Zero ad trackers.We never sell your data.

Rare Disease Trial Updates: October 2026

In October 2026, Trial Friend tracked 654 rare disease clinical trial updates across 130 conditions, including 1 FDA-related actions and 88 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 ChangeDuchenne Muscular DystrophyOct 31stMassachusetts General HospitalNCT05016908
No longer enrolling new participants

No longer enrolling new participants

Status ChangeInherited ErythromelalgiaOct 31stBiohaven Therapeutics Ltd.NCT07262268
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaOct 31stMedicenna Therapeutics, Inc.NCT05086692
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMerkel Cell CarcinomaOct 31stMedicenna Therapeutics, Inc.NCT05086692
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTuberous SclerosisOct 31stDarcy KruegerNCT05104983
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThalassemiaOct 31stChildren's Hospital of PhiladelphiaNCT06364774
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinocerebellar AtaxiaOct 31stFirst Affiliated Hospital of Wannan Medical CollegeNCT07838883
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

FDA ApprovalRecurrent Respiratory PapillomatosisOct 30th
FDA Approves First Plasmid DNA-based Immunotherapy for Adults with Recurrent Respiratory Papillomatosis

The U.S. Food and Drug Administration today granted accelerated approval for Voceryq (doruxapapogene ralaplasmid and lalikinogene sifuplasmid-gxfd) for treatment of adults with recurrent respiratory papillomatosis (RRP).

Status ChangePulmonary Arterial HypertensionOct 30thNational Heart, Lung, and Blood Institute (NHLBI)NCT05967299
No longer enrolling new participants

No longer enrolling new participants

Status ChangeRett SyndromeOct 30thGeorgetown UniversityNCT05012475
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeuroendocrine TumorsOct 29thUniversity of California, DavisNCT05160480
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeMyotonic DystrophyOct 29thMassachusetts General HospitalNCT05020002
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisOct 29thVertex Pharmaceuticals IncorporatedNCT07501702
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisOct 29thargenxNCT04833894
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisOct 29thUCB Biopharma SRLNCT07246564
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNTRK Fusion CancerOct 29thBayerNCT04142437
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNeuroendocrine TumorsOct 29thChimeric TherapeuticsNCT06055439
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesOct 29thMemorial Sloan Kettering Cancer CenterNCT05884333
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisOct 29thAston UniversityNCT07689682
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeMultiple SclerosisOct 29thCentre Hospitalier Universitaire de NīmesNCT03981003
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple MyelomaOct 29thSanofiNCT06356571
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyOct 28thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06740526
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisOct 28thHospital de GranollersNCT07486206
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHereditary Spastic ParaplegiaOct 27thFirst Affiliated Hospital of Wannan Medical CollegeNCT07838883
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeHemophilia AOct 27thSwedish Orphan BiovitrumNCT06716814
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGraves’ DiseaseOct 27thNorthwell HealthNCT07369063
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGeneralized Pustular PsoriasisOct 27thCorEvitasNCT06100991
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGastrointestinal Stromal TumorOct 27thDeciphera Pharmaceuticals, LLCNCT05957367
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGastrointestinal Stromal TumorOct 27thCentre Leon BerardNCT05152472
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyOct 26thWave Life Sciences USA, Inc.NCT04906460
No longer enrolling new participants

No longer enrolling new participants

Status ChangeDravet SyndromeOct 26thStoke Therapeutics, IncNCT06872125
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAlpha-1 Antitrypsin DeficiencyOct 24thWave Life Sciences USA, Inc.NCT06405633
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeAdrenocortical CarcinomaOct 24thMaria Sklodowska-Curie National Research Institute of OncologyNCT05563467
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSpinal Muscular AtrophyOct 22ndHoffmann-La RocheNCT05861999
No longer enrolling new participants

No longer enrolling new participants

Status ChangeShort Bowel SyndromeOct 22ndChongqing Peg-Bio Biopharm Co., Ltd.NCT06512584
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSystemic SclerosisOct 22ndargenxNCT06655155
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSystemic SclerosisOct 22ndUniversity Hospital, MontpellierNCT07493395
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeSystemic SclerosisOct 22ndHospital for Special Surgery, New YorkNCT03844061
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSjögren's SyndromeOct 22ndResolve TherapeuticsNCT06440525
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSickle Cell DiseaseOct 22ndVanderbilt University Medical CenterNCT05153967
No longer enrolling new participants

No longer enrolling new participants

Status ChangePulmonary Arterial HypertensionOct 21stMerck Sharp & Dohme LLCNCT07218029
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrader-Willi SyndromeOct 21stMaimonides Medical CenterNCT05939453
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrader-Willi SyndromeOct 21stRhythm Pharmaceuticals, Inc.NCT06239116
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisOct 20thShirley Ryan AbilityLabNCT07132775
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple MyelomaOct 20thCentre Hospitalier Universitaire de Saint EtienneNCT05259553
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick Disease Type COct 20thAzafaros B.V.NCT07399704
No longer enrolling new participants

No longer enrolling new participants

Status ChangeNiemann-Pick DiseaseOct 20thAzafaros B.V.NCT07399704
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelodysplastic SyndromesOct 20thEric Stephen Winer, MDNCT05703542
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisOct 20thUCB Biopharma SRLNCT06540144
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisOct 20thVor BiopharmaNCT06456580
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaOct 19thInstituto do Cancer do Estado de São PauloNCT06638931
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyOct 18thRigerna Therapeutics Co., Ltd.; Rigerna Therapeutics (Beijing) Co., Ltd.NCT07305974
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisOct 18thMediar TherapeuticsNCT06967805
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisOct 17thUniversity of MichiganNCT06817213
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCushing DiseaseOct 16thH. Lundbeck A/SNCT06471829
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaOct 16thInstitut de Recherches Internationales ServierNCT06501625
No longer enrolling new participants

