgium

sites_addedBecker Muscular DystrophyAug 19thSolid Biosciences Inc.NCT07160634
Added 1 new site: Toronto, Canada

Added 1 new site: Toronto, Canada

eligibility_changeBecker Muscular DystrophyAug 19thSolid Biosciences Inc.NCT07160634
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Aug 19thAlnylam PharmaceuticalsNCT07052903
Added 10 new sites: Laguna Hills, California, Chicago, Illinois, Edegem, Belgium and 7 more

Added 10 new sites: Laguna Hills, California, Chicago, Illinois, Edegem, Belgium and 7 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Aug 19thAlnylam PharmaceuticalsNCT07223203
Added 7 new sites: Nashville, Tennessee, Vancouver, Canada, Heidelberg, Germany and 4 more

Added 7 new sites: Nashville, Tennessee, Vancouver, Canada, Heidelberg, Germany and 4 more

sites_addedAchondroplasiaAug 19thAscendis Pharma Growth Disorders A/SNCT05929807
Added 2 new sites: Paris, France, London, United Kingdom

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

sites_addedSpinal Muscular AtrophyAug 18thBiogenNCT07221669
Added 4 new sites: Randwick, Australia, Porto Alegre, Brazil, Sai Paula, Brazil and 1 more

Added 4 new sites: Randwick, Australia, Porto Alegre, Brazil, Sai Paula, Brazil and 1 more

sites_addedSickle Cell DiseaseAug 18thPfizerNCT05431088
Added 34 new sites: Mobile, Alabama, Hartford, Connecticut, Plantation, Florida and 31 more

Added 34 new sites: Mobile, Alabama, Hartford, Connecticut, Plantation, Florida and 31 more

eligibility_changeSickle Cell DiseaseAug 18thPfizerNCT05431088
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedWaldenström MacroglobulinemiaAug 18thExscientia AI Ltd., a wholly owned subsidiary of Recursion Pharmaceuticals, Inc.NCT06980116
Added 8 new sites: Athens, Greece, Pátrai, Greece, Thessaloniki, Greece and 5 more

Added 8 new sites: Athens, Greece, Pátrai, Greece, Thessaloniki, Greece and 5 more

sites_addedVon Willebrand DiseaseAug 18thAmerican Thrombosis and Hemostasis NetworkNCT04398628
Added 5 new sites: Miami, Florida, Tampa, Florida, Columbus, Ohio and 2 more

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

phase_changeUrea Cycle DisordersAug 18thiECURE, Inc.NCT06255782
Moved from Phase 1, Phase 2 to Phase 3

Moved from Phase 1, Phase 2 to Phase 3

sites_addedThalassemiaAug 18thNovo Nordisk A/SNCT06609226
Expanded from 105 to 106 sites

Expanded from 105 to 106 sites

sites_addedThalassemiaAug 18thAmerican Thrombosis and Hemostasis NetworkNCT04398628
Added 5 new sites: Miami, Florida, Tampa, Florida, Columbus, Ohio and 2 more

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

sites_addedSystemic Juvenile Idiopathic ArthritisAug 18thAbbVieNCT05609630
Added 1 new site: Berlin, Germany

Added 1 new site: Berlin, Germany

sites_addedSickle Cell DiseaseAug 18thNovo Nordisk A/SNCT06609226
Expanded from 105 to 106 sites

Expanded from 105 to 106 sites

sites_addedSickle Cell DiseaseAug 18thNovo Nordisk A/SNCT06612268
Added 2 new sites: Amsterdam, Netherlands, Al Ahsa, Saudi Arabia

Added 2 new sites: Amsterdam, Netherlands, Al Ahsa, Saudi Arabia

Enrollment UpdateSickle Cell DiseaseAug 18thUniversity of CincinnatiNCT06725550
Target enrollment increased from 10 to 120

Target enrollment increased from 10 to 120

sites_addedSickle Cell DiseaseAug 18thCSL BehringNCT06699849
Added 7 new sites: Atlanta, Georgia, Chicago, Illinois, St Louis, Missouri and 4 more

Added 7 new sites: Atlanta, Georgia, Chicago, Illinois, St Louis, Missouri and 4 more

sites_addedSickle Cell DiseaseAug 18thAmerican Thrombosis and Hemostasis NetworkNCT04398628
Added 5 new sites: Miami, Florida, Tampa, Florida, Columbus, Ohio and 2 more

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

sites_addedPompe DiseaseAug 17thDenali Therapeutics Inc.NCT07354724
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedShort Bowel SyndromeAug 17thHanmi Pharmaceutical Company LimitedNCT04775706
Added 1 new site: Skawina, Poland

Added 1 new site: Skawina, Poland

sites_addedShort Bowel SyndromeAug 17thZealand PharmaNCT07197944
Added 11 new sites: Chicago, Illinois, Leuven, Belgium, Helsinki, Finland and 8 more

Added 11 new sites: Chicago, Illinois, Leuven, Belgium, Helsinki, Finland and 8 more

Enrollment UpdateSarcoidosisAug 17thMontefiore Medical CenterNCT06610019
Target enrollment increased from 5000 to 10000

Target enrollment increased from 5000 to 10000

Enrollment UpdatePulmonary Arterial HypertensionAug 17thBastiaan DriehuysNCT04991454
Target enrollment decreased from 60 to 20

Target enrollment decreased from 60 to 20

sites_addedPulmonary Arterial HypertensionAug 17thMerck Sharp & Dohme LLCNCT07218029
Added 4 new sites: Belo Horizonte, Brazil, Lille, France, Mexico City, Mexico and 1 more

Added 4 new sites: Belo Horizonte, Brazil, Lille, France, Mexico City, Mexico and 1 more

sites_addedPulmonary Arterial HypertensionAug 17thAllRock Bio, Inc.NCT07175038
Added 4 new sites: Houston, Texas, Foggia, Italy, Rome, Italy and 1 more

Added 4 new sites: Houston, Texas, Foggia, Italy, Rome, Italy and 1 more

eligibility_changePulmonary Arterial HypertensionAug 17thAllRock Bio, Inc.NCT07175038
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPrimary Biliary CholangitisAug 17thGilead SciencesNCT06051617
Added 6 new sites: New York, New York, Prague, Czechia, Villejuif, France and 3 more

Added 6 new sites: New York, New York, Prague, Czechia, Villejuif, France and 3 more

sites_addedPrader-Willi SyndromeAug 17thSoleno Therapeutics, Inc.NCT07450664
Added 23 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 20 more

Added 23 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 20 more

eligibility_changePolycythemia VeraAug 17thChengdu Zenitar Biomedical Technology Co., LtdNCT07232290
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraAug 17thDisc Medicine, IncNCT05320198
Added 2 new sites: Los Angeles, California, Saint Albans, Australia

Added 2 new sites: Los Angeles, California, Saint Albans, Australia

Enrollment UpdatePolycythemia VeraAug 17thNational Cancer Institute (NCI)NCT06661915
Target enrollment increased from 62 to 78

Target enrollment increased from 62 to 78

eligibility_changePolycythemia VeraAug 17thNational Cancer Institute (NCI)NCT06661915
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycystic Kidney DiseaseAug 17thUniversity Medical Center GroningenNCT05373264
Added 10 new sites: Antwerp, Belgium, Bordeaux, France, Lyon, France and 7 more

Added 10 new sites: Antwerp, Belgium, Bordeaux, France, Lyon, France and 7 more

eligibility_changePhenylketonuriaAug 17thNGGT (Suzhou) Biotechnology Co., Ltd.NCT06687733
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPhelan-McDermid SyndromeAug 17thNeuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Birmingham, Alabama, Ann Arbor, Michigan

Added 2 new sites: Birmingham, Alabama, Ann Arbor, Michigan

sites_addedPhelan-McDermid SyndromeAug 17thNeuren Pharmaceuticals LimitedNCT07593391
Added 4 new sites: Glendale, California, Lexington, Massachusetts, Cincinnati, Ohio and 1 more

