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Rare Disease Trial Updates: June 2026

In June 2026, Trial Friend tracked 466 rare disease clinical trial updates across 98 conditions, including 1 FDA-related actions and 71 trial status changes, alongside enrollment updates and new trial site openings sourced from ClinicalTrials.gov and FDA announcements.

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

Major Updates: FDA Actions, Status Changes & Results

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

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

Status ChangeSpinocerebellar AtaxiaJun 30thArrowhead PharmaceuticalsNCT06672445
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaJun 29thBeacon TherapeuticsNCT07174726
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

No longer enrolling new participants

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

Now recruiting (was Enrolling By Invitation)

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

No longer enrolling new participants

Status ChangeC3 GlomerulopathyJun 24thNovartis PharmaceuticalsNCT07156149
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

No longer enrolling new participants

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

Now recruiting (was Enrolling By Invitation)

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

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 21stNovartis PharmaceuticalsNCT06551519
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeThalassemiaJun 16thLantu BiopharmaNCT06308159
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 14thAmgenNCT06700343
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaJun 14thAstraZenecaNCT06097728
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaJun 14thHyogo Medical UniversityNCT06318286
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeTuberous SclerosisJun 9thJazz PharmaceuticalsNCT05485831
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeMultiple SclerosisJun 7thBiogenNCT05688436
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeMesotheliomaJun 7thUdai KammulaNCT03935893
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeIgA NephropathyJun 6thNovartis PharmaceuticalsNCT04557462
No longer enrolling new participants

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

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

Status changed from Recruiting to Enrolling By Invitation

Status ChangeDravet SyndromeJun 4thJazz PharmaceuticalsNCT05485831
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

Status ChangeCholangiocarcinomaJun 4thUdai KammulaNCT03801083
No longer enrolling new participants

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

No longer enrolling new participants

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

Now recruiting (was Enrolling By Invitation)

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

No longer enrolling new participants

Status ChangeRetinitis PigmentosaJun 1stPYC TherapeuticsNCT06852963
No longer enrolling new participants

No longer enrolling new participants

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

Now recruiting (was Active Not Recruiting)

Status ChangeOsteogenesis ImperfectaJun 1stAmgenNCT05972551
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

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

Added 1 new site: Maywood, Illinois

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

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

eligibility_changeVon Willebrand DiseaseJun 30thHemab ApSNCT06754852
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Added 1 new site: Greenville, North Carolina

sites_addedWild-Type ATTR Cardiac AmyloidosisJun 30thBayerNCT07298044
Added 13 new sites: Feldkirch, Austria, Brussels, Belgium, Bruges, Belgium and 10 more

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

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

Added 1 new site: Coon Rapids, Minnesota

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

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

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

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

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

Eligibility criteria have been updated

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

Eligibility criteria have been updated

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

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

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

Eligibility criteria have been updated

sites_removedParoxysmal Nocturnal HemoglobinuriaJun 29thAstraZenecaNCT07457151
Reduced from 5 to 2 sites

Reduced from 5 to 2 sites

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

Eligibility criteria have been updated

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

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

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

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

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

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

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

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

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

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

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

Added 1 new site: Genova, Italy

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

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

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

Eligibility criteria have been updated

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

Eligibility criteria have been updated

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

Reduced from 21 to 16 sites

sites_addedMyelofibrosisJun 28thiOncturaNCT06887803
Added 1 new site: Meldola, Italy

Added 1 new site: Meldola, Italy

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

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

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

Added 1 new site: Duarte, California

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

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

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

Target enrollment increased from 20 to 40

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

Eligibility criteria have been updated

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

Added 1 new site: Memphis, Tennessee

eligibility_changeMyelodysplastic SyndromesJun 28thEllipses PharmaNCT04581512
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

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

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

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

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

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

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

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

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

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

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

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

Reduced from 108 to 106 sites

sites_addedMultiple SclerosisJun 28thSanofiNCT07325292
Added 2 new sites: Columbus, Ohio, Beijing, China

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

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

Added 1 new site: Livingston, New Jersey

eligibility_changeMultiple SclerosisJun 28thUniversity of PatrasNCT07630311
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

