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Rare Disease Trial Updates: July 2026

In July 2026, Trial Friend tracked 517 rare disease clinical trial updates across 122 conditions, including 3 FDA-related actions and 127 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 ChangeCerebral AdrenoleukodystrophyJul 30thMinoryx Therapeutics, S.L.NCT05819866
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thAkcea TherapeuticsNCT04850105
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeJul 29thMavriX Bio, LLCNCT07181837
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeAmyotrophic Lateral SclerosisJul 29thZydus Therapeutics Inc.NCT07023835
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeSystemic MastocytosisJul 28thMacroGenicsNCT05362773
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 28thUniversity of California, San FranciscoNCT07064941
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 28thInstitute of Hematology & Blood Diseases Hospital, ChinaNCT06931912
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJul 28thCity of Hope Medical CenterNCT05384756
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyelofibrosisJul 26thPharmaEssentiaNCT06468033
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJul 26thOssium Health, Inc.NCT05589896
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesJul 26thUniversity of RochesterNCT03739502
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisJul 25thEuropean Myeloma Network B.V.NCT06649695
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgG4-Related DiseaseJul 25thStanford UniversityNCT07025330
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseJul 25thUniversity of VirginiaNCT07213648
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaJul 25thDavid Bond, MDNCT05821088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFacioscapulohumeral Muscular DystrophyJul 24thEpicrispr Biotechnologies, Inc.NCT06907875
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFriedreich AtaxiaJul 24thInstitut National de la Santé Et de la Recherche Médicale, FranceNCT05874388
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal Segmental GlomerulosclerosisJul 24thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06799520
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisJul 23rdMayo ClinicNCT07156357
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEhlers-Danlos SyndromeJul 23rdDeltaHealth Hospital · ShanghaiNCT07672210
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeDuchenne Muscular DystrophyJul 23rdREGENXBIO Inc.NCT05693142
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBecker Muscular DystrophyJul 22ndREGENXBIO Inc.NCT05693142
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisJul 22ndMerit E. Cudkowicz, MDNCT07410806
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisJul 22ndMerit E. Cudkowicz, MDNCT04297683
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 21stBioray LaboratoriesNCT05577312
No longer enrolling new participants

No longer enrolling new participants

Status ChangeVascular Ehlers-Danlos SyndromeJul 21stDeltaHealth Hospital · ShanghaiNCT07672210
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeSystemic Juvenile Idiopathic ArthritisJul 21stEli Lilly and CompanyNCT04088396
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionJul 20thUniversity of RochesterNCT06477640
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycystic Kidney DiseaseJul 20thGlaxoSmithKlineNCT06734234
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParoxysmal Nocturnal HemoglobinuriaJul 20thOmeros CorporationNCT06298955
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyotonic DystrophyJul 19thWake Forest University Health SciencesNCT05854433
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMyelofibrosisJul 19thKaren Ballen, MDNCT05233618
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 19thHoffmann-La RocheNCT07483450
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 19thEDMUS FoundationNCT05622643
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaJul 19thUNICANCERNCT05078047
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMarfan SyndromeJul 18thDeltaHealth Hospital · ShanghaiNCT07672210
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeLight Chain AmyloidosisJul 18thNexcella Inc.NCT06097832
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisJul 18thDaewoong Pharmaceutical Co. LTD.NCT05389215
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisJul 18thBoehringer IngelheimNCT06241560
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Hemorrhagic TelangiectasiaJul 18thAlnylam PharmaceuticalsNCT06659640
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaJul 17thMerck Sharp & Dohme LLCNCT06456346
No longer enrolling new participants

No longer enrolling new participants

FDA DesignationHomocystinuriaJul 17th
FDA grants orphan drug designation to betaine for Homocystinuria

The FDA granted orphan drug designation to betaine, developed by Cycle Pharmaceuticals Ltd., on 2026-07-17 for the treatment of homocystinuria. 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 ChangeFragile X SyndromeJul 17thRush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseJul 16thFred Hutchinson Cancer CenterNCT04431479
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 16thUniversity Hospital, GhentNCT05277766
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)Jul 15thAstraZenecaNCT07608354
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeJul 15thRush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisJul 15thSunnybrook Health Sciences CentreNCT07193953
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUsher SyndromeJul 14thLaboratoires TheaNCT06627179
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTuberous SclerosisJul 14thRush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyJul 14thNovartis PharmaceuticalsNCT05335876
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRett SyndromeJul 13thRush University Medical CenterNCT06139172
No longer enrolling new participants

No longer enrolling new participants

Status ChangeShort Bowel SyndromeJul 13thChildren's Hospital of PhiladelphiaNCT05432648
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeRett SyndromeJul 13thTaysha Gene Therapies, Inc.NCT05606614
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaJul 13thLaboratoires TheaNCT06627179
No longer enrolling new participants

No longer enrolling new participants

Status ChangeProgressive Supranuclear PalsyJul 13thBeth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraJul 13thFROM- Fondazione per la Ricerca Ospedale di Bergamo- ETSNCT06506084
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyasthenia GravisJul 12thLeiden University Medical CenterNCT06659627
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyJul 12thBeth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyJul 12thWest China HospitalNCT07657520
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMultiple SclerosisJul 12thUniversity of Colorado, DenverNCT06641271
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMultiple SclerosisJul 12thUniversity of MichiganNCT06933160
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisJul 11thColumbia UniversityNCT05451771
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseJul 11thBeth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

FDA NewsGastrointestinal Stromal TumorJul 10th
FDA Proposes Rule to Modernize Drug Manufacturing Registration

The U.S. Food and Drug Administration today issued a proposed rule that, if finalized, would create a streamlined registration pathway for distributed manufacturing establishments that operate as a single establishment using a “hub-and-spoke” model.

Status ChangeChronic Graft-versus-Host DiseaseJul 9thNovartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 9thNataliya UbohaNCT06048133
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyJul 9thREGENXBIO Inc.NCT05683379
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCorticobasal DegenerationJul 9thBeth Israel Deaconess Medical CenterNCT02994719
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 9thTransThera Sciences (Nanjing), Inc.NCT05948475
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBecker Muscular DystrophyJul 8thREGENXBIO Inc.NCT05683379
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlagille SyndromeJul 8thAlbireo, an Ipsen CompanyNCT05035030
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWilson DiseaseJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeVon Willebrand DiseaseJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUsher SyndromeJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeUrea Cycle DisordersJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTyrosinemia Type IJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTuberous SclerosisJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 7thNovartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaJul 7thRegeneron PharmaceuticalsNCT06421636
No longer enrolling new participants

No longer enrolling new participants

Status ChangeStickler SyndromeJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinocerebellar AtaxiaJul 7thNational Eye Institute (NEI)NCT02741440
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSjögren's SyndromeJul 7thNational Center for Complementary and Integrative Health (NCCIH)NCT03952624
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJul 7thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseJul 7thInstituto Nacional de Investigacao em Saude, AngolaNCT06417411
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSevere Combined ImmunodeficiencyJul 6thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRett SyndromeJul 6thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrimary HyperoxaluriaJul 6thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrader-Willi SyndromeJul 6thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePompe DiseaseJul 6thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraJul 6thNovartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangePhenylketonuriaJul 6thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangePhelan-McDermid SyndromeJul 6thPeking University First HospitalNCT07014020
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick DiseaseJul 5thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisJul 5thNovartis PharmaceuticalsNCT02386800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisJul 5thTiziana Life Sciences LTDNCT06292923
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMethylmalonic AcidemiaJul 5thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMetachromatic LeukodystrophyJul 5thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMenkes DiseaseJul 5thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMarfan SyndromeJul 4thIRCCS Policlinico S. DonatoNCT05700175
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMaple Syrup Urine DiseaseJul 4thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLeber Congenital AmaurosisJul 4thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeKrabbe DiseaseJul 4thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomocystinuriaJul 4thRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary AngioedemaJul 4thAstria Therapeutics, Inc.NCT06842823
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia BJul 3rdRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia AJul 3rdNovo Nordisk A/SNCT05685238
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHemophilia AJul 3rdRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeJul 3rdRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGeographic AtrophyJul 3rdAstellas Pharma IncNCT06970665
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGastrointestinal Stromal TumorJul 3rdIDRX, Inc., a wholly owned subsidiary of GSK, LLCNCT05489237
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGalactosemiaJul 3rdRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFragile X SyndromeJul 3rdRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal EpilepsyJul 3rdBiohaven Therapeutics Ltd.NCT06309966
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaJul 2ndJonsson Comprehensive Cancer CenterNCT07118176
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisJul 2ndPhathom Pharmaceuticals, Inc.NCT06851559
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyJul 2ndRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDravet SyndromeJul 2ndRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisJul 2ndRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCommon Variable ImmunodeficiencyJul 2ndAvni JoshiNCT05593588
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Granulomatous DiseaseJul 2ndRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCanavan DiseaseJul 2ndRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalSickle Cell DiseaseJul 1st
FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent β thalassemia (TDT).