No longer enrolling new participants

Status ChangeCholangiocarcinomaOct 16thInstituto do Cancer do Estado de São PauloNCT06638931
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisOct 15thUniversity of UlmNCT06280079
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisOct 15thCoya TherapeuticsNCT07161999
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAchondroplasiaOct 15thAscendis Pharma A/SNCT06732895
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAchondroplasiaOct 15thKyowa Kirin Co., Ltd.NCT06926491
No longer enrolling new participants

No longer enrolling new participants

Status ChangeTay-Sachs DiseaseOct 13thAzafaros B.V.NCT07399704
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSystemic SclerosisOct 13thNovartis PharmaceuticalsNCT06470048
No longer enrolling new participants

No longer enrolling new participants

Status ChangeThyroid Eye DiseaseOct 13thMinghui Pharmaceutical (Hangzhou) LtdNCT06989918
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyotonic DystrophyOct 12thSarepta Therapeutics, Inc.NCT06138743
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeMultiple System AtrophyOct 12thTeva Branded Pharmaceutical Products R&D LLCNCT06568237
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMultiple SclerosisOct 12thUniversity of Kansas Medical CenterNCT06428006
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIgA NephropathyOct 10thNovartis PharmaceuticalsNCT04557462
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeFamilial Hypertrophic CardiomyopathyOct 9thMontreal Heart InstituteNCT05100420
Now recruiting (was Enrolling By Invitation)

Now recruiting (was Enrolling By Invitation)

Status ChangeFacioscapulohumeral Muscular DystrophyOct 9thSarepta Therapeutics, Inc.NCT06131983
Status changed from Recruiting to Enrolling By Invitation

Status changed from Recruiting to Enrolling By Invitation

Status ChangeFriedreich AtaxiaOct 9thBiogenNCT06953583
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFanconi AnemiaOct 9thNational Cancer Institute (NCI)NCT06744283
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseOct 8thIncyte CorporationNCT06843408
No longer enrolling new participants

No longer enrolling new participants

Status ChangeChronic Graft-versus-Host DiseaseOct 8thJanssen Research & Development, LLCNCT01804686
No longer enrolling new participants

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisOct 7thNational Institute of Neurological Disorders and Stroke (NINDS)NCT07204977
Now recruiting (was Active Not Recruiting)

Now recruiting (was Active Not Recruiting)

Status ChangeUrea Cycle DisordersOct 6thiECURE, Inc.NCT06255782
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSarcoidosisOct 5thXentria, Inc.NCT05890729
No longer enrolling new participants

No longer enrolling new participants

Status ChangePrimary Biliary CholangitisOct 4thIpsenNCT06447168
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyelofibrosisOct 3rdMarina KremyanskayaNCT05025488
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMyasthenia GravisOct 3rdNovartis PharmaceuticalsNCT06744920
No longer enrolling new participants

No longer enrolling new participants

Status ChangeSanfilippo SyndromeOct 3rdJCR Pharmaceuticals Co., Ltd.NCT06488924
No longer enrolling new participants

No longer enrolling new participants

Status ChangeMesotheliomaOct 3rdNuvectis Pharma, Inc.NCT05873686
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHodgkin LymphomaOct 2ndECOG-ACRIN Cancer Research GroupNCT06002828
No longer enrolling new participants

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisOct 2ndBoehringer IngelheimNCT07036523
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia AOct 2ndSwedish Orphan BiovitrumNCT06940830
No longer enrolling new participants

No longer enrolling new participants

Status ChangeHemophilia AOct 2ndSanofiNCT06684314
No longer enrolling new participants

No longer enrolling new participants

Status ChangeGeographic AtrophyOct 1stVisionCare, Inc.NCT04796545
No longer enrolling new participants

No longer enrolling new participants

Status ChangeFocal EpilepsyOct 1stBiohaven Therapeutics Ltd.NCT06132893
No longer enrolling new participants

No longer enrolling new participants

Status ChangeEssential ThrombocythemiaOct 1stMarina KremyanskayaNCT05025488
No longer enrolling new participants

No longer enrolling new participants

Trial Site & Enrollment Activity

sites_addedHereditary Hemorrhagic TelangiectasiaOct 31stAtavistik Bio, IncNCT07601425
Added 1 new site: Chapel Hill, North Carolina

Added 1 new site: Chapel Hill, North Carolina

sites_addedAcute Myeloid LeukemiaOct 31stNational Cancer Institute (NCI)NCT07437950
Added 9 new sites: O'Fallon, Illinois, O'Fallon, Illinois, City of Saint Peters, Missouri and 6 more

Added 9 new sites: O'Fallon, Illinois, O'Fallon, Illinois, City of Saint Peters, Missouri and 6 more

sites_addedLupus NephritisOct 31stAutolus LimitedNCT07053800
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedCholangiocarcinomaOct 31stFull-Life Technologies UK LimitedNCT07873450
Added 1 new site: Minneapolis, Minnesota

Added 1 new site: Minneapolis, Minnesota

sites_addedMultiple SclerosisOct 31stSundance Biosciences, Inc.NCT07840326
Added 4 new sites: Tampa, Florida, Stony Brook, New York, Oklahoma City, Oklahoma and 1 more

Added 4 new sites: Tampa, Florida, Stony Brook, New York, Oklahoma City, Oklahoma and 1 more

sites_addedHypoparathyroidismOct 31stMBX BiosciencesNCT07699471
Added 1 new site: Madison, Wisconsin