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

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

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

Target enrollment decreased from 154 to 27

eligibility_changeMesotheliomaJun 28thHospices Civils de LyonNCT06790706
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

eligibility_changeMesotheliomaJun 28thNovartis PharmaceuticalsNCT04104776
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Eligibility criteria have been updated

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

Added 1 new site: Paris, France

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

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

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

Added 1 new site: Chicago, Illinois

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

Added 1 new site: Boston, Massachusetts

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

Added 1 new site: Barcelona, Spain

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

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

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

Target enrollment increased from 476 to 750

sites_addedIgA NephropathyJun 27thBiogenNCT06935357
Added 2 new sites: Bogotá, Colombia, London, United Kingdom

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

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

Reduced from 267 to 246 sites

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

Target enrollment increased from 510 to 579

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

Eligibility criteria have been updated

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

Target enrollment increased from 200 to 250

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

Eligibility criteria have been updated

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

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

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

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

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

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

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

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

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

Eligibility criteria have been updated

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

Reduced from 24 to 21 sites

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

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

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

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

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

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

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

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

sites_removedGranulomatosis with PolyangiitisJun 26thGlaxoSmithKlineNCT05263934
Reduced from 76 to 63 sites

Reduced from 76 to 63 sites

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

Added 1 new site: London, United Kingdom

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

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

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

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

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

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

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

Added 1 new site: Colorado Springs, Colorado

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

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

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

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

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

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

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

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

sites_removedEosinophilic Granulomatosis with PolyangiitisJun 25thGlaxoSmithKlineNCT05263934
Reduced from 76 to 63 sites

Reduced from 76 to 63 sites

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

Eligibility criteria have been updated

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

Added 1 new site: St Louis, Missouri

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

Eligibility criteria have been updated

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

Added 1 new site: Cordova, Tennessee

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

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJun 24thPrileniaNCT07322003
Added 4 new sites: Jerusalem, Israel, Tel Aviv, Israel, Santiago de Compostela, Spain and 1 more

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

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

Target enrollment decreased from 50 to 32

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jun 24thAstraZenecaNCT06465810
Added 7 new sites: Wuhan, China, Nanjing, China, Suzhou, China and 4 more

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

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

Eligibility criteria have been updated

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

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

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

Added 1 new site: Umeå, Sweden

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

Target enrollment increased from 42 to 54

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

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

eligibility_changeAmyotrophic Lateral SclerosisJun 24thNura BioNCT07369076
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJun 24thPrileniaNCT07322003
Added 8 new sites: San Francisco, California, Chicago, Illinois, St Louis, Missouri and 5 more

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

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

Added 1 new site: New York, New York

sites_addedWild-Type ATTR Cardiac AmyloidosisJun 23rdAstraZenecaNCT06465810
Added 7 new sites: Wuhan, China, Nanjing, China, Suzhou, China and 4 more

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

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

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

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

Added 1 new site: Salt Lake City, Utah

sites_removedWaldenström MacroglobulinemiaJun 23rdBeOne MedicinesNCT05006716
Reduced from 130 to 127 sites

Reduced from 130 to 127 sites

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

Added 1 new site: Belo Horizonte, Brazil

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

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

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

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

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

Added 1 new site: Lahore, Pakistan

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

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

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

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

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

Added 1 new site: Porto Alegre, Brazil

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

Reduced from 5 to 3 sites

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

Target enrollment decreased from 20 to 9

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

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

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

Added 1 new site: Belo Horizonte, Brazil

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

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

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

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

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

Added 1 new site: Tours, France

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

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

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

Added 1 new site: London, United Kingdom

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

Added 1 new site: Seoul, South Korea

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

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

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

Added 1 new site: Lahore, Pakistan

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

Added 1 new site: Lahore, Pakistan

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

Added 1 new site: Lahore, Pakistan

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

Added 1 new site: Lahore, Pakistan

sites_removedMyelofibrosisJun 21stGeron CorporationNCT04576156
Reduced from 174 to 144 sites

Reduced from 174 to 144 sites

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

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJun 21stTakedaNCT07422480
Added 43 new sites: Gilbert, Arizona, Duarte, California, Santa Rosa, California and 40 more