Status ChangeBecker Muscular DystrophyJul 1stRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBarth SyndromeJul 1stRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAngelman SyndromeJul 1stRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlport SyndromeJul 1stRTI InternationalNCT03655223
No longer enrolling new participants

No longer enrolling new participants

Status ChangeBecker Muscular DystrophyJul 1stBioMarin PharmaceuticalNCT06280209
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedGraves’ DiseaseJul 31stBiohaven Therapeutics Ltd.NCT06980649
Added 1 new site: Herston, Australia

Added 1 new site: Herston, Australia

sites_addedGeographic AtrophyJul 31stADARx Pharmaceuticals, Inc.NCT06990269
Added 6 new sites: Walnut Creek, California, Malvern, Australia, Sydney, Australia and 3 more

Added 6 new sites: Walnut Creek, California, Malvern, Australia, Sydney, Australia and 3 more

sites_addedFocal EpilepsyJul 31stRapport Therapeutics Inc.NCT07563881
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

sites_addedEssential ThrombocythemiaJul 31stPrelude Therapeutics IncorporatedNCT07469891
Added 3 new sites: Tampa, Florida, Nashville, Tennessee, Houston, Texas

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

sites_addedHemophilia BJul 31stCSL BehringNCT06003387
Added 3 new sites: São Paulo, Brazil, São Paulo, Brazil, Sofia, Bulgaria

Added 3 new sites: São Paulo, Brazil, São Paulo, Brazil, Sofia, Bulgaria

eligibility_changeHemophilia BJul 31stCSL BehringNCT06003387
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedGraves’ DiseaseJul 31stYarrow Bioscience, Inc.NCT07682896
Reduced from 30 to 28 sites

Reduced from 30 to 28 sites

sites_addedGraves’ DiseaseJul 31stBiohaven Therapeutics Ltd.NCT07661056
Added 3 new sites: Huntington Beach, California, Greenville, North Carolina, Raleigh, North Carolina

Added 3 new sites: Huntington Beach, California, Greenville, North Carolina, Raleigh, North Carolina

sites_addedGraves’ DiseaseJul 31stImmunovant Sciences GmbHNCT07286006
Added 12 new sites: South Brisbane, Australia, Sliven, Bulgaria, Smolyan, Bulgaria and 9 more

Added 12 new sites: South Brisbane, Australia, Sliven, Bulgaria, Smolyan, Bulgaria and 9 more

sites_addedGraves’ DiseaseJul 31stImmunovant Sciences GmbHNCT07018323
Added 4 new sites: Santiago, Chile, Acquaviva delle Fonti, Italy, Livorno, Italy and 1 more

Added 4 new sites: Santiago, Chile, Acquaviva delle Fonti, Italy, Livorno, Italy and 1 more

sites_addedGeographic AtrophyJul 31stRegeneron PharmaceuticalsNCT06541704
Added 6 new sites: Los Angeles, California, Albury, Australia, Hurstville, Australia and 3 more

Added 6 new sites: Los Angeles, California, Albury, Australia, Hurstville, Australia and 3 more

eligibility_changeFragile X SyndromeJul 31stHolland Bloorview Kids Rehabilitation HospitalNCT06081348
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 30thImmunovant Sciences GmbHNCT07032662
Added 3 new sites: Mission Hills, California, Belo Horizonte, Brazil, Miraflores, Peru

Added 3 new sites: Mission Hills, California, Belo Horizonte, Brazil, Miraflores, Peru

sites_addedDravet SyndromeJul 30thLongboard PharmaceuticalsNCT06660394
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedDermatomyositisJul 30thCartesian TherapeuticsNCT07089121
Added 1 new site: Cincinnati, Ohio

Added 1 new site: Cincinnati, Ohio

sites_addedCongenital Adrenal HyperplasiaJul 30thCrinetics Pharmaceuticals Inc.NCT07159841
Added 6 new sites: New York, New York, Porto Alegre, Brazil, Ribeirão Preto, Brazil and 3 more

Added 6 new sites: New York, New York, Porto Alegre, Brazil, Ribeirão Preto, Brazil and 3 more

sites_addedCongenital Adrenal HyperplasiaJul 30thCrinetics Pharmaceuticals Inc.NCT07144163
Added 8 new sites: Baltimore, Maryland, Fortaleza, Brazil, Porto Alegre, Brazil and 5 more

Added 8 new sites: Baltimore, Maryland, Fortaleza, Brazil, Porto Alegre, Brazil and 5 more

sites_addedCharcot-Marie-Tooth DiseaseJul 30thNovartis PharmaceuticalsNCT07140614
Added 1 new site: Montreal, Canada

Added 1 new site: Montreal, Canada

sites_removedCerebral AdrenoleukodystrophyJul 30thMinoryx Therapeutics, S.L.NCT05819866
Reduced from 13 to 11 sites

Reduced from 13 to 11 sites

sites_addedAngelman SyndromeJul 29thIonis Pharmaceuticals, Inc.NCT06914609
Added 2 new sites: Los Angeles, California, Heidelberg, Australia

Added 2 new sites: Los Angeles, California, Heidelberg, Australia

sites_addedAmyotrophic Lateral SclerosisJul 29thPrileniaNCT07322003
Added 9 new sites: Edmonton, Canada, Hamilton, Canada, Lille, France and 6 more

Added 9 new sites: Edmonton, Canada, Hamilton, Canada, Lille, France and 6 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thNovo Nordisk A/SNCT07207811
Added 3 new sites: Genk, Belgium, Jinan, China, Guangzhou, China

Added 3 new sites: Genk, Belgium, Jinan, China, Guangzhou, China

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thAlnylam PharmaceuticalsNCT07223203
Added 12 new sites: Houston, Texas, Woolloongabba, Australia, Ribeirão Preto, Brazil and 9 more

Added 12 new sites: Houston, Texas, Woolloongabba, Australia, Ribeirão Preto, Brazil and 9 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thAlnylam PharmaceuticalsNCT07052903
Added 7 new sites: Columbus, Ohio, Córdoba, Argentina, Binhai, China and 4 more

Added 7 new sites: Columbus, Ohio, Córdoba, Argentina, Binhai, China and 4 more

Enrollment UpdateATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thAlnylam PharmaceuticalsNCT07052903
Target enrollment increased from 1250 to 1750

Target enrollment increased from 1250 to 1750

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thAlnylam PharmaceuticalsNCT07052903
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 29thAstraZenecaNCT06465810
Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

eligibility_changeAngelman SyndromeJul 29thUltragenyx Pharmaceutical IncNCT06415344
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 29thPrileniaNCT07322003
Added 10 new sites: Phoenix, Arizona, Aurora, Colorado, Baltimore, Maryland and 7 more

Added 10 new sites: Phoenix, Arizona, Aurora, Colorado, Baltimore, Maryland and 7 more

sites_addedAmyotrophic Lateral SclerosisJul 29thPhenoNet, Inc.NCT07142291
Added 1 new site: Hradec Králové, Czechia

Added 1 new site: Hradec Králové, Czechia

sites_addedAlpha-1 Antitrypsin DeficiencyJul 29thAIRNA CorporationNCT07431112
Added 2 new sites: Cambridge, United Kingdom, Southampton, United Kingdom

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

sites_addedAchondroplasiaJul 29thTyra Biosciences, IncNCT06842355
Added 3 new sites: Paris, France, Utrecht, Netherlands, Madrid, Spain

Added 3 new sites: Paris, France, Utrecht, Netherlands, Madrid, Spain

sites_addedAchondroplasiaJul 29thBioMarin PharmaceuticalNCT07441876
Added 1 new site: Osaka, Japan

Added 1 new site: Osaka, Japan

sites_addedWild-Type ATTR Cardiac AmyloidosisJul 28thAstraZenecaNCT06465810
Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

Added 6 new sites: Hefei, China, Chongqing, China, Guiyang, China and 3 more

sites_addedTurner SyndromeJul 28thAscendis 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

eligibility_changeTuberous SclerosisJul 28thHolland Bloorview Kids Rehabilitation HospitalNCT06081348
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeThalassemiaJul 28thInstitute of Hematology & Blood Diseases Hospital, ChinaNCT06931912
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSickle Cell DiseaseJul 28thNew York Medical CollegeNCT02675959
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateSickle Cell DiseaseJul 28thCity of Hope Medical CenterNCT05384756
Target enrollment increased from 2 to 20

Target enrollment increased from 2 to 20

sites_addedPulmonary Arterial HypertensionJul 27thRegeneron PharmaceuticalsNCT07318597
Added 4 new sites: Halle, Germany, Mainz, Germany, Bialystok, Poland and 1 more

Added 4 new sites: Halle, Germany, Mainz, Germany, Bialystok, Poland and 1 more

sites_addedPolycystic Kidney DiseaseJul 27thPYC TherapeuticsNCT06714006
Added 2 new sites: Randwick, Australia, Shatin, Hong Kong

Added 2 new sites: Randwick, Australia, Shatin, Hong Kong

sites_removedPulmonary Arterial HypertensionJul 27thImmuneCare Biopharmaceuticals (Shanghai) Co., Ltd.NCT07593027
Reduced from 32 to 29 sites

Reduced from 32 to 29 sites

eligibility_changePulmonary Arterial HypertensionJul 27thGustavo A Heresi, MD, MSNCT06992440
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePulmonary Arterial HypertensionJul 27thImmuneCare Biopharmaceuticals (Shanghai) Co., Ltd.NCT07593027
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJul 27thNovartis PharmaceuticalsNCT06649110
Expanded from 85 to 87 sites

Expanded from 85 to 87 sites

sites_addedPhenylketonuriaJul 27thPTC TherapeuticsNCT06302348
Added 2 new sites: Lille, France, Paris, France

Added 2 new sites: Lille, France, Paris, France

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 27thAstraZenecaNCT07457151
Added 1 new site: Busan, South Korea

Added 1 new site: Busan, South Korea

eligibility_changeNarcolepsy Type 1Jul 26thXijing HospitalNCT07654803
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyotonic DystrophyJul 26thAvidity Biosciences, Inc.NCT07008469
Added 9 new sites: Minneapolis, Minnesota, Cincinnati, Ohio, Aarhus, Denmark and 6 more

Added 9 new sites: Minneapolis, Minnesota, Cincinnati, Ohio, Aarhus, Denmark and 6 more

sites_addedMyotonic DystrophyJul 26thDyne TherapeuticsNCT07486934
Added 1 new site: Roma, Italy

Added 1 new site: Roma, Italy

sites_addedMyelofibrosisJul 26thPharmaEssentiaNCT06468033
Added 1 new site: Charlotte, North Carolina

Added 1 new site: Charlotte, North Carolina

sites_addedMyelofibrosisJul 26thDisc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changeMyelofibrosisJul 26thDisc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisJul 26thAspera Biomedicines, Inc.NCT07250646
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedMyelodysplastic SyndromesJul 26thDisc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changeMyelodysplastic SyndromesJul 26thDisc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJul 26thTakedaNCT07422480
Added 2 new sites: Tampa, Florida, Manchester, United Kingdom

Added 2 new sites: Tampa, Florida, Manchester, United Kingdom

sites_addedMyelodysplastic SyndromesJul 26thNational Cancer Institute (NCI)NCT05554406
Added 1 new site: Charleston, South Carolina

Added 1 new site: Charleston, South Carolina

sites_addedMyasthenia GravisJul 26thVertex Pharmaceuticals IncorporatedNCT07501702
Added 7 new sites: Chattanooga, Tennessee, Bydgoszcz, Poland, Poznan, Poland and 4 more

Added 7 new sites: Chattanooga, Tennessee, Bydgoszcz, Poland, Poznan, Poland and 4 more

sites_addedMyasthenia GravisJul 26thMerck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 1 new site: Kocaeli, Turkey (Türkiye)