Added 1 new site: Madison, Wisconsin

sites_addedGraves’ DiseaseOct 31stYarrow Bioscience, Inc.NCT07682896
Added 3 new sites: Ocoee, Florida, Durham, North Carolina, St Leonards, Australia

Added 3 new sites: Ocoee, Florida, Durham, North Carolina, St Leonards, Australia

sites_addedMultiple SclerosisOct 31stGenentech, Inc.NCT07609719
Added 3 new sites: Birmingham, Alabama, Fort Collins, Colorado, Savannah, Georgia

Added 3 new sites: Birmingham, Alabama, Fort Collins, Colorado, Savannah, Georgia

sites_addedCystic FibrosisOct 31stAnagram Therapeutics, Inc.NCT07450547
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

Enrollment UpdateCushing DiseaseOct 31stEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT03831958
Target enrollment decreased from 600 to 240

Target enrollment decreased from 600 to 240

eligibility_changeCushing DiseaseOct 31stEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT03831958
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseOct 31stMerida BiosciencesNCT07305818
Added 3 new sites: Barcelona, Spain, Santander, Spain, Seville, Spain

Added 3 new sites: Barcelona, Spain, Santander, Spain, Seville, Spain

sites_removedAlport SyndromeOct 31stBayerNCT07211685
Reduced from 58 to 51 sites

Reduced from 58 to 51 sites

eligibility_changeCholangiocarcinomaOct 31stNational Cancer Institute (NCI)NCT05849480
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeDermatomyositisOct 31stNational Institute of Environmental Health Sciences (NIEHS)NCT07111065
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyOct 31stargenxNCT06920004
Added 2 new sites: Knoxville, Tennessee, Tel HaShomer, Israel

Added 2 new sites: Knoxville, Tennessee, Tel HaShomer, Israel

eligibility_changeAcute Myeloid LeukemiaOct 31stRemix TherapeuticsNCT06297941
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyOct 31stNeurona TherapeuticsNCT05135091
Added 1 new site: Buffalo, New York

Added 1 new site: Buffalo, New York

sites_addedDravet SyndromeOct 31stEpygenixNCT04462770
Added 18 new sites: Beijing, China, Beijing, China, Chongqing, China and 15 more

Added 18 new sites: Beijing, China, Beijing, China, Chongqing, China and 15 more

eligibility_changeCystic FibrosisOct 31stNational Heart, Lung, and Blood Institute (NHLBI)NCT00943514
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeOct 31stNational Heart, Lung, and Blood Institute (NHLBI)NCT00943514
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCommon Variable ImmunodeficiencyOct 31stNational Heart, Lung, and Blood Institute (NHLBI)NCT00943514
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisOct 31stContineum TherapeuticsNCT07284459
Added 1 new site: Marseille, France

Added 1 new site: Marseille, France

sites_addedNeuroendocrine TumorsOct 31stExelixisNCT06943755
Added 2 new sites: São Paulo, Brazil, Mexico City, Mexico

Added 2 new sites: São Paulo, Brazil, Mexico City, Mexico

sites_addedIdiopathic Pulmonary FibrosisOct 31stAstraZenecaNCT07652658
Added 2 new sites: Fountain Hill, Pennsylvania, Coswig, Germany

Added 2 new sites: Fountain Hill, Pennsylvania, Coswig, Germany

sites_addedEssential ThrombocythemiaOct 31stPrelude Therapeutics IncorporatedNCT07469891
Added 4 new sites: Sacramento, California, Miami, Florida, Nice, France and 1 more

Added 4 new sites: Sacramento, California, Miami, Florida, Nice, France and 1 more

sites_removedGeographic AtrophyOct 31stAstellas Pharma IncNCT06970665
Reduced from 14 to 11 sites

Reduced from 14 to 11 sites

sites_addedHodgkin LymphomaOct 31stNational Cancer Institute (NCI)NCT05675410
Added 2 new sites: Edina, Minnesota, Seneca, South Carolina

Added 2 new sites: Edina, Minnesota, Seneca, South Carolina

sites_addedAcute Myeloid LeukemiaOct 31stNational Cancer Institute (NCI)NCT05554406
Added 1 new site: Edina, Minnesota

Added 1 new site: Edina, Minnesota

sites_addedWaldenström MacroglobulinemiaOct 31stNational Cancer Institute (NCI)NCT04840602
Added 7 new sites: Brownstown, Michigan, Dearborn, Michigan, Jackson, Michigan and 4 more

Added 7 new sites: Brownstown, Michigan, Dearborn, Michigan, Jackson, Michigan and 4 more

sites_addedMultiple SclerosisOct 31stHoffmann-La RocheNCT07827586
Added 1 new site: Pardubice, Czechia

Added 1 new site: Pardubice, Czechia

sites_addedEssential ThrombocythemiaOct 31stIncyte CorporationNCT07623200
Added 20 new sites: La Jolla, California, Atlanta, Georgia, Kurralta Park, Australia and 17 more

Added 20 new sites: La Jolla, California, Atlanta, Georgia, Kurralta Park, Australia and 17 more

sites_addedIgA NephropathyOct 31stJade Biosciences, Inc.NCT07541287
Added 2 new sites: Kaohsiung City, Taiwan, Taiyuan, Taiwan

Added 2 new sites: Kaohsiung City, Taiwan, Taiyuan, Taiwan

sites_addedMultiple MyelomaOct 31stAstraZenecaNCT06106945
Added 3 new sites: Leeds, United Kingdom, Manchester, United Kingdom, Plymouth, United Kingdom

Added 3 new sites: Leeds, United Kingdom, Manchester, United Kingdom, Plymouth, United Kingdom

eligibility_changeMultiple MyelomaOct 31stAstraZenecaNCT06106945
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple MyelomaOct 31stC4 Therapeutics, Inc.NCT07284758
Added 39 new sites: Duarte, California, Boston, Massachusetts, Boston, Massachusetts and 36 more