Added 4 new sites: Glendale, California, Lexington, Massachusetts, Cincinnati, Ohio and 1 more

Enrollment UpdateParoxysmal Nocturnal HemoglobinuriaAug 17thAstraZenecaNCT07457151
Target enrollment increased from 8 to 27

Target enrollment increased from 8 to 27

sites_addedNoonan SyndromeAug 16thAscendis Pharma A/SNCT07221851
Added 2 new sites: Wilmington, Delaware, Seattle, Washington

Added 2 new sites: Wilmington, Delaware, Seattle, Washington

phase_changeNeuromyelitis OpticaAug 16thTianjin Medical University General HospitalNCT05792462
Moved from Phase 1, Phase 2 to Phase 1

Moved from Phase 1, Phase 2 to Phase 1

sites_addedNarcolepsy Type 1Aug 16thCentessa Pharmaceuticals (UK) LimitedNCT07598708
Added 2 new sites: Orlando, Florida, Atlanta, Georgia

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

sites_addedMyotonic DystrophyAug 16thSarepta Therapeutics, Inc.NCT06138743
Added 35 new sites: Liverpool, Australia, Birtinya, Australia, Herston, Australia and 32 more

Added 35 new sites: Liverpool, Australia, Birtinya, Australia, Herston, Australia and 32 more

eligibility_changeMyotonic DystrophyAug 16thSarepta Therapeutics, Inc.NCT06138743
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyotonic DystrophyAug 16thVirginia Commonwealth UniversityNCT06747884
Added 1 new site: Cerqueira César, Brazil

Added 1 new site: Cerqueira César, Brazil

sites_addedMyotonic DystrophyAug 16thAvidity Biosciences, Inc.NCT07587242
Added 3 new sites: Madrid, Spain, Seville, Spain, Newcastle, United Kingdom

Added 3 new sites: Madrid, Spain, Seville, Spain, Newcastle, United Kingdom

sites_addedMyotonic DystrophyAug 16thDyne TherapeuticsNCT07486934
Added 8 new sites: Los Angeles, California, Edegem, Belgium, Garches, France and 5 more

Added 8 new sites: Los Angeles, California, Edegem, Belgium, Garches, France and 5 more

sites_addedMyotonic DystrophyAug 16thVertex Pharmaceuticals IncorporatedNCT06926621
Added 2 new sites: St Louis, Missouri, Richmond, Virginia

Added 2 new sites: St Louis, Missouri, Richmond, Virginia

sites_addedMyelofibrosisAug 16thDisc Medicine, IncNCT05320198
Added 2 new sites: Los Angeles, California, Saint Albans, Australia

Added 2 new sites: Los Angeles, California, Saint Albans, Australia

sites_addedMyelofibrosisAug 16thPharmaEssentiaNCT06468033
Added 71 new sites: Los Angeles, California, Washington D.C., District of Columbia, Kansas City, Kansas and 68 more

Added 71 new sites: Los Angeles, California, Washington D.C., District of Columbia, Kansas City, Kansas and 68 more

Enrollment UpdateMyelofibrosisAug 16thNational Cancer Institute (NCI)NCT06661915
Target enrollment increased from 62 to 78

Target enrollment increased from 62 to 78

eligibility_changeMyelofibrosisAug 16thNational Cancer Institute (NCI)NCT06661915
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesAug 16thDisc Medicine, IncNCT05320198
Added 2 new sites: Los Angeles, California, Saint Albans, Australia

Added 2 new sites: Los Angeles, California, Saint Albans, Australia

sites_addedMyelodysplastic SyndromesAug 16thNational Cancer Institute (NCI)NCT05564390
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

Enrollment UpdateMyelodysplastic SyndromesAug 16thTaiho Pharmaceutical Co., Ltd.NCT04608110
Target enrollment increased from 40 to 59

Target enrollment increased from 40 to 59

eligibility_changeMyelodysplastic SyndromesAug 16thTaiho Pharmaceutical Co., Ltd.NCT04608110
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisAug 16thMerck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 5 new sites: Rome, Italy, Rome, Italy, Kragujevac, Serbia and 2 more

Added 5 new sites: Rome, Italy, Rome, Italy, Kragujevac, Serbia and 2 more

Enrollment UpdateMyasthenia GravisAug 16thCabaletta BioNCT06359041
Target enrollment increased from 12 to 24

Target enrollment increased from 12 to 24

sites_addedMyasthenia GravisAug 16thUCB Biopharma SRLNCT06540144
Added 2 new sites: Lodz, Poland, Yenimahalle, Turkey (Türkiye)

Added 2 new sites: Lodz, Poland, Yenimahalle, Turkey (Türkiye)

sites_addedMyasthenia GravisAug 16thKite, A Gilead CompanyNCT07304154
Added 2 new sites: Newport Beach, California, Montreal, Canada

Added 2 new sites: Newport Beach, California, Montreal, Canada

eligibility_changeMyasthenia GravisAug 16thargenxNCT06298552
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMyasthenia GravisAug 16thNovartis PharmaceuticalsNCT06517758
Reduced from 125 to 116 sites

Reduced from 125 to 116 sites

Enrollment UpdateMyasthenia GravisAug 16thCSPC ZhongQi Pharmaceutical Technology Co., Ltd.NCT06688435
Target enrollment increased from 50 to 60

Target enrollment increased from 50 to 60

eligibility_changeMyasthenia GravisAug 16thCSPC ZhongQi Pharmaceutical Technology Co., Ltd.NCT06688435
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMultiple SclerosisAug 16thNovartis PharmaceuticalsNCT06869785
Reduced from 65 to 63 sites

Reduced from 65 to 63 sites

eligibility_changeMultiple SclerosisAug 16thHoffmann-La RocheNCT05704361
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 16thSanofiNCT07325292
Added 4 new sites: Milan, Italy, Foggia, Italy, Roma, Italy and 1 more

Added 4 new sites: Milan, Italy, Foggia, Italy, Roma, Italy and 1 more

sites_addedMultiple SclerosisAug 16thZenas BioPharma (USA), LLCNCT07299019
Added 7 new sites: Torrance, California, Choceň, Czechia, Kaunas, Lithuania and 4 more

Added 7 new sites: Torrance, California, Choceň, Czechia, Kaunas, Lithuania and 4 more

sites_addedMultiple SclerosisAug 16thBiogenNCT05658497
Added 1 new site: Salford, United Kingdom

Added 1 new site: Salford, United Kingdom

sites_addedMultiple SclerosisAug 16thKite, A Gilead CompanyNCT07304154
Added 2 new sites: Newport Beach, California, Montreal, Canada

Added 2 new sites: Newport Beach, California, Montreal, Canada

sites_addedMultiple SclerosisAug 16thNovartis PharmaceuticalsNCT06846281
Added 7 new sites: Hollywood, Florida, Miami, Florida, Chicago, Illinois and 4 more

Added 7 new sites: Hollywood, Florida, Miami, Florida, Chicago, Illinois and 4 more

Enrollment UpdateMultiple SclerosisAug 16thSomogy Megyei Kaposi Mór Teaching HospitalNCT07411664
Target enrollment increased from 60 to 151

Target enrollment increased from 60 to 151

sites_addedMesotheliomaAug 16thIntergroupe Francophone de Cancerologie ThoraciqueNCT06840834
Added 1 new site: Lyon, France

Added 1 new site: Lyon, France

eligibility_changeMesotheliomaAug 16thIntergroupe Francophone de Cancerologie ThoraciqueNCT06840834
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaAug 16thKupando GmbHNCT07600476
Added 1 new site: Munich, Germany

Added 1 new site: Munich, Germany

sites_addedMembranous NephropathyAug 16thAstraZenecaNCT07571746
Added 1 new site: München, Germany

Added 1 new site: München, Germany

eligibility_changeMembranous NephropathyAug 16thBiogenNCT06962800
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMastocytosisAug 16thBlueprint Medicines CorporationNCT07143669
Added 4 new sites: Evanston, Illinois, Rochester, Minnesota, Omaha, Nebraska and 1 more

