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Rare Disease Trial Updates: May 2026

In May 2026, Trial Friend tracked 546 rare disease clinical trial updates across 105 conditions, including 2 FDA-related actions and 61 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 ChangeMultiple SclerosisMay 31stSandozNCT06847724
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 31stNYU Langone HealthNCT06816004
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaMay 31stUniversity of Wisconsin, MadisonNCT06608511
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaMay 31stH. Lee Moffitt Cancer Center and Research InstituteNCT04975152
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeLight Chain AmyloidosisMay 30thLantheus Germany GmbHNCT05184088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGranulomatosis with PolyangiitisMay 29thNS Pharma, Inc.NCT06046222
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFragile X SyndromeMay 29thZynerba Pharmaceuticals, Inc.NCT03802799
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 28thVertex Pharmaceuticals IncorporatedNCT05422222
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic Granulomatosis with PolyangiitisMay 28thNS Pharma, Inc.NCT06046222
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 28thVertex Pharmaceuticals IncorporatedNCT05668741
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 28thVertex Pharmaceuticals IncorporatedNCT06154447
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)May 27thPfizerNCT07494656
No longer enrolling new participants

No longer enrolling new participants

Status ChangeATTR Amyloidosis (Transthyretin Amyloidosis)May 27thLantheus Germany GmbHNCT05184088
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAplastic AnemiaMay 27thFred Hutchinson Cancer CenterNCT06752694
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWaldenström MacroglobulinemiaMay 26thShayna Sarosiek, MDNCT05190705
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaMay 25thOctant, Inc.NCT07408232
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParoxysmal Nocturnal HemoglobinuriaMay 25thNovartis PharmaceuticalsNCT06606314
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRetinitis PigmentosaMay 18thSparingVisionNCT05748873
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrimary Biliary CholangitisMay 18thZydus Therapeutics Inc.NCT06427395
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 17thStanford UniversityNCT05088356
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple System AtrophyMay 17thWashington University School of MedicineNCT05826457
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMultiple SclerosisMay 17thSanofiNCT06141473
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 17thSanofiNCT06141486
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 17thTG Therapeutics, Inc.NCT07211633
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaMay 16thUniversity of UtahNCT05723055
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary AngioedemaMay 16thCSL BehringNCT06806657
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFacioscapulohumeral Muscular DystrophyMay 15thAvidity Biosciences, Inc.NCT06547216
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFriedreich AtaxiaMay 15thBiogenNCT06054893
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisMay 13thUniversity of AlbertaNCT03362658
No longer enrolling new participants

No longer enrolling new participants

Status ChangeWilson DiseaseMay 12thPrime Medicine, Inc.NCT07226622
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseMay 12thCity of Hope Medical CenterNCT03279094
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

FDA DesignationChronic Graft-versus-Host DiseaseMay 12th
FDA grants orphan drug designation to rituximab for Chronic Graft-versus-Host Disease

The FDA granted orphan drug designation to rituximab, developed by ODDIFACT SAS, on 2026-05-12 for the treatment of chronic graft-versus-host disease. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.

Status ChangeRetinitis PigmentosaMay 11thUniversity Hospital TuebingenNCT06323772
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraMay 11thJohn MascarenhasNCT05467800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeurofibromatosis Type 1May 10thNovartis PharmaceuticalsNCT03975829
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMyelofibrosisMay 10thJohn MascarenhasNCT05467800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 10thM.D. Anderson Cancer CenterNCT04493138
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 10thH. Lee Moffitt Cancer Center and Research InstituteNCT05732961
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesMay 10thBristol-Myers SquibbNCT05949684
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 10thUniversity of ExeterNCT06274671
No longer enrolling new participants

No longer enrolling new participants

Status ChangeLight Chain AmyloidosisMay 9thNational Cancer Institute (NCI)NCT04847453
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseMay 9thUniversitaire Ziekenhuizen KU LeuvenNCT06626412
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomocystinuriaMay 9thTravere Therapeutics, Inc.NCT06247085
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHereditary Spastic ParaplegiaMay 9thNational Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
No longer enrolling new participants

No longer enrolling new participants

FDA ApprovalCholangiocarcinomaMay 8th
FDA Grants Seventh Approval under the National Priority Voucher Pilot Program

FDA issued an approval for Bizengri (zenocutuzumab-zbco), a drug that treats NRG1 fusion-positive cholangiocarcinoma, an ultra-rare, aggressive cancer that forms in the bile ducts.

Status ChangeGeographic AtrophyMay 8thHoffmann-La RocheNCT06961370
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaMay 8thJohn MascarenhasNCT05467800
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 7thCentre Hospitalier Universitaire de la RéunionNCT06371261
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCystic FibrosisMay 7thSionna Therapeutics Inc.NCT07108153
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlpha-1 Antitrypsin DeficiencyMay 6thBioMarin PharmaceuticalNCT06738017
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAdrenocortical CarcinomaMay 6thColumbia UniversityNCT03127774
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic Paraplegia Type 4May 5thNational Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
No longer enrolling new participants

No longer enrolling new participants

Status ChangePolycythemia VeraMay 4thSwedish Orphan BiovitrumNCT03165734
No longer enrolling new participants

No longer enrolling new participants

Status ChangeParoxysmal Nocturnal HemoglobinuriaMay 4thRegeneron PharmaceuticalsNCT05133531
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisMay 3rdSwedish Orphan BiovitrumNCT03165734
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisMay 3rdUniversity of VirginiaNCT04593082
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisMay 2ndCalluna Pharma ASNCT06736990
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseMay 2ndUniversity of IowaNCT05707663
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHuntington DiseaseMay 2ndSkyhawk Therapeutics, Inc.NCT06873334
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHomozygous Familial HypercholesterolemiaMay 2ndNovartis PharmaceuticalsNCT05682378
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeGeographic AtrophyMay 1stClinOmicsAINCT07298291
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGranulomatosis with PolyangiitisMay 1stImperial College LondonNCT06230354
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFabry DiseaseMay 1stUniversität MünsterNCT06095713
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedNoonan SyndromeMay 31stAscendis Pharma A/SNCT07221851
Added 1 new site: Sejong, South Korea

Added 1 new site: Sejong, South Korea

sites_addedNeurofibromatosis Type 1May 31stUniversity of Alabama at BirminghamNCT06188741
Added 2 new sites: Palo Alto, California, St Louis, Missouri

Added 2 new sites: Palo Alto, California, St Louis, Missouri

sites_addedMyelofibrosisMay 31stPrelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

Enrollment UpdateMyelodysplastic SyndromesMay 31stMayo ClinicNCT03017820
Target enrollment decreased from 127 to 99

Target enrollment decreased from 127 to 99

sites_addedMyasthenia GravisMay 31stNovartis PharmaceuticalsNCT06744920
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedMyasthenia GravisMay 31stAmgenNCT06987539
Added 2 new sites: Paris, France, Valencia, Spain

Added 2 new sites: Paris, France, Valencia, Spain

sites_addedMyasthenia GravisMay 31stargenxNCT07284420
Added 1 new site: Bergamo, Italy

Added 1 new site: Bergamo, Italy

sites_addedMyasthenia GravisMay 31stNovartis PharmaceuticalsNCT06517758
Added 7 new sites: Boston, Massachusetts, Detroit, Michigan, CABA, Argentina and 4 more

Added 7 new sites: Boston, Massachusetts, Detroit, Michigan, CABA, Argentina and 4 more

sites_addedMultiple System AtrophyMay 31stTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Tel Aviv, Israel, Milan, Italy

Added 2 new sites: Tel Aviv, Israel, Milan, Italy

eligibility_changeMultiple SclerosisMay 31stHoffmann-La RocheNCT04586010
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 31stSanofiNCT07325292
Added 8 new sites: Phoenix, Arizona, Northbrook, Illinois, Teaneck, New Jersey and 5 more

Added 8 new sites: Phoenix, Arizona, Northbrook, Illinois, Teaneck, New Jersey and 5 more

eligibility_changeMultiple SclerosisMay 31stHoffmann-La RocheNCT05119569
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 31stAmgenNCT06700343
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_removedMultiple SclerosisMay 31stNovartis PharmaceuticalsNCT05156281
Reduced from 225 to 223 sites

Reduced from 225 to 223 sites

sites_addedMultiple SclerosisMay 31stNovartis PharmaceuticalsNCT07225504
Added 11 new sites: Chicago, Illinois, Rosario, Argentina, Murdoch, Australia and 8 more

Added 11 new sites: Chicago, Illinois, Rosario, Argentina, Murdoch, Australia and 8 more

sites_addedMultiple SclerosisMay 31stHoffmann-La RocheNCT06780150
Added 6 new sites: Arnsdorf, Germany, Berg, Germany, Frankfurt, Germany and 3 more

Added 6 new sites: Arnsdorf, Germany, Berg, Germany, Frankfurt, Germany and 3 more

sites_addedMultiple SclerosisMay 31stZenas BioPharma (USA), LLCNCT07299019
Added 3 new sites: Bradenton, Florida, Rochester Hills, Michigan, St Louis, Missouri

Added 3 new sites: Bradenton, Florida, Rochester Hills, Michigan, St Louis, Missouri

sites_addedMultiple SclerosisMay 31stNovartis PharmaceuticalsNCT06846281
Added 2 new sites: Irvine, California, Hradec Králové, Czechia

Added 2 new sites: Irvine, California, Hradec Králové, Czechia

sites_addedMultiple SclerosisMay 31stTG Therapeutics, Inc.NCT07211633
Added 3 new sites: Tulza, Bosnia and Herzegovina, Kragujevac, Serbia, Niš, Serbia

Added 3 new sites: Tulza, Bosnia and Herzegovina, Kragujevac, Serbia, Niš, Serbia

sites_addedMultiple SclerosisMay 31stTG Therapeutics, Inc.NCT05877963
Added 1 new site: Savannah, Georgia

Added 1 new site: Savannah, Georgia

sites_addedMultiple SclerosisMay 31stTG Therapeutics, Inc.NCT06433752
Added 3 new sites: Billings, Montana, Norman, Oklahoma, Ogden, Utah

Added 3 new sites: Billings, Montana, Norman, Oklahoma, Ogden, Utah

sites_addedMultiple SclerosisMay 31stZenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Pheonix, Arizona, West Hollywood, California

Added 2 new sites: Pheonix, Arizona, West Hollywood, California

sites_addedMesotheliomaMay 31stAstraZenecaNCT06535607
Added 31 new sites: New York, New York, Philadelphia, Pennsylvania, Clayton, Australia and 28 more

Added 31 new sites: New York, New York, Philadelphia, Pennsylvania, Clayton, Australia and 28 more

Enrollment UpdateMesotheliomaMay 31stGenmabNCT05579366
Target enrollment increased from 764 to 884

Target enrollment increased from 764 to 884

eligibility_changeMesotheliomaMay 31stGenmabNCT05579366
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaMay 31stMerck Sharp & Dohme LLCNCT07302347
Added 1 new site: Chūō, Japan

Added 1 new site: Chūō, Japan

sites_addedIgA NephropathyMay 30thBiogenNCT06935357
Added 4 new sites: Los Angeles, California, New York, New York, Shanghai, China and 1 more

Added 4 new sites: Los Angeles, California, New York, New York, Shanghai, China and 1 more

sites_addedIgA NephropathyMay 30thNovartis PharmaceuticalsNCT07146906
Added 2 new sites: Opole, Poland, Seoul, South Korea

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

eligibility_changeIgA NephropathyMay 30thNovartis PharmaceuticalsNCT07146906
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyMay 30thHaisco Pharmaceutical Group Co., Ltd.NCT07390123
Added 8 new sites: Huangshan City, China, Wuhu, China, Zhanjiang, China and 5 more

Added 8 new sites: Huangshan City, China, Wuhu, China, Zhanjiang, China and 5 more

sites_addedHuntington DiseaseMay 30thHoffmann-La RocheNCT07246941
Added 1 new site: Parkville, Australia

Added 1 new site: Parkville, Australia

sites_addedHuntington DiseaseMay 30thNovartis PharmaceuticalsNCT07326709
Added 2 new sites: Georgetown, Texas, Southampton, United Kingdom

Added 2 new sites: Georgetown, Texas, Southampton, United Kingdom

sites_addedHomozygous Familial HypercholesterolemiaMay 30thArrowhead PharmaceuticalsNCT07037771
Added 7 new sites: Innsbruck, Austria, Linz, Austria, Vienna, Austria and 4 more