Added 39 new sites: Duarte, California, Boston, Massachusetts, Boston, Massachusetts and 36 more

sites_addedMultiple SclerosisOct 31stTG Therapeutics, Inc.NCT07220252
Added 3 new sites: Salt Lake City, Utah, Rzeszów, Poland, Bratislava, Slovakia

Added 3 new sites: Salt Lake City, Utah, Rzeszów, Poland, Bratislava, Slovakia

sites_addedMembranous NephropathyOct 31stVertex Pharmaceuticals IncorporatedNCT07204275
Added 2 new sites: Gastonia, North Carolina, Goiânia, Brazil

Added 2 new sites: Gastonia, North Carolina, Goiânia, Brazil

sites_addedMultiple SclerosisOct 31stTG Therapeutics, Inc.NCT06433752
Added 3 new sites: Paramus, New Jersey, Lake Success, New York, The Bronx, New York

Added 3 new sites: Paramus, New Jersey, Lake Success, New York, The Bronx, New York

sites_removedMesotheliomaOct 31stMedicenna Therapeutics, Inc.NCT05086692
Reduced from 28 to 25 sites

Reduced from 28 to 25 sites

sites_removedMerkel Cell CarcinomaOct 31stMedicenna Therapeutics, Inc.NCT05086692
Reduced from 28 to 25 sites

Reduced from 28 to 25 sites

sites_removedTuberous SclerosisOct 31stDarcy KruegerNCT05104983
Reduced from 11 to 8 sites

Reduced from 11 to 8 sites

sites_addedThyroid Eye DiseaseOct 31stEthyreal Bio, Inc.NCT07885605
Added 2 new sites: Orland Park, Illinois, Wilmington, North Carolina

Added 2 new sites: Orland Park, Illinois, Wilmington, North Carolina

sites_addedSystemic SclerosisOct 31stAstraZenecaNCT07691970
Added 23 new sites: Indianapolis, Indiana, Columbus, Ohio, Dallas, Texas and 20 more

Added 23 new sites: Indianapolis, Indiana, Columbus, Ohio, Dallas, Texas and 20 more

sites_removedSystemic SclerosisOct 31stNkarta, Inc.NCT06733935
Reduced from 18 to 12 sites

Reduced from 18 to 12 sites

eligibility_changeSystemic SclerosisOct 31stNkarta, Inc.NCT06733935
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedStickler SyndromeOct 31stHelen Keller Eye Research FoundationNCT07146516
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedStargardt DiseaseOct 31stAstellas Institute for Regenerative MedicineNCT07734064
Added 1 new site: Southaven, Mississippi

Added 1 new site: Southaven, Mississippi

sites_addedSpinal Muscular AtrophyOct 31stBiogenNCT07221669
Added 1 new site: Lo Barnechea, Chile

Added 1 new site: Lo Barnechea, Chile

sites_addedSpinal Muscular AtrophyOct 31stNovartis PharmaceuticalsNCT07448610
Added 5 new sites: Little Rock, Arkansas, Stanford, California, Boston, Massachusetts and 2 more

Added 5 new sites: Little Rock, Arkansas, Stanford, California, Boston, Massachusetts and 2 more

sites_addedSjögren's SyndromeOct 31stNovartis PharmaceuticalsNCT07737743
Added 4 new sites: Memphis, Tennessee, Hefei, China, Bogota, Colombia and 1 more

Added 4 new sites: Memphis, Tennessee, Hefei, China, Bogota, Colombia and 1 more

sites_addedSjögren's SyndromeOct 31stLubris Bio Pty LtdNCT07118241
Added 1 new site: Melbourne, Australia

Added 1 new site: Melbourne, Australia

sites_addedSickle Cell DiseaseOct 31stAgios Pharmaceuticals, Inc.NCT07656415
Added 2 new sites: New York, New York, Houston, Texas

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

sites_addedSickle Cell DiseaseOct 31stNovartis PharmaceuticalsNCT06546670
Added 1 new site: São Paulo, Brazil

Added 1 new site: São Paulo, Brazil

sites_addedPulmonary Arterial HypertensionOct 30th35Pharma Inc., a GSK companyNCT07143448
Added 4 new sites: Boston, Massachusetts, Barcelona, Spain, Madrid, Spain and 1 more

Added 4 new sites: Boston, Massachusetts, Barcelona, Spain, Madrid, Spain and 1 more

Enrollment UpdatePulmonary Arterial HypertensionOct 30thNational Heart, Lung, and Blood Institute (NHLBI)NCT05967299
Target enrollment decreased from 96 to 40

Target enrollment decreased from 96 to 40

eligibility_changeRett SyndromeOct 30thChildren's Hospital of PhiladelphiaNCT05932589
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changePulmonary Langerhans Cell HistiocytosisOct 30thCarl AllenNCT04079179
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionOct 30thAOP Orphan Pharmaceuticals AGNCT06317805
Added 9 new sites: Bahía Blanca, Argentina, Buenos Aires, Argentina, Buenos Aires, Argentina and 6 more

Added 9 new sites: Bahía Blanca, Argentina, Buenos Aires, Argentina, Buenos Aires, Argentina and 6 more

eligibility_changePulmonary Arterial HypertensionOct 30thAOP Orphan Pharmaceuticals AGNCT06317805
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionOct 30thRegeneron PharmaceuticalsNCT07318597
Added 1 new site: Katowice, Poland