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

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

Eligibility criteria have been updated

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

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

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

Added 1 new site: Schaumburg, Illinois

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

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

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

Added 1 new site: Sendai, Japan

sites_removedMultiple SclerosisJun 21stNovartis PharmaceuticalsNCT06551519
Reduced from 109 to 89 sites

Reduced from 109 to 89 sites

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

Target enrollment decreased from 700 to 489

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

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

sites_addedMembranous NephropathyJun 21stBiogenNCT06962800
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

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

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

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

Eligibility criteria have been updated

sites_addedIgA NephropathyJun 20thBiogenNCT06935357
Added 4 new sites: St Leonards, Australia, Guangzhou, China, Wuhan, China and 1 more

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

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

Eligibility criteria have been updated

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

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

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

Eligibility criteria have been updated

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

Added 1 new site: Pittsburgh, Pennsylvania

eligibility_changeHemophilia BJun 19thCSL BehringNCT06003387
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

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

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

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

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

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

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

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

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

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

Eligibility criteria have been updated

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

Added 1 new site: Tbilisi, Georgia

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

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

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

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

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

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

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

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

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

Eligibility criteria have been updated

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

Added 1 new site: Bethesda, Maryland

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

Added 1 new site: San Francisco, California

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

Added 1 new site: Palo Alto, California

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

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

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

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

sites_addedDermatomyositisJun 18thargenxNCT05979441
Added 2 new sites: Guangzhou, China, Taichung, Taiwan

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

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

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

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

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

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18thSanofiNCT06859099
Added 2 new sites: Bunkyo-Ku, Japan, Konya, Turkey (Türkiye)

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

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18thSanofiNCT06290141
Added 5 new sites: Centennial, Colorado, Miami Lakes, Florida, Lille, France and 2 more

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

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18thargenxNCT06920004
Added 2 new sites: Paris, France, Salford, United Kingdom

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

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18thSanofiNCT06290128
Added 4 new sites: Miami Lakes, Florida, Belo Horizonte, Brazil, Bunkyo-Ku, Japan and 1 more

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

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 18thargenxNCT07091630
Added 5 new sites: Birmingham, Alabama, Rosario, Argentina, Lille, France and 2 more

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

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

Target enrollment increased from 241 to 296

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

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

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

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

eligibility_changeAmyotrophic Lateral SclerosisJun 17thCorcept TherapeuticsNCT05407324
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Added 1 new site: Ann Arbor, Michigan

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

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

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

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

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

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

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

Added 1 new site: Philadelphia, Pennsylvania

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

Eligibility criteria have been updated

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

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

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

Eligibility criteria have been updated

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

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

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

Added 1 new site: Santa Barbara, California

sites_removedPolycythemia VeraJun 15thNovartis PharmaceuticalsNCT05548062
Reduced from 35 to 33 sites

Reduced from 35 to 33 sites

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

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

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

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

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

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

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

Added 1 new site: Atlanta, Georgia

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

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

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

Added 1 new site: Huntersville, North Carolina

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

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

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

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

eligibility_changeMyelodysplastic SyndromesJun 14thMayo ClinicNCT03017820
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Added 1 new site: Winston-Salem, North Carolina

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

Target enrollment decreased from 180 to 82

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

Target enrollment decreased from 40 to 10

sites_addedMyasthenia GravisJun 14thargenxNCT07294170
Added 3 new sites: Detroit, Michigan, Durham, North Carolina, Milan, Italy

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

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

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

sites_addedMyasthenia GravisJun 14thargenxNCT07284420
Added 2 new sites: Detroit, Michigan, Durham, North Carolina

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

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

Added 1 new site: Palo Alto, California

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

Eligibility criteria have been updated

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

Added 1 new site: Kassel, Germany

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

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

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

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

Enrollment UpdateMultiple SclerosisJun 14thAmgenNCT06700343
Target enrollment decreased from 444 to 152

Target enrollment decreased from 444 to 152

sites_addedMultiple SclerosisJun 14thNovartis PharmaceuticalsNCT05344469
Expanded from 127 to 129 sites