Added 7 new sites: Innsbruck, Austria, Linz, Austria, Vienna, Austria and 4 more

sites_addedHomocystinuriaMay 30thTravere Therapeutics, Inc.NCT06431893
Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

eligibility_changeHomocystinuriaMay 30thTravere Therapeutics, Inc.NCT06431893
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHodgkin LymphomaMay 30thUniversity of Southern CaliforniaNCT07259304
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

sites_addedHodgkin LymphomaMay 30thNational Cancer Institute (NCI)NCT05675410
Added 4 new sites: Los Angeles, California, Jacksonville, Florida, Miami Beach, Florida and 1 more

Added 4 new sites: Los Angeles, California, Jacksonville, Florida, Miami Beach, Florida and 1 more

eligibility_changeHodgkin LymphomaMay 30thMayo ClinicNCT07285044
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary AngioedemaMay 30thIonis Pharmaceuticals, Inc.NCT07298447
Added 2 new sites: Padova, Italy, San Donato Milanese, Italy

Added 2 new sites: Padova, Italy, San Donato Milanese, Italy

eligibility_changeGiant Cell ArteritisMay 29thUniversity of AarhusNCT07552636
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFacioscapulohumeral Muscular DystrophyMay 29thAvidity Biosciences, Inc.NCT07038200
Added 24 new sites: Aarhus, Denmark, Copenhagen, Denmark, Marseille, France and 21 more

Added 24 new sites: Aarhus, Denmark, Copenhagen, Denmark, Marseille, France and 21 more

sites_addedFocal Segmental GlomerulosclerosisMay 29thBoehringer IngelheimNCT07220083
Added 15 new sites: Fortaleza, Brazil, Porto Alegre, Brazil, São Paulo, Brazil and 12 more

Added 15 new sites: Fortaleza, Brazil, Porto Alegre, Brazil, São Paulo, Brazil and 12 more

sites_addedEssential ThrombocythemiaMay 29thPrelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_removedHemophilia BMay 29thCSL BehringNCT07080905
Reduced from 11 to 2 sites

Reduced from 11 to 2 sites

eligibility_changeHemophilia BMay 29thCSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BMay 29thPfizerNCT05568719
Added 1 new site: Taichung, Taiwan

Added 1 new site: Taichung, Taiwan

sites_addedHemophilia AMay 29thPfizerNCT05568719
Added 1 new site: Taichung, Taiwan

Added 1 new site: Taichung, Taiwan

sites_addedGuillain-Barré SyndromeMay 29thAnnexon, Inc.NCT07020819
Added 4 new sites: Phoenix, Arizona, Carlsbad, California, Jacksonville, Florida and 1 more

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

sites_addedGraves’ DiseaseMay 29thMerida BiosciencesNCT07305818
Added 1 new site: Wesley Chapel, Florida

Added 1 new site: Wesley Chapel, Florida

sites_addedGeographic AtrophyMay 29thRegeneron PharmaceuticalsNCT07230834
Added 2 new sites: Bucharest, Romania, Cluj-Napoca, Romania

Added 2 new sites: Bucharest, Romania, Cluj-Napoca, Romania

sites_addedGeographic AtrophyMay 29thNovartis PharmaceuticalsNCT07441642
Added 3 new sites: Pécs, Hungary, Bydgoszcz, Poland, Krakow, Poland

Added 3 new sites: Pécs, Hungary, Bydgoszcz, Poland, Krakow, Poland

sites_addedGeographic AtrophyMay 29thComplement TherapeuticsNCT07144137
Added 2 new sites: London, United Kingdom, Southampton, United Kingdom

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

eligibility_changeGastrointestinal Stromal TumorMay 29thUniversity of California, San DiegoNCT07171203
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateFragile X SyndromeMay 29thZynerba Pharmaceuticals, Inc.NCT03802799
Target enrollment decreased from 550 to 476

Target enrollment decreased from 550 to 476

eligibility_changeFocal EpilepsyMay 29thNational Institute of Neurological Disorders and Stroke (NINDS)NCT03478852
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyMay 29thRapport Therapeutics Inc.NCT07563881
Added 1 new site: Fort Wayne, Indiana

Added 1 new site: Fort Wayne, Indiana

sites_addedDravet SyndromeMay 28thEncoded TherapeuticsNCT05419492
Added 1 new site: Ann Arbor, Michigan

Added 1 new site: Ann Arbor, Michigan

sites_addedDermatomyositisMay 28thAstraZenecaNCT06455449
Added 1 new site: Amarillo, Texas

Added 1 new site: Amarillo, Texas

sites_addedDermatomyositisMay 28thargenxNCT05979441
Added 1 new site: Nanjing, China

Added 1 new site: Nanjing, China

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28thargenxNCT07091630
Added 13 new sites: Linz, Austria, Sofia, Bulgaria, Fuzhou, China and 10 more

Added 13 new sites: Linz, Austria, Sofia, Bulgaria, Fuzhou, China and 10 more

eligibility_changeEosinophilic EsophagitisMay 28thNational Institute of Allergy and Infectious Diseases (NIAID)NCT02504853
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 28thEncoded TherapeuticsNCT05419492
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_addedDermatomyositisMay 28thargenxNCT05979441
Added 1 new site: Baltimore, Maryland

Added 1 new site: Baltimore, Maryland

sites_removedCystic FibrosisMay 28thVertex Pharmaceuticals IncorporatedNCT05668741
Reduced from 43 to 26 sites

Reduced from 43 to 26 sites

Enrollment UpdateCystic FibrosisMay 28thVertex Pharmaceuticals IncorporatedNCT05668741
Target enrollment decreased from 39 to 26

Target enrollment decreased from 39 to 26

Enrollment UpdateCystic FibrosisMay 28thVertex Pharmaceuticals IncorporatedNCT06154447
Target enrollment decreased from 255 to 165

Target enrollment decreased from 255 to 165

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28thSanofiNCT06290128
Added 5 new sites: Centennial, Colorado, Bradenton, Florida, Kansas City, Kansas and 2 more

Added 5 new sites: Centennial, Colorado, Bradenton, Florida, Kansas City, Kansas and 2 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28thSanofiNCT06859099
Added 2 new sites: Kansas City, Kansas, Philadelphia, Pennsylvania

Added 2 new sites: Kansas City, Kansas, Philadelphia, Pennsylvania

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 28thSanofiNCT06290141
Added 1 new site: Budapest, Hungary

Added 1 new site: Budapest, Hungary

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 27thNovo Nordisk A/SNCT07207811
Added 4 new sites: Cleveland, Ohio, Adelaide, Australia, Roeselare, Belgium and 1 more

Added 4 new sites: Cleveland, Ohio, Adelaide, Australia, Roeselare, Belgium and 1 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 27thAstraZenecaNCT06465810
Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

Enrollment UpdateAplastic AnemiaMay 27thFred Hutchinson Cancer CenterNCT06752694
Target enrollment decreased from 20 to 1

Target enrollment decreased from 20 to 1

sites_addedAmyotrophic Lateral SclerosisMay 27thNura BioNCT07369076
Added 2 new sites: Brisbane, Australia, Fredericton, Canada

Added 2 new sites: Brisbane, Australia, Fredericton, Canada

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 26thAstraZenecaNCT06465810
Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

Added 7 new sites: Portland, Oregon, Fuzhou, China, Guangzhou, China and 4 more

sites_addedWaldenström MacroglobulinemiaMay 26thNational Cancer Institute (NCI)NCT04840602
Added 2 new sites: Saint Louis Park, Minnesota, Appleton, Wisconsin

Added 2 new sites: Saint Louis Park, Minnesota, Appleton, Wisconsin

sites_addedSystemic Juvenile Idiopathic ArthritisMay 26thAbbVieNCT05609630
Added 14 new sites: Little Rock, Arkansas, Hollywood, Florida, Columbus, Ohio and 11 more

Added 14 new sites: Little Rock, Arkansas, Hollywood, Florida, Columbus, Ohio and 11 more

sites_addedStickler SyndromeMay 26thHelen Keller Eye Research FoundationNCT07146516
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

eligibility_changeStickler SyndromeMay 26thHelen Keller Eye Research FoundationNCT07146516
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedWaldenström MacroglobulinemiaMay 26thNational Cancer Institute (NCI)NCT04840602
Added 1 new site: Nampa, Idaho

Added 1 new site: Nampa, Idaho

Enrollment UpdateWaldenström MacroglobulinemiaMay 26thShayna Sarosiek, MDNCT05190705
Target enrollment decreased from 36 to 21

Target enrollment decreased from 36 to 21

sites_addedTurner SyndromeMay 26thAscendis Pharma A/SNCT07221851
Added 1 new site: Hanover, Germany

Added 1 new site: Hanover, Germany

sites_addedSystemic MastocytosisMay 26thBlueprint Medicines CorporationNCT04910685
Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

sites_addedSystemic Juvenile Idiopathic ArthritisMay 26thSanofiNCT02991469
Added 1 new site: Lisbon, Portugal

Added 1 new site: Lisbon, Portugal

sites_removedSpinal Muscular AtrophyMay 26thBiogenNCT05067790
Reduced from 44 to 22 sites

Reduced from 44 to 22 sites

sites_addedSjögren's SyndromeMay 26thArtiva Biotherapeutics, Inc.NCT06991114
Added 27 new sites: Tucson, Arizona, Chula Vista, California, Los Angeles, California and 24 more

Added 27 new sites: Tucson, Arizona, Chula Vista, California, Los Angeles, California and 24 more

sites_addedSickle Cell DiseaseMay 26thDisc Medicine, IncNCT07187973
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

Enrollment UpdateRetinitis PigmentosaMay 25thOctant, Inc.NCT07408232
Target enrollment decreased from 50 to 32

Target enrollment decreased from 50 to 32

sites_addedPulmonary Arterial HypertensionMay 25thPfizerNCT06137742
Added 1 new site: Edmonton, Canada

Added 1 new site: Edmonton, Canada

sites_addedPulmonary Arterial HypertensionMay 25thMerck Sharp & Dohme LLCNCT07218029
Added 5 new sites: Orange, California, Dallas, Texas, Blumenau, Brazil and 2 more

Added 5 new sites: Orange, California, Dallas, Texas, Blumenau, Brazil and 2 more

sites_addedProgressive Supranuclear PalsyMay 25thNovartis PharmaceuticalsNCT07498426
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedPrader-Willi SyndromeMay 25thSoleno Therapeutics, Inc.NCT07450664
Added 4 new sites: Chicago, Illinois, Ann Arbor, Michigan, Chapel Hill, North Carolina and 1 more

Added 4 new sites: Chicago, Illinois, Ann Arbor, Michigan, Chapel Hill, North Carolina and 1 more

Enrollment UpdateParoxysmal Nocturnal HemoglobinuriaMay 25thNovartis PharmaceuticalsNCT06606314
Target enrollment increased from 100 to 132

Target enrollment increased from 100 to 132

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 25thAstraZenecaNCT07457151
Expanded from 4 to 5 sites

Expanded from 4 to 5 sites

sites_addedNoonan SyndromeMay 24thAscendis Pharma A/SNCT07221851
Added 3 new sites: Le Kremlin-Bicêtre, France, Hanover, Germany, Seoul, South Korea

Added 3 new sites: Le Kremlin-Bicêtre, France, Hanover, Germany, Seoul, South Korea

sites_addedNeurofibromatosis Type 1May 24thHealx LimitedNCT06541847
Added 1 new site: Palo Alto, California

Added 1 new site: Palo Alto, California

eligibility_changeMyelodysplastic SyndromesMay 24thMayo ClinicNCT07285044
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesMay 24thCity of Hope Medical CenterNCT06287944
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisMay 24thUCB Biopharma SRLNCT07246564
Added 1 new site: Changchun, China

Added 1 new site: Changchun, China

sites_addedMyasthenia GravisMay 24thVertex Pharmaceuticals IncorporatedNCT07501702
Added 3 new sites: Boca Raton, Florida, Houston, Texas, Waxahachie, Texas

Added 3 new sites: Boca Raton, Florida, Houston, Texas, Waxahachie, Texas

sites_addedMyasthenia GravisMay 24thImmunovant Sciences GmbHNCT07039916
Added 2 new sites: Huntsville, Alabama, Rosario, Argentina