Added 1 new site: Katowice, Poland

sites_addedPrimary Central Nervous System LymphomaOct 30thOno Pharmaceutical Co., Ltd.NCT07104032
Added 4 new sites: Rochester, New York, Darlinghurst, Australia, Cuneo, Italy and 1 more

Added 4 new sites: Rochester, New York, Darlinghurst, Australia, Cuneo, Italy and 1 more

sites_addedPompe DiseaseOct 30thGenzyme, a Sanofi CompanyNCT00231400
Added 44 new sites: Los Angeles, California, Orange, California, Sacramento, California and 41 more

Added 44 new sites: Los Angeles, California, Orange, California, Sacramento, California and 41 more

sites_addedPolycythemia VeraOct 30thNovartis PharmaceuticalsNCT07357727
Added 8 new sites: Salzburg, Austria, Curitiba, Brazil, Palermo, Italy and 5 more

Added 8 new sites: Salzburg, Austria, Curitiba, Brazil, Palermo, Italy and 5 more

sites_addedPolycythemia VeraOct 30thOno Pharmaceutical Co., Ltd.NCT07429266
Added 6 new sites: Houston, Texas, Heidelberg, Australia, Graz, Austria and 3 more

Added 6 new sites: Houston, Texas, Heidelberg, Australia, Graz, Austria and 3 more

sites_addedPolycythemia VeraOct 30thNational Cancer Institute (NCI)NCT06661915
Added 1 new site: Winston-Salem, North Carolina

Added 1 new site: Winston-Salem, North Carolina

sites_addedPolycythemia VeraOct 30thUniversity of BirminghamNCT04116502
Added 8 new sites: Bodelwyddan, United Kingdom, Coventry, United Kingdom, Ipswich, United Kingdom and 5 more

Added 8 new sites: Bodelwyddan, United Kingdom, Coventry, United Kingdom, Ipswich, United Kingdom and 5 more

sites_addedPolycythemia VeraOct 30thDisc Medicine, IncNCT05320198
Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

sites_addedPolycythemia VeraOct 30thPrelude Therapeutics IncorporatedNCT07469891
Added 4 new sites: Sacramento, California, Miami, Florida, Nice, France and 1 more

Added 4 new sites: Sacramento, California, Miami, Florida, Nice, France and 1 more

sites_addedPhenylketonuriaOct 30thMaze TherapeuticsNCT07694440
Added 2 new sites: Tours, France, London, United Kingdom

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

sites_addedPhenylketonuriaOct 30thPTC TherapeuticsNCT06302348
Added 4 new sites: Nedlands, Australia, South Brisbane, Australia, Stockholm, Sweden and 1 more

Added 4 new sites: Nedlands, Australia, South Brisbane, Australia, Stockholm, Sweden and 1 more

sites_addedMyelofibrosisOct 29thPrelude Therapeutics IncorporatedNCT07469891
Added 4 new sites: Sacramento, California, Miami, Florida, Nice, France and 1 more

Added 4 new sites: Sacramento, California, Miami, Florida, Nice, France and 1 more

eligibility_changeMyelodysplastic SyndromesOct 29thRemix TherapeuticsNCT06297941
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisOct 29thVertex Pharmaceuticals IncorporatedNCT07501702
Added 3 new sites: Tampa, Florida, Snellville, Georgia, Chapel Hill, North Carolina

Added 3 new sites: Tampa, Florida, Snellville, Georgia, Chapel Hill, North Carolina

Enrollment UpdateMyasthenia GravisOct 29thargenxNCT04833894
Target enrollment increased from 12 to 15

Target enrollment increased from 12 to 15

sites_addedMyasthenia GravisOct 29thTG Therapeutics, Inc.NCT07673744
Added 1 new site: Maitland, Florida

Added 1 new site: Maitland, Florida

eligibility_changeMultiple SclerosisOct 29thUniversity of South FloridaNCT07615725
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisOct 29thNovartis PharmaceuticalsNCT06846281
Added 2 new sites: Chapel Hill, North Carolina, Marburg, Germany

Added 2 new sites: Chapel Hill, North Carolina, Marburg, Germany

sites_addedMultiple MyelomaOct 29thPfizerNCT05623020
Added 20 new sites: Daphne, Alabama, Daphne, Alabama, Fairhope, Alabama and 17 more

Added 20 new sites: Daphne, Alabama, Daphne, Alabama, Fairhope, Alabama and 17 more

sites_addedMultiple MyelomaOct 29thUniversity of UtahNCT06517017
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_removedMultiple MyelomaOct 29thBeOne MedicinesNCT04973605
Reduced from 77 to 74 sites

Reduced from 77 to 74 sites

sites_addedNTRK Fusion CancerOct 29thTeligene USNCT06010342
Added 2 new sites: Dallas, Texas, Lewisville, Texas

Added 2 new sites: Dallas, Texas, Lewisville, Texas

sites_removedNTRK Fusion CancerOct 29thBayerNCT04142437
Reduced from 76 to 47 sites

Reduced from 76 to 47 sites

Enrollment UpdateNTRK Fusion CancerOct 29thBayerNCT04142437
Target enrollment decreased from 150 to 54

Target enrollment decreased from 150 to 54

eligibility_changeNeurofibromatosis Type 1Oct 29thThe Children's Tumor FoundationNCT01885767
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNeuroendocrine TumorsOct 29thExelixisNCT06943755
Added 2 new sites: Bethesda, Maryland, Tainan, Taiwan

Added 2 new sites: Bethesda, Maryland, Tainan, Taiwan

sites_addedNeuroendocrine TumorsOct 29thExelixisNCT07620574
Added 2 new sites: Memphis, Tennessee, Houston, Texas

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

sites_addedNarcolepsy Type 1Oct 29thTakedaNCT07646678
Added 7 new sites: Santa Ana, California, Miami, Florida, Lansing, Michigan and 4 more