Added 4 new sites: Evanston, Illinois, Rochester, Minnesota, Omaha, Nebraska and 1 more

Enrollment UpdateMastocytosisAug 16thBlueprint Medicines CorporationNCT07143669
Target enrollment increased from 450 to 750

Target enrollment increased from 450 to 750

eligibility_changeMastocytosisAug 16thBlueprint Medicines CorporationNCT07143669
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyAug 15thNovartis PharmaceuticalsNCT07146906
Added 3 new sites: Recife, Brazil, Natal, Brazil, Prague, Czechia

Added 3 new sites: Recife, Brazil, Natal, Brazil, Prague, Czechia

sites_removedIgA NephropathyAug 15thHoffmann-La RocheNCT05797610
Reduced from 204 to 178 sites

Reduced from 204 to 178 sites

sites_addedIdiopathic Pulmonary FibrosisAug 15thContineum TherapeuticsNCT07284459
Added 4 new sites: San Luis, Argentina, Greenslopes, Australia, Montreal, Canada and 1 more

Added 4 new sites: San Luis, Argentina, Greenslopes, Australia, Montreal, Canada and 1 more

sites_addedIdiopathic Pulmonary FibrosisAug 15thMediar TherapeuticsNCT06967805
Added 1 new site: Rotterdam, Netherlands

Added 1 new site: Rotterdam, Netherlands

sites_addedIdiopathic Pulmonary FibrosisAug 15thRein TherapeuticsNCT06968845
Added 6 new sites: Los Angeles, California, Temple, Texas, Camperdown, Australia and 3 more

Added 6 new sites: Los Angeles, California, Temple, Texas, Camperdown, Australia and 3 more

sites_addedIdiopathic Pulmonary FibrosisAug 15thAvalyn Pharma Inc.NCT07194382
Added 21 new sites: Camperdown, Australia, Westmead, Australia, Vancouver, Canada and 18 more

Added 21 new sites: Camperdown, Australia, Westmead, Australia, Vancouver, Canada and 18 more

eligibility_changeIdiopathic Pulmonary FibrosisAug 15thJoseph C. WuNCT05988463
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisAug 15thDuke UniversityNCT01915511
Added 3 new sites: Detroit, Michigan, Philadelphia, Pennsylvania, Colchester, Vermont

Added 3 new sites: Detroit, Michigan, Philadelphia, Pennsylvania, Colchester, Vermont

sites_addedHypoparathyroidismAug 15thAmorphical Ltd.NCT06547151
Added 1 new site: Haifa, Israel

Added 1 new site: Haifa, Israel

Enrollment UpdateHypoparathyroidismAug 15thAmorphical Ltd.NCT06547151
Target enrollment decreased from 81 to 62

Target enrollment decreased from 81 to 62

sites_addedHuntington DiseaseAug 15thNovartis PharmaceuticalsNCT07326709
Added 13 new sites: Sacramento, California, Farmington, Connecticut, Durham, North Carolina and 10 more

Added 13 new sites: Sacramento, California, Farmington, Connecticut, Durham, North Carolina and 10 more

sites_addedHomocystinuriaAug 15thTravere Therapeutics, Inc.NCT03406611
Added 5 new sites: Chicago, Illinois, New York, New York, Morrisville, North Carolina and 2 more

Added 5 new sites: Chicago, Illinois, New York, New York, Morrisville, North Carolina and 2 more

Enrollment UpdateHomocystinuriaAug 15thTravere Therapeutics, Inc.NCT03406611
Target enrollment increased from 32 to 39

Target enrollment increased from 32 to 39

eligibility_changeHomocystinuriaAug 15thTravere Therapeutics, Inc.NCT03406611
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaAug 15thChildren's Oncology GroupNCT05705531
Added 4 new sites: Madera, California, Jacksonville, Florida, Orlando, Florida and 1 more

Added 4 new sites: Madera, California, Jacksonville, Florida, Orlando, Florida and 1 more

eligibility_changeHodgkin LymphomaAug 15thNational Cancer Institute (NCI)NCT01676805
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHodgkin LymphomaAug 15thDivaya BhutaniNCT05346809
Target enrollment decreased from 39 to 8

Target enrollment decreased from 39 to 8

sites_addedHodgkin LymphomaAug 15thNational Cancer Institute (NCI)NCT05675410
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedGeographic AtrophyAug 14thGalimedix Therapeutics IncNCT06659549
Added 7 new sites: Lyon, France, Paris, France, Tours, France and 4 more

Added 7 new sites: Lyon, France, Paris, France, Tours, France and 4 more

sites_addedHemophilia BAug 14thPfizerNCT05611801
Added 32 new sites: Salt Lake City, Utah, Maruípe, Brazil, Edmonton, Canada and 29 more

Added 32 new sites: Salt Lake City, Utah, Maruípe, Brazil, Edmonton, Canada and 29 more

sites_addedHemophilia AAug 14thPfizerNCT05611801
Added 32 new sites: Salt Lake City, Utah, Maruípe, Brazil, Edmonton, Canada and 29 more

Added 32 new sites: Salt Lake City, Utah, Maruípe, Brazil, Edmonton, Canada and 29 more

sites_addedGraves’ DiseaseAug 14thImmunovant Sciences GmbHNCT07018323
Added 2 new sites: Thessaloniki, Greece, Torino, Italy

Added 2 new sites: Thessaloniki, Greece, Torino, Italy

sites_addedGraves’ DiseaseAug 14thargenxNCT07570316
Added 4 new sites: Houston, Texas, Tbilisi, Georgia, Tbilisi, Georgia and 1 more

Added 4 new sites: Houston, Texas, Tbilisi, Georgia, Tbilisi, Georgia and 1 more

eligibility_changeGiant Cell ArteritisAug 14thNational Institute of Allergy and Infectious Diseases (NIAID)NCT07108387
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeGiant Cell ArteritisAug 14thNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NCT02257866
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyAug 14thRegeneron PharmaceuticalsNCT06541704
Added 1 new site: Waltham, Massachusetts

Added 1 new site: Waltham, Massachusetts

sites_removedGeographic AtrophyAug 14thi-Lumen Scientific AUS PTY LTDNCT06662162
Reduced from 15 to 12 sites

Reduced from 15 to 12 sites

sites_addedGeographic AtrophyAug 14thKriya Therapeutics, Inc.NCT06765980
Added 1 new site: Erie, Pennsylvania

Added 1 new site: Erie, Pennsylvania

Enrollment UpdateGaucher DiseaseAug 14thShanghai Jiao Tong University School of MedicineNCT06528080
Target enrollment decreased from 9 to 3

Target enrollment decreased from 9 to 3

sites_addedGastrointestinal Stromal TumorAug 14thKumquat Biosciences Inc.NCT07406633
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

eligibility_changeFacioscapulohumeral Muscular DystrophyAug 14thSarepta Therapeutics, Inc.NCT06131983
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal Segmental GlomerulosclerosisAug 14thAkebia TherapeuticsNCT07268638
Added 2 new sites: Hialeah, Florida, Worcester, Massachusetts

Added 2 new sites: Hialeah, Florida, Worcester, Massachusetts

sites_removedFocal EpilepsyAug 14thBiohaven Therapeutics Ltd.NCT06309966
Reduced from 152 to 150 sites

Reduced from 152 to 150 sites

sites_addedFocal EpilepsyAug 14thRapport Therapeutics Inc.NCT07563881
Added 3 new sites: Atlanta, Georgia, Boise, Idaho, Cleveland, Ohio

Added 3 new sites: Atlanta, Georgia, Boise, Idaho, Cleveland, Ohio

sites_removedFocal EpilepsyAug 14thSK Life Science, Inc.NCT05067634
Reduced from 54 to 35 sites

Reduced from 54 to 35 sites

eligibility_changeFamilial Hypertrophic CardiomyopathyAug 14thProlaioNCT07420907
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateFamilial Hypertrophic CardiomyopathyAug 14thMontefiore Medical CenterNCT06610019
Target enrollment increased from 5000 to 10000