Added 2 new sites: Huntsville, Alabama, Rosario, Argentina

sites_addedMyasthenia GravisMay 24thMerck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 5 new sites: Farmington, Connecticut, Cincinnati, Ohio, Camperdown, Australia and 2 more

Added 5 new sites: Farmington, Connecticut, Cincinnati, Ohio, Camperdown, Australia and 2 more

eligibility_changeMyasthenia GravisMay 24thMerck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyasthenia GravisMay 24thNovartis PharmaceuticalsNCT06744920
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple System AtrophyMay 24thTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: La Jolla, California, Roma, Italy

Added 2 new sites: La Jolla, California, Roma, Italy

sites_addedMultiple SclerosisMay 24thHoffmann-La RocheNCT07282574
Added 26 new sites: Heidelberg, Australia, Nîmes, France, Saint-Herblain, France and 23 more

Added 26 new sites: Heidelberg, Australia, Nîmes, France, Saint-Herblain, France and 23 more

sites_addedMultiple SclerosisMay 24thNovartis PharmaceuticalsNCT07225504
Added 5 new sites: Hradec Králové, Czechia, Tallinn, Estonia, 's-Hertogenbosch, Netherlands and 2 more

Added 5 new sites: Hradec Králové, Czechia, Tallinn, Estonia, 's-Hertogenbosch, Netherlands and 2 more

Enrollment UpdateMultiple SclerosisMay 24thDaishi TianNCT07303790
Target enrollment decreased from 15 to 9

Target enrollment decreased from 15 to 9

eligibility_changeMultiple SclerosisMay 24thDaishi TianNCT07303790
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisMay 24thNovartis PharmaceuticalsNCT06869785
Added 3 new sites: Miami, Florida, Weston, Florida, Columbus, Ohio

Added 3 new sites: Miami, Florida, Weston, Florida, Columbus, Ohio

sites_addedMultiple SclerosisMay 24thSetPoint Medical CorporationNCT06796504
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedMultiple SclerosisMay 24thZenas BioPharma (USA), LLCNCT07067463
Added 7 new sites: Scottsdale, Arizona, Bradenton, Florida, St Louis, Missouri and 4 more

Added 7 new sites: Scottsdale, Arizona, Bradenton, Florida, St Louis, Missouri and 4 more

sites_addedMesotheliomaMay 24thA2 Biotherapeutics Inc.NCT06885424
Added 1 new site: Gilbert, Arizona

Added 1 new site: Gilbert, Arizona

Enrollment UpdateMesotheliomaMay 24thNational Cancer Institute (NCI)NCT04431024
Target enrollment decreased from 800 to 300

Target enrollment decreased from 800 to 300

eligibility_changeMesotheliomaMay 24thNational Cancer Institute (NCI)NCT04431024
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaMay 24thMayo ClinicNCT07285044
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMerkel Cell CarcinomaMay 24thSystImmune Inc.NCT07080242
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyMay 24thBiogenNCT06962800
Added 4 new sites: Stanford, California, Nashville, Tennessee, Lucknow, India and 1 more

Added 4 new sites: Stanford, California, Nashville, Tennessee, Lucknow, India and 1 more

sites_addedMembranous NephropathyMay 24thNkarta, Inc.NCT06557265
Added 1 new site: Parkville, Australia

Added 1 new site: Parkville, Australia

Enrollment UpdateMembranous NephropathyMay 24thNkarta, Inc.NCT06557265
Target enrollment increased from 96 to 120

Target enrollment increased from 96 to 120

eligibility_changeMembranous NephropathyMay 24thNkarta, Inc.NCT06557265
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMastocytosisMay 24thBlueprint Medicines CorporationNCT07143669
Added 1 new site: Kansas City, Kansas

Added 1 new site: Kansas City, Kansas

sites_addedMastocytosisMay 24thBlueprint Medicines CorporationNCT04910685
Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

Added 4 new sites: Buenos Aires, Argentina, Ghent, Belgium, Brno, Czechia and 1 more

sites_addedIgA NephropathyMay 23rdBiogenNCT06935357
Added 2 new sites: Hong Kong, China, Sheffield, United Kingdom

Added 2 new sites: Hong Kong, China, Sheffield, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisMay 23rdContineum TherapeuticsNCT07284459
Added 9 new sites: Midland, Australia, Spearwood, Australia, Trois-Rivières, Canada and 6 more

Added 9 new sites: Midland, Australia, Spearwood, Australia, Trois-Rivières, Canada and 6 more

eligibility_changeHypoparathyroidismMay 23rdPeking Union Medical College HospitalNCT07197450
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseMay 23rdNovartis PharmaceuticalsNCT07326709
Added 2 new sites: Philadelphia, Pennsylvania, Montreal, Canada

Added 2 new sites: Philadelphia, Pennsylvania, Montreal, Canada

eligibility_changeHomozygous Familial HypercholesterolemiaMay 23rdEddingpharm (Zhuhai) Co., Ltd.NCT07489209
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary AngioedemaMay 23rdADARx Pharmaceuticals, Inc.NCT06960213
Added 1 new site: Krakow, Poland

Added 1 new site: Krakow, Poland

sites_addedHereditary AngioedemaMay 23rdAstria Therapeutics, Inc.NCT07204938
Added 9 new sites: Little Rock, Arkansas, San Diego, California, Santa Monica, California and 6 more

Added 9 new sites: Little Rock, Arkansas, San Diego, California, Santa Monica, California and 6 more

sites_addedHereditary AngioedemaMay 23rdIonis Pharmaceuticals, Inc.NCT07298447
Added 2 new sites: Miami, Florida, Hershey, Pennsylvania

Added 2 new sites: Miami, Florida, Hershey, Pennsylvania

sites_addedGraves’ DiseaseMay 22ndImmunovant Sciences GmbHNCT06727604
Added 1 new site: Cambridge, United Kingdom

Added 1 new site: Cambridge, United Kingdom

sites_addedGraves’ DiseaseMay 22ndImmunovant Sciences GmbHNCT07018323
Added 10 new sites: Fortaleza, Brazil, Brasília, Brazil, Curitiba, Brazil and 7 more

Added 10 new sites: Fortaleza, Brazil, Brasília, Brazil, Curitiba, Brazil and 7 more

sites_addedGaucher DiseaseMay 22ndPrevail TherapeuticsNCT05487599
Added 1 new site: Los Angeles, California

Added 1 new site: Los Angeles, California

eligibility_changeGastrointestinal Stromal TumorMay 22ndCogent Biosciences, Inc.NCT05208047
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeGastrointestinal Stromal TumorMay 22ndAffiliated Hospital to Academy of Military Medical SciencesNCT07581977
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedFibrodysplasia Ossificans ProgressivaMay 22ndIncyte CorporationNCT05090891
Reduced from 27 to 24 sites

Reduced from 27 to 24 sites

eligibility_changeFibrodysplasia Ossificans ProgressivaMay 22ndIncyte CorporationNCT05090891
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BMay 22ndRegeneron PharmaceuticalsNCT06379789
Added 1 new site: Nashville, Tennessee

Added 1 new site: Nashville, Tennessee

sites_addedHemophilia BMay 22ndPfizerNCT05568719
Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

sites_addedHemophilia AMay 22ndPfizerNCT05568719
Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

Added 2 new sites: Toronto, Canada, Adana, Turkey (Türkiye)

sites_addedHemophilia AMay 22ndTakedaNCT06550882
Added 3 new sites: Seoul, South Korea, Seoul, South Korea, Ulsan, South Korea

Added 3 new sites: Seoul, South Korea, Seoul, South Korea, Ulsan, South Korea

sites_addedHemophilia AMay 22ndPfizerNCT04370054
Added 10 new sites: Oakland, California, Redwood City, California, San Francisco, California and 7 more

Added 10 new sites: Oakland, California, Redwood City, California, San Francisco, California and 7 more

sites_addedGastrointestinal Stromal TumorMay 22ndKumquat Biosciences Inc.NCT07406633
Added 7 new sites: Los Angeles, California, Jacksonville, Florida, Tampa, Florida and 4 more

Added 7 new sites: Los Angeles, California, Jacksonville, Florida, Tampa, Florida and 4 more

sites_addedEpidermolysis BullosaMay 22ndXinnate ABNCT06594393
Added 3 new sites: Rome, Italy, Barcelona, Spain, Uppsala, Sweden

Added 3 new sites: Rome, Italy, Barcelona, Spain, Uppsala, Sweden

sites_addedEosinophilic EsophagitisMay 21stRegeneron PharmaceuticalsNCT07112378
Added 2 new sites: Boston, Massachusetts, Philadelphia, Pennsylvania

Added 2 new sites: Boston, Massachusetts, Philadelphia, Pennsylvania

sites_removedDuchenne Muscular DystrophyMay 21stHoffmann-La RocheNCT06450639
Reduced from 22 to 19 sites

Reduced from 22 to 19 sites

eligibility_changeDuchenne Muscular DystrophyMay 21stHoffmann-La RocheNCT06450639
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 21stLongboard PharmaceuticalsNCT06660394
Added 12 new sites: Washington D.C., District of Columbia, Edegem, Belgium, Porto Alegre, Brazil and 9 more

Added 12 new sites: Washington D.C., District of Columbia, Edegem, Belgium, Porto Alegre, Brazil and 9 more

sites_addedDravet SyndromeMay 21stIonis Pharmaceuticals, Inc.NCT07531745
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_removedDravet SyndromeMay 21stUniversity of Colorado, DenverNCT06598449
Reduced from 3 to 1 sites

Reduced from 3 to 1 sites

Enrollment UpdateDravet SyndromeMay 21stUniversity of Colorado, DenverNCT06598449
Target enrollment decreased from 12 to 5

Target enrollment decreased from 12 to 5

eligibility_changeDravet SyndromeMay 21stUniversity of Colorado, DenverNCT06598449
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 21stEncoded TherapeuticsNCT05419492
Added 1 new site: Rochester, Minnesota

Added 1 new site: Rochester, Minnesota

sites_addedDermatomyositisMay 21stPfizerNCT06698796
Added 17 new sites: Allen, Texas, Beckley, West Virginia, SAN M. de Tucuman, Argentina and 14 more

Added 17 new sites: Allen, Texas, Beckley, West Virginia, SAN M. de Tucuman, Argentina and 14 more

sites_addedCongenital Adrenal HyperplasiaMay 21stCrinetics Pharmaceuticals Inc.NCT07144163
Added 10 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Dallas, Texas and 7 more

Added 10 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Dallas, Texas and 7 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 21stSanofiNCT06290141
Added 4 new sites: Hershey, Pennsylvania, Houston, Texas, Belo Horizonte, Brazil and 1 more

Added 4 new sites: Hershey, Pennsylvania, Houston, Texas, Belo Horizonte, Brazil and 1 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 21stGrifols Therapeutics LLCNCT07540221
Added 3 new sites: Rancho Mirage, California, New York, New York, Sherman, Texas

Added 3 new sites: Rancho Mirage, California, New York, New York, Sherman, Texas

sites_addedCholangiocarcinomaMay 21stBold Therapeutics, Inc.NCT04421820
Added 6 new sites: Milan, Italy, Naples, Italy, Pisa, Italy and 3 more

Added 6 new sites: Milan, Italy, Naples, Italy, Pisa, Italy and 3 more

eligibility_changeCentral Diabetes InsipidusMay 21stMassachusetts General HospitalNCT07568509
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisMay 20thMassachusetts General HospitalNCT05966038
Added 2 new sites: Grand Rapids, Michigan, Philadelphia, Pennsylvania

Added 2 new sites: Grand Rapids, Michigan, Philadelphia, Pennsylvania

sites_removedBecker Muscular DystrophyMay 20thSatellos Bioscience, Inc.NCT07287189
Reduced from 25 to 21 sites

Reduced from 25 to 21 sites

sites_removedBecker Muscular DystrophyMay 20thHoffmann-La RocheNCT06450639
Reduced from 22 to 19 sites

Reduced from 22 to 19 sites

eligibility_changeBecker Muscular DystrophyMay 20thHoffmann-La RocheNCT06450639
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 20thAlnylam PharmaceuticalsNCT07223203
Added 11 new sites: Baltimore, Maryland, São Paulo, Brazil, Le Kremlin-Bicêtre, France and 8 more