Added 7 new sites: Santa Ana, California, Miami, Florida, Lansing, Michigan and 4 more

sites_addedMyelofibrosisOct 29thDisc Medicine, IncNCT05320198
Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

eligibility_changeMyelofibrosisOct 29thGlaxoSmithKlineNCT03441113
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisOct 29thNovartis PharmaceuticalsNCT07357727
Added 8 new sites: Salzburg, Austria, Curitiba, Brazil, Palermo, Italy and 5 more

Added 8 new sites: Salzburg, Austria, Curitiba, Brazil, Palermo, Italy and 5 more

eligibility_changeMyelofibrosisOct 29thUniversity of UtahNCT05535764
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelofibrosisOct 29thNational Cancer Institute (NCI)NCT06661915
Added 1 new site: Winston-Salem, North Carolina

Added 1 new site: Winston-Salem, North Carolina

sites_addedMyelofibrosisOct 29thSwedish Orphan BiovitrumNCT07774455
Added 2 new sites: Tampa, Florida, Nashville, Tennessee

Added 2 new sites: Tampa, Florida, Nashville, Tennessee

sites_addedMyelodysplastic SyndromesOct 29thNational Cancer Institute (NCI)NCT05554406
Added 1 new site: Edina, Minnesota

Added 1 new site: Edina, Minnesota

sites_addedMyelodysplastic SyndromesOct 29thDisc Medicine, IncNCT05320198
Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

sites_addedMyelodysplastic SyndromesOct 29thGenmabNCT07384715
Added 3 new sites: Bergamo, Italy, Milan, Italy, Pisa, Italy

Added 3 new sites: Bergamo, Italy, Milan, Italy, Pisa, Italy

phase_changeMyelodysplastic SyndromesOct 29thCullinan Therapeutics Inc.NCT05143996
Moved from Phase 1 to Phase 1, Phase 2

Moved from Phase 1 to Phase 1, Phase 2

sites_addedMyelodysplastic SyndromesOct 29thCullinan Therapeutics Inc.NCT05143996
Added 6 new sites: Phoenix, Arizona, Duarte, California, Aurora, Colorado and 3 more

Added 6 new sites: Phoenix, Arizona, Duarte, California, Aurora, Colorado and 3 more

Enrollment UpdateMyelodysplastic SyndromesOct 29thCullinan Therapeutics Inc.NCT05143996
Target enrollment increased from 60 to 275

Target enrollment increased from 60 to 275

eligibility_changeMyelodysplastic SyndromesOct 29thCullinan Therapeutics Inc.NCT05143996
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesOct 29thNational Cancer Institute (NCI)NCT05564390
Added 2 new sites: Bemidji, Minnesota, Edina, Minnesota

Added 2 new sites: Bemidji, Minnesota, Edina, Minnesota

eligibility_changeMyelodysplastic SyndromesOct 29thFred Hutchinson Cancer CenterNCT06013423
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesOct 29thNational Cancer Institute (NCI)NCT06577441
Added 2 new sites: Edina, Minnesota, Saint Paul, Minnesota

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

eligibility_changeMyelodysplastic SyndromesOct 29thApollo Therapeutics LtdNCT06372717
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMyelodysplastic SyndromesOct 29thMemorial Sloan Kettering Cancer CenterNCT05884333
Target enrollment decreased from 54 to 17

Target enrollment decreased from 54 to 17

eligibility_changeMyasthenia GravisOct 29thUCB Biopharma SRLNCT07465289
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisOct 29thDianthus TherapeuticsNCT07647510
Added 8 new sites: Kutaisi, Georgia, Tbilisi, Georgia, Tbilisi, Georgia and 5 more

Added 8 new sites: Kutaisi, Georgia, Tbilisi, Georgia, Tbilisi, Georgia and 5 more

sites_addedMyasthenia GravisOct 29thAmgenNCT06987539
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

sites_addedMyasthenia GravisOct 29thMerck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, GermanyNCT06463587
Added 1 new site: Greenfield, Wisconsin

Added 1 new site: Greenfield, Wisconsin

sites_removedMyasthenia GravisOct 29thUCB Biopharma SRLNCT06540144
Reduced from 11 to 9 sites

Reduced from 11 to 9 sites

sites_addedMyasthenia GravisOct 29thUCB Biopharma SRLNCT07463521
Added 14 new sites: Beijing, China, Chongqing, China, Dijon, France and 11 more

Added 14 new sites: Beijing, China, Chongqing, China, Dijon, France and 11 more

sites_addedMyasthenia GravisOct 29thKite, A Gilead CompanyNCT06626919
Added 8 new sites: Los Angeles, California, Orange, California, Palo Alto, California and 5 more

Added 8 new sites: Los Angeles, California, Orange, California, Palo Alto, California and 5 more

eligibility_changeMyasthenia GravisOct 29thKite, A Gilead CompanyNCT06626919
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyasthenia GravisOct 29thRegeneron PharmaceuticalsNCT05070858
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMultiple SclerosisOct 29thHoffmann-La RocheNCT05269004
Reduced from 236 to 232 sites

Reduced from 236 to 232 sites

sites_addedMultiple SclerosisOct 29thHoffmann-La RocheNCT06675955
Added 13 new sites: Pleven, Bulgaria, Pleven, Bulgaria, Sofia, Bulgaria and 10 more

Added 13 new sites: Pleven, Bulgaria, Pleven, Bulgaria, Sofia, Bulgaria and 10 more

sites_addedMultiple SclerosisOct 29thAston UniversityNCT07689682
Added 1 new site: Birmingham, United Kingdom