Expanded from 127 to 129 sites

eligibility_changeMultiple SclerosisJun 14thJohns Hopkins UniversityNCT06170970
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Eligibility criteria have been updated

sites_removedMesotheliomaJun 14thAstraZenecaNCT06097728
Reduced from 178 to 151 sites

Reduced from 178 to 151 sites

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

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

eligibility_changeMesotheliomaJun 14thIDEAYA BiosciencesNCT07277413
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaJun 14thAstraZenecaNCT07115043
Expanded from 11 to 13 sites

Expanded from 11 to 13 sites

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

Added 1 new site: Kyoto, Japan

sites_addedMembranous NephropathyJun 14thBiogenNCT06962800
Added 5 new sites: Cleveland, Ohio, London, Canada, Xi'an, China and 2 more

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

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

Reduced from 12 to 9 sites

sites_addedIgA NephropathyJun 13thBiogenNCT06935357
Added 1 new site: Herston, Australia

Added 1 new site: Herston, Australia

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

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

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

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

eligibility_changeIdiopathic Pulmonary FibrosisJun 13thMediar TherapeuticsNCT06967805
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

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

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

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

Added 1 new site: Barcelona, Spain

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

Target enrollment decreased from 44 to 24

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

Added 1 new site: Chicago, Illinois

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

Added 1 new site: Dublin, Ireland

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

Eligibility criteria have been updated

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

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

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

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

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

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

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

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

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

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

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

Moved from Phase 1 to Phase 2

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

Added 1 new site: Poitiers, France

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

Target enrollment increased from 20 to 50

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

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

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

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

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

Added 1 new site: Southport, Australia

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

Added 1 new site: Sunderland, United Kingdom

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

Target enrollment decreased from 1000 to 500

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

Eligibility criteria have been updated

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

Eligibility criteria have been updated

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

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

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

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

sites_addedFocal Segmental GlomerulosclerosisJun 12thSanofiNCT06500702
Added 2 new sites: Atlanta, Georgia, São Paulo, Brazil

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

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

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

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

Added 1 new site: Bentonville, Arkansas

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

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

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

Added 1 new site: Nashville, Tennessee

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

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

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

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

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

Added 1 new site: Palo Alto, California

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

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

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

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

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

Added 1 new site: Atlanta, Georgia

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

Added 1 new site: Ankara, Turkey (Türkiye)

sites_addedAmyotrophic Lateral SclerosisJun 10thPrileniaNCT07322003
Added 11 new sites: Florida City, Florida, Atlanta, Georgia, Philadelphia, Pennsylvania and 8 more

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

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

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

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

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

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

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

sites_addedSpinal Muscular AtrophyJun 9thBiogenNCT07221669
Added 2 new sites: Chicago, Illinois, Shanghai, China

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

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

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

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

Added 1 new site: Boston, Massachusetts

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

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

eligibility_changeWaldenström MacroglobulinemiaJun 9thBeOne MedicinesNCT05006716
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Added 1 new site: Dallas, Texas

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

Added 1 new site: Cincinnati, Ohio

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

Added 1 new site: Bethesda, Maryland

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

Eligibility criteria have been updated

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

Added 1 new site: Barranquilla, Colombia

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

Added 1 new site: Dresden, Germany

eligibility_changePrimary Sclerosing CholangitisJun 8thIpsenNCT05627362
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Target enrollment increased from 116 to 166

eligibility_changePolycystic Kidney DiseaseJun 8thPYC TherapeuticsNCT06714006
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Eligibility criteria have been updated

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

Eligibility criteria have been updated

sites_addedPrimary Biliary CholangitisJun 8thIpsenNCT06016842
Added 3 new sites: Lodz, Poland, Hat Yai, Thailand, Khon Kaen, Thailand

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

eligibility_changePrimary Biliary CholangitisJun 8thIpsenNCT06016842
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Added 1 new site: Kurashiki, Japan

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

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

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

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

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

Eligibility criteria have been updated

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

Target enrollment increased from 60 to 76

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

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

eligibility_changeMyotonic DystrophyJun 7thSanofiNCT06844214
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Target enrollment increased from 500 to 1000

eligibility_changeMyotonic DystrophyJun 7thHuashan HospitalNCT06101940
Eligibility criteria have been updated