Added 1 new site: Kocaeli, Turkey (Türkiye)

sites_addedMultiple System AtrophyJul 26thTeva Branded Pharmaceutical Products R&D LLCNCT06568237
Added 1 new site: Belgrade, Serbia

Added 1 new site: Belgrade, Serbia

sites_addedMultiple SclerosisJul 26thTG Therapeutics, Inc.NCT06433752
Expanded from 90 to 91 sites

Expanded from 90 to 91 sites

sites_removedMultiple SclerosisJul 26thHoffmann-La RocheNCT03523858
Reduced from 124 to 120 sites

Reduced from 124 to 120 sites

phase_changeMultiple SclerosisJul 26thMGH Institute of Health ProfessionsNCT06136728
Moved from Phase 4 to Phase 3

Moved from Phase 4 to Phase 3

sites_addedMultiple SclerosisJul 26thHoffmann-La RocheNCT07282574
Added 12 new sites: Ulm, Germany, Düsseldorf, Germany, Hamburg, Germany and 9 more

Added 12 new sites: Ulm, Germany, Düsseldorf, Germany, Hamburg, Germany and 9 more

Enrollment UpdateMixed Connective Tissue DiseaseJul 26thTerry L. WahlsNCT07180537
Target enrollment increased from 200 to 400

Target enrollment increased from 200 to 400

sites_addedMesotheliomaJul 26thIDEAYA BiosciencesNCT07277413
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

eligibility_changeMesotheliomaJul 26thNational Cancer Institute (NCI)NCT00242723
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaJul 26thUniversity of ArizonaNCT06814496
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyJul 26thBiogenNCT06962800
Added 1 new site: Shanghai, China

Added 1 new site: Shanghai, China

sites_addedMembranous NephropathyJul 26thVertex Pharmaceuticals IncorporatedNCT07204275
Added 6 new sites: Shenyang, China, Prague, Czechia, Cork, Ireland and 3 more

Added 6 new sites: Shenyang, China, Prague, Czechia, Cork, Ireland and 3 more

eligibility_changeMedullary Thyroid CancerJul 26thUniversity of ArizonaNCT06814496
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMedullary Thyroid CancerJul 26thUniversity of PennsylvaniaNCT04877613
Target enrollment decreased from 18 to 9

Target enrollment decreased from 18 to 9

eligibility_changeLight Chain AmyloidosisJul 25thGracell Biotechnologies (Shanghai) Co., Ltd.NCT07250269
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyJul 25thNovartis PharmaceuticalsNCT06858319
Added 3 new sites: Houston, Texas, Catamarca, Argentina, Toronto, Canada

Added 3 new sites: Houston, Texas, Catamarca, Argentina, Toronto, Canada

sites_addedIgA NephropathyJul 25thBiogenNCT06935357
Added 3 new sites: Shanghai, China, Prague, Czechia, Modena, Italy

Added 3 new sites: Shanghai, China, Prague, Czechia, Modena, Italy

sites_addedIdiopathic Pulmonary FibrosisJul 25thRein TherapeuticsNCT06968845
Added 9 new sites: Indianapolis, Indiana, New York, New York, Cincinnati, Ohio and 6 more

Added 9 new sites: Indianapolis, Indiana, New York, New York, Cincinnati, Ohio and 6 more

sites_addedIdiopathic Pulmonary FibrosisJul 25thContineum TherapeuticsNCT07284459
Added 3 new sites: Córdoba, Argentina, Camperdown, Australia, London, United Kingdom

Added 3 new sites: Córdoba, Argentina, Camperdown, Australia, London, United Kingdom

sites_addedHuntington DiseaseJul 25thSkyhawk Therapeutics, Inc.NCT07537075
Added 6 new sites: New Lambton Heights, Australia, Herston, Australia, Nedlands, Australia and 3 more

Added 6 new sites: New Lambton Heights, Australia, Herston, Australia, Nedlands, Australia and 3 more

sites_addedHomozygous Familial HypercholesterolemiaJul 25thCRISPR Therapeutics AGNCT07491172
Added 19 new sites: Hialeah, Florida, Jacksonville, Florida, Orlando, Florida and 16 more

Added 19 new sites: Hialeah, Florida, Jacksonville, Florida, Orlando, Florida and 16 more

sites_addedHodgkin LymphomaJul 25thUniversity College, LondonNCT04685616
Added 3 new sites: Basingstoke, United Kingdom, Cardiff, United Kingdom, Nottingham, United Kingdom

Added 3 new sites: Basingstoke, United Kingdom, Cardiff, United Kingdom, Nottingham, United Kingdom

sites_addedHodgkin LymphomaJul 25thAstraZenecaNCT06137144
Added 3 new sites: Birtinya, Australia, Dijon, France, Bunkyō City, Japan

Added 3 new sites: Birtinya, Australia, Dijon, France, Bunkyō City, Japan

sites_removedHereditary AngioedemaJul 25thPharvaris Netherlands B.V.NCT05396105
Reduced from 63 to 60 sites

Reduced from 63 to 60 sites

sites_addedFocal EpilepsyJul 24thRapport Therapeutics Inc.NCT07563881
Added 1 new site: Hackensack, New Jersey

Added 1 new site: Hackensack, New Jersey

sites_addedHemophilia BJul 24thCSL BehringNCT07080905
Added 12 new sites: Orange, California, Gainesville, Florida, Atlanta, Georgia and 9 more

Added 12 new sites: Orange, California, Gainesville, Florida, Atlanta, Georgia and 9 more

eligibility_changeHemophilia BJul 24thCSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseJul 24thBiohaven Therapeutics Ltd.NCT07661056
Added 6 new sites: Champaign, Illinois, Las Vegas, Nevada, Asheville, North Carolina and 3 more

Added 6 new sites: Champaign, Illinois, Las Vegas, Nevada, Asheville, North Carolina and 3 more

eligibility_changeJul 24thAskBio IncNCT07282847
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyJul 24thONL TherapeuticsNCT06659445
Added 4 new sites: Glendale, California, Greenville, North Carolina, McAllen, Texas and 1 more

Added 4 new sites: Glendale, California, Greenville, North Carolina, McAllen, Texas and 1 more

sites_addedGeographic AtrophyJul 24thi-Lumen Scientific AUS PTY LTDNCT06662162
Added 2 new sites: Reno, Nevada, Belfast, United Kingdom

Added 2 new sites: Reno, Nevada, Belfast, United Kingdom

sites_addedGastrointestinal Stromal TumorJul 24thDeciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedGastrointestinal Stromal TumorJul 24thAstraZenecaNCT07124000
Added 1 new site: Farmington, New Mexico

Added 1 new site: Farmington, New Mexico

sites_addedFocal Segmental GlomerulosclerosisJul 24thBoehringer IngelheimNCT07220083
Added 5 new sites: Humble, Texas, San Miguel de Tucumán, Argentina, Pleven, Bulgaria and 2 more

Added 5 new sites: Humble, Texas, San Miguel de Tucumán, Argentina, Pleven, Bulgaria and 2 more

eligibility_changeFocal Segmental GlomerulosclerosisJul 24thSanofiNCT06500702
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyJul 24thRapport Therapeutics Inc.NCT07594119
Added 2 new sites: Raleigh, North Carolina, Houston, Texas

Added 2 new sites: Raleigh, North Carolina, Houston, Texas

sites_addedEssential ThrombocythemiaJul 24thDisc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changeEssential ThrombocythemiaJul 24thDisc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaJul 24thPharmaEssentiaNCT04285086
Added 2 new sites: Scottsdale, Arizona, Incheon, South Korea

Added 2 new sites: Scottsdale, Arizona, Incheon, South Korea

sites_addedEhlers-Danlos SyndromeJul 23rdDeltaHealth Hospital · ShanghaiNCT07672210
Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

sites_addedDuchenne Muscular DystrophyJul 23rdCatalyst Pharmaceuticals, Inc.NCT06564974
Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

sites_addedDuchenne Muscular DystrophyJul 23rdItalfarmacoNCT05933057
Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

sites_addedDuchenne Muscular DystrophyJul 23rdREGENXBIO Inc.NCT05693142
Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

eligibility_changeDuchenne Muscular DystrophyJul 23rdREGENXBIO Inc.NCT05693142
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeDiamond-Blackfan AnemiaJul 23rdApriligen, Inc.NCT07476183
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisJul 23rdChugai PharmaceuticalNCT06371417
Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

eligibility_changeDermatomyositisJul 23rdChugai PharmaceuticalNCT06371417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDermatomyositisJul 23rdChugai PharmaceuticalNCT06723106
Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

sites_addedDermatomyositisJul 23rdargenxNCT05979441
Added 2 new sites: Rozzano, Italy, Yamaguchi, Japan

Added 2 new sites: Rozzano, Italy, Yamaguchi, Japan

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 23rdargenxNCT06920004
Added 7 new sites: Birmingham, Alabama, Bogotá, Colombia, Medellín, Colombia and 4 more

Added 7 new sites: Birmingham, Alabama, Bogotá, Colombia, Medellín, Colombia and 4 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 23rdargenxNCT07091630
Added 10 new sites: Córdoba, Argentina, Rosario, Argentina, São Paulo, Brazil and 7 more

Added 10 new sites: Córdoba, Argentina, Rosario, Argentina, São Paulo, Brazil and 7 more

sites_addedChronic Graft-versus-Host DiseaseJul 23rdIncyte CorporationNCT06585774
Added 3 new sites: Tucson, Arizona, San Francisco, California, Ulm, Germany

Added 3 new sites: Tucson, Arizona, San Francisco, California, Ulm, Germany

sites_addedBullous PemphigoidJul 22ndChugai PharmaceuticalNCT06371417
Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

eligibility_changeBullous PemphigoidJul 22ndChugai PharmaceuticalNCT06371417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedBehçet DiseaseJul 22ndChugai PharmaceuticalNCT06371417
Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

Added 10 new sites: Sofia, Bulgaria, Rüdersdorf, Germany, Himeji, Japan and 7 more

eligibility_changeBehçet DiseaseJul 22ndChugai PharmaceuticalNCT06371417
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 22ndRegeneron PharmaceuticalsNCT06351592
Added 3 new sites: Ulm, Germany, Zabrze, Poland, Warsaw, Poland

Added 3 new sites: Ulm, Germany, Zabrze, Poland, Warsaw, Poland

sites_addedBullous PemphigoidJul 22ndChugai PharmaceuticalNCT06723106
Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

sites_addedBehçet DiseaseJul 22ndChugai PharmaceuticalNCT06723106
Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