Added 11 new sites: Baltimore, Maryland, São Paulo, Brazil, Le Kremlin-Bicêtre, France and 8 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 20thAlnylam PharmaceuticalsNCT07052903
Added 15 new sites: Evanston, Illinois, Portland, Oregon, Charleston, South Carolina and 12 more

Added 15 new sites: Evanston, Illinois, Portland, Oregon, Charleston, South Carolina and 12 more

sites_removedAmyotrophic Lateral SclerosisMay 20thNovartis PharmaceuticalsNCT06643481
Reduced from 86 to 74 sites

Reduced from 86 to 74 sites

eligibility_changeAmyotrophic Lateral SclerosisMay 20thQurAlis CorporationNCT05633459
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedWaldenström MacroglobulinemiaMay 19thAbbVieNCT07387471
Added 5 new sites: Nagoya, Japan, Fukuoka, Japan, Koto-ku, Japan and 2 more

Added 5 new sites: Nagoya, Japan, Fukuoka, Japan, Koto-ku, Japan and 2 more

sites_addedTurner SyndromeMay 19thAscendis Pharma A/SNCT07221851
Added 2 new sites: Le Kremlin-Bicêtre, France, Seoul, South Korea

Added 2 new sites: Le Kremlin-Bicêtre, France, Seoul, South Korea

sites_addedWaldenström MacroglobulinemiaMay 19thAbbVieNCT07387471
Added 4 new sites: Kyoto, Japan, Sakai-shi, Japan, Chuo-shi, Japan and 1 more

Added 4 new sites: Kyoto, Japan, Sakai-shi, Japan, Chuo-shi, Japan and 1 more

sites_addedStargardt DiseaseMay 19thAstellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Southaven, Mississippi

Added 1 new site: Southaven, Mississippi

eligibility_changeSpinal Muscular AtrophyMay 19thColumbia UniversityNCT06955897
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 19thNovartis PharmaceuticalsNCT06546670
Added 1 new site: Providence, Rhode Island

Added 1 new site: Providence, Rhode Island

sites_addedSickle Cell DiseaseMay 19thNovartis PharmaceuticalsNCT06439082
Added 1 new site: Orange, California

Added 1 new site: Orange, California

eligibility_changeSickle Cell DiseaseMay 19thNew York UniversityNCT06318143
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 19thCSL BehringNCT06699849
Added 2 new sites: Louisville, Kentucky, Greenville, North Carolina

Added 2 new sites: Louisville, Kentucky, Greenville, North Carolina

eligibility_changePrimary Sclerosing CholangitisMay 18thNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT04685200
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 18thNovartis PharmaceuticalsNCT07229235
Added 2 new sites: Milan, Italy, Torino, Italy

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

eligibility_changeRetinitis PigmentosaMay 18thSparingVisionNCT05748873
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionMay 18thPfizerNCT06137742
Added 36 new sites: Costa Mesa, California, Orange, California, Sacramento, California and 33 more

Added 36 new sites: Costa Mesa, California, Orange, California, Sacramento, California and 33 more

sites_addedPulmonary Arterial HypertensionMay 18thMerck Sharp & Dohme LLCNCT07218029
Added 6 new sites: Houston, Texas, Calgary, Canada, Hanover, Germany and 3 more

Added 6 new sites: Houston, Texas, Calgary, Canada, Hanover, Germany and 3 more

sites_addedPulmonary Arterial HypertensionMay 18thApollo Therapeutics LtdNCT06846554
Added 1 new site: Cambridge, United Kingdom

Added 1 new site: Cambridge, United Kingdom

sites_addedProgressive Supranuclear PalsyMay 18thNovartis PharmaceuticalsNCT07498426
Added 1 new site: Ulm, Germany

Added 1 new site: Ulm, Germany

sites_addedPrimary Biliary CholangitisMay 18thUmecrine Cognition ABNCT07304843
Added 4 new sites: Balçova, Turkey (Türkiye), Rize, Turkey (Türkiye), Sanliurfa, Turkey (Türkiye) and 1 more

Added 4 new sites: Balçova, Turkey (Türkiye), Rize, Turkey (Türkiye), Sanliurfa, Turkey (Türkiye) and 1 more

Enrollment UpdatePrimary Biliary CholangitisMay 18thZydus Therapeutics Inc.NCT06427395
Target enrollment decreased from 150 to 102

Target enrollment decreased from 150 to 102

sites_addedPolycythemia VeraMay 18thPrelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

sites_addedPolycythemia VeraMay 18thDisc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedPolycythemia VeraMay 18thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Genolier, Switzerland

Added 1 new site: Genolier, Switzerland

sites_addedPolycythemia VeraMay 18thNational Cancer Institute (NCI)NCT06661915
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedPolycystic Kidney DiseaseMay 18thVertex Pharmaceuticals IncorporatedNCT07161037
Added 2 new sites: Nashville, Tennessee, Groningen, Netherlands

Added 2 new sites: Nashville, Tennessee, Groningen, Netherlands

sites_addedPolycystic Kidney DiseaseMay 18thPYC TherapeuticsNCT06714006
Added 1 new site: Concord, Australia

Added 1 new site: Concord, Australia

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 18thHoffmann-La RocheNCT04654468
Added 1 new site: Guangzhou, China

Added 1 new site: Guangzhou, China

sites_addedParoxysmal Nocturnal HemoglobinuriaMay 18thRegeneron PharmaceuticalsNCT07154745
Added 3 new sites: Seoul, South Korea, Murcia, Spain, Salamanca, Spain

Added 3 new sites: Seoul, South Korea, Murcia, Spain, Salamanca, Spain

sites_addedNoonan SyndromeMay 17thBioMarin PharmaceuticalNCT06668805
Added 12 new sites: Los Angeles, California, Atlanta, Georgia, Chicago, Illinois and 9 more

Added 12 new sites: Los Angeles, California, Atlanta, Georgia, Chicago, Illinois and 9 more

Enrollment UpdateNoonan SyndromeMay 17thBioMarin PharmaceuticalNCT06668805
Target enrollment decreased from 54 to 30

Target enrollment decreased from 54 to 30

phase_changeNeuromyelitis OpticaMay 17thTianjin Medical University General HospitalNCT07420296
Moved from Phase 4 to Phase 3

Moved from Phase 4 to Phase 3

sites_addedMyotonic DystrophyMay 17thDyne TherapeuticsNCT05481879
Added 3 new sites: Stanford, California, Gainesville, Florida, St Louis, Missouri

Added 3 new sites: Stanford, California, Gainesville, Florida, St Louis, Missouri

sites_addedMyelofibrosisMay 17thPrelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

sites_addedMyelofibrosisMay 17thAjax Therapeutics, Inc., a wholly owned subsidiary of Eli Lilly and CompanyNCT06343805
Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

Added 5 new sites: Bologna, Italy, Milan, Italy, Madrid, Spain and 2 more

sites_addedMyelofibrosisMay 17thDisc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedMyelofibrosisMay 17thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Genolier, Switzerland

Added 1 new site: Genolier, Switzerland

sites_addedMyelofibrosisMay 17thNational Cancer Institute (NCI)NCT06661915
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_removedMyelodysplastic SyndromesMay 17thMemorial Sloan Kettering Cancer CenterNCT07566377
Reduced from 7 to 1 sites

Reduced from 7 to 1 sites

eligibility_changeMyelodysplastic SyndromesMay 17thGCP-Service International West GmbHNCT05583552
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesMay 17thNational Cancer Institute (NCI)NCT05564390
Added 2 new sites: Los Angeles, California, Stony Brook, New York

Added 2 new sites: Los Angeles, California, Stony Brook, New York

sites_addedMyelodysplastic SyndromesMay 17thDisc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedMyelodysplastic SyndromesMay 17thNational Cancer Institute (NCI)NCT06577441
Added 1 new site: Stony Brook, New York

Added 1 new site: Stony Brook, New York

eligibility_changeMyelodysplastic SyndromesMay 17thFred Hutchinson Cancer CenterNCT06928662
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesMay 17thTakedaNCT06499285
Added 26 new sites: Kansas City, Missouri, Lake Success, New York, Winston-Salem, North Carolina and 23 more

Added 26 new sites: Kansas City, Missouri, Lake Success, New York, Winston-Salem, North Carolina and 23 more

sites_addedMyasthenia GravisMay 17thAstraZenecaNCT07247279
Added 1 new site: Saint Petersburg, Russia

Added 1 new site: Saint Petersburg, Russia

sites_addedMyasthenia GravisMay 17thKyverna TherapeuticsNCT06193889
Added 2 new sites: Columbia, Missouri, Munich, Germany

Added 2 new sites: Columbia, Missouri, Munich, Germany

sites_addedMyasthenia GravisMay 17thNovartis PharmaceuticalsNCT06517758
Added 1 new site: Porto Alegre, Brazil

Added 1 new site: Porto Alegre, Brazil

sites_addedMultiple System AtrophyMay 17thTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 1 new site: Düsseldorf, Germany

Added 1 new site: Düsseldorf, Germany

sites_removedMultiple SclerosisMay 17thSanofiNCT06141473
Reduced from 384 to 382 sites

Reduced from 384 to 382 sites

Enrollment UpdateMultiple SclerosisMay 17thSanofiNCT06141473
Target enrollment increased from 1600 to 1655

Target enrollment increased from 1600 to 1655

Enrollment UpdateMultiple SclerosisMay 17thTG Therapeutics, Inc.NCT06433752
Target enrollment increased from 500 to 2000

Target enrollment increased from 500 to 2000

sites_addedMultiple SclerosisMay 17thNovartis PharmaceuticalsNCT07225504
Added 15 new sites: Homestead, Florida, Foxborough, Massachusetts, Greenville, South Carolina and 12 more

Added 15 new sites: Homestead, Florida, Foxborough, Massachusetts, Greenville, South Carolina and 12 more

sites_removedMultiple SclerosisMay 17thSanofiNCT06141486
Reduced from 338 to 311 sites

Reduced from 338 to 311 sites

sites_addedMultiple SclerosisMay 17thNovartis PharmaceuticalsNCT06846281
Added 3 new sites: Buffalo, New York, São Paulo, Brazil, Bochum, Germany

Added 3 new sites: Buffalo, New York, São Paulo, Brazil, Bochum, Germany

sites_addedMultiple SclerosisMay 17thSanofiNCT07325292
Added 6 new sites: Fort Collins, Colorado, Ghent, Belgium, Chengdu, China and 3 more

Added 6 new sites: Fort Collins, Colorado, Ghent, Belgium, Chengdu, China and 3 more

sites_addedMultiple SclerosisMay 17thAmgenNCT06700343
Added 1 new site: Pensacola, Florida

Added 1 new site: Pensacola, Florida

sites_addedMesotheliomaMay 17thVerismo TherapeuticsNCT05568680
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

sites_addedMembranous NephropathyMay 17thNkarta, Inc.NCT06557265
Added 2 new sites: Tustin, California, Summit, New Jersey

Added 2 new sites: Tustin, California, Summit, New Jersey

sites_addedMembranous NephropathyMay 17thVertex Pharmaceuticals IncorporatedNCT07204275
Added 7 new sites: Belo Horizonte, Brazil, Harbin, China, Shijiazhuang, China and 4 more

Added 7 new sites: Belo Horizonte, Brazil, Harbin, China, Shijiazhuang, China and 4 more

sites_addedLight Chain AmyloidosisMay 16thAbbVieNCT06158854
Added 2 new sites: Nashville, Tennessee, Pavia, Italy

Added 2 new sites: Nashville, Tennessee, Pavia, Italy

sites_addedIgA NephropathyMay 16thHoffmann-La RocheNCT05797610
Added 2 new sites: Doral, Florida, Cleveland, Ohio

Added 2 new sites: Doral, Florida, Cleveland, Ohio

Enrollment UpdateIdiopathic Pulmonary FibrosisMay 16thAssiut UniversityNCT05842681
Target enrollment increased from 30 to 1000

Target enrollment increased from 30 to 1000

eligibility_changeIdiopathic Pulmonary FibrosisMay 16thAssiut UniversityNCT05842681
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisMay 16thBoehringer IngelheimNCT06238622
Added 3 new sites: The Woodlands, Texas, Vienna, Austria, Exeter, United Kingdom

Added 3 new sites: The Woodlands, Texas, Vienna, Austria, Exeter, United Kingdom

sites_addedHunter SyndromeMay 16thDenali Therapeutics Inc.NCT06075537
Added 1 new site: Manchester, United Kingdom