Eligibility criteria have been updated

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

Added 1 new site: Columbia, Missouri

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

Added 1 new site: Maastricht, Netherlands

eligibility_changeMyelofibrosisJun 7thUniversity of UtahNCT05535764
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

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

Added 1 new site: Kurashiki, Japan

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

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

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

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

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

Target enrollment decreased from 255 to 155

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

Eligibility criteria have been updated

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

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

eligibility_changeMyelodysplastic SyndromesJun 7thGlaxoSmithKlineNCT06847867
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

sites_addedMyasthenia GravisJun 7thargenxNCT07284420
Added 3 new sites: Tampa, Florida, Columbus, Ohio, San Antonio, Texas

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

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

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

sites_addedMyasthenia GravisJun 7thargenxNCT07294170
Added 5 new sites: Tampa, Florida, Columbus, Ohio, Chattanooga, Tennessee and 2 more

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

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

Added 1 new site: Madrid, Spain

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

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

eligibility_changeMultiple SclerosisJun 7thBiogenNCT05688436
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

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

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

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

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

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

Added 1 new site: Miami, Florida

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

Target enrollment decreased from 240 to 20

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

Added 1 new site: Philadelphia, Pennsylvania

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

Added 1 new site: Durham, North Carolina

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

Eligibility criteria have been updated

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

Target enrollment decreased from 240 to 20

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

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

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

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

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

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

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

Target enrollment decreased from 50 to 26

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

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

sites_removedIgA NephropathyJun 6thNovartis PharmaceuticalsNCT04557462
Reduced from 161 to 155 sites

Reduced from 161 to 155 sites

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

Target enrollment decreased from 540 to 448

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

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

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

Added 1 new site: London, United Kingdom

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

Target enrollment increased from 12 to 24

eligibility_changeIgA NephropathyJun 6thUniversity of LeicesterNCT04663204
Eligibility criteria have been updated

Eligibility criteria have been updated

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

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

eligibility_changeIgA NephropathyJun 6thNovartis PharmaceuticalsNCT04573478
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJun 6thContineum TherapeuticsNCT07284459
Added 7 new sites: Westmead, Australia, Vancouver, Canada, Ramat Gan, Israel and 4 more

Added 7 new sites: Westmead, Australia, Vancouver, Canada, Ramat Gan, Israel and 4 more

sites_addedIdiopathic Pulmonary FibrosisJun 6thRein TherapeuticsNCT06968845
Added 5 new sites: Denver, Colorado, Weston, Florida, Chapel Hill, North Carolina and 2 more

Added 5 new sites: Denver, Colorado, Weston, Florida, Chapel Hill, North Carolina and 2 more

sites_addedHuntington DiseaseJun 6thSkyhawk Therapeutics, Inc.NCT07378644
Added 5 new sites: CABA, Argentina, CABA, Argentina, CABA, Argentina and 2 more

Added 5 new sites: CABA, Argentina, CABA, Argentina, CABA, Argentina and 2 more

sites_addedHunter SyndromeJun 6thJCR Pharmaceuticals Co., Ltd.NCT04573023
Added 2 new sites: Lyon, France, Montpellier, France

Added 2 new sites: Lyon, France, Montpellier, France

sites_addedHereditary AngioedemaJun 6thAstria Therapeutics, Inc.NCT07204938
Added 18 new sites: Colorado Springs, Colorado, Wheaton, Maryland, Santo André, Brazil and 15 more

Added 18 new sites: Colorado Springs, Colorado, Wheaton, Maryland, Santo André, Brazil and 15 more

sites_addedHereditary AngioedemaJun 6thCSL BehringNCT07001280
Added 4 new sites: Miami, Florida, Louisville, Kentucky, Linz, Austria and 1 more

Added 4 new sites: Miami, Florida, Louisville, Kentucky, Linz, Austria and 1 more

sites_addedGeographic AtrophyJun 5thNovartis PharmaceuticalsNCT07441642
Added 6 new sites: Sofia, Bulgaria, Bonn, Germany, Milan, Italy and 3 more