Added 5 new sites: Himeji, Japan, Isehara, Japan, Sendai, Japan and 2 more

sites_addedBecker Muscular DystrophyJul 22ndCatalyst Pharmaceuticals, Inc.NCT06564974
Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

Added 6 new sites: Loma Linda, California, Madera, California, Washington D.C., District of Columbia and 3 more

sites_addedBecker Muscular DystrophyJul 22ndSolid Biosciences Inc.NCT07160634
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

sites_addedBecker Muscular DystrophyJul 22ndItalfarmacoNCT05933057
Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

Added 9 new sites: Prague, Czechia, Essen, Germany, Hamburg, Germany and 6 more

sites_addedBecker Muscular DystrophyJul 22ndREGENXBIO Inc.NCT05693142
Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

Added 7 new sites: Orange, California, Kansas City, Kansas, Grand Rapids, Michigan and 4 more

eligibility_changeBecker Muscular DystrophyJul 22ndREGENXBIO Inc.NCT05693142
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 22ndAstraZenecaNCT06355934
Added 3 new sites: Lecce, Italy, Messina, Italy, Milan, Italy

Added 3 new sites: Lecce, Italy, Messina, Italy, Milan, Italy

Enrollment UpdateAmyotrophic Lateral SclerosisJul 22ndMerit E. Cudkowicz, MDNCT07410806
Target enrollment increased from 160 to 240

Target enrollment increased from 160 to 240

eligibility_changeAmyotrophic Lateral SclerosisJul 22ndMerit E. Cudkowicz, MDNCT07410806
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 22ndEli Lilly and CompanyNCT07571200
Added 1 new site: London, Canada

Added 1 new site: London, Canada

sites_addedAmyotrophic Lateral SclerosisJul 22ndEli Lilly and CompanyNCT07571174
Added 1 new site: London, Canada

Added 1 new site: London, Canada

sites_addedAlpha-1 Antitrypsin DeficiencyJul 22ndTessera Therapeutics, Inc.NCT07227207
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

Enrollment UpdateTurner SyndromeJul 21stDong-A ST Co., Ltd.NCT04798690
Target enrollment increased from 2500 to 5000

Target enrollment increased from 2500 to 5000

sites_addedThalassemiaJul 21stBioray LaboratoriesNCT05577312
Added 3 new sites: Guanzhou, China, Shenzhen, China, Nanning, China

Added 3 new sites: Guanzhou, China, Shenzhen, China, Nanning, China

sites_removedSystemic Juvenile Idiopathic ArthritisJul 21stAbbVieNCT05609630
Reduced from 56 to 47 sites

Reduced from 56 to 47 sites

sites_addedVascular Ehlers-Danlos SyndromeJul 21stDeltaHealth Hospital · ShanghaiNCT07672210
Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

sites_addedStargardt DiseaseJul 21stAstellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Pittsburgh, Pennsylvania

Added 1 new site: Pittsburgh, Pennsylvania

eligibility_changeStargardt DiseaseJul 21stAstellas Pharma Global Development, Inc.NCT07425574
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseJul 21stNovartis PharmaceuticalsNCT06546670
Added 2 new sites: Philadelphia, Pennsylvania, Ankara, Turkey (Türkiye)

Added 2 new sites: Philadelphia, Pennsylvania, Ankara, Turkey (Türkiye)

eligibility_changePompe DiseaseJul 20thAskBio IncNCT07282847
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraJul 20thDisc Medicine, IncNCT05320198
Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

Added 3 new sites: Gilbert, Arizona, San Francisco, California, New York, New York

eligibility_changePolycythemia VeraJul 20thDisc Medicine, IncNCT05320198
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionJul 20thMerck Sharp & Dohme LLCNCT07218029
Added 1 new site: São Paulo, Brazil

Added 1 new site: São Paulo, Brazil

sites_addedPulmonary Arterial HypertensionJul 20thRegeneron PharmaceuticalsNCT07318597
Added 9 new sites: South Brisbane, Australia, Leuven, Belgium, Brussels, Belgium and 6 more

Added 9 new sites: South Brisbane, Australia, Leuven, Belgium, Brussels, Belgium and 6 more

sites_addedPrimary Biliary CholangitisJul 20thGilead SciencesNCT06051617
Added 2 new sites: South San Francisco, California, Petah Tikva, Israel

Added 2 new sites: South San Francisco, California, Petah Tikva, Israel

sites_addedPolycythemia VeraJul 20thNovartis PharmaceuticalsNCT07357727
Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

sites_addedPolycystic Kidney DiseaseJul 20thGlaxoSmithKlineNCT06734234
Added 8 new sites: Rochester, Minnesota, Ghent, Belgium, Toronto, Canada and 5 more

Added 8 new sites: Rochester, Minnesota, Ghent, Belgium, Toronto, Canada and 5 more

eligibility_changePolycystic Kidney DiseaseJul 20thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT04782258
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPhenylketonuriaJul 20thMaze TherapeuticsNCT07694440
Added 1 new site: Indianapolis, Indiana

Added 1 new site: Indianapolis, Indiana

sites_addedNarcolepsy Type 1Jul 19thCentessa Pharmaceuticals (UK) LimitedNCT07598708
Added 2 new sites: Miami, Florida, Miami Lakes, Florida

Added 2 new sites: Miami, Florida, Miami Lakes, Florida

sites_addedMyotonic DystrophyJul 19thDyne TherapeuticsNCT07486934
Added 2 new sites: Richmond, Virginia, Milan, Italy

Added 2 new sites: Richmond, Virginia, Milan, Italy

sites_addedMyelofibrosisJul 19thNovartis PharmaceuticalsNCT07357727
Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

Added 5 new sites: Buenos Aires, Argentina, Hefei, China, Johor Bahru, Malaysia and 2 more

sites_addedMyelodysplastic SyndromesJul 19thNational Cancer Institute (NCI)NCT05564390
Added 1 new site: Reno, Nevada

Added 1 new site: Reno, Nevada

sites_addedMyelodysplastic SyndromesJul 19thM.D. Anderson Cancer CenterNCT06566742
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

eligibility_changeMyelodysplastic SyndromesJul 19thM.D. Anderson Cancer CenterNCT06566742
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJul 19thAstraZenecaNCT07247279
Added 1 new site: Ufa, Russia

Added 1 new site: Ufa, Russia

sites_removedMyasthenia GravisJul 19thUCB Biopharma SRLNCT06149559
Reduced from 13 to 9 sites

Reduced from 13 to 9 sites

sites_addedMultiple SclerosisJul 19thNovartis PharmaceuticalsNCT05344469
Added 6 new sites: Aalen, Germany, Bogen, Germany, Dillingen Saar, Germany and 3 more

Added 6 new sites: Aalen, Germany, Bogen, Germany, Dillingen Saar, Germany and 3 more

sites_addedMultiple SclerosisJul 19thZenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Livingston, New Jersey, Lublin, Poland

Added 2 new sites: Livingston, New Jersey, Lublin, Poland

sites_addedMultiple SclerosisJul 19thNovartis PharmaceuticalsNCT06675864
Added 3 new sites: Mainz, Germany, Barcelona, Spain, Madrid, Spain

Added 3 new sites: Mainz, Germany, Barcelona, Spain, Madrid, Spain

sites_addedMultiple SclerosisJul 19thZenas BioPharma (USA), LLCNCT07299019
Added 5 new sites: Boca Raton, Florida, Clearwater, Florida, Indianapolis, Indiana and 2 more

Added 5 new sites: Boca Raton, Florida, Clearwater, Florida, Indianapolis, Indiana and 2 more

sites_removedMultiple SclerosisJul 19thHoffmann-La RocheNCT05123703
Reduced from 102 to 71 sites

Reduced from 102 to 71 sites

eligibility_changeMultiple SclerosisJul 19thHoffmann-La RocheNCT05123703
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 19thHoffmann-La RocheNCT06780150
Added 2 new sites: Lübeck, Germany, Neuruppin, Germany

Added 2 new sites: Lübeck, Germany, Neuruppin, Germany

eligibility_changeMultiple SclerosisJul 19thHoffmann-La RocheNCT06780150
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 19thNovartis PharmaceuticalsNCT07225504
Added 10 new sites: Aurora, Colorado, Wellesley, Massachusetts, Dallas, Texas and 7 more

Added 10 new sites: Aurora, Colorado, Wellesley, Massachusetts, Dallas, Texas and 7 more

eligibility_changeMultiple SclerosisJul 19thNovartis PharmaceuticalsNCT07225504
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMesotheliomaJul 19thUniversity College, LondonNCT05628376
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMerkel Cell CarcinomaJul 19thUNICANCERNCT05078047
Reduced from 40 to 33 sites

Reduced from 40 to 33 sites

sites_addedMerkel Cell CarcinomaJul 19thNeonc Technologies, Inc.NCT06047379
Added 1 new site: Boynton Beach, Florida

Added 1 new site: Boynton Beach, Florida

sites_removedMembranous NephropathyJul 19thClimb Bio, Inc.NCT07096843
Reduced from 45 to 43 sites

Reduced from 45 to 43 sites

sites_addedMarfan SyndromeJul 18thDeltaHealth Hospital · ShanghaiNCT07672210
Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

Added 4 new sites: Shanghai, China, Beijing, China, Guangzhou, China and 1 more

sites_addedLight Chain AmyloidosisJul 18thRajshekhar Chakraborty, MDNCT07110844
Added 2 new sites: Boston, Massachusetts, Milwaukee, Wisconsin

Added 2 new sites: Boston, Massachusetts, Milwaukee, Wisconsin

sites_addedIgA NephropathyJul 18thBiogenNCT06935357
Added 1 new site: Guiyang, China

Added 1 new site: Guiyang, China

sites_addedIdiopathic Pulmonary FibrosisJul 18thContineum TherapeuticsNCT07284459
Added 2 new sites: San Miguel de Tucumán, Argentina, London, United Kingdom

Added 2 new sites: San Miguel de Tucumán, Argentina, London, United Kingdom

sites_addedHuntington DiseaseJul 18thNovartis PharmaceuticalsNCT07326709
Added 14 new sites: Tampa, Florida, Indianapolis, Indiana, Iowa City, Iowa and 11 more

Added 14 new sites: Tampa, Florida, Indianapolis, Indiana, Iowa City, Iowa and 11 more

sites_addedHuntington DiseaseJul 18thSkyhawk Therapeutics, Inc.NCT07378644
Added 9 new sites: Buenos Aires, Argentina, Belo Horizonte, Brazil, Campina Grande do Sul, Brazil and 6 more