Added 1 new site: Manchester, United Kingdom

sites_removedHodgkin LymphomaMay 16thMemorial Sloan Kettering Cancer CenterNCT07566377
Reduced from 7 to 1 sites

Reduced from 7 to 1 sites

eligibility_changeHodgkin LymphomaMay 16thUniversity of UtahNCT05723055
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHereditary AngioedemaMay 16thIntellia TherapeuticsNCT06634420
Target enrollment increased from 60 to 80

Target enrollment increased from 60 to 80

Enrollment UpdateHereditary AngioedemaMay 16thCSL BehringNCT06806657
Target enrollment decreased from 30 to 18

Target enrollment decreased from 30 to 18

Enrollment UpdateFragile X SyndromeMay 15thHospices Civils de LyonNCT06868979
Target enrollment increased from 88 to 118

Target enrollment increased from 88 to 118

eligibility_changeFragile X SyndromeMay 15thHospices Civils de LyonNCT06868979
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BMay 15thCSL BehringNCT07080905
Added 1 new site: Madrid, Spain

Added 1 new site: Madrid, Spain

sites_addedHemophilia BMay 15thPfizerNCT03861273
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedHemophilia BMay 15thPfizerNCT05568719
Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

sites_addedHemophilia BMay 15thPfizerNCT03307980
Added 13 new sites: Sacramento, California, Sacramento, California, Metairie, Louisiana and 10 more

Added 13 new sites: Sacramento, California, Sacramento, California, Metairie, Louisiana and 10 more

sites_addedHemophilia AMay 15thOctapharmaNCT05936580
Added 1 new site: Stasbourg, France

Added 1 new site: Stasbourg, France

sites_addedHemophilia AMay 15thPfizerNCT05568719
Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

Added 11 new sites: Rancho Cordova, California, Sacramento, California, Sacramento, California and 8 more

sites_addedGraves’ DiseaseMay 15thBiohaven Therapeutics Ltd.NCT06980649
Added 4 new sites: Greenville, North Carolina, Canton, Ohio, Memphis, Tennessee and 1 more

Added 4 new sites: Greenville, North Carolina, Canton, Ohio, Memphis, Tennessee and 1 more

sites_addedGeographic AtrophyMay 15thNovartis PharmaceuticalsNCT07441642
Added 2 new sites: Erie, Pennsylvania, Bellaire, Texas

Added 2 new sites: Erie, Pennsylvania, Bellaire, Texas

sites_addedFocal Segmental GlomerulosclerosisMay 15thBoehringer IngelheimNCT07220083
Added 10 new sites: Brunswick, Georgia, Ahmedabad, India, Dehradun, India and 7 more

Added 10 new sites: Brunswick, Georgia, Ahmedabad, India, Dehradun, India and 7 more

sites_addedFocal EpilepsyMay 15thXenon Pharmaceuticals Inc.NCT05718817
Added 3 new sites: Buenos Aires, Argentina, San Luis, Argentina, Kuopio, Finland

Added 3 new sites: Buenos Aires, Argentina, San Luis, Argentina, Kuopio, Finland

sites_addedFocal EpilepsyMay 15thXenon Pharmaceuticals Inc.NCT05716100
Added 1 new site: Portland, Oregon

Added 1 new site: Portland, Oregon

sites_addedEssential ThrombocythemiaMay 15thNational Cancer Institute (NCI)NCT06661915
Added 1 new site: Sacramento, California

Added 1 new site: Sacramento, California

sites_addedEssential ThrombocythemiaMay 15thMerck Sharp & Dohme LLCNCT06456346
Added 3 new sites: Amarillo, Texas, Dallas, Texas, Webster, Texas

Added 3 new sites: Amarillo, Texas, Dallas, Texas, Webster, Texas

sites_addedEssential ThrombocythemiaMay 15thPrelude Therapeutics IncorporatedNCT07469891
Added 1 new site: Coral Springs, Florida

Added 1 new site: Coral Springs, Florida

sites_addedEssential ThrombocythemiaMay 15thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Genolier, Switzerland

Added 1 new site: Genolier, Switzerland

sites_addedEssential ThrombocythemiaMay 15thDisc Medicine, IncNCT05320198
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_removedDuchenne Muscular DystrophyMay 14thSatellos Bioscience, Inc.NCT07287189
Reduced from 25 to 21 sites

Reduced from 25 to 21 sites

sites_addedDravet SyndromeMay 14thEpygenixNCT04462770
Added 13 new sites: Birmingham, Alabama, Little Rock, Arkansas, Morrisville, North Carolina and 10 more

Added 13 new sites: Birmingham, Alabama, Little Rock, Arkansas, Morrisville, North Carolina and 10 more

sites_addedCystic FibrosisMay 14thSpliSense Ltd.NCT06429176
Added 2 new sites: Los Angeles, California, Denver, Colorado

Added 2 new sites: Los Angeles, California, Denver, Colorado

Enrollment UpdateCystic FibrosisMay 14thSpliSense Ltd.NCT06429176
Target enrollment increased from 24 to 64

Target enrollment increased from 24 to 64

eligibility_changeCystic FibrosisMay 14thSpliSense Ltd.NCT06429176
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaMay 14thEli Lilly and CompanyNCT07213791
Added 1 new site: Chiba, Japan

Added 1 new site: Chiba, Japan

Enrollment UpdateEhlers-Danlos SyndromeMay 14thHospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

Enrollment UpdateDuchenne Muscular DystrophyMay 14thShanghai Siponuoyin Biotechnology Co LtdNCT07188012
Target enrollment increased from 6 to 9

Target enrollment increased from 6 to 9

eligibility_changeDuchenne Muscular DystrophyMay 14thShanghai Siponuoyin Biotechnology Co LtdNCT07188012
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateDilated CardiomyopathyMay 14thHospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_removedDermatomyositisMay 14thPfizerNCT06698796
Reduced from 24 to 7 sites

Reduced from 24 to 7 sites

sites_addedCystic FibrosisMay 14thKrystal Biotech, Inc.NCT05504837
Added 1 new site: Boise, Idaho

Added 1 new site: Boise, Idaho

Enrollment UpdateCystic FibrosisMay 14thKrystal Biotech, Inc.NCT05504837
Target enrollment increased from 12 to 20

Target enrollment increased from 12 to 20

eligibility_changeCystic FibrosisMay 14thKrystal Biotech, Inc.NCT05504837
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCystic FibrosisMay 14thArcturus Therapeutics, Inc.NCT06747858
Added 6 new sites: Washington D.C., District of Columbia, St Louis, Missouri, Cleveland, Ohio and 3 more

Added 6 new sites: Washington D.C., District of Columbia, St Louis, Missouri, Cleveland, Ohio and 3 more

Enrollment UpdateCystic FibrosisMay 14thArcturus Therapeutics, Inc.NCT06747858
Target enrollment increased from 12 to 33

Target enrollment increased from 12 to 33

eligibility_changeCystic FibrosisMay 14thArcturus Therapeutics, Inc.NCT06747858
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14thDianthus TherapeuticsNCT06858579
Added 14 new sites: Nashville, Tennessee, Buenos Aires, Argentina, Liverpool, Australia and 11 more

Added 14 new sites: Nashville, Tennessee, Buenos Aires, Argentina, Liverpool, Australia and 11 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14thJanssen Research & Development, LLCNCT05327114
Added 5 new sites: Marseille, France, Nîmes, France, Milan, Italy and 2 more

Added 5 new sites: Marseille, France, Nîmes, France, Milan, Italy and 2 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14thargenxNCT06920004
Added 27 new sites: Tampa, Florida, Rosario, Argentina, Vienna, Austria and 24 more

Added 27 new sites: Tampa, Florida, Rosario, Argentina, Vienna, Austria and 24 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 14thargenxNCT07091630
Added 24 new sites: Rancho Mirage, California, Naples, Florida, Philadelphia, Pennsylvania and 21 more

Added 24 new sites: Rancho Mirage, California, Naples, Florida, Philadelphia, Pennsylvania and 21 more

sites_addedCholangiocarcinomaMay 14thVerismo TherapeuticsNCT05568680
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

eligibility_changeCholangiocarcinomaMay 14thNantes University HospitalNCT06735560
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaMay 14thInstitut de Recherches Internationales ServierNCT06501625
Added 1 new site: Los Alamitos, California

Added 1 new site: Los Alamitos, California

Enrollment UpdateCatecholaminergic Polymorphic Ventricular TachycardiaMay 14thHospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

Enrollment UpdateBecker Muscular DystrophyMay 13thShanghai Siponuoyin Biotechnology Co LtdNCT07188012
Target enrollment increased from 6 to 9

Target enrollment increased from 6 to 9

eligibility_changeBecker Muscular DystrophyMay 13thShanghai Siponuoyin Biotechnology Co LtdNCT07188012
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAtypical Hemolytic Uremic SyndromeMay 13thNovartis PharmaceuticalsNCT05795140
Added 2 new sites: Iruma-gun, Japan, Mersin, Turkey (Türkiye)

Added 2 new sites: Iruma-gun, Japan, Mersin, Turkey (Türkiye)

Enrollment UpdateAtypical Hemolytic Uremic SyndromeMay 13thNovartis PharmaceuticalsNCT05795140
Target enrollment decreased from 125 to 65

Target enrollment decreased from 125 to 65

eligibility_changeAtypical Hemolytic Uremic SyndromeMay 13thNovartis PharmaceuticalsNCT05795140
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 13thEidos Therapeutics, a BridgeBio companyNCT06563895
Added 3 new sites: Seattle, Washington, Murdoch, Australia, Woolloongabba, Australia

Added 3 new sites: Seattle, Washington, Murdoch, Australia, Woolloongabba, Australia

eligibility_changeATTR Amyloidosis (Transthyretin Amyloidosis)May 13thEidos Therapeutics, a BridgeBio companyNCT06563895
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedAmyotrophic Lateral SclerosisMay 13thUniversity of AlbertaNCT03362658
Reduced from 9 to 7 sites

Reduced from 9 to 7 sites

Enrollment UpdateAmyotrophic Lateral SclerosisMay 13thUniversity of AlbertaNCT03362658
Target enrollment decreased from 700 to 119

Target enrollment decreased from 700 to 119

sites_addedAchondroplasiaMay 13thBioMarin PharmaceuticalNCT07441876
Added 2 new sites: California City, California, Parkville, Australia

Added 2 new sites: California City, California, Parkville, Australia

sites_removedWaldenström MacroglobulinemiaMay 12thChristian BuskeNCT04263480
Reduced from 20 to 17 sites

Reduced from 20 to 17 sites

sites_removedSystemic Juvenile Idiopathic ArthritisMay 12thEli Lilly and CompanyNCT03773965
Reduced from 82 to 80 sites

Reduced from 82 to 80 sites

sites_addedSickle Cell DiseaseMay 12thDisc Medicine, IncNCT07187973
Added 2 new sites: Birmingham, Alabama, Augusta, Georgia

Added 2 new sites: Birmingham, Alabama, Augusta, Georgia

sites_addedSickle Cell DiseaseMay 12thCSL BehringNCT07224360
Added 2 new sites: Little Rock, Arkansas, Bethesda, Maryland

Added 2 new sites: Little Rock, Arkansas, Bethesda, Maryland

sites_addedWaldenström MacroglobulinemiaMay 12thBeOne MedicinesNCT05006716
Added 1 new site: Shenyang, China

Added 1 new site: Shenyang, China

sites_addedWaldenström MacroglobulinemiaMay 12thJanssen Research & Development, LLCNCT01804686
Added 2 new sites: Melbourne, Australia, Warsaw, Poland

Added 2 new sites: Melbourne, Australia, Warsaw, Poland

Enrollment UpdateVascular Ehlers-Danlos SyndromeMay 12thHospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_addedTuberous SclerosisMay 12thNational Tuberous Sclerosis AssociationNCT05676099
Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

sites_addedStickler SyndromeMay 12thHelen Keller Eye Research FoundationNCT07146516
Added 2 new sites: Royal Oak, Michigan, Westbury, New York

Added 2 new sites: Royal Oak, Michigan, Westbury, New York

sites_addedStargardt DiseaseMay 12thAstellas Pharma Global Development, Inc.NCT07425574
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

phase_changeSickle Cell DiseaseMay 12thChildren's Hospital Medical Center, CincinnatiNCT06290401
Moved from Phase 2 to N/A