Added 6 new sites: Sofia, Bulgaria, Bonn, Germany, Milan, Italy and 3 more

sites_addedFocal Segmental GlomerulosclerosisJun 5thAkebia TherapeuticsNCT07268638
Added 4 new sites: Los Angeles, California, San Dimas, California, Arvada, Colorado and 1 more

Added 4 new sites: Los Angeles, California, San Dimas, California, Arvada, Colorado and 1 more

sites_addedHemophilia BJun 5thPfizerNCT03861273
Added 1 new site: Hamilton, Pennsylvania

Added 1 new site: Hamilton, Pennsylvania

eligibility_changeHemophilia BJun 5thCSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia AJun 5thOctapharmaNCT03695978
Added 4 new sites: St Louis, Missouri, San Antonio, Texas, Chambéry, France and 1 more

Added 4 new sites: St Louis, Missouri, San Antonio, Texas, Chambéry, France and 1 more

sites_addedHemophilia AJun 5thPfizerNCT06703606
Added 3 new sites: Greenville, North Carolina, Greenville, North Carolina, Fatih, Turkey (Türkiye)

Added 3 new sites: Greenville, North Carolina, Greenville, North Carolina, Fatih, Turkey (Türkiye)

sites_addedGeographic AtrophyJun 5thNovartis PharmaceuticalsNCT07441642
Added 3 new sites: Santa Barbara, California, Prague, Czechia, Créteil, France

Added 3 new sites: Santa Barbara, California, Prague, Czechia, Créteil, France

sites_addedGeographic AtrophyJun 5thApellis Pharmaceuticals, Inc.NCT07215390
Added 25 new sites: Gilbert, Arizona, Huntington Beach, California, Oxnard, California and 22 more

Added 25 new sites: Gilbert, Arizona, Huntington Beach, California, Oxnard, California and 22 more

sites_addedGastrointestinal Stromal TumorJun 5thDeciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Genova, Italy

Added 1 new site: Genova, Italy

eligibility_changeFriedreich AtaxiaJun 5thBiogenNCT06953583
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeFocal EpilepsyJun 5thSupernus Pharmaceuticals, Inc.NCT06798896
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaJun 5thNovartis PharmaceuticalsNCT07340138
Added 1 new site: Kurashiki, Japan

Added 1 new site: Kurashiki, Japan

sites_addedEssential ThrombocythemiaJun 5thJubilant Therapeutics Inc.NCT07612280
Added 6 new sites: Sydney, Australia, Sydney, Australia, Adelaide, Australia and 3 more

Added 6 new sites: Sydney, Australia, Sydney, Australia, Adelaide, Australia and 3 more

sites_addedEssential ThrombocythemiaJun 5thNovartis PharmaceuticalsNCT07357727
Added 4 new sites: Clayton, Australia, Tianjin, China, Suwon, South Korea and 1 more

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

sites_addedCongenital Adrenal HyperplasiaJun 4thCrinetics Pharmaceuticals Inc.NCT07144163
Added 8 new sites: Los Angeles, California, Minneapolis, Minnesota, Milwaukee, Wisconsin and 5 more

Added 8 new sites: Los Angeles, California, Minneapolis, Minnesota, Milwaukee, Wisconsin and 5 more

sites_addedChronic Graft-versus-Host DiseaseJun 4thIncyte CorporationNCT06585774
Added 3 new sites: Chicago, Illinois, Bari, Italy, Catania, Italy

Added 3 new sites: Chicago, Illinois, Bari, Italy, Catania, Italy

sites_addedCholangiocarcinomaJun 4thEli Lilly and CompanyNCT07213791
Added 1 new site: Shanghai, China

Added 1 new site: Shanghai, China

sites_addedCholangiocarcinomaJun 4thInstitut für Klinische Krebsforschung IKF GmbH at Krankenhaus NordwestNCT07260175
Added 4 new sites: Essen, Germany, Giessen, Germany, Mainz, Germany and 1 more