Added 9 new sites: Buenos Aires, Argentina, Belo Horizonte, Brazil, Campina Grande do Sul, Brazil and 6 more

Enrollment UpdateHereditary Hemorrhagic TelangiectasiaJul 18thAlnylam PharmaceuticalsNCT06659640
Target enrollment decreased from 120 to 89

Target enrollment decreased from 120 to 89

sites_addedEssential ThrombocythemiaJul 17thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Hefei, China

Added 1 new site: Hefei, China

sites_addedGraves’ DiseaseJul 17thBiohaven Therapeutics Ltd.NCT07661056
Added 3 new sites: Doral, Florida, El Paso, Texas, Midlothian, Virginia

Added 3 new sites: Doral, Florida, El Paso, Texas, Midlothian, Virginia

sites_addedGeographic AtrophyJul 17thComplement TherapeuticsNCT07144137
Added 1 new site: Oak Forest, Illinois

Added 1 new site: Oak Forest, Illinois

sites_addedGeographic AtrophyJul 17thAbbVieNCT07160179
Added 1 new site: Reno, Nevada

Added 1 new site: Reno, Nevada

sites_addedGeographic AtrophyJul 17thONL TherapeuticsNCT06659445
Added 26 new sites: Bakersfield, California, Beverly Hills, California, Huntington Beach, California and 23 more

Added 26 new sites: Bakersfield, California, Beverly Hills, California, Huntington Beach, California and 23 more

sites_addedGastrointestinal Stromal TumorJul 17thDeciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Paris, France

Added 1 new site: Paris, France

sites_addedFocal Segmental GlomerulosclerosisJul 17thAkebia TherapeuticsNCT07268638
Added 9 new sites: Boca Raton, Florida, Livonia, Michigan, New Bern, North Carolina and 6 more

Added 9 new sites: Boca Raton, Florida, Livonia, Michigan, New Bern, North Carolina and 6 more

eligibility_changeFocal Segmental GlomerulosclerosisJul 17thAkebia TherapeuticsNCT07268638
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal Segmental GlomerulosclerosisJul 17thBoehringer IngelheimNCT07220083
Added 11 new sites: Brunswick, Georgia, Greensboro, North Carolina, Plovdiv, Bulgaria and 8 more

Added 11 new sites: Brunswick, Georgia, Greensboro, North Carolina, Plovdiv, Bulgaria and 8 more

eligibility_changeFocal Segmental GlomerulosclerosisJul 17thBoehringer IngelheimNCT07220083
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaJul 17thNovartis PharmaceuticalsNCT07357727
Added 4 new sites: Buenos Aires, Argentina, Johor Bahru, Malaysia, Kuala Selangor, Malaysia and 1 more

Added 4 new sites: Buenos Aires, Argentina, Johor Bahru, Malaysia, Kuala Selangor, Malaysia and 1 more

sites_addedDuchenne Muscular DystrophyJul 16thSolid Biosciences Inc.NCT07160634
Added 1 new site: Calgary, Canada

Added 1 new site: Calgary, Canada

Enrollment UpdateChronic Graft-versus-Host DiseaseJul 16thFred Hutchinson Cancer CenterNCT04431479
Target enrollment decreased from 300 to 211

Target enrollment decreased from 300 to 211

sites_addedCentral Diabetes InsipidusJul 16thChildren's Oncology GroupNCT06368817
Added 6 new sites: New Haven, Connecticut, Scarborough, Maine, Boston, Massachusetts and 3 more

Added 6 new sites: New Haven, Connecticut, Scarborough, Maine, Boston, Massachusetts and 3 more

sites_addedEosinophilic EsophagitisJul 16thDr. Falk Pharma GmbHNCT06596252
Added 1 new site: Plymouth, Michigan

Added 1 new site: Plymouth, Michigan

sites_addedCushing DiseaseJul 16thUniversity Hospital, AngersNCT05881005
Added 2 new sites: Lille, France, Vandœuvre-lès-Nancy, France

Added 2 new sites: Lille, France, Vandœuvre-lès-Nancy, France

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16thImmunovant Sciences GmbHNCT07032662
Added 3 new sites: Chicago, Illinois, Rosario, Argentina, Lublin, Poland

Added 3 new sites: Chicago, Illinois, Rosario, Argentina, Lublin, Poland

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16thDianthus TherapeuticsNCT06858579
Added 11 new sites: New Haven, Connecticut, Niigata, Japan, Kawasaki, Japan and 8 more

Added 11 new sites: New Haven, Connecticut, Niigata, Japan, Kawasaki, Japan and 8 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16thSanofiNCT06290141
Added 2 new sites: New York, New York, Rozzano, Italy

Added 2 new sites: New York, New York, Rozzano, Italy

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 16thSanofiNCT06859099
Added 2 new sites: Münster, Germany, Tlalnepantla, Mexico

Added 2 new sites: Münster, Germany, Tlalnepantla, Mexico

Enrollment UpdateCholangiocarcinomaJul 16thGenfitNCT05874414
Target enrollment increased from 74 to 98

Target enrollment increased from 74 to 98

eligibility_changeCholangiocarcinomaJul 16thGenfitNCT05874414
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedAutoimmune HepatitisJul 15thHannover Medical SchoolNCT05810480
Reduced from 8 to 6 sites

Reduced from 8 to 6 sites

Enrollment UpdateAutoimmune HepatitisJul 15thHannover Medical SchoolNCT05810480
Target enrollment increased from 200 to 400

Target enrollment increased from 200 to 400

eligibility_changeAutoimmune HepatitisJul 15thHannover Medical SchoolNCT05810480
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedC3 GlomerulopathyJul 15thNovartis PharmaceuticalsNCT03955445
Added 3 new sites: Heidelberg, Germany, Bari, Italy, Roma, Italy

Added 3 new sites: Heidelberg, Germany, Bari, Italy, Roma, Italy

sites_addedBarth SyndromeJul 15thLMU KlinikumNCT05554835
Added 15 new sites: Bologna, Italy, Bologna, Italy, Bologna, Italy and 12 more

Added 15 new sites: Bologna, Italy, Bologna, Italy, Bologna, Italy and 12 more

sites_removedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 15thAstraZenecaNCT07608354
Reduced from 78 to 76 sites

Reduced from 78 to 76 sites

sites_addedAmyotrophic Lateral SclerosisJul 15thPrileniaNCT07322003
Added 1 new site: Ulm, Germany

Added 1 new site: Ulm, Germany

sites_addedAmyotrophic Lateral SclerosisJul 15thPhenoNet, Inc.NCT07142291
Added 1 new site: Málaga, Spain

Added 1 new site: Málaga, Spain

sites_addedAlpha-1 Antitrypsin DeficiencyJul 15thGrifols Therapeutics LLCNCT07555483
Added 22 new sites: Birmingham, Alabama, Phoenix, Arizona, Scottsdale, Arizona and 19 more

Added 22 new sites: Birmingham, Alabama, Phoenix, Arizona, Scottsdale, Arizona and 19 more

sites_addedAchondroplasiaJul 15thTyra Biosciences, IncNCT06842355
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedWaldenström MacroglobulinemiaJul 14thNational Cancer Institute (NCI)NCT04840602
Added 1 new site: Irvine, California

Added 1 new site: Irvine, California

sites_addedSickle Cell DiseaseJul 14thNovartis PharmaceuticalsNCT06546670
Added 1 new site: Adana, Turkey (Türkiye)

Added 1 new site: Adana, Turkey (Türkiye)

Enrollment UpdateX-Linked HypophosphatemiaJul 14thKyowa Kirin Pharmaceutical Development LtdNCT03193476
Target enrollment increased from 1343 to 1489

Target enrollment increased from 1343 to 1489

sites_addedVon Willebrand DiseaseJul 14thHoffmann-La RocheNCT06998524
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

sites_removedUsher SyndromeJul 14thLaboratoires TheaNCT06627179
Reduced from 28 to 26 sites

Reduced from 28 to 26 sites

sites_addedTurner SyndromeJul 14thNovo Nordisk A/SNCT05723835
Added 1 new site: Yangsan, South Korea

Added 1 new site: Yangsan, South Korea

sites_addedTurner SyndromeJul 14thAscendis Pharma A/SNCT07221851
Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

eligibility_changeThalassemiaJul 14thNational Heart, Lung, and Blood Institute (NHLBI)NCT07599176
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeThalassemiaJul 14thCelgeneNCT04143724
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeSpinal Muscular AtrophyJul 14thSt. Jude Children's Research HospitalNCT06532474
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateSpinal Muscular AtrophyJul 14thNovartis PharmaceuticalsNCT05335876
Target enrollment decreased from 175 to 20

Target enrollment decreased from 175 to 20

eligibility_changeSickle Cell DiseaseJul 14thNational Heart, Lung, and Blood Institute (NHLBI)NCT07599176
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPhelan-McDermid SyndromeJul 13thNeuren Pharmaceuticals LimitedNCT07593391
Added 1 new site: Chevy Chase, Maryland

Added 1 new site: Chevy Chase, Maryland

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 13thRegeneron PharmaceuticalsNCT05744921
Added 1 new site: Chandigarh, India

Added 1 new site: Chandigarh, India

sites_addedRett SyndromeJul 13thTaysha Gene Therapies, Inc.NCT05606614
Added 3 new sites: Duarte, California, Oakland, California, Baltimore, Maryland

Added 3 new sites: Duarte, California, Oakland, California, Baltimore, Maryland

sites_removedRetinitis PigmentosaJul 13thLaboratoires TheaNCT06627179
Reduced from 28 to 26 sites

Reduced from 28 to 26 sites

sites_addedPulmonary Arterial HypertensionJul 13thMerck Sharp & Dohme LLCNCT07218029
Added 1 new site: Adelaide, Australia

Added 1 new site: Adelaide, Australia

sites_addedPrimary Biliary CholangitisJul 13thCascade Pharmaceuticals, IncNCT07282353
Added 5 new sites: Hefei, China, Xiamen, China, Wuhan, China and 2 more

Added 5 new sites: Hefei, China, Xiamen, China, Wuhan, China and 2 more

eligibility_changePrimary Biliary CholangitisJul 13thCascade Pharmaceuticals, IncNCT07282353
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraJul 13thNovartis PharmaceuticalsNCT07357727
Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

sites_addedPolycythemia VeraJul 13thOno Pharmaceutical Co., Ltd.NCT07429266
Added 1 new site: The Bronx, New York