Target enrollment increased from 5000 to 10000

Enrollment UpdateFabry DiseaseAug 14thMontefiore Medical CenterNCT06610019
Target enrollment increased from 5000 to 10000

Target enrollment increased from 5000 to 10000

sites_addedEssential ThrombocythemiaAug 14thDisc Medicine, IncNCT05320198
Added 2 new sites: Los Angeles, California, Saint Albans, Australia

Added 2 new sites: Los Angeles, California, Saint Albans, Australia

Enrollment UpdateEssential ThrombocythemiaAug 14thNational Cancer Institute (NCI)NCT06661915
Target enrollment increased from 62 to 78

Target enrollment increased from 62 to 78

eligibility_changeEssential ThrombocythemiaAug 14thNational Cancer Institute (NCI)NCT06661915
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeEpidermolysis BullosaAug 14thBioMendics, LLCNCT07027345
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEpidermolysis BullosaAug 14thXinnate ABNCT06594393
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

eligibility_changeEpidermolysis BullosaAug 14thParadigm TherapeuticsNCT07482787
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDuchenne Muscular DystrophyAug 13thSolid Biosciences Inc.NCT07160634
Added 1 new site: Toronto, Canada

Added 1 new site: Toronto, Canada

eligibility_changeDuchenne Muscular DystrophyAug 13thSolid Biosciences Inc.NCT07160634
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeAug 13thEpygenixNCT04462770
Added 16 new sites: Buenos Aires, Argentina, Córdoba, Argentina, Córdoba, Argentina and 13 more

Added 16 new sites: Buenos Aires, Argentina, Córdoba, Argentina, Córdoba, Argentina and 13 more

eligibility_changeCushing DiseaseAug 13thCorcept TherapeuticsNCT03604198
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaAug 13thNational Cancer Institute (NCI)NCT05211323
Target enrollment decreased from 88 to 4

Target enrollment decreased from 88 to 4

sites_addedDuchenne Muscular DystrophyAug 13thAvidity Biosciences, Inc.NCT07587242
Added 3 new sites: Madrid, Spain, Seville, Spain, Newcastle, United Kingdom

Added 3 new sites: Madrid, Spain, Seville, Spain, Newcastle, United Kingdom

sites_addedDravet SyndromeAug 13thLongboard PharmaceuticalsNCT06660394
Added 34 new sites: Little Rock, Arkansas, San Francisco, California, Bethesda, Maryland and 31 more

Added 34 new sites: Little Rock, Arkansas, San Francisco, California, Bethesda, Maryland and 31 more

Enrollment UpdateDravet SyndromeAug 13thLongboard PharmaceuticalsNCT06660394
Target enrollment decreased from 160 to 104

Target enrollment decreased from 160 to 104

eligibility_changeDilated CardiomyopathyAug 13thProlaioNCT07420907
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDilated CardiomyopathyAug 13thNuevocor Pte. Ltd.NCT07606274
Added 2 new sites: Ann Arbor, Michigan, Columbus, Ohio

Added 2 new sites: Ann Arbor, Michigan, Columbus, Ohio

eligibility_changeDermatomyositisAug 13thNational Institute of Environmental Health Sciences (NIEHS)NCT07111065
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateDermatomyositisAug 13thNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NCT00059748
Target enrollment decreased from 5000 to 756

Target enrollment decreased from 5000 to 756

sites_addedCongenital Adrenal HyperplasiaAug 13thCrinetics Pharmaceuticals Inc.NCT07144163
Added 8 new sites: Aurora, Colorado, Philadelphia, Pennsylvania, Ribeirão Preto, Brazil and 5 more

Added 8 new sites: Aurora, Colorado, Philadelphia, Pennsylvania, Ribeirão Preto, Brazil and 5 more

sites_addedCongenital Adrenal HyperplasiaAug 13thCrinetics Pharmaceuticals Inc.NCT07159841
Added 1 new site: Talcahuano, Chile

Added 1 new site: Talcahuano, Chile

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 13thSanofiNCT06290128
Added 3 new sites: New York, New York, São Paulo, Brazil, Lisbon, Portugal

Added 3 new sites: New York, New York, São Paulo, Brazil, Lisbon, Portugal

Enrollment UpdateChronic Inflammatory Demyelinating PolyneuropathyAug 13thSanofiNCT06290128
Target enrollment decreased from 140 to 109

Target enrollment decreased from 140 to 109

Enrollment UpdateChronic Graft-versus-Host DiseaseAug 13thNational Cancer Institute (NCI)NCT04044365
Target enrollment decreased from 420 to 139

Target enrollment decreased from 420 to 139

sites_addedCholangiocarcinomaAug 13thInstitut für Klinische Krebsforschung IKF GmbH at Krankenhaus NordwestNCT07260175
Added 3 new sites: Hamburg, Germany, Oldenburg, Germany, Regensburg, Germany

Added 3 new sites: Hamburg, Germany, Oldenburg, Germany, Regensburg, Germany

eligibility_changeAlport SyndromeAug 12thBayerNCT07211685
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAcromegalyAug 12thCamurus ABNCT07583563
Added 1 new site: Leeds, United Kingdom

Added 1 new site: Leeds, United Kingdom

sites_addedBecker Muscular DystrophyAug 12thAvidity Biosciences, Inc.NCT07587242
Added 3 new sites: Madrid, Spain, Seville, Spain, Newcastle, United Kingdom

Added 3 new sites: Madrid, Spain, Seville, Spain, Newcastle, United Kingdom

sites_addedBarth SyndromeAug 12thLMU KlinikumNCT05554835
Added 3 new sites: Munich, Germany, Halle, Germany, München, Germany

Added 3 new sites: Munich, Germany, Halle, Germany, München, Germany

Enrollment UpdateArrhythmogenic CardiomyopathyAug 12thMontefiore Medical CenterNCT06610019
Target enrollment increased from 5000 to 10000

Target enrollment increased from 5000 to 10000

sites_addedAmyotrophic Lateral SclerosisAug 12thUniversity Hospital, LimogesNCT06147843
Added 2 new sites: Hanover, Germany, Ulm, Germany

Added 2 new sites: Hanover, Germany, Ulm, Germany

phase_changeAmyotrophic Lateral SclerosisAug 12thCoya TherapeuticsNCT07161999
Moved from Phase 2 to Phase 2, Phase 3

Moved from Phase 2 to Phase 2, Phase 3

eligibility_changeAmyotrophic Lateral SclerosisAug 12thCoya TherapeuticsNCT07161999
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeAmyotrophic Lateral SclerosisAug 12thJohns Hopkins UniversityNCT06656702
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAlpha-1 Antitrypsin DeficiencyAug 12thTessera Therapeutics, Inc.NCT07227207
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedTurner SyndromeAug 11thAscendis Pharma A/SNCT07221851
Added 2 new sites: Wilmington, Delaware, Seattle, Washington

Added 2 new sites: Wilmington, Delaware, Seattle, Washington

eligibility_changeTakayasu ArteritisAug 11thNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NCT02257866
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedWaldenström MacroglobulinemiaAug 11thBeOne MedicinesNCT05006716
Reduced from 127 to 114 sites

Reduced from 127 to 114 sites

sites_removedWaldenström MacroglobulinemiaAug 11thBeiGeneNCT07169331
Reduced from 8 to 6 sites

Reduced from 8 to 6 sites

sites_addedStargardt DiseaseAug 11thAstellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Jacksonville, Florida

Added 1 new site: Jacksonville, Florida

sites_addedSpinal Muscular AtrophyAug 11thargenxNCT07287982
Added 7 new sites: Cambridge, United Kingdom, London, United Kingdom, Oxford, United Kingdom and 4 more

Added 7 new sites: Cambridge, United Kingdom, London, United Kingdom, Oxford, United Kingdom and 4 more

sites_addedSickle Cell DiseaseAug 11thCSL BehringNCT07224360
Added 11 new sites: Orange, California, Atlanta, Georgia, Columbus, Ohio and 8 more