Added 4 new sites: Essen, Germany, Giessen, Germany, Mainz, Germany and 1 more

sites_addedCholangiocarcinomaJun 4thContext Therapeutics Inc.NCT06756035
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

sites_addedDuchenne Muscular DystrophyJun 4thSolid Biosciences Inc.NCT07160634
Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Enrollment UpdateDilated CardiomyopathyJun 4thPeerbridge Health, IncNCT06699056
Target enrollment increased from 1500 to 2000

Target enrollment increased from 1500 to 2000

sites_addedDilated CardiomyopathyJun 4thKardigan, Inc.NCT07210723
Added 10 new sites: San Diego, California, Jacksonville, Florida, Chicago, Illinois and 7 more

Added 10 new sites: San Diego, California, Jacksonville, Florida, Chicago, Illinois and 7 more

sites_addedCystic FibrosisJun 4thArkansas Children's Hospital Research InstituteNCT07148739
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

eligibility_changeCystic FibrosisJun 4thGeorge SolomonNCT03161808
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCongenital Adrenal HyperplasiaJun 4thCrinetics Pharmaceuticals Inc.NCT07159841
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

eligibility_changeCongenital Adrenal HyperplasiaJun 4thCrinetics Pharmaceuticals Inc.NCT07159841
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJun 4thDianthus TherapeuticsNCT06858579
Added 1 new site: Manchester, United Kingdom

Added 1 new site: Manchester, United Kingdom

eligibility_changeCholangiocarcinomaJun 4thSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityNCT02757859
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaJun 4thUdai KammulaNCT03801083
Target enrollment decreased from 59 to 4

Target enrollment decreased from 59 to 4

sites_addedAlagille SyndromeJun 3rdMirum Pharmaceuticals, Inc.NCT07290257
Added 2 new sites: Athens, Greece, Coimbra, Portugal

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

Enrollment UpdateAlagille SyndromeJun 3rdMirum Pharmaceuticals, Inc.NCT07290257
Target enrollment increased from 100 to 230

Target enrollment increased from 100 to 230

eligibility_changeAlagille SyndromeJun 3rdMirum Pharmaceuticals, Inc.NCT07290257
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedC3 GlomerulopathyJun 3rdNovartis PharmaceuticalsNCT07156149
Added 5 new sites: Fukuoka, Japan, Yokohama, Japan, Tosa-gun, Japan and 2 more

Added 5 new sites: Fukuoka, Japan, Yokohama, Japan, Tosa-gun, Japan and 2 more

sites_addedBecker Muscular DystrophyJun 3rdSolid Biosciences Inc.NCT07160634
Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Added 3 new sites: Little Rock, Arkansas, Flower Mound, Texas, Norfolk, Virginia

Enrollment UpdateATTR Amyloidosis (Transthyretin Amyloidosis)Jun 3rdSteen Hvitfeldt PoulsenNCT06318260
Target enrollment decreased from 35 to 15

Target enrollment decreased from 35 to 15

Enrollment UpdateAplastic AnemiaJun 3rdNational Heart, Lung, and Blood Institute (NHLBI)NCT05998408
Target enrollment decreased from 145 to 13

Target enrollment decreased from 145 to 13

sites_addedAmyotrophic Lateral SclerosisJun 3rdPhenoNet, Inc.NCT07142291
Added 1 new site: Seville, Spain

Added 1 new site: Seville, Spain

sites_addedAmyotrophic Lateral SclerosisJun 3rdLeigh R. Hochberg, MD, PhD.NCT00912041
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedAlagille SyndromeJun 3rdIpsenNCT06850038
Added 6 new sites: Los Angeles, California, San Francisco, California, St Louis, Missouri and 3 more

Added 6 new sites: Los Angeles, California, San Francisco, California, St Louis, Missouri and 3 more

sites_addedAchondroplasiaJun 3rdBioMarin PharmaceuticalNCT07441876
Added 7 new sites: Chicago, Illinois, Baltimore, Maryland, San Antonio, Texas and 4 more

Added 7 new sites: Chicago, Illinois, Baltimore, Maryland, San Antonio, Texas and 4 more

sites_addedSickle Cell DiseaseJun 2ndNovartis PharmaceuticalsNCT06546670
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedWaldenström MacroglobulinemiaJun 2ndAbbVieNCT07387471
Added 2 new sites: Hiroshima, Japan, Nagano, Japan