Added 1 new site: The Bronx, New York

Enrollment UpdatePolycythemia VeraJul 13thFROM- Fondazione per la Ricerca Ospedale di Bergamo- ETSNCT06506084
Target enrollment increased from 319 to 429

Target enrollment increased from 319 to 429

sites_addedPhenylketonuriaJul 13thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Added 1 new site: Mainz, Germany

Added 1 new site: Mainz, Germany

sites_addedPhelan-McDermid SyndromeJul 13thNeuren Pharmaceuticals LimitedNCT07281079
Added 3 new sites: Palo Alto, California, Houston, Texas, Toronto, Canada

Added 3 new sites: Palo Alto, California, Houston, Texas, Toronto, Canada

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 13thRegeneron PharmaceuticalsNCT07154745
Added 3 new sites: Santo André, Brazil, Lévis, Canada, Barcelona, Spain

Added 3 new sites: Santo André, Brazil, Lévis, Canada, Barcelona, Spain

sites_addedNoonan SyndromeJul 12thNovo Nordisk A/SNCT05723835
Added 1 new site: Yangsan, South Korea

Added 1 new site: Yangsan, South Korea

sites_addedNoonan SyndromeJul 12thAscendis Pharma A/SNCT07221851
Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

Added 4 new sites: Aurora, Colorado, Lake Success, New York, Edinburg, Texas and 1 more

sites_removedNeurofibromatosis Type 1Jul 12thSpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, GermanyNCT03962543
Reduced from 50 to 43 sites

Reduced from 50 to 43 sites

sites_addedNarcolepsy Type 1Jul 12thAlkermes, Inc.NCT07540897
Added 5 new sites: Miami, Florida, Winter Park, Florida, Lansing, Michigan and 2 more

Added 5 new sites: Miami, Florida, Winter Park, Florida, Lansing, Michigan and 2 more

sites_addedNarcolepsy Type 1Jul 12thTakedaNCT07363720
Added 23 new sites: Redwood City, California, Brandon, Florida, Miami, Florida and 20 more

Added 23 new sites: Redwood City, California, Brandon, Florida, Miami, Florida and 20 more

sites_addedMyotonic DystrophyJul 12thDyne TherapeuticsNCT07486934
Added 3 new sites: La Jolla, California, Nantes, France, Toulouse, France

Added 3 new sites: La Jolla, California, Nantes, France, Toulouse, France

sites_addedMyelofibrosisJul 12thNovartis PharmaceuticalsNCT07357727
Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

sites_removedMyelofibrosisJul 12thFROM- Fondazione per la Ricerca Ospedale di Bergamo- ETSNCT06533813
Reduced from 36 to 27 sites

Reduced from 36 to 27 sites

sites_addedMyelodysplastic SyndromesJul 12thUniversity of Southern CaliforniaNCT02935361
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_removedMyelodysplastic SyndromesJul 12thGenmabNCT07384715
Reduced from 9 to 6 sites

Reduced from 9 to 6 sites

sites_addedMyelodysplastic SyndromesJul 12thJanssen Research & Development, LLCNCT06788509
Added 3 new sites: Randwick, Australia, Madrid, Spain, Salamanca, Spain

Added 3 new sites: Randwick, Australia, Madrid, Spain, Salamanca, Spain

eligibility_changeMyelodysplastic SyndromesJul 12thJennifer SaultzNCT03874052
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesJul 12thNational Cancer Institute (NCI)NCT06577441
Added 3 new sites: Fairway, Kansas, Kansas City, Kansas, Westwood, Kansas

Added 3 new sites: Fairway, Kansas, Kansas City, Kansas, Westwood, Kansas

sites_addedMyelodysplastic SyndromesJul 12thOrca Biosystems, Inc.NCT07216443
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedMyasthenia GravisJul 12thAmgenNCT06987539
Added 1 new site: Headington, United Kingdom

Added 1 new site: Headington, United Kingdom

sites_addedMyasthenia GravisJul 12thJanssen Research & Development, LLCNCT07217587
Added 1 new site: New Orleans, Louisiana

Added 1 new site: New Orleans, Louisiana

sites_addedMyasthenia GravisJul 12thKyverna TherapeuticsNCT06193889
Added 5 new sites: Los Angeles, California, Portland, Oregon, Riyadh, Saudi Arabia and 2 more

Added 5 new sites: Los Angeles, California, Portland, Oregon, Riyadh, Saudi Arabia and 2 more

sites_addedMultiple System AtrophyJul 12thTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: München, Germany, Münster, Germany

Added 2 new sites: München, Germany, Münster, Germany

eligibility_changeMultiple SclerosisJul 12thUniversity of Colorado, DenverNCT06641271
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 12thZenas BioPharma (USA), LLCNCT07067463
Added 6 new sites: Roeland Park, Kansas, Plovdiv, Bulgaria, Sofia, Bulgaria and 3 more

Added 6 new sites: Roeland Park, Kansas, Plovdiv, Bulgaria, Sofia, Bulgaria and 3 more

sites_addedMultiple SclerosisJul 12thJohns Hopkins UniversityNCT07497399
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedMultiple SclerosisJul 12thHoffmann-La RocheNCT07282574
Added 18 new sites: New Lambton Heights, Australia, Box Hill, Australia, Strasbourg, France and 15 more

Added 18 new sites: New Lambton Heights, Australia, Box Hill, Australia, Strasbourg, France and 15 more

sites_addedMesotheliomaJul 12thAstraZenecaNCT06535607
Added 1 new site: Natal, Brazil

Added 1 new site: Natal, Brazil

sites_addedMesotheliomaJul 12thDutch Society of Physicians for Pulmonology and TuberculosisNCT06996249
Added 13 new sites: Alkmaar, Netherlands, Amsterdam, Netherlands, Breda, Netherlands and 10 more

Added 13 new sites: Alkmaar, Netherlands, Amsterdam, Netherlands, Breda, Netherlands and 10 more

sites_addedMesotheliomaJul 12thPerspective TherapeuticsNCT06710756
Added 3 new sites: Kansas City, Kansas, Rochester, Minnesota, Madison, Wisconsin

Added 3 new sites: Kansas City, Kansas, Rochester, Minnesota, Madison, Wisconsin

sites_addedMesotheliomaJul 12thIDEAYA BiosciencesNCT07277413
Added 1 new site: Washington D.C., District of Columbia

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

sites_addedMesotheliomaJul 12thNuvectis Pharma, Inc.NCT05873686
Added 3 new sites: La Jolla, California, New York, New York, Cleveland, Ohio

Added 3 new sites: La Jolla, California, New York, New York, Cleveland, Ohio

eligibility_changeMesotheliomaJul 12thNuvectis Pharma, Inc.NCT05873686
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMembranous NephropathyJul 12thNanjing University School of MedicineNCT06285279
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLight Chain AmyloidosisJul 11thJin Lu, MDNCT06192979
Added 1 new site: Qingdao, China

Added 1 new site: Qingdao, China

eligibility_changeIgG4-Related DiseaseJul 11thNanjing University School of MedicineNCT06285279
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyJul 11thJade Biosciences, Inc.NCT07541287
Added 2 new sites: Seoul, South Korea, Seoul, South Korea

Added 2 new sites: Seoul, South Korea, Seoul, South Korea

sites_addedIgA NephropathyJul 11thBiogenNCT06935357
Added 1 new site: Konya, Turkey (Türkiye)

Added 1 new site: Konya, Turkey (Türkiye)

sites_addedIdiopathic Pulmonary FibrosisJul 11thContineum TherapeuticsNCT07284459
Added 1 new site: San Miguel de Tucumán, Argentina

Added 1 new site: San Miguel de Tucumán, Argentina

sites_addedIdiopathic Pulmonary FibrosisJul 11thBoehringer IngelheimNCT06238622
Added 5 new sites: The Woodlands, Texas, Brussels, Belgium, Belo Horizonte,Minas Gerais, Brazil and 2 more

Added 5 new sites: The Woodlands, Texas, Brussels, Belgium, Belo Horizonte,Minas Gerais, Brazil and 2 more

sites_addedHodgkin LymphomaJul 11thNational Cancer Institute (NCI)NCT05675410
Added 3 new sites: Madera, California, Carroll, Iowa, Houston, Texas

Added 3 new sites: Madera, California, Carroll, Iowa, Houston, Texas

sites_addedGraves’ DiseaseJul 10thBiohaven Therapeutics Ltd.NCT07661056
Added 1 new site: Layton, Utah

Added 1 new site: Layton, Utah

sites_addedGraves’ DiseaseJul 10thImmunovant Sciences GmbHNCT07018323
Added 5 new sites: Santiago, Chile, Nagoya, Japan, Kamogawa, Japan and 2 more

Added 5 new sites: Santiago, Chile, Nagoya, Japan, Kamogawa, Japan and 2 more

Enrollment UpdateFriedreich AtaxiaJul 10thBiogenNCT06628687
Target enrollment decreased from 20 to 10

Target enrollment decreased from 20 to 10

sites_addedHemophilia BJul 10thRegeneron PharmaceuticalsNCT06379789
Added 2 new sites: Herston, Australia, Adelaide, Australia

Added 2 new sites: Herston, Australia, Adelaide, Australia

sites_addedHemophilia AJul 10thHoffmann-La RocheNCT07416604
Added 4 new sites: Iowa City, Iowa, Madrid, Spain, Málaga, Spain and 1 more

Added 4 new sites: Iowa City, Iowa, Madrid, Spain, Málaga, Spain and 1 more

sites_addedGraves’ DiseaseJul 10thBiohaven Therapeutics Ltd.NCT07661056
Added 6 new sites: San Diego, California, Hialeah, Florida, Morehead City, North Carolina and 3 more

Added 6 new sites: San Diego, California, Hialeah, Florida, Morehead City, North Carolina and 3 more

sites_addedGraves’ DiseaseJul 10thSanofiNCT06984627
Added 2 new sites: Merano, Italy, Milan, Italy

Added 2 new sites: Merano, Italy, Milan, Italy

sites_addedGeographic AtrophyJul 10thRegeneron PharmaceuticalsNCT06541704
Added 1 new site: Lille, France

Added 1 new site: Lille, France

sites_addedGeographic AtrophyJul 10thNovartis PharmaceuticalsNCT07441642
Added 10 new sites: Philadelphia, Pennsylvania, London, Canada, Ludwigshafen, Germany and 7 more