Moved from Phase 2 to N/A

Enrollment UpdateSickle Cell DiseaseMay 12thSt. Jude Children's Research HospitalNCT05810181
Target enrollment increased from 70 to 145

Target enrollment increased from 70 to 145

eligibility_changeSickle Cell DiseaseMay 12thSt. Jude Children's Research HospitalNCT05810181
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 12thDisc Medicine, IncNCT07187973
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

eligibility_changeSickle Cell DiseaseMay 12thBoston Children's HospitalNCT06555939
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedRetinitis PigmentosaMay 11thJanssen Research & Development, LLCNCT04794101
Added 3 new sites: Bellaire, Texas, Milan, Italy, Rome, Italy

Added 3 new sites: Bellaire, Texas, Milan, Italy, Rome, Italy

sites_removedPulmonary Arterial HypertensionMay 11thPfizerNCT06137742
Reduced from 67 to 31 sites

Reduced from 67 to 31 sites

sites_addedPrimary Sclerosing CholangitisMay 11thPSC Partners Seeking a CureNCT06297993
Added 2 new sites: Dallas, Texas, Oxford, United Kingdom

Added 2 new sites: Dallas, Texas, Oxford, United Kingdom

sites_addedPolycythemia VeraMay 11thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Berkeley Heights, New Jersey

Added 1 new site: Berkeley Heights, New Jersey

Enrollment UpdatePolycythemia VeraMay 11thIcahn School of Medicine at Mount SinaiNCT05835466
Target enrollment decreased from 26 to 10

Target enrollment decreased from 26 to 10

sites_addedPhenylketonuriaMay 11thPTC TherapeuticsNCT06302348
Added 3 new sites: Tours, France, Dublin, Ireland, Warsaw, Poland

Added 3 new sites: Tours, France, Dublin, Ireland, Warsaw, Poland

sites_addedPhelan-McDermid SyndromeMay 11thNeuren Pharmaceuticals LimitedNCT07281079
Added 2 new sites: Brookline, Massachusetts, Cincinnati, Ohio

Added 2 new sites: Brookline, Massachusetts, Cincinnati, Ohio

sites_addedOsteogenesis ImperfectaMay 11thAmgenNCT07366086
Added 1 new site: Okayama, Japan

Added 1 new site: Okayama, Japan

sites_addedNarcolepsy Type 1May 10thTakedaNCT07363720
Added 11 new sites: Middletown, New Jersey, Durham, North Carolina, Winston-Salem, North Carolina and 8 more

Added 11 new sites: Middletown, New Jersey, Durham, North Carolina, Winston-Salem, North Carolina and 8 more

sites_addedNarcolepsy Type 1May 10thAlkermes, Inc.NCT07455383
Added 6 new sites: Brandon, Florida, Macon, Georgia, Lansing, Michigan and 3 more

Added 6 new sites: Brandon, Florida, Macon, Georgia, Lansing, Michigan and 3 more

sites_addedMyotonic DystrophyMay 10thDyne TherapeuticsNCT07486934
Added 1 new site: Toyonaka-Shi, Japan

Added 1 new site: Toyonaka-Shi, Japan

sites_addedMyelofibrosisMay 10thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Berkeley Heights, New Jersey

Added 1 new site: Berkeley Heights, New Jersey

Enrollment UpdateMyelofibrosisMay 10thIcahn School of Medicine at Mount SinaiNCT05835466
Target enrollment decreased from 26 to 10

Target enrollment decreased from 26 to 10

sites_removedMyelofibrosisMay 10thBristol-Myers SquibbNCT04817007
Reduced from 53 to 49 sites

Reduced from 53 to 49 sites

Enrollment UpdateMyelodysplastic SyndromesMay 10thM.D. Anderson Cancer CenterNCT04493138
Target enrollment decreased from 58 to 30

Target enrollment decreased from 58 to 30

Enrollment UpdateMyelodysplastic SyndromesMay 10thH. Lee Moffitt Cancer Center and Research InstituteNCT05732961
Target enrollment decreased from 70 to 6

Target enrollment decreased from 70 to 6

sites_addedMyelodysplastic SyndromesMay 10thNational Cancer Institute (NCI)NCT05554406
Added 2 new sites: Los Angeles, California, Pembroke Pines, Florida

Added 2 new sites: Los Angeles, California, Pembroke Pines, Florida

sites_addedMyelodysplastic SyndromesMay 10thGenmabNCT07384715
Added 9 new sites: Aarhus, Denmark, Copenhagen, Denmark, Berlin, Germany and 6 more

Added 9 new sites: Aarhus, Denmark, Copenhagen, Denmark, Berlin, Germany and 6 more

sites_addedMyelodysplastic SyndromesMay 10thPedAL BCU, LLCNCT04726241
Added 1 new site: Winfield, Illinois

Added 1 new site: Winfield, Illinois

sites_removedMyelodysplastic SyndromesMay 10thBristol-Myers SquibbNCT05949684
Reduced from 169 to 144 sites

Reduced from 169 to 144 sites

phase_changeMyelodysplastic SyndromesMay 10thUniversity of VirginiaNCT04482894
Moved from Phase 2 to N/A

Moved from Phase 2 to N/A

sites_addedMyasthenia GravisMay 10thNovartis PharmaceuticalsNCT06517758
Added 2 new sites: Córdoba, Argentina, Joinville, Brazil

Added 2 new sites: Córdoba, Argentina, Joinville, Brazil

sites_addedMyasthenia GravisMay 10thJanssen Research & Development, LLCNCT07217587
Added 4 new sites: Pomona, California, Fort Collins, Colorado, Raleigh, North Carolina and 1 more

Added 4 new sites: Pomona, California, Fort Collins, Colorado, Raleigh, North Carolina and 1 more

sites_addedMyasthenia GravisMay 10thNovartis PharmaceuticalsNCT06744920
Added 1 new site: Niš, Serbia

Added 1 new site: Niš, Serbia

sites_addedMyasthenia GravisMay 10thUCB Biopharma SRLNCT06149559
Added 2 new sites: Denton, Texas, Ankara, Turkey (Türkiye)

Added 2 new sites: Denton, Texas, Ankara, Turkey (Türkiye)

sites_addedMyasthenia GravisMay 10thVertex Pharmaceuticals IncorporatedNCT07501702
Added 3 new sites: Boca Raton, Florida, Homestead, Florida, Las Vegas, Nevada

Added 3 new sites: Boca Raton, Florida, Homestead, Florida, Las Vegas, Nevada

sites_addedMyasthenia GravisMay 10thVor BiopharmaNCT06456580
Added 34 new sites: Tampa, Florida, Mendoza, Argentina, Liège, Belgium and 31 more

Added 34 new sites: Tampa, Florida, Mendoza, Argentina, Liège, Belgium and 31 more

sites_addedMultiple System AtrophyMay 10thTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 2 new sites: Salerno, Italy, Niigata, Japan

Added 2 new sites: Salerno, Italy, Niigata, Japan

Enrollment UpdateMultiple SclerosisMay 10thNYU Langone HealthNCT07011914
Target enrollment decreased from 386 to 300

Target enrollment decreased from 386 to 300

Enrollment UpdateMultiple SclerosisMay 10thPoitiers University HospitalNCT05491031
Target enrollment decreased from 250 to 50

Target enrollment decreased from 250 to 50

sites_addedMultiple SclerosisMay 10thNovartis PharmaceuticalsNCT07225504
Added 22 new sites: Washington D.C., District of Columbia, Miami, Florida, Albuquerque, New Mexico and 19 more

Added 22 new sites: Washington D.C., District of Columbia, Miami, Florida, Albuquerque, New Mexico and 19 more

sites_addedMultiple SclerosisMay 10thCelgeneNCT06782490
Added 4 new sites: Birmingham, Alabama, Southfield, Michigan, Hradec Králové, Czechia and 1 more

Added 4 new sites: Birmingham, Alabama, Southfield, Michigan, Hradec Králové, Czechia and 1 more

sites_addedMultiple SclerosisMay 10thSandozNCT06847724
Added 1 new site: Lublin, Poland

Added 1 new site: Lublin, Poland

sites_addedMultiple SclerosisMay 10thZenas BioPharma (USA), LLCNCT07299019
Added 4 new sites: Scottsdale, Arizona, Boston, Massachusetts, San Antonio, Texas and 1 more

Added 4 new sites: Scottsdale, Arizona, Boston, Massachusetts, San Antonio, Texas and 1 more

sites_addedMultiple SclerosisMay 10thNovartis PharmaceuticalsNCT05344469
Added 4 new sites: Albstadt, Germany, Magdeburg, Germany, Schneverdingen, Germany and 1 more

Added 4 new sites: Albstadt, Germany, Magdeburg, Germany, Schneverdingen, Germany and 1 more

sites_removedMesotheliomaMay 10thUniversity of NebraskaNCT02012699
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedMembranous NephropathyMay 10thBiogenNCT06962800
Added 2 new sites: St Albans, Australia, Chandigarh, India

Added 2 new sites: St Albans, Australia, Chandigarh, India

Enrollment UpdateMarfan SyndromeMay 9thHospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_addedLymphangioleiomyomatosisMay 9thNational Tuberous Sclerosis AssociationNCT05676099
Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

Added 3 new sites: Kansas City, Missouri, Rochester, New York, Montreal, Canada

sites_addedLight Chain AmyloidosisMay 9thShanghai Chia Tai Tianqing Pharmaceutical Technology Development Co., Ltd.NCT07266116
Added 4 new sites: Guangzhou, China, Changchun, China, Changchun, China and 1 more

Added 4 new sites: Guangzhou, China, Changchun, China, Changchun, China and 1 more

eligibility_changeLight Chain AmyloidosisMay 9thShanghai Chia Tai Tianqing Pharmaceutical Technology Development Co., Ltd.NCT07266116
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLight Chain AmyloidosisMay 9thSWOG Cancer Research NetworkNCT06022939
Added 84 new sites: Goodyear, Arizona, Tucson, Arizona, Tucson, Arizona and 81 more

Added 84 new sites: Goodyear, Arizona, Tucson, Arizona, Tucson, Arizona and 81 more

sites_addedIgA NephropathyMay 9thBiogenNCT06935357
Added 5 new sites: Philadelphia, Pennsylvania, Santa Fe, Argentina, Fontevivo, Italy and 2 more

Added 5 new sites: Philadelphia, Pennsylvania, Santa Fe, Argentina, Fontevivo, Italy and 2 more

sites_addedIgA NephropathyMay 9thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT05248659
Added 158 new sites: Birmingham, Alabama, Los Angeles, California, Palo Alto, California and 155 more

Added 158 new sites: Birmingham, Alabama, Los Angeles, California, Palo Alto, California and 155 more

eligibility_changeIgA NephropathyMay 9thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT05248659
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyMay 9thNovartis PharmaceuticalsNCT06858319
Added 10 new sites: Orlando, Florida, New York, New York, Dallas, Texas and 7 more

Added 10 new sites: Orlando, Florida, New York, New York, Dallas, Texas and 7 more

sites_addedIdiopathic Pulmonary FibrosisMay 9thMediar TherapeuticsNCT06967805
Added 3 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Curitiba, Brazil

Added 3 new sites: Atlanta, Georgia, Pittsburgh, Pennsylvania, Curitiba, Brazil

sites_addedIdiopathic Pulmonary FibrosisMay 9thDuke UniversityNCT01915511
Added 3 new sites: Aurora, Colorado, Houston, Texas, Salt Lake City, Utah

Added 3 new sites: Aurora, Colorado, Houston, Texas, Salt Lake City, Utah

sites_addedIdiopathic Pulmonary FibrosisMay 9thVejle HospitalNCT06714123
Added 3 new sites: Copenhagen, Denmark, Leicester, United Kingdom, Norwich, United Kingdom

Added 3 new sites: Copenhagen, Denmark, Leicester, United Kingdom, Norwich, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisMay 9thRein TherapeuticsNCT06968845
Added 2 new sites: New Haven, Connecticut, Edinburgh, United Kingdom

Added 2 new sites: New Haven, Connecticut, Edinburgh, United Kingdom

sites_addedIdiopathic Pulmonary FibrosisMay 9thMannkind CorporationNCT07344558
Added 1 new site: McKinney, Texas