Added 11 new sites: Orange, California, Atlanta, Georgia, Columbus, Ohio and 8 more

eligibility_changeSickle Cell DiseaseAug 11thCSL BehringNCT07224360
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionAug 10thRegeneron PharmaceuticalsNCT07318597
Added 1 new site: Salamanca, Spain

Added 1 new site: Salamanca, Spain

Enrollment UpdatePulmonary Arterial HypertensionAug 10thSheffield Teaching Hospitals NHS Foundation TrustNCT05825417
Target enrollment increased from 40 to 70

Target enrollment increased from 40 to 70

eligibility_changePulmonary Arterial HypertensionAug 10thSheffield Teaching Hospitals NHS Foundation TrustNCT05825417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraAug 10thTakedaNCT07648030
Added 5 new sites: Nagoya, Japan, Maebashi, Japan, Shinjuku-ku, Japan and 2 more

Added 5 new sites: Nagoya, Japan, Maebashi, Japan, Shinjuku-ku, Japan and 2 more

sites_addedPolycystic Kidney DiseaseAug 10thCalico Life Sciences LLCNCT06902558
Added 90 new sites: Birmingham, Alabama, Huntington Park, California, Los Angeles, California and 87 more

Added 90 new sites: Birmingham, Alabama, Huntington Park, California, Los Angeles, California and 87 more

eligibility_changePhenylketonuriaAug 10thThe First Affiliated Hospital of Bengbu Medical UniversityNCT06061614
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedParoxysmal Nocturnal HemoglobinuriaAug 10thRegeneron PharmaceuticalsNCT07154745
Added 1 new site: São Paulo, Brazil

Added 1 new site: São Paulo, Brazil

sites_addedParoxysmal Nocturnal HemoglobinuriaAug 10thNovartis PharmaceuticalsNCT06934967
Added 2 new sites: Atlanta, Georgia, Cologne, Germany

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

eligibility_changeNeuromyelitis OpticaAug 9thTianjin Medical University General HospitalNCT05792462
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeNeurofibromatosis Type 1Aug 9thNational Cancer Institute (NCI)NCT06880991
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1Aug 9thAlkermes, Inc.NCT07540897
Added 4 new sites: Charlotte, North Carolina, Colombia, South Carolina, Markham, Canada and 1 more

Added 4 new sites: Charlotte, North Carolina, Colombia, South Carolina, Markham, Canada and 1 more

sites_addedNarcolepsy Type 1Aug 9thAlkermes, Inc.NCT07455383
Added 7 new sites: Phoenix, Arizona, Charlotte, North Carolina, Namur, Belgium and 4 more

Added 7 new sites: Phoenix, Arizona, Charlotte, North Carolina, Namur, Belgium and 4 more

sites_addedNarcolepsy Type 1Aug 9thAlkermes, Inc.NCT06767683
Added 3 new sites: Cullman, Alabama, Boulder, Colorado, Kansas City, Kansas

Added 3 new sites: Cullman, Alabama, Boulder, Colorado, Kansas City, Kansas

sites_addedMyotonic DystrophyAug 9thVirginia Commonwealth UniversityNCT06747884
Added 2 new sites: Little Rock, Arkansas, Kansas City, Kansas

Added 2 new sites: Little Rock, Arkansas, Kansas City, Kansas

sites_addedMyelofibrosisAug 9thSumitomo Pharma America, Inc.NCT04176198
Added 6 new sites: Fitzroy, Australia, Halle, Germany, Lübeck, Germany and 3 more

Added 6 new sites: Fitzroy, Australia, Halle, Germany, Lübeck, Germany and 3 more

sites_removedMyelofibrosisAug 9thTakedaNCT07623161
Reduced from 207 to 194 sites

Reduced from 207 to 194 sites

Enrollment UpdateMyelodysplastic SyndromesAug 9thM.D. Anderson Cancer CenterNCT02392572
Target enrollment decreased from 120 to 58

Target enrollment decreased from 120 to 58

sites_addedMyasthenia GravisAug 9thAurinia Pharmaceuticals Inc.NCT07596901
Added 7 new sites: Pasadena, California, Homestead, Florida, Chicago, Illinois and 4 more

Added 7 new sites: Pasadena, California, Homestead, Florida, Chicago, Illinois and 4 more

sites_addedMyasthenia GravisAug 9thVertex Pharmaceuticals IncorporatedNCT07501702
Added 2 new sites: Mission Hills, California, Poznan, Poland

Added 2 new sites: Mission Hills, California, Poznan, Poland

sites_addedMultiple System AtrophyAug 9thTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Pittsburgh, Pennsylvania, Barcelona, Spain

Added 2 new sites: Pittsburgh, Pennsylvania, Barcelona, Spain

sites_removedMultiple SclerosisAug 9thHoffmann-La RocheNCT07074886
Reduced from 56 to 50 sites

Reduced from 56 to 50 sites

eligibility_changeMultiple SclerosisAug 9thAssistance Publique Hopitaux De MarseilleNCT03600779
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 9thZenas BioPharma (USA), LLCNCT07067463
Added 5 new sites: Gdansk, Poland, Lublin, Poland, Constanța, Romania and 2 more

Added 5 new sites: Gdansk, Poland, Lublin, Poland, Constanța, Romania and 2 more

eligibility_changeMultiple SclerosisAug 9thHospital Universitari Vall d'Hebron Research InstituteNCT07168772
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisAug 9thSetPoint Medical CorporationNCT06796504
Added 3 new sites: Irvine, California, Austin, Texas, Milwaukee, Wisconsin

Added 3 new sites: Irvine, California, Austin, Texas, Milwaukee, Wisconsin

eligibility_changeMultiple SclerosisAug 9thSetPoint Medical CorporationNCT06796504
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaAug 9thSHY TherapeuticsNCT07705334
Added 1 new site: Scottsdale, Arizona

Added 1 new site: Scottsdale, Arizona

sites_addedMesotheliomaAug 9thIDEAYA BiosciencesNCT07277413
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

eligibility_changeMesotheliomaAug 9thDavid Bartlett, MDNCT07192900
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyAug 9thVertex Pharmaceuticals IncorporatedNCT07204275
Added 3 new sites: Roseville, Missouri, New York, New York, Kawasaki-shi, Japan

Added 3 new sites: Roseville, Missouri, New York, New York, Kawasaki-shi, Japan

eligibility_changeMembranous NephropathyAug 9thNanjing Bioheng Biotech Co., Ltd.NCT07203404
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMastocytosisAug 9thBlueprint Medicines CorporationNCT04910685
Added 4 new sites: Cali, Colombia, Maastricht, Netherlands, Krakow, Poland and 1 more

Added 4 new sites: Cali, Colombia, Maastricht, Netherlands, Krakow, Poland and 1 more

sites_addedMastocytosisAug 9thBlueprint Medicines CorporationNCT07264959
Added 2 new sites: Wheaton, Maryland, Ann Arbor, Michigan

Added 2 new sites: Wheaton, Maryland, Ann Arbor, Michigan

sites_addedIgA NephropathyAug 8thNovartis PharmaceuticalsNCT07498335
Added 3 new sites: Beijing, China, Chiba, Japan, Seoul, South Korea

Added 3 new sites: Beijing, China, Chiba, Japan, Seoul, South Korea

sites_addedIgA NephropathyAug 8thBiogenNCT06935357
Added 5 new sites: Woolloongabba, Australia, Heidelberg, Germany, Leipzig, Germany and 2 more

Added 5 new sites: Woolloongabba, Australia, Heidelberg, Germany, Leipzig, Germany and 2 more

sites_addedIdiopathic Pulmonary FibrosisAug 8thContineum TherapeuticsNCT07284459
Added 3 new sites: Curicó, Chile, Ñuñoa, Chile, Santiago, Chile