Added 2 new sites: Hiroshima, Japan, Nagano, Japan

sites_addedVon Willebrand DiseaseJun 2ndTakedaNCT05582993
Added 7 new sites: East Lansing, Michigan, Las Vegas, Nevada, Narita, Japan and 4 more

Added 7 new sites: East Lansing, Michigan, Las Vegas, Nevada, Narita, Japan and 4 more

sites_addedTurner SyndromeJun 2ndAscendis Pharma A/SNCT07221851
Added 1 new site: Sejong, South Korea

Added 1 new site: Sejong, South Korea

sites_addedStargardt DiseaseJun 2ndAAVantgarde Bio SrlNCT07161544
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedStargardt DiseaseJun 2ndAstellas Pharma Global Development, Inc.NCT07425574
Added 2 new sites: Sacramento, California, Portland, Oregon

Added 2 new sites: Sacramento, California, Portland, Oregon

sites_addedPulmonary Arterial HypertensionJun 1stMassachusetts General HospitalNCT07312227
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

Enrollment UpdatePulmonary Arterial HypertensionJun 1stMassachusetts General HospitalNCT07312227
Target enrollment increased from 22 to 27

Target enrollment increased from 22 to 27

eligibility_changePulmonary Arterial HypertensionJun 1stMassachusetts General HospitalNCT07312227
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJun 1stAllRock Bio, Inc.NCT07175038
Added 2 new sites: Auchenflower, Australia, Pavia, Italy

Added 2 new sites: Auchenflower, Australia, Pavia, Italy

sites_addedPulmonary Arterial HypertensionJun 1stMerck Sharp & Dohme LLCNCT07218029
Added 3 new sites: Scottsdale, Arizona, Edmonton, Canada, Giessen, Germany

Added 3 new sites: Scottsdale, Arizona, Edmonton, Canada, Giessen, Germany

sites_addedPulmonary Arterial HypertensionJun 1stRegeneron PharmaceuticalsNCT07318597
Added 3 new sites: Riga, Latvia, Namdong-gu, South Korea, Seoul, South Korea

Added 3 new sites: Riga, Latvia, Namdong-gu, South Korea, Seoul, South Korea

sites_addedProgressive Supranuclear PalsyJun 1stNovartis PharmaceuticalsNCT07498426
Added 3 new sites: Leipzig, Germany, Berlin, Germany, Stadtroda, Germany

Added 3 new sites: Leipzig, Germany, Berlin, Germany, Stadtroda, Germany

sites_addedPrimary Sclerosing CholangitisJun 1stIpsenNCT07387549
Added 1 new site: Chandler, Arizona

Added 1 new site: Chandler, Arizona

sites_addedPrimary Biliary CholangitisJun 1stGilead SciencesNCT06051617
Added 3 new sites: Birmingham, Alabama, Padova, Italy, Chiayi City, Taiwan

Added 3 new sites: Birmingham, Alabama, Padova, Italy, Chiayi City, Taiwan

sites_addedPrimary Biliary CholangitisJun 1stIpsenNCT06447168
Added 3 new sites: Fitzroy, Australia, Linz, Austria, Montreal, Canada

Added 3 new sites: Fitzroy, Australia, Linz, Austria, Montreal, Canada

sites_addedPolycythemia VeraJun 1stDisc Medicine, IncNCT06985147
Added 1 new site: Cleveland, Ohio

Added 1 new site: Cleveland, Ohio

sites_addedPolycythemia VeraJun 1stPrelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedPhenylketonuriaJun 1stAgios Pharmaceuticals, Inc.NCT07241234
Added 1 new site: Szczecin, Poland

Added 1 new site: Szczecin, Poland

sites_addedPhelan-McDermid SyndromeJun 1stNeuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Chicago, Illinois, Lexington, Massachusetts

Added 2 new sites: Chicago, Illinois, Lexington, Massachusetts

sites_addedParoxysmal Nocturnal HemoglobinuriaJun 1stRegeneron PharmaceuticalsNCT07154745
Added 2 new sites: Ribeirão Preto, Brazil, Istanbul, Turkey (Türkiye)

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