Added 10 new sites: Philadelphia, Pennsylvania, London, Canada, Ludwigshafen, Germany and 7 more

sites_addedGastrointestinal Stromal TumorJul 10thKumquat Biosciences Inc.NCT07406633
Added 8 new sites: Miami, Florida, Chicago, Illinois, Iowa City, Iowa and 5 more

Added 8 new sites: Miami, Florida, Chicago, Illinois, Iowa City, Iowa and 5 more

sites_addedFocal EpilepsyJul 10thXenon Pharmaceuticals Inc.NCT05718817
Added 2 new sites: Philadelphia, Pennsylvania, Nuevo León, Mexico

Added 2 new sites: Philadelphia, Pennsylvania, Nuevo León, Mexico

sites_addedEssential ThrombocythemiaJul 10thNovartis PharmaceuticalsNCT07357727
Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

Added 5 new sites: Chongqing, China, Shenyang, China, Kolkata, India and 2 more

sites_addedDravet SyndromeJul 9thEncoded TherapeuticsNCT05419492
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedDilated CardiomyopathyJul 9thAffinia TherapeuticsNCT07426419
Added 1 new site: Kansas City, Missouri

Added 1 new site: Kansas City, Missouri

sites_removedChronic Graft-versus-Host DiseaseJul 9thNovartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

sites_addedCatecholaminergic Polymorphic Ventricular TachycardiaJul 9thCardurion Pharmaceuticals, Inc.NCT06658899
Added 3 new sites: Salt Lake City, Utah, Bron, France, Paris, France

Added 3 new sites: Salt Lake City, Utah, Bron, France, Paris, France

sites_addedDilated CardiomyopathyJul 9thKardigan, Inc.NCT07210723
Added 14 new sites: Minneapolis, Minnesota, Durham, North Carolina, Cincinnati, Ohio and 11 more

Added 14 new sites: Minneapolis, Minnesota, Durham, North Carolina, Cincinnati, Ohio and 11 more

Enrollment UpdateCystic FibrosisJul 9thUniversity of Alabama at BirminghamNCT03256773
Target enrollment increased from 60 to 160

Target enrollment increased from 60 to 160

sites_addedCongenital Adrenal HyperplasiaJul 9thCrinetics Pharmaceuticals Inc.NCT07159841
Added 2 new sites: San Francisco, California, Tübingen, Germany

Added 2 new sites: San Francisco, California, Tübingen, Germany

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 9thGrifols Therapeutics LLCNCT07540221
Added 1 new site: Port Charlotte, Florida

Added 1 new site: Port Charlotte, Florida

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 9thJanssen Research & Development, LLCNCT05327114
Added 3 new sites: Miami, Florida, Halle, Germany, Catania, Italy

Added 3 new sites: Miami, Florida, Halle, Germany, Catania, Italy

sites_addedAmyotrophic Lateral SclerosisJul 8thPrileniaNCT07322003
Added 3 new sites: Jacksonville, Florida, Berlin, Germany, Madrid, Spain

Added 3 new sites: Jacksonville, Florida, Berlin, Germany, Madrid, Spain

sites_addedAmyotrophic Lateral SclerosisJul 8thPrileniaNCT07322003
Added 2 new sites: Paris, France, Jena, Germany

Added 2 new sites: Paris, France, Jena, Germany

sites_removedAlagille SyndromeJul 8thAlbireo, an Ipsen CompanyNCT05035030
Reduced from 39 to 35 sites

Reduced from 39 to 35 sites

sites_addedAchondroplasiaJul 8thBioMarin PharmaceuticalNCT07441876
Added 10 new sites: Wilmington, Delaware, Baltimore, Maryland, Cincinnati, Ohio and 7 more

Added 10 new sites: Wilmington, Delaware, Baltimore, Maryland, Cincinnati, Ohio and 7 more

sites_addedUsher SyndromeJul 7thBlueRock TherapeuticsNCT06789445
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_removedThalassemiaJul 7thNovartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

Enrollment UpdateThalassemiaJul 7thRegeneron PharmaceuticalsNCT06421636
Target enrollment decreased from 95 to 19

Target enrollment decreased from 95 to 19

sites_addedStargardt DiseaseJul 7thAlkeus Pharmaceuticals, Inc.NCT07419334
Added 1 new site: Fort Worth, Texas

Added 1 new site: Fort Worth, Texas

sites_addedSpinal Muscular AtrophyJul 7thBiogenNCT07221669
Added 1 new site: Tokyo, Japan

Added 1 new site: Tokyo, Japan

Enrollment UpdateSjögren's SyndromeJul 7thNational Center for Complementary and Integrative Health (NCCIH)NCT03952624
Target enrollment decreased from 692 to 37

Target enrollment decreased from 692 to 37

sites_addedSickle Cell DiseaseJul 7thKamau TherapeuticsNCT04819841
Added 2 new sites: New York, New York, New York, New York

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

Enrollment UpdateSickle Cell DiseaseJul 7thBristol-Myers SquibbNCT06481306
Target enrollment increased from 184 to 224

Target enrollment increased from 184 to 224

eligibility_changeSickle Cell DiseaseJul 7thBristol-Myers SquibbNCT06481306
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedShort Bowel SyndromeJul 6thBoston Children's HospitalNCT03530852
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

eligibility_changeShort Bowel SyndromeJul 6thBoston Children's HospitalNCT03530852
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedShort Bowel SyndromeJul 6thBoston Children's HospitalNCT05635747
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedRetinitis PigmentosaJul 6thBlueRock TherapeuticsNCT06789445
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

Enrollment UpdatePulmonary Arterial HypertensionJul 6thUniversity of Sao Paulo General HospitalNCT07013149
Target enrollment increased from 121 to 183

Target enrollment increased from 121 to 183

eligibility_changePulmonary Arterial HypertensionJul 6thUniversity of Sao Paulo General HospitalNCT07013149
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeNTRK Fusion CancerJul 6thSt. Jude Children's Research HospitalNCT06239272
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSarcoidosisJul 6thOttawa Heart Institute Research CorporationNCT03593759
Added 1 new site: Auckland, New Zealand

Added 1 new site: Auckland, New Zealand

sites_addedRett SyndromeJul 6thInternational Rett Syndrome FoundationNCT05432349
Added 2 new sites: San Diego, California, Miami, Florida

Added 2 new sites: San Diego, California, Miami, Florida

sites_addedProgressive Familial Intrahepatic CholestasisJul 6thIpsenNCT07588880
Added 2 new sites: Beijing, China, Hunan, China

Added 2 new sites: Beijing, China, Hunan, China

sites_addedPrimary Sclerosing CholangitisJul 6thIpsenNCT07387549
Added 2 new sites: Colorado Springs, Colorado, Johnson City, Tennessee

Added 2 new sites: Colorado Springs, Colorado, Johnson City, Tennessee

sites_addedPrimary Biliary CholangitisJul 6thIpsenNCT06016842
Added 3 new sites: Rochester, Minnesota, Huntington, West Virginia, Coimbra, Portugal

Added 3 new sites: Rochester, Minnesota, Huntington, West Virginia, Coimbra, Portugal

sites_removedPolycythemia VeraJul 6thNovartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

sites_addedPolycythemia VeraJul 6thOno Pharmaceutical Co., Ltd.NCT07429266
Added 4 new sites: Plantation, Florida, Tamarac, Florida, Bethesda, Maryland and 1 more

Added 4 new sites: Plantation, Florida, Tamarac, Florida, Bethesda, Maryland and 1 more

sites_addedPolycythemia VeraJul 6thNovartis PharmaceuticalsNCT07357727
Added 2 new sites: Guangzhou, China, Yantai, China

Added 2 new sites: Guangzhou, China, Yantai, China

phase_changePhelan-McDermid SyndromeJul 6thPeking University First HospitalNCT07014020
Moved from N/A to Phase 1

Moved from N/A to Phase 1

sites_addedParoxysmal Nocturnal HemoglobinuriaJul 6thSwedish Orphan BiovitrumNCT05776472
Added 15 new sites: Garran, Australia, Anderlecht, Belgium, Turnhout, Belgium and 12 more

Added 15 new sites: Garran, Australia, Anderlecht, Belgium, Turnhout, Belgium and 12 more

sites_addedNarcolepsy Type 1Jul 5thTakedaNCT07633301
Added 17 new sites: Redwood City, California, Miami, Florida, Cincinnati, Ohio and 14 more

Added 17 new sites: Redwood City, California, Miami, Florida, Cincinnati, Ohio and 14 more

sites_addedNarcolepsy Type 1Jul 5thCentessa Pharmaceuticals (UK) LimitedNCT07598708
Added 3 new sites: Denver, North Carolina, Cincinnati, Ohio, Sugar Land, Texas

Added 3 new sites: Denver, North Carolina, Cincinnati, Ohio, Sugar Land, Texas

sites_addedNarcolepsy Type 1Jul 5thAlkermes, Inc.NCT07455383
Added 5 new sites: Cullman, Alabama, Kansas City, Kansas, Middletown, New Jersey and 2 more

Added 5 new sites: Cullman, Alabama, Kansas City, Kansas, Middletown, New Jersey and 2 more

sites_addedMyotonic DystrophyJul 5thVertex Pharmaceuticals IncorporatedNCT06926621
Added 1 new site: München, Germany

Added 1 new site: München, Germany

sites_addedMyotonic DystrophyJul 5thDyne TherapeuticsNCT07486934
Added 8 new sites: Indianapolis, Indiana, Leuven, Belgium, Copenhagen, Denmark and 5 more

Added 8 new sites: Indianapolis, Indiana, Leuven, Belgium, Copenhagen, Denmark and 5 more

sites_removedMyelofibrosisJul 5thNovartis PharmaceuticalsNCT02386800
Reduced from 101 to 97 sites

Reduced from 101 to 97 sites

sites_addedMyelofibrosisJul 5thNovartis PharmaceuticalsNCT07357727
Added 2 new sites: Guangzhou, China, Yantai, China

Added 2 new sites: Guangzhou, China, Yantai, China

sites_addedMyelodysplastic SyndromesJul 5thOrca Biosystems, Inc.NCT03802695
Added 1 new site: Hackensack, New Jersey

Added 1 new site: Hackensack, New Jersey

Enrollment UpdateMyelodysplastic SyndromesJul 5thOHSU Knight Cancer InstituteNCT06357182
Target enrollment increased from 24 to 30

Target enrollment increased from 24 to 30

eligibility_changeMyelodysplastic SyndromesJul 5thOHSU Knight Cancer InstituteNCT06357182
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisJul 5thKite, A Gilead CompanyNCT07304154
Added 2 new sites: New York, New York, Westmead, Australia