Added 3 new sites: Curicó, Chile, Ñuñoa, Chile, Santiago, Chile

sites_addedIdiopathic Pulmonary FibrosisAug 8thAvalyn Pharma Inc.NCT06951217
Added 14 new sites: Houston, Texas, San Miguel de Tucumán, Argentina, Buenos Aires, Argentina and 11 more

Added 14 new sites: Houston, Texas, San Miguel de Tucumán, Argentina, Buenos Aires, Argentina and 11 more

eligibility_changeHypoparathyroidismAug 8thAscendis Pharma Bone Diseases A/SNCT07081997
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseAug 8thHoffmann-La RocheNCT07246941
Added 1 new site: Montral, Canada

Added 1 new site: Montral, Canada

eligibility_changeHodgkin LymphomaAug 8thMemorial Sloan Kettering Cancer CenterNCT03618550
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary Hemorrhagic TelangiectasiaAug 8thDiagonal Therapeutics, Inc.NCT07623525
Added 7 new sites: Camperdown, Australia, Herston, Australia, Herston, Australia and 4 more

Added 7 new sites: Camperdown, Australia, Herston, Australia, Herston, Australia and 4 more

sites_addedHereditary AngioedemaAug 8thIonis Pharmaceuticals, Inc.NCT07298447
Added 3 new sites: Baton Rouge, Louisiana, Cleveland, Ohio, Roma, Italy

Added 3 new sites: Baton Rouge, Louisiana, Cleveland, Ohio, Roma, Italy

sites_addedHemophilia BAug 7thCSL BehringNCT06003387
Added 1 new site: Warsaw, Poland

Added 1 new site: Warsaw, Poland

sites_addedGraves’ DiseaseAug 7thBiohaven Therapeutics Ltd.NCT07661056
Added 2 new sites: Morehead City, North Carolina, Chattanooga, Tennessee

Added 2 new sites: Morehead City, North Carolina, Chattanooga, Tennessee

sites_addedHemophilia BAug 7thCSL BehringNCT07080905
Added 1 new site: Marseille, France

Added 1 new site: Marseille, France

sites_addedHemophilia BAug 7thRegeneron PharmaceuticalsNCT06379789
Added 2 new sites: Houston, Texas, Melbourne, Australia

Added 2 new sites: Houston, Texas, Melbourne, Australia

sites_addedHemophilia AAug 7thHoffmann-La RocheNCT07416526
Added 17 new sites: Aurora, Colorado, Iowa City, Iowa, Bonn, Germany and 14 more

Added 17 new sites: Aurora, Colorado, Iowa City, Iowa, Bonn, Germany and 14 more

sites_addedHemophilia AAug 7thHoffmann-La RocheNCT07226206
Added 1 new site: San Francisco, California

Added 1 new site: San Francisco, California

sites_addedHemophilia AAug 7thHoffmann-La RocheNCT07416604
Added 18 new sites: Aurora, Colorado, Tampa, Florida, Leuven, Belgium and 15 more

Added 18 new sites: Aurora, Colorado, Tampa, Florida, Leuven, Belgium and 15 more

sites_addedGraves’ DiseaseAug 7thBiohaven Therapeutics Ltd.NCT07661056
Added 4 new sites: Farmington Hills, Michigan, Dallas, Texas, San Antonio, Texas and 1 more

Added 4 new sites: Farmington Hills, Michigan, Dallas, Texas, San Antonio, Texas and 1 more

sites_addedGeographic AtrophyAug 7thRegeneron PharmaceuticalsNCT07230834
Added 1 new site: Arecibo, Puerto Rico

Added 1 new site: Arecibo, Puerto Rico

sites_addedGeographic AtrophyAug 7thCellio Therapeutics IncNCT07594236
Added 2 new sites: Palo Alto, California, Sacramento, California

Added 2 new sites: Palo Alto, California, Sacramento, California

sites_addedGeographic AtrophyAug 7thComplement TherapeuticsNCT07144137
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedGeographic AtrophyAug 7thGalimedix Therapeutics IncNCT06659549
Added 6 new sites: Bordeaux, France, Lyon, France, Saint-Cyr-sur-Loire, France and 3 more

Added 6 new sites: Bordeaux, France, Lyon, France, Saint-Cyr-sur-Loire, France and 3 more

sites_addedFocal Segmental GlomerulosclerosisAug 7thBoehringer IngelheimNCT07220083
Added 9 new sites: Northridge, California, Kansas City, Kansas, Columbus, Mississippi and 6 more

Added 9 new sites: Northridge, California, Kansas City, Kansas, Columbus, Mississippi and 6 more

sites_addedFocal EpilepsyAug 7thRapport Therapeutics Inc.NCT07594119
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

sites_addedFocal EpilepsyAug 7thRapport Therapeutics Inc.NCT07563881
Added 2 new sites: Gulf Breeze, Florida, Philadelphia, Pennsylvania

Added 2 new sites: Gulf Breeze, Florida, Philadelphia, Pennsylvania

sites_addedEosinophilic EsophagitisAug 6thRegeneron PharmaceuticalsNCT07112378
Added 1 new site: Milwaukee, Wisconsin

Added 1 new site: Milwaukee, Wisconsin

sites_addedDravet SyndromeAug 6thStoke Therapeutics, IncNCT06872125
Added 3 new sites: Beijing, China, Guangzhou, China, Wuhan, China

Added 3 new sites: Beijing, China, Guangzhou, China, Wuhan, China

sites_addedDravet SyndromeAug 6thIonis Pharmaceuticals, Inc.NCT07531745
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedDilated CardiomyopathyAug 6thKardigan, Inc.NCT07210723
Added 22 new sites: Los Angeles, California, Stanford, California, Baltimore, Maryland and 19 more

Added 22 new sites: Los Angeles, California, Stanford, California, Baltimore, Maryland and 19 more

sites_addedDesmoplastic Small Round Cell TumorAug 6thNational Cancer Institute (NCI)NCT04616560
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedChronic Inflammatory Demyelinating PolyneuropathyAug 6thJanssen Research & Development, LLCNCT05327114
Added 1 new site: Buenos Aires, Argentina

Added 1 new site: Buenos Aires, Argentina

Enrollment UpdateChronic Granulomatous DiseaseAug 6thNational Institute of Allergy and Infectious Diseases (NIAID)NCT06325709
Target enrollment increased from 10 to 15

Target enrollment increased from 10 to 15

eligibility_changeChronic Granulomatous DiseaseAug 6thNational Institute of Allergy and Infectious Diseases (NIAID)NCT06325709
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaAug 6thServier Affaires MédicalesNCT05876754
Target enrollment increased from 220 to 299

Target enrollment increased from 220 to 299

sites_addedCholangiocarcinomaAug 6thiOMEDICO AGNCT06607302
Added 3 new sites: Augsburg, Germany, Berlin, Germany, Passau, Germany

Added 3 new sites: Augsburg, Germany, Berlin, Germany, Passau, Germany

sites_addedC3 GlomerulopathyAug 5theleva GmbHNCT07483827
Added 1 new site: Belgrade, Serbia

Added 1 new site: Belgrade, Serbia

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Aug 5thEidos Therapeutics, a BridgeBio companyNCT06563895
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

sites_addedAmyotrophic Lateral SclerosisAug 5thSt. Joseph's Hospital and Medical Center, PhoenixNCT06578195
Added 5 new sites: Los Angeles, California, Davie, Florida, Chicago, Illinois and 2 more

Added 5 new sites: Los Angeles, California, Davie, Florida, Chicago, Illinois and 2 more

eligibility_changeAmyotrophic Lateral SclerosisAug 5thUniversity of MinnesotaNCT06735014
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateAmyotrophic Lateral SclerosisAug 5thNational Center of Neurology and Psychiatry, JapanNCT07257302
Target enrollment increased from 15 to 25

Target enrollment increased from 15 to 25

sites_addedAchondroplasiaAug 5thTyra Biosciences, IncNCT06842355
Added 2 new sites: London, United Kingdom, Sheffield, United Kingdom