Added 1 new site: Birmingham, United Kingdom

sites_addedMultiple SclerosisOct 29thZenas BioPharma (USA), LLCNCT07067463
Added 5 new sites: Pleven, Bulgaria, Tbilisi, Georgia, Palermo, Italy and 2 more

Added 5 new sites: Pleven, Bulgaria, Tbilisi, Georgia, Palermo, Italy and 2 more

sites_addedMultiple SclerosisOct 29thTG Therapeutics, Inc.NCT07503873
Added 5 new sites: Mostar, Bosnia and Herzegovina, Rustavi, Georgia, Krakow, Poland and 2 more

Added 5 new sites: Mostar, Bosnia and Herzegovina, Rustavi, Georgia, Krakow, Poland and 2 more

sites_addedMultiple SclerosisOct 29thZenas BioPharma (USA), LLCNCT07299019
Added 17 new sites: Kogarah, Australia, Sofia, Bulgaria, Veliko Tarnovo, Bulgaria and 14 more

Added 17 new sites: Kogarah, Australia, Sofia, Bulgaria, Veliko Tarnovo, Bulgaria and 14 more

sites_addedMultiple SclerosisOct 29thKite, A Gilead CompanyNCT07304154
Added 2 new sites: Montreal, Canada, Ottawa, Canada

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

eligibility_changeMultiple SclerosisOct 29thKite, A Gilead CompanyNCT07304154
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple MyelomaOct 29thCanadian Cancer Trials GroupNCT06182774
Added 2 new sites: Mississauga, Canada, Oakville, Canada

Added 2 new sites: Mississauga, Canada, Oakville, Canada

sites_addedMultiple MyelomaOct 29thNational Cancer Institute (NCI)NCT06465316
Added 1 new site: Washington D.C., District of Columbia

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

sites_addedMultiple MyelomaOct 29thRegeneron PharmaceuticalsNCT07609940
Added 2 new sites: Reno, Nevada, Lubbock, Texas

Added 2 new sites: Reno, Nevada, Lubbock, Texas

sites_addedMultiple MyelomaOct 29thRegeneron PharmaceuticalsNCT07393282
Added 7 new sites: New York, New York, Katowice, Poland, Hwasun, South Korea and 4 more

Added 7 new sites: New York, New York, Katowice, Poland, Hwasun, South Korea and 4 more

sites_addedMultiple MyelomaOct 29thK36 Therapeutics, Inc.NCT05651932
Added 4 new sites: Ann Arbor, Michigan, Houston, Texas, Tacoma, Washington and 1 more

Added 4 new sites: Ann Arbor, Michigan, Houston, Texas, Tacoma, Washington and 1 more

sites_addedMultiple MyelomaOct 29thAbbVieNCT06953960
Added 6 new sites: New Haven, Connecticut, Hillsborough, North Carolina, Winston-Salem, North Carolina and 3 more

Added 6 new sites: New Haven, Connecticut, Hillsborough, North Carolina, Winston-Salem, North Carolina and 3 more

sites_addedMultiple MyelomaOct 29thNational Cancer Institute (NCI)NCT06948084
Added 5 new sites: O'Fallon, Illinois, O'Fallon, Illinois, Springfield, Illinois and 2 more

Added 5 new sites: O'Fallon, Illinois, O'Fallon, Illinois, Springfield, Illinois and 2 more

sites_addedMerkel Cell CarcinomaOct 28thSystImmune Inc.NCT07080242
Added 8 new sites: Glendale, California, Batumi, Georgia, Kutaisi, Georgia and 5 more

Added 8 new sites: Glendale, California, Batumi, Georgia, Kutaisi, Georgia and 5 more

sites_removedIgA NephropathyOct 28thHoffmann-La RocheNCT05797610
Reduced from 171 to 162 sites

Reduced from 171 to 162 sites

sites_addedIdiopathic Pulmonary FibrosisOct 28thBoehringer IngelheimNCT06238622
Added 3 new sites: New York, New York, Pessac, France, Taichung, Taiwan

Added 3 new sites: New York, New York, Pessac, France, Taichung, Taiwan

sites_addedMesotheliomaOct 28thOrion Corporation, Orion PharmaNCT07563738
Added 21 new sites: Los Angeles, California, Orange, California, Fort Worth, Texas and 18 more

Added 21 new sites: Los Angeles, California, Orange, California, Fort Worth, Texas and 18 more

sites_addedMesotheliomaOct 28thNational Cancer Institute (NCI)NCT05001880
Added 1 new site: Edina, Minnesota

Added 1 new site: Edina, Minnesota

eligibility_changeMerkel Cell CarcinomaOct 28thTuHURA Biosciences, Inc.NCT06940440
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMembranous NephropathyOct 28thAstraZenecaNCT07571746
Added 3 new sites: Beijing, China, Beijing, China, Hanover, Germany

Added 3 new sites: Beijing, China, Beijing, China, Hanover, Germany

eligibility_changeMembranous NephropathyOct 28thNational Human Genome Research Institute (NHGRI)NCT06664814
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLupus NephritisOct 28thCRISPR TherapeuticsNCT06925542
Added 1 new site: Valencia, Spain

Added 1 new site: Valencia, Spain

sites_addedLupus NephritisOct 28thRegeneron PharmaceuticalsNCT06975787
Added 1 new site: Gainesville, Florida

Added 1 new site: Gainesville, Florida

sites_addedLeber Congenital AmaurosisOct 28thLaboratoires TheaNCT06891443
Added 1 new site: Sydney, Australia

Added 1 new site: Sydney, Australia

sites_addedImmune ThrombocytopeniaOct 28thNovartis PharmaceuticalsNCT07421167
Added 1 new site: Chandler, Arizona