Added 2 new sites: New York, New York, Westmead, Australia

sites_addedMyasthenia GravisJul 5thVertex Pharmaceuticals IncorporatedNCT07501702
Added 6 new sites: Jacksonville, Florida, Miami, Florida, Box Hill, Australia and 3 more

Added 6 new sites: Jacksonville, Florida, Miami, Florida, Box Hill, Australia and 3 more

sites_addedMultiple SclerosisJul 5thZenas BioPharma (USA), LLCNCT07299019
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_addedMultiple SclerosisJul 5thKite, A Gilead CompanyNCT07304154
Added 3 new sites: Palo Alto, California, New York, New York, Westmead, Australia

Added 3 new sites: Palo Alto, California, New York, New York, Westmead, Australia

eligibility_changeMultiple SclerosisJul 5thCentre Hospitalier Universitaire de NīmesNCT07292480
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisJul 5thZenas BioPharma (USA), LLCNCT07067463
Added 1 new site: Poznan, Poland

Added 1 new site: Poznan, Poland

eligibility_changeMesotheliomaJul 5thMemorial Sloan Kettering Cancer CenterNCT07532902
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyJul 5thAstraZenecaNCT07571746
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedMembranous NephropathyJul 5thVertex Pharmaceuticals IncorporatedNCT07204275
Added 4 new sites: Clayton, Australia, Concord, Australia, Bari, Italy and 1 more

Added 4 new sites: Clayton, Australia, Concord, Australia, Bari, Italy and 1 more

eligibility_changeLight Chain AmyloidosisJul 4thAlexion Pharmaceuticals, Inc.NCT07081646
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLennox-Gastaut SyndromeJul 4thEpygenixNCT05066217
Added 6 new sites: Orlando, Florida, New York, New York, Philadelphia, Pennsylvania and 3 more

Added 6 new sites: Orlando, Florida, New York, New York, Philadelphia, Pennsylvania and 3 more

eligibility_changeIdiopathic Pulmonary FibrosisJul 4thHospital de GranollersNCT07482917
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJul 4thContineum TherapeuticsNCT07284459
Added 6 new sites: San Miguel de Tucumán, Argentina, Buenos Aires, Argentina, Santa Fe, Argentina and 3 more

Added 6 new sites: San Miguel de Tucumán, Argentina, Buenos Aires, Argentina, Santa Fe, Argentina and 3 more

eligibility_changeIdiopathic Pulmonary FibrosisJul 4thContineum TherapeuticsNCT07284459
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisJul 4thAvalyn Pharma Inc.NCT06951217
Added 7 new sites: Kansas City, Kansas, Baltimore, Maryland, Salt Lake City, Utah and 4 more

Added 7 new sites: Kansas City, Kansas, Baltimore, Maryland, Salt Lake City, Utah and 4 more

sites_addedIdiopathic Pulmonary FibrosisJul 4thRein TherapeuticsNCT06968845
Added 2 new sites: Salt Lake City, Utah, Warsaw, Poland

Added 2 new sites: Salt Lake City, Utah, Warsaw, Poland

sites_addedHypersensitivity PneumonitisJul 4thAvalyn Pharma Inc.NCT06329401
Added 2 new sites: Mount Kisco, New York, Beaverton, Oregon

Added 2 new sites: Mount Kisco, New York, Beaverton, Oregon

sites_addedHuntington DiseaseJul 4thHoffmann-La RocheNCT07246941
Added 2 new sites: Clayton, Australia, Cambridge, United Kingdom

Added 2 new sites: Clayton, Australia, Cambridge, United Kingdom

sites_addedHodgkin LymphomaJul 4thBaylor College of MedicineNCT04664179
Added 2 new sites: Houston, Texas, Houston, Texas

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

sites_addedHemophilia AJul 3rdSwedish Orphan BiovitrumNCT06940830
Added 2 new sites: Catania, Italy, Padua, Italy

Added 2 new sites: Catania, Italy, Padua, Italy

sites_addedHemophilia AJul 3rdWayne State UniversityNCT05022459
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedHemophilia AJul 3rdNovo Nordisk A/SNCT05685238
Added 1 new site: Tianjin, China

Added 1 new site: Tianjin, China

sites_addedGraves’ DiseaseJul 3rdMerida BiosciencesNCT07305818
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

eligibility_changeGraves’ DiseaseJul 3rdMerida BiosciencesNCT07305818
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseJul 3rdBiohaven Therapeutics Ltd.NCT07661056
Added 4 new sites: Burbank, California, Walnut Creek, California, Lenexa, Kansas and 1 more

Added 4 new sites: Burbank, California, Walnut Creek, California, Lenexa, Kansas and 1 more

sites_addedJul 3rdDenali Therapeutics Inc.NCT07354724
Added 2 new sites: Irvine, California, Durham, North Carolina

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

sites_addedGeographic AtrophyJul 3rdSanofiNCT07215234
Added 1 new site: Austin, Texas

Added 1 new site: Austin, Texas

sites_removedFocal EpilepsyJul 3rdBiohaven Therapeutics Ltd.NCT06309966
Reduced from 174 to 152 sites

Reduced from 174 to 152 sites

sites_addedEssential ThrombocythemiaJul 3rdNovartis PharmaceuticalsNCT07357727
Added 2 new sites: Guangzhou, China, Yantai, China

Added 2 new sites: Guangzhou, China, Yantai, China

sites_addedDuchenne Muscular DystrophyJul 2ndITF Therapeutics LLCNCT07127978
Added 2 new sites: Durham, North Carolina, Portland, Oregon

Added 2 new sites: Durham, North Carolina, Portland, Oregon

sites_addedDilated CardiomyopathyJul 2ndAffinia TherapeuticsNCT07426419
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

eligibility_changeCholangiocarcinomaJul 2ndUniversity of California, DavisNCT06101277
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaJul 2ndUniversity of MiamiNCT06260943
Target enrollment increased from 100 to 210

Target enrollment increased from 100 to 210

sites_addedCholangiocarcinomaJul 2ndAlentis Therapeutics AGNCT07169734
Added 13 new sites: Chicago, Illinois, Dijon, France, Marseille, France and 10 more

Added 13 new sites: Chicago, Illinois, Dijon, France, Marseille, France and 10 more

sites_addedDravet SyndromeJul 2ndEpygenixNCT04462770
Added 2 new sites: New Haven, Connecticut, New Delhi, India

Added 2 new sites: New Haven, Connecticut, New Delhi, India

eligibility_changeDravet SyndromeJul 2ndEpygenixNCT04462770
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedDravet SyndromeJul 2ndStoke Therapeutics, IncNCT06872125
Reduced from 61 to 58 sites

Reduced from 61 to 58 sites

eligibility_changeDilated CardiomyopathyJul 2ndEmory UniversityNCT06464588
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeDesmoplastic Small Round Cell TumorJul 2ndUniversity of FloridaNCT06625190
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCongenital Adrenal HyperplasiaJul 2ndCrinetics Pharmaceuticals Inc.NCT06712823
Added 4 new sites: Philadelphia, Pennsylvania, Curitiba, Brazil, Porto Alegre, Brazil and 1 more

Added 4 new sites: Philadelphia, Pennsylvania, Curitiba, Brazil, Porto Alegre, Brazil and 1 more

Enrollment UpdateCongenital Adrenal HyperplasiaJul 2ndCrinetics Pharmaceuticals Inc.NCT06712823
Target enrollment increased from 150 to 200

Target enrollment increased from 150 to 200

eligibility_changeCongenital Adrenal HyperplasiaJul 2ndCrinetics Pharmaceuticals Inc.NCT06712823
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 2ndKite, A Gilead CompanyNCT07304154
Added 2 new sites: New York, New York, Westmead, Australia

Added 2 new sites: New York, New York, Westmead, Australia

sites_addedChronic Inflammatory Demyelinating PolyneuropathyJul 2ndKedrion S.p.A.NCT06752356
Added 1 new site: Lebanon, New Hampshire

Added 1 new site: Lebanon, New Hampshire

eligibility_changeChronic Inflammatory Demyelinating PolyneuropathyJul 2ndKedrion S.p.A.NCT06752356
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCholangiocarcinomaJul 2ndNational Cancer Institute (NCI)NCT06050252
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaJul 2ndUniversity Hospital, AngersNCT06990659
Added 5 new sites: Marseille, France, Marseille, France, Nantes, France and 2 more

Added 5 new sites: Marseille, France, Marseille, France, Nantes, France and 2 more

eligibility_changeCholangiocarcinomaJul 2ndUniversity Hospital, AngersNCT06990659
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCholangiocarcinomaJul 2ndGuangxi Medical UniversityNCT07658586
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisJul 1stPrileniaNCT07322003
Added 2 new sites: Tampa, Florida, Milan, Italy

Added 2 new sites: Tampa, Florida, Milan, Italy

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 1stAstraZenecaNCT07338942
Added 40 new sites: Beijing, China, Beijing, China, Beijing, China and 37 more

Added 40 new sites: Beijing, China, Beijing, China, Beijing, China and 37 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Jul 1stEidos Therapeutics, a BridgeBio companyNCT06563895
Added 3 new sites: CABA, Argentina, Aarhus, Denmark, A Coruña, Spain

Added 3 new sites: CABA, Argentina, Aarhus, Denmark, A Coruña, Spain

sites_addedAmyotrophic Lateral SclerosisJul 1stCentre hospitalier de l'Université de Montréal (CHUM)NCT06051123
Added 2 new sites: Fredericton, Canada, Saskatoon, Canada

Added 2 new sites: Fredericton, Canada, Saskatoon, Canada

sites_addedAmyotrophic Lateral SclerosisJul 1stPrileniaNCT07322003
Added 2 new sites: Milan, Italy, Umeå, Sweden

Added 2 new sites: Milan, Italy, Umeå, Sweden

sites_addedAmyotrophic Lateral SclerosisJul 1stWashington University School of MedicineNCT07294144
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedAcromegalyJul 1stCamurus ABNCT07583563
Added 1 new site: Würzburg, Germany

Added 1 new site: Würzburg, Germany

sites_addedAchondroplasiaJul 1stBioMarin PharmaceuticalNCT06168201
Added 1 new site: Baltimore, Maryland

Added 1 new site: Baltimore, Maryland

sites_addedAchondroplasiaJul 1stTyra Biosciences, IncNCT06842355
Added 1 new site: Stockholm, Sweden

Added 1 new site: Stockholm, Sweden