Added 2 new sites: London, United Kingdom, Sheffield, United Kingdom

sites_addedSystemic MastocytosisAug 4thBlueprint Medicines CorporationNCT04910685
Added 4 new sites: Cali, Colombia, Maastricht, Netherlands, Krakow, Poland and 1 more

Added 4 new sites: Cali, Colombia, Maastricht, Netherlands, Krakow, Poland and 1 more

sites_addedSystemic MastocytosisAug 4thBlueprint Medicines CorporationNCT07264959
Added 2 new sites: Wheaton, Maryland, Ann Arbor, Michigan

Added 2 new sites: Wheaton, Maryland, Ann Arbor, Michigan

sites_addedStargardt DiseaseAug 4thCellio Therapeutics IncNCT07594236
Added 2 new sites: Palo Alto, California, Sacramento, California

Added 2 new sites: Palo Alto, California, Sacramento, California

sites_addedWaldenström MacroglobulinemiaAug 4thNational Cancer Institute (NCI)NCT04840602
Added 2 new sites: Detroit, Michigan, Farmington Hills, Michigan

Added 2 new sites: Detroit, Michigan, Farmington Hills, Michigan

sites_addedStargardt DiseaseAug 4thAAVantgarde Bio SrlNCT07161544
Added 1 new site: Jacksonville, Florida

Added 1 new site: Jacksonville, Florida

sites_addedSpinal Muscular AtrophyAug 4thBiogenNCT07221669
Added 2 new sites: Distrito Federal, Brazil, Guangzhou, China

Added 2 new sites: Distrito Federal, Brazil, Guangzhou, China

Enrollment UpdateSickle Cell DiseaseAug 4thUniversity of CincinnatiNCT06725550
Target enrollment decreased from 120 to 10

Target enrollment decreased from 120 to 10

Enrollment UpdateSickle Cell DiseaseAug 4thIndiana UniversityNCT05045820
Target enrollment increased from 60 to 106

Target enrollment increased from 60 to 106

sites_addedSickle Cell DiseaseAug 4thDisc Medicine, IncNCT07187973
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

Enrollment UpdateSickle Cell DiseaseAug 4thIndiana UniversityNCT06511453
Target enrollment decreased from 300 to 59

Target enrollment decreased from 300 to 59

sites_addedPulmonary Arterial HypertensionAug 3rdDiagonal Therapeutics, Inc.NCT07623525
Added 7 new sites: Camperdown, Australia, Herston, Australia, Herston, Australia and 4 more

Added 7 new sites: Camperdown, Australia, Herston, Australia, Herston, Australia and 4 more

sites_addedProgressive Familial Intrahepatic CholestasisAug 3rdIpsenNCT07588880
Added 2 new sites: Nanjing, China, Xiamen, China

Added 2 new sites: Nanjing, China, Xiamen, China

sites_addedPrimary Sclerosing CholangitisAug 3rdIpsenNCT07387549
Added 2 new sites: Houston, Texas, Seattle, Washington

Added 2 new sites: Houston, Texas, Seattle, Washington

sites_addedPrimary Biliary CholangitisAug 3rdIpsenNCT06447168
Added 6 new sites: Camperdown, Australia, Busan, South Korea, Seongnam-si, South Korea and 3 more

Added 6 new sites: Camperdown, Australia, Busan, South Korea, Seongnam-si, South Korea and 3 more

Enrollment UpdateShort Bowel SyndromeAug 3rdZealand PharmaNCT07228403
Target enrollment decreased from 35 to 28

Target enrollment decreased from 35 to 28

Enrollment UpdateRetinitis PigmentosaAug 3rdjCyte, IncNCT06912633
Target enrollment decreased from 60 to 31

Target enrollment decreased from 60 to 31

sites_removedRetinitis PigmentosaAug 3rdjCyte, IncNCT06912633
Reduced from 14 to 12 sites

Reduced from 14 to 12 sites

sites_addedPulmonary Arterial HypertensionAug 3rdMerck Sharp & Dohme LLCNCT07218029
Added 1 new site: Washington D.C., District of Columbia

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

sites_addedPulmonary Arterial HypertensionAug 3rdGossamer Bio USA, Inc.NCT06274801
Added 5 new sites: Anderlecht, Belgium, Dresden, Germany, Rotterdam, Netherlands and 2 more

Added 5 new sites: Anderlecht, Belgium, Dresden, Germany, Rotterdam, Netherlands and 2 more

sites_addedProgressive Supranuclear PalsyAug 3rdNovartis PharmaceuticalsNCT07498426
Added 16 new sites: Scottsdale, Arizona, Westmead, Australia, Shanghai, China and 13 more

Added 16 new sites: Scottsdale, Arizona, Westmead, Australia, Shanghai, China and 13 more

sites_addedPrimary Sclerosing CholangitisAug 3rdAssistance Publique - Hôpitaux de ParisNCT06675604
Added 33 new sites: Brussels, Belgium, Leuven, Belgium, Leuven, Belgium and 30 more

Added 33 new sites: Brussels, Belgium, Leuven, Belgium, Leuven, Belgium and 30 more

eligibility_changePrimary Biliary CholangitisAug 3rdXijing Hospital of Digestive DiseasesNCT07104201
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPrimary Biliary CholangitisAug 3rdAssistance Publique - Hôpitaux de ParisNCT06675604
Added 33 new sites: Brussels, Belgium, Leuven, Belgium, Leuven, Belgium and 30 more

Added 33 new sites: Brussels, Belgium, Leuven, Belgium, Leuven, Belgium and 30 more

sites_addedPolycythemia VeraAug 3rdPrelude Therapeutics IncorporatedNCT07469891
Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

sites_addedParoxysmal Nocturnal HemoglobinuriaAug 3rdRegeneron PharmaceuticalsNCT05744921
Added 1 new site: Changhua, Taiwan

Added 1 new site: Changhua, Taiwan

sites_addedNoonan SyndromeAug 2ndAscendis Pharma A/SNCT07221851
Added 3 new sites: Paris, France, Florence, Italy, Târgu Mureş, Romania

Added 3 new sites: Paris, France, Florence, Italy, Târgu Mureş, Romania

sites_addedNarcolepsy Type 1Aug 2ndCentessa Pharmaceuticals (UK) LimitedNCT07598708
Added 3 new sites: Colorado Springs, Colorado, Flowood, Mississippi, Dublin, Ohio

Added 3 new sites: Colorado Springs, Colorado, Flowood, Mississippi, Dublin, Ohio

sites_addedMyotonic DystrophyAug 2ndPepGen IncNCT06667453
Added 4 new sites: Pusan, South Korea, Daegu, South Korea, Seoul, South Korea and 1 more

Added 4 new sites: Pusan, South Korea, Daegu, South Korea, Seoul, South Korea and 1 more

sites_addedMyotonic DystrophyAug 2ndDyne TherapeuticsNCT07486934
Added 5 new sites: Salt Lake City, Utah, Marseille, France, Bonn, Germany and 2 more

Added 5 new sites: Salt Lake City, Utah, Marseille, France, Bonn, Germany and 2 more

sites_addedMyelofibrosisAug 2ndPrelude Therapeutics IncorporatedNCT07469891
Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

eligibility_changeMyelofibrosisAug 2ndMemorial Sloan Kettering Cancer CenterNCT06773195
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMyelodysplastic SyndromesAug 2ndSawa Ito, MDNCT06529731
Target enrollment increased from 45 to 57

Target enrollment increased from 45 to 57

eligibility_changeMyelodysplastic SyndromesAug 2ndSawa Ito, MDNCT06529731
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesAug 2ndJanssen Research & Development, LLCNCT06788509
Added 3 new sites: Macquarie University, Australia, Haifa, Israel, Tel Aviv, Israel

Added 3 new sites: Macquarie University, Australia, Haifa, Israel, Tel Aviv, Israel

Enrollment UpdateMyelodysplastic SyndromesAug 2ndUniversity of NebraskaNCT04898790
Target enrollment increased from 88 to 108