Added 1 new site: McKinney, Texas

sites_addedIdiopathic Pulmonary FibrosisMay 9thUnited TherapeuticsNCT04905693
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedHypoparathyroidismMay 9thAscendis Pharma Bone Diseases A/SNCT07081997
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedHomozygous Familial HypercholesterolemiaMay 9thArrowhead PharmaceuticalsNCT07037771
Added 15 new sites: La Louvière, Belgium, Leuven, Belgium, Cerqueira César, Brazil and 12 more

Added 15 new sites: La Louvière, Belgium, Leuven, Belgium, Cerqueira César, Brazil and 12 more

sites_addedHomocystinuriaMay 9thTravere Therapeutics, Inc.NCT06247085
Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

Added 52 new sites: Phoenix, Arizona, New Haven, Connecticut, Atlanta, Georgia and 49 more

eligibility_changeHomocystinuriaMay 9thTravere Therapeutics, Inc.NCT06247085
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaMay 9thUniversity of WashingtonNCT07097363
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateHereditary Spastic ParaplegiaMay 9thNational Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
Target enrollment decreased from 9300 to 2323

Target enrollment decreased from 9300 to 2323

sites_addedHereditary AngioedemaMay 9thAstria Therapeutics, Inc.NCT06842823
Added 2 new sites: Dallas, Texas, Milan, Italy

Added 2 new sites: Dallas, Texas, Milan, Italy

sites_addedHereditary AngioedemaMay 9thPharvaris Netherlands B.V.NCT06679881
Added 37 new sites: Birmingham, Alabama, Scottsdale, Arizona, Little Rock, Arkansas and 34 more

Added 37 new sites: Birmingham, Alabama, Scottsdale, Arizona, Little Rock, Arkansas and 34 more

sites_addedGraves’ DiseaseMay 8thImmunovant Sciences GmbHNCT06727604
Added 3 new sites: Metairie, Louisiana, Nashville, Tennessee, Lublin, Poland

Added 3 new sites: Metairie, Louisiana, Nashville, Tennessee, Lublin, Poland

sites_addedGraves’ DiseaseMay 8thImmunovant Sciences GmbHNCT07018323
Added 6 new sites: Parkville, Australia, Campinas, Brazil, Rio de Janeiro, Brazil and 3 more

Added 6 new sites: Parkville, Australia, Campinas, Brazil, Rio de Janeiro, Brazil and 3 more

sites_addedGastrointestinal Stromal TumorMay 8thCancer Research UKNCT06380816
Added 2 new sites: London, United Kingdom, Newcastle upon Tyne, United Kingdom

Added 2 new sites: London, United Kingdom, Newcastle upon Tyne, United Kingdom

Enrollment UpdateFamilial Hypertrophic CardiomyopathyMay 8thHospital do CoracaoNCT06546137
Target enrollment increased from 600 to 1211

Target enrollment increased from 600 to 1211

sites_addedEssential ThrombocythemiaMay 8thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Berkeley Heights, New Jersey

Added 1 new site: Berkeley Heights, New Jersey

sites_removedHemophilia BMay 8thPfizerNCT03307980
Reduced from 25 to 12 sites

Reduced from 25 to 12 sites

sites_removedHemophilia BMay 8thPfizerNCT05568719
Reduced from 17 to 8 sites

Reduced from 17 to 8 sites

sites_removedHemophilia BMay 8thPfizerNCT05611801
Reduced from 66 to 34 sites

Reduced from 66 to 34 sites

sites_addedHemophilia AMay 8thHoffmann-La RocheNCT07416604
Added 3 new sites: Orange, California, Indianapolis, Indiana, Seattle, Washington

Added 3 new sites: Orange, California, Indianapolis, Indiana, Seattle, Washington

sites_removedHemophilia AMay 8thPfizerNCT05568719
Reduced from 17 to 8 sites

Reduced from 17 to 8 sites

sites_removedHemophilia AMay 8thPfizerNCT05611801
Reduced from 66 to 34 sites

Reduced from 66 to 34 sites

sites_addedGeographic AtrophyMay 8thNovartis PharmaceuticalsNCT07441642
Added 7 new sites: Sacramento, California, Toms River, New Jersey, Houston, Texas and 4 more

Added 7 new sites: Sacramento, California, Toms River, New Jersey, Houston, Texas and 4 more

sites_addedGeographic AtrophyMay 8thComplement TherapeuticsNCT07392255
Added 1 new site: La Crosse, Wisconsin

Added 1 new site: La Crosse, Wisconsin

Enrollment UpdateGeographic AtrophyMay 8thHoffmann-La RocheNCT06961370
Target enrollment decreased from 132 to 27

Target enrollment decreased from 132 to 27

sites_addedGeographic AtrophyMay 8thComplement TherapeuticsNCT07144137
Added 2 new sites: Gloucester, United Kingdom, London, United Kingdom

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

sites_addedGeographic AtrophyMay 8thAnnexon, Inc.NCT06510816
Added 18 new sites: Fullerton, California, Tampa, Florida, Elmhurst, Illinois and 15 more

Added 18 new sites: Fullerton, California, Tampa, Florida, Elmhurst, Illinois and 15 more

eligibility_changeGeographic AtrophyMay 8thAnnexon, Inc.NCT06510816
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGeographic AtrophyMay 8thi-Lumen Scientific AUS PTY LTDNCT06662162
Added 4 new sites: Manchester, Connecticut, Fort Worth, Texas, Tyler, Texas and 1 more

Added 4 new sites: Manchester, Connecticut, Fort Worth, Texas, Tyler, Texas and 1 more

sites_addedGastrointestinal Stromal TumorMay 8thIDRX, Inc., a wholly owned subsidiary of GSK, LLCNCT05489237
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

eligibility_changeGastrointestinal Stromal TumorMay 8thIDRX, Inc., a wholly owned subsidiary of GSK, LLCNCT05489237
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedGastrointestinal Stromal TumorMay 8thUniversity of NebraskaNCT02012699
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedFacioscapulohumeral Muscular DystrophyMay 8thUniversity of Kansas Medical CenterNCT04635891
Added 1 new site: Durham, North Carolina

Added 1 new site: Durham, North Carolina

sites_addedEssential ThrombocythemiaMay 8thMerck Sharp & Dohme LLCNCT06079879
Added 3 new sites: Edmonton, Canada, Santiago de Compostela, Spain, Taoyuan, Taiwan

Added 3 new sites: Edmonton, Canada, Santiago de Compostela, Spain, Taoyuan, Taiwan

sites_addedEssential ThrombocythemiaMay 8thDisc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

Enrollment UpdateEssential ThrombocythemiaMay 8thIcahn School of Medicine at Mount SinaiNCT05835466
Target enrollment decreased from 26 to 10

Target enrollment decreased from 26 to 10

sites_addedEssential ThrombocythemiaMay 8thMerck Sharp & Dohme LLCNCT06456346
Added 3 new sites: Eugene, Oregon, L'Hospitalet de Llobregat, Spain, Taoyuan, Taiwan

Added 3 new sites: Eugene, Oregon, L'Hospitalet de Llobregat, Spain, Taoyuan, Taiwan

sites_addedEssential ThrombocythemiaMay 8thNovartis PharmaceuticalsNCT07357727
Expanded from 3 to 4 sites

Expanded from 3 to 4 sites

eligibility_changeDuchenne Muscular DystrophyMay 7thSarepta Therapeutics, Inc.NCT06270719
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDravet SyndromeMay 7thIonis Pharmaceuticals, Inc.NCT07531745
Added 1 new site: Memphis, Tennessee

Added 1 new site: Memphis, Tennessee

Enrollment UpdateCystic FibrosisMay 7thCentre Hospitalier Universitaire de la RéunionNCT06371261
Target enrollment decreased from 35 to 17

Target enrollment decreased from 35 to 17

eligibility_changeCushing DiseaseMay 7thNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NCT06635629
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 7thSanofiNCT06290128
Added 1 new site: Las Vegas, Nevada

Added 1 new site: Las Vegas, Nevada

sites_addedChronic Inflammatory Demyelinating PolyneuropathyMay 7thSanofiNCT06290141
Added 3 new sites: Bradenton, Florida, Las Vegas, Nevada, Milan, Italy

Added 3 new sites: Bradenton, Florida, Las Vegas, Nevada, Milan, Italy

sites_addedCholangiocarcinomaMay 7thBold Therapeutics, Inc.NCT04421820
Added 2 new sites: Bonn, Germany, Ulm, Germany

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

sites_removedCholangiocarcinomaMay 7thUniversity of NebraskaNCT02012699
Reduced from 45 to 42 sites

Reduced from 45 to 42 sites

sites_addedCentral Diabetes InsipidusMay 7thUniversity Hospital, Basel, SwitzerlandNCT06036004
Added 1 new site: Berlin, Germany

Added 1 new site: Berlin, Germany

sites_addedCentral Diabetes InsipidusMay 7thChildren's Oncology GroupNCT06368817
Added 2 new sites: Indianapolis, Indiana, Winnipeg, Canada

Added 2 new sites: Indianapolis, Indiana, Winnipeg, Canada

Enrollment UpdateAlpha-1 Antitrypsin DeficiencyMay 6thBioMarin PharmaceuticalNCT06738017
Target enrollment decreased from 12 to 6

Target enrollment decreased from 12 to 6

eligibility_changeBecker Muscular DystrophyMay 6thSarepta Therapeutics, Inc.NCT06270719
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)May 6thAstraZenecaNCT06465810
Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

eligibility_changeAmyotrophic Lateral SclerosisMay 6thNational Institute of Neurological Disorders and Stroke (NINDS)NCT07204977
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAmyotrophic Lateral SclerosisMay 6thMerit E. Cudkowicz, MDNCT04297683
Added 1 new site: Rochester, New York

Added 1 new site: Rochester, New York

eligibility_changeAmyotrophic Lateral SclerosisMay 6thCentre hospitalier de l'Université de Montréal (CHUM)NCT06051123
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAlagille SyndromeMay 6thAlbireo, an Ipsen CompanyNCT05035030
Added 4 new sites: New York, New York, South Brisbane, Australia, Petah Tikva, Israel and 1 more

Added 4 new sites: New York, New York, South Brisbane, Australia, Petah Tikva, Israel and 1 more

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 5thAstraZenecaNCT06465810
Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

Added 5 new sites: Shanghai, China, Hebei Sheng, China, Shaanxi, China and 2 more

sites_addedWaldenström MacroglobulinemiaMay 5thChristian BuskeNCT05099471
Added 3 new sites: Mutlangen, Germany, Paderborn, Germany, Rostock, Germany

Added 3 new sites: Mutlangen, Germany, Paderborn, Germany, Rostock, Germany

sites_addedStargardt DiseaseMay 5thAlkeus Pharmaceuticals, Inc.NCT07419334
Added 2 new sites: Phoenix, Arizona, Gainesville, Florida

Added 2 new sites: Phoenix, Arizona, Gainesville, Florida

Enrollment UpdateHereditary Spastic Paraplegia Type 4May 5thNational Institute of Neurological Disorders and Stroke (NINDS)NCT01568658
Target enrollment decreased from 9300 to 2323

Target enrollment decreased from 9300 to 2323

sites_addedWild-Type ATTR Cardiac AmyloidosisMay 5thBayerNCT07298044
Added 1 new site: Braunau am Inn, Austria

Added 1 new site: Braunau am Inn, Austria

sites_addedWaldenström MacroglobulinemiaMay 5thModeX Therapeutics, An OPKO Health CompanyNCT07249905
Added 1 new site: Richmond, Australia

Added 1 new site: Richmond, Australia

sites_addedSpinal Muscular AtrophyMay 5thScholar Rock, Inc.NCT07047144
Added 2 new sites: Atlanta, Georgia, Leeds, United Kingdom

Added 2 new sites: Atlanta, Georgia, Leeds, United Kingdom

sites_addedPulmonary Arterial HypertensionMay 4thMerck Sharp & Dohme LLCNCT07218029
Added 8 new sites: Pittsburgh, Pennsylvania, Providence, Rhode Island, Charleston, South Carolina and 5 more

Added 8 new sites: Pittsburgh, Pennsylvania, Providence, Rhode Island, Charleston, South Carolina and 5 more

sites_addedPrimary Biliary CholangitisMay 4thGilead SciencesNCT03301506
Added 1 new site: Saint Petersburg, Russia