Target enrollment increased from 88 to 108

eligibility_changeMyelodysplastic SyndromesAug 2ndCity of Hope Medical CenterNCT06287944
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesAug 2ndNational Cancer Institute (NCI)NCT03816319
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesAug 2ndNational Cancer Institute (NCI)NCT05554406
Added 5 new sites: Oakland, California, Roseville, California, Santa Clara, California and 2 more

Added 5 new sites: Oakland, California, Roseville, California, Santa Clara, California and 2 more

eligibility_changeMyelodysplastic SyndromesAug 2ndNational Cancer Institute (NCI)NCT05554406
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesAug 2ndM.D. Anderson Cancer CenterNCT06616636
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

Enrollment UpdateMyelodysplastic SyndromesAug 2ndChildren's Oncology GroupNCT05457556
Target enrollment decreased from 435 to 66

Target enrollment decreased from 435 to 66

eligibility_changeMyelodysplastic SyndromesAug 2ndNational Cancer Institute (NCI)NCT06577441
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisAug 2ndargenxNCT07294170
Added 10 new sites: Boston, Massachusetts, Chattanooga, Tennessee, Berlin, Germany and 7 more

Added 10 new sites: Boston, Massachusetts, Chattanooga, Tennessee, Berlin, Germany and 7 more

sites_addedMyasthenia GravisAug 2ndCartesian TherapeuticsNCT07089121
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

sites_addedMyasthenia GravisAug 2ndJanssen Research & Development, LLCNCT07217587
Added 2 new sites: New York, New York, Cordova, Tennessee

Added 2 new sites: New York, New York, Cordova, Tennessee

sites_addedMyasthenia GravisAug 2ndNovartis PharmaceuticalsNCT06744920
Added 4 new sites: Boston, Massachusetts, São Paulo, Brazil, Chiba, Japan and 1 more

Added 4 new sites: Boston, Massachusetts, São Paulo, Brazil, Chiba, Japan and 1 more

eligibility_changeMyasthenia GravisAug 2ndNovartis PharmaceuticalsNCT06744920
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisAug 2ndImmunovant Sciences GmbHNCT07039916
Added 4 new sites: Houston, Texas, Caba, Argentina, Mexico City, Mexico and 1 more

Added 4 new sites: Houston, Texas, Caba, Argentina, Mexico City, Mexico and 1 more

sites_addedMyasthenia GravisAug 2ndargenxNCT07284420
Added 9 new sites: Boston, Massachusetts, Berlin, Germany, Athens, Greece and 6 more

Added 9 new sites: Boston, Massachusetts, Berlin, Germany, Athens, Greece and 6 more

sites_addedMultiple System AtrophyAug 2ndTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 1 new site: Valencia, Spain

Added 1 new site: Valencia, Spain

sites_addedMultiple SclerosisAug 2ndHoffmann-La RocheNCT05269004
Added 4 new sites: Seattle, Washington, Calgary, Canada, Saint-Herblain, France and 1 more

Added 4 new sites: Seattle, Washington, Calgary, Canada, Saint-Herblain, France and 1 more

sites_addedMultiple SclerosisAug 2ndNovartis PharmaceuticalsNCT07225504
Added 20 new sites: Dallas, Texas, CABA, Argentina, San Juan, Argentina and 17 more

Added 20 new sites: Dallas, Texas, CABA, Argentina, San Juan, Argentina and 17 more

sites_addedMultiple SclerosisAug 2ndNovartis PharmaceuticalsNCT05344469
Added 3 new sites: Siegen, Germany, Görlitz, Germany, Oldenburg, Germany

Added 3 new sites: Siegen, Germany, Görlitz, Germany, Oldenburg, Germany

sites_removedMultiple SclerosisAug 2ndHoffmann-La RocheNCT04548999
Reduced from 149 to 147 sites

Reduced from 149 to 147 sites

sites_addedMultiple SclerosisAug 2ndZenas BioPharma (USA), LLCNCT07067463
Added 17 new sites: Pleven, Bulgaria, Zagreb, Croatia, Tbilisi, Georgia and 14 more

Added 17 new sites: Pleven, Bulgaria, Zagreb, Croatia, Tbilisi, Georgia and 14 more

sites_addedMultiple SclerosisAug 2ndTG Therapeutics, Inc.NCT07503873
Added 2 new sites: Shtip, North Macedonia, Skopje, North Macedonia

Added 2 new sites: Shtip, North Macedonia, Skopje, North Macedonia

sites_addedMultiple SclerosisAug 2ndZenas BioPharma (USA), LLCNCT07299019
Added 5 new sites: Naples, Florida, Nowa Sól, Poland, Rzeszów, Poland and 2 more

Added 5 new sites: Naples, Florida, Nowa Sól, Poland, Rzeszów, Poland and 2 more

eligibility_changeMastocytosisAug 2ndNational Institute of Allergy and Infectious Diseases (NIAID)NCT00044122
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyAug 1stAlexion Pharmaceuticals, Inc.NCT07024563
Added 1 new site: Almería, Spain

Added 1 new site: Almería, Spain

Enrollment UpdateIgA NephropathyAug 1stAlexion Pharmaceuticals, Inc.NCT07024563
Target enrollment increased from 24 to 36

Target enrollment increased from 24 to 36

eligibility_changeIgA NephropathyAug 1stAlexion Pharmaceuticals, Inc.NCT07024563
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyAug 1stNovartis PharmaceuticalsNCT06797518
Added 1 new site: Kuala Lumpur, Malaysia

Added 1 new site: Kuala Lumpur, Malaysia

sites_addedIgA NephropathyAug 1stADARx Pharmaceuticals, Inc.NCT06989359
Added 5 new sites: Bergamo, Italy, Pavia, Italy, Gyeonggi-do, South Korea and 2 more

Added 5 new sites: Bergamo, Italy, Pavia, Italy, Gyeonggi-do, South Korea and 2 more

eligibility_changeIdiopathic Pulmonary FibrosisAug 1stBoehringer IngelheimNCT07036523
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisAug 1stContineum TherapeuticsNCT07284459
Added 4 new sites: Rosario, Argentina, Rosario, Argentina, San Miguel de Tucumán, Argentina and 1 more

Added 4 new sites: Rosario, Argentina, Rosario, Argentina, San Miguel de Tucumán, Argentina and 1 more

eligibility_changeIdiopathic Pulmonary FibrosisAug 1stNational Institute of Environmental Health Sciences (NIEHS)NCT06325696
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedIdiopathic Pulmonary FibrosisAug 1stUnited TherapeuticsNCT04905693
Reduced from 194 to 192 sites

Reduced from 194 to 192 sites

sites_addedHypoparathyroidismAug 1stAscendis Pharma A/SNCT07706764
Added 2 new sites: Bron, France, Le Kremlin-Bicêtre, France

Added 2 new sites: Bron, France, Le Kremlin-Bicêtre, France

sites_addedHuntington DiseaseAug 1stNovartis PharmaceuticalsNCT07326709
Added 10 new sites: Stanford, California, Detroit, Michigan, Chapel Hill, North Carolina and 7 more

Added 10 new sites: Stanford, California, Detroit, Michigan, Chapel Hill, North Carolina and 7 more

sites_addedHodgkin LymphomaAug 1stCompass TherapeuticsNCT06150664
Added 1 new site: Fort Wayne, Indiana

Added 1 new site: Fort Wayne, Indiana

sites_addedHodgkin LymphomaAug 1stUniversity of Southern CaliforniaNCT07259304
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

Enrollment UpdateHodgkin LymphomaAug 1stUniversity of Southern CaliforniaNCT07259304
Target enrollment decreased from 250 to 118

Target enrollment decreased from 250 to 118

eligibility_changeHodgkin LymphomaAug 1stUniversity of Southern CaliforniaNCT07259304
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary AngioedemaAug 1stAstria Therapeutics, Inc.NCT07204938
Added 13 new sites: Prague, Czechia, Paris, France, Ashkelon, Israel and 10 more

Added 13 new sites: Prague, Czechia, Paris, France, Ashkelon, Israel and 10 more