Added 1 new site: Saint Petersburg, Russia

Enrollment UpdatePrimary Biliary CholangitisMay 4thGilead SciencesNCT03301506
Target enrollment decreased from 500 to 340

Target enrollment decreased from 500 to 340

sites_addedPhenylketonuriaMay 4thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

eligibility_changePhenylketonuriaMay 4thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseMay 4thNovartis PharmaceuticalsNCT06439082
Added 1 new site: Masaka, Uganda

Added 1 new site: Masaka, Uganda

sites_addedShort Bowel SyndromeMay 4thHanmi Pharmaceutical Company LimitedNCT04775706
Added 1 new site: Salford, United Kingdom

Added 1 new site: Salford, United Kingdom

sites_addedPulmonary Arterial HypertensionMay 4thRegeneron PharmaceuticalsNCT07318597
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedPulmonary Arterial HypertensionMay 4thNovartis PharmaceuticalsNCT06649110
Added 54 new sites: Scottsdale, Arizona, Orange, California, Orlando, Florida and 51 more

Added 54 new sites: Scottsdale, Arizona, Orange, California, Orlando, Florida and 51 more

sites_addedProgressive Familial Intrahepatic CholestasisMay 4thIpsenNCT07185919
Added 1 new site: Gyeongsang, South Korea

Added 1 new site: Gyeongsang, South Korea

sites_addedPrimary Biliary CholangitisMay 4thIpsenNCT06016842
Added 3 new sites: Guadalajara, Mexico, Monterrey, Mexico, Chiang Mai, Thailand

Added 3 new sites: Guadalajara, Mexico, Monterrey, Mexico, Chiang Mai, Thailand

sites_addedPrimary Biliary CholangitisMay 4thGilead SciencesNCT06051617
Added 6 new sites: Bobigny, France, Beersheba, Israel, Tel Aviv, Israel and 3 more

Added 6 new sites: Bobigny, France, Beersheba, Israel, Tel Aviv, Israel and 3 more

sites_addedPompe DiseaseMay 4thGenzyme, a Sanofi CompanyNCT00231400
Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

sites_addedPolycythemia VeraMay 4thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedPolycythemia VeraMay 4thDisc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

sites_addedPolycythemia VeraMay 4thNational Cancer Institute (NCI)NCT06661915
Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

sites_addedPheochromocytomaMay 4thPerspective TherapeuticsNCT05636618
Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

eligibility_changePheochromocytomaMay 4thPerspective TherapeuticsNCT05636618
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdatePhenylketonuriaMay 4thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06628128
Target enrollment increased from 45 to 240

Target enrollment increased from 45 to 240

eligibility_changePhenylketonuriaMay 4thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06628128
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedParoxysmal Nocturnal HemoglobinuriaMay 4thHoffmann-La RocheNCT04434092
Reduced from 63 to 61 sites

Reduced from 63 to 61 sites

sites_addedParagangliomaMay 4thPerspective TherapeuticsNCT05636618
Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

Added 2 new sites: Cleveland, Ohio, Milwaukee, Wisconsin

eligibility_changeParagangliomaMay 4thPerspective TherapeuticsNCT05636618
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1May 3rdCentessa Pharmaceuticals (UK) LimitedNCT06752668
Added 8 new sites: El Paso, Texas, Léon, France, Bologna, Italy and 5 more

Added 8 new sites: El Paso, Texas, Léon, France, Bologna, Italy and 5 more

Enrollment UpdateNarcolepsy Type 1May 3rdCentessa Pharmaceuticals (UK) LimitedNCT06752668
Target enrollment increased from 96 to 248

Target enrollment increased from 96 to 248

sites_addedMyotonic DystrophyMay 3rdSanofiNCT06844214
Added 1 new site: Richmond, Virginia

Added 1 new site: Richmond, Virginia

eligibility_changeMyotonic DystrophyMay 3rdSanofiNCT06844214
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyotonic DystrophyMay 3rdPepGen IncNCT06667453
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisMay 3rdNovartis PharmaceuticalsNCT07357727
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedMyelofibrosisMay 3rdDisc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

sites_addedMyelofibrosisMay 3rdNational Cancer Institute (NCI)NCT06661915
Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

sites_addedMyelofibrosisMay 3rdiOncturaNCT06887803
Added 4 new sites: Madrid, Spain, Boston, United Kingdom, Belfast, United Kingdom and 1 more

Added 4 new sites: Madrid, Spain, Boston, United Kingdom, Belfast, United Kingdom and 1 more

sites_addedMyelofibrosisMay 3rdSumitomo Pharma America, Inc.NCT04176198
Added 1 new site: Aomori, Japan

Added 1 new site: Aomori, Japan

sites_addedMyelodysplastic SyndromesMay 3rdDisc Medicine, IncNCT05320198
Added 1 new site: West Perth, Australia

Added 1 new site: West Perth, Australia

sites_addedMultiple System AtrophyMay 3rdIonis Pharmaceuticals, Inc.NCT04165486
Added 1 new site: Göttingen, Germany

Added 1 new site: Göttingen, Germany

sites_addedMultiple SclerosisMay 3rdHoffmann-La RocheNCT05269004
Added 3 new sites: Innsbruck, Austria, Calgary, Canada, Stockholm, Sweden

Added 3 new sites: Innsbruck, Austria, Calgary, Canada, Stockholm, Sweden

sites_addedMultiple SclerosisMay 3rdHoffmann-La RocheNCT06780150
Added 6 new sites: Amberg, Germany, Celle, Germany, Frankfurt, Germany and 3 more

Added 6 new sites: Amberg, Germany, Celle, Germany, Frankfurt, Germany and 3 more

sites_removedMultiple SclerosisMay 3rdHoffmann-La RocheNCT04544436
Reduced from 122 to 120 sites

Reduced from 122 to 120 sites

sites_addedMultiple SclerosisMay 3rdNovartis PharmaceuticalsNCT06846281
Added 4 new sites: Orlando, Florida, Las Vegas, Nevada, Overpelt, Belgium and 1 more

Added 4 new sites: Orlando, Florida, Las Vegas, Nevada, Overpelt, Belgium and 1 more

Enrollment UpdateMucopolysaccharidosis Type IMay 3rdREGENXBIO Inc.NCT06103487
Target enrollment increased from 7 to 21

Target enrollment increased from 7 to 21

Enrollment UpdateMesotheliomaMay 3rdNational Cancer Institute (NCI)NCT00242723
Target enrollment increased from 1310 to 1559

Target enrollment increased from 1310 to 1559

eligibility_changeMesotheliomaMay 3rdNational Cancer Institute (NCI)NCT00242723
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaMay 3rdPerspective TherapeuticsNCT06710756
Added 4 new sites: Miami, Florida, Lexington, Kentucky, Columbus, Ohio and 1 more

Added 4 new sites: Miami, Florida, Lexington, Kentucky, Columbus, Ohio and 1 more

sites_addedMerkel Cell CarcinomaMay 3rdUniversity of ArizonaNCT06814496
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

eligibility_changeMerkel Cell CarcinomaMay 3rdMemorial Sloan Kettering Cancer CenterNCT06021626
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMerkel Cell CarcinomaMay 3rdMerck Sharp & Dohme LLCNCT07302347
Added 1 new site: Sapporo, Japan

Added 1 new site: Sapporo, Japan

sites_addedMembranous NephropathyMay 2ndClimb Bio, Inc.NCT07096843
Added 12 new sites: Temuco, Chile, Valdivia, Chile, Santiago, Chile and 9 more

Added 12 new sites: Temuco, Chile, Valdivia, Chile, Santiago, Chile and 9 more

sites_addedMedullary Thyroid CancerMay 2ndUniversity of ArizonaNCT06814496
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedLight Chain AmyloidosisMay 2ndRegeneron PharmaceuticalsNCT06292780
Added 1 new site: Athens, Greece

Added 1 new site: Athens, Greece

sites_addedIgA NephropathyMay 2ndBiogenNCT06935357
Added 2 new sites: Pátrai, Greece, Taoyuan, Taiwan

Added 2 new sites: Pátrai, Greece, Taoyuan, Taiwan

eligibility_changeIgA NephropathyMay 2ndBiogenNCT06935357
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyMay 2ndNovartis PharmaceuticalsNCT06797518
Added 2 new sites: Córdoba, Argentina, Riyadh, Saudi Arabia

Added 2 new sites: Córdoba, Argentina, Riyadh, Saudi Arabia

sites_addedIdiopathic Pulmonary FibrosisMay 2ndAvalyn Pharma Inc.NCT06951217
Added 11 new sites: Birmingham, Alabama, Los Angeles, California, Baltimore, Maryland and 8 more

Added 11 new sites: Birmingham, Alabama, Los Angeles, California, Baltimore, Maryland and 8 more

sites_addedHypoparathyroidismMay 2ndAscendis Pharma Bone Diseases A/SNCT07081997
Added 1 new site: Spokane Valley, Washington

Added 1 new site: Spokane Valley, Washington

sites_addedHuntington DiseaseMay 2ndUniversity College, LondonNCT02855476
Added 1 new site: Lisbon, Portugal

Added 1 new site: Lisbon, Portugal

eligibility_changeHuntington DiseaseMay 2ndUniversity of IowaNCT05707663
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseMay 2ndNovartis PharmaceuticalsNCT07326709
Added 6 new sites: Albany, New York, Houston, Texas, Seattle, Washington and 3 more

Added 6 new sites: Albany, New York, Houston, Texas, Seattle, Washington and 3 more

Enrollment UpdateHomozygous Familial HypercholesterolemiaMay 2ndNovartis PharmaceuticalsNCT05682378
Target enrollment increased from 131 to 195

Target enrollment increased from 131 to 195

eligibility_changeHomozygous Familial HypercholesterolemiaMay 2ndNovartis PharmaceuticalsNCT05682378
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHereditary Hemorrhagic TelangiectasiaMay 2ndAlnylam PharmaceuticalsNCT06659640
Added 8 new sites: Birmingham, Alabama, Gainesville, Florida, Indianapolis, Indiana and 5 more

Added 8 new sites: Birmingham, Alabama, Gainesville, Florida, Indianapolis, Indiana and 5 more

sites_addedGastrointestinal Stromal TumorMay 1stAstraZenecaNCT07124000
Added 7 new sites: Bullhead City, Arizona, Boulder, Colorado, St. Petersburg, Florida and 4 more

Added 7 new sites: Bullhead City, Arizona, Boulder, Colorado, St. Petersburg, Florida and 4 more

sites_addedGastrointestinal Stromal TumorMay 1stDeciphera Pharmaceuticals, LLCNCT05957367
Added 1 new site: Frankfurt, Germany

Added 1 new site: Frankfurt, Germany

sites_addedHereditary AngioedemaMay 1stIonis Pharmaceuticals, Inc.NCT07298447
Added 1 new site: Warsaw, Poland

Added 1 new site: Warsaw, Poland

Enrollment UpdateGranulomatosis with PolyangiitisMay 1stImperial College LondonNCT06230354
Target enrollment decreased from 66 to 42

Target enrollment decreased from 66 to 42

eligibility_changeGranulomatosis with PolyangiitisMay 1stImperial College LondonNCT06230354
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMay 1stGenzyme, a Sanofi CompanyNCT00231400
Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

Added 7 new sites: Grand Rapids, Michigan, Minneapolis, Minnesota, Minneapolis, Minnesota and 4 more

sites_addedGeographic AtrophyMay 1stKriya Therapeutics, Inc.NCT06765980
Added 1 new site: Tel Aviv, Israel

Added 1 new site: Tel Aviv, Israel

sites_addedFocal Segmental GlomerulosclerosisMay 1stBoehringer IngelheimNCT07220083
Added 7 new sites: Denver, Colorado, Washington D.C., District of Columbia, Kalamazoo, Michigan and 4 more

Added 7 new sites: Denver, Colorado, Washington D.C., District of Columbia, Kalamazoo, Michigan and 4 more

sites_addedFabry DiseaseMay 1stGenzyme, a Sanofi CompanyNCT00196742
Added 9 new sites: Kansas City, Kansas, Grand Rapids, Michigan, Minneapolis, Minnesota and 6 more

Added 9 new sites: Kansas City, Kansas, Grand Rapids, Michigan, Minneapolis, Minnesota and 6 more

sites_addedFabry DiseaseMay 1stAmicus TherapeuticsNCT06906367
Added 1 new site: New York, New York

Added 1 new site: New York, New York