Added 1 new site: Chandler, Arizona

sites_addedImmune ThrombocytopeniaOct 28thCRISPR Therapeutics AGNCT07549698
Added 3 new sites: Boston, Massachusetts, Dresden, Germany, Barcelona, Spain

Added 3 new sites: Boston, Massachusetts, Dresden, Germany, Barcelona, Spain

sites_addedIgA NephropathyOct 28thNovartis PharmaceuticalsNCT07146906
Added 1 new site: San Francisco, California

Added 1 new site: San Francisco, California

sites_addedIgA NephropathyOct 28thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06740526
Added 19 new sites: Birmingham, Alabama, Stanford, California, Denver, Colorado and 16 more

Added 19 new sites: Birmingham, Alabama, Stanford, California, Denver, Colorado and 16 more

Enrollment UpdateIgA NephropathyOct 28thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06740526
Target enrollment increased from 25 to 56

Target enrollment increased from 25 to 56

eligibility_changeIgA NephropathyOct 28thOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06740526
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisOct 28thContineum TherapeuticsNCT07284459
Added 6 new sites: Chermside, Australia, Rennes, France, Amiens, France and 3 more

Added 6 new sites: Chermside, Australia, Rennes, France, Amiens, France and 3 more

sites_addedIdiopathic Pulmonary FibrosisOct 28thPureTechNCT07284602
Added 8 new sites: Hialeah, Florida, Kissimmee, Florida, Greensboro, North Carolina and 5 more

Added 8 new sites: Hialeah, Florida, Kissimmee, Florida, Greensboro, North Carolina and 5 more

sites_addedHunter SyndromeOct 27thDenali Therapeutics Inc.NCT06075537
Added 1 new site: Porto Alegre, Brazil

Added 1 new site: Porto Alegre, Brazil

sites_addedHodgkin LymphomaOct 27thChildren's Hospital Medical Center, CincinnatiNCT06131801
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

sites_addedGranulomatosis with PolyangiitisOct 27thAstraZenecaNCT07444567
Added 2 new sites: Calgary, Canada, Petah Tikva, Israel

Added 2 new sites: Calgary, Canada, Petah Tikva, Israel

eligibility_changeGastrointestinal Stromal TumorOct 27thKura Oncology, Inc.NCT06655246
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHypoparathyroidismOct 27thMBX BiosciencesNCT07699471
Added 2 new sites: Omaha, Nebraska, Oakville, Canada

Added 2 new sites: Omaha, Nebraska, Oakville, Canada

sites_addedHypoparathyroidismOct 27thAscendis Pharma Bone Diseases A/SNCT07081997
Expanded from 8 to 9 sites

Expanded from 8 to 9 sites

sites_addedHuntington DiseaseOct 27thSarepta Therapeutics, Inc.NCT07536061
Added 1 new site: Parkville, Australia

Added 1 new site: Parkville, Australia

sites_addedHomozygous Familial HypercholesterolemiaOct 27thArrowhead PharmaceuticalsNCT07473843
Added 1 new site: Warsaw, Poland

Added 1 new site: Warsaw, Poland

sites_addedHereditary Hemorrhagic TelangiectasiaOct 27thAtavistik Bio, IncNCT07601425
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

sites_addedHemophilia BOct 27thRegeneron PharmaceuticalsNCT06379789
Added 2 new sites: Dallas, Texas, Oxford, United Kingdom

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

sites_addedHemophilia BOct 27thCSL BehringNCT07080905
Added 1 new site: San Diego, California

Added 1 new site: San Diego, California

eligibility_changeHemophilia BOct 27thCSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BOct 27thPfizerNCT05145127
Added 3 new sites: Belgrade, Serbia, Taipei, Taiwan, Birmingham, United Kingdom

Added 3 new sites: Belgrade, Serbia, Taipei, Taiwan, Birmingham, United Kingdom

sites_removedHemophilia BOct 27thPfizerNCT03861273
Reduced from 67 to 65 sites

Reduced from 67 to 65 sites

sites_addedHemophilia AOct 27thPfizerNCT05145127
Added 3 new sites: Belgrade, Serbia, Taipei, Taiwan, Birmingham, United Kingdom

Added 3 new sites: Belgrade, Serbia, Taipei, Taiwan, Birmingham, United Kingdom

sites_removedHemophilia AOct 27thHoffmann-La RocheNCT05181618
Reduced from 28 to 26 sites

Reduced from 28 to 26 sites

sites_addedHemophilia AOct 27thHoffmann-La RocheNCT07416526
Added 4 new sites: Rosario, Argentina, Vienna, Austria, Campinas, Brazil and 1 more

Added 4 new sites: Rosario, Argentina, Vienna, Austria, Campinas, Brazil and 1 more

sites_addedGuillain-Barré SyndromeOct 27thAnnexon, Inc.NCT07020819
Added 18 new sites: Phoenix, Arizona, Loma Linda, California, Palo Alto, California and 15 more

Added 18 new sites: Phoenix, Arizona, Loma Linda, California, Palo Alto, California and 15 more

eligibility_changeGuillain-Barré SyndromeOct 27thAnnexon, Inc.NCT07020819
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedGraves’ DiseaseOct 27thBiohaven Therapeutics Ltd.NCT07661056
Added 1 new site: San Juan, Puerto Rico

Added 1 new site: San Juan, Puerto Rico

sites_addedGraves’ DiseaseOct 27thBiogenNCT07722546
Added 1 new site: Humble, Texas

Added 1 new site: Humble, Texas

sites_addedGiant Cell ArteritisOct 27thCentre Integre Universitaire de Sante et Services Sociaux du Nord de l'ile de MontrealNCT05749094
Added 1 new site: Ottawa, Canada

Added 1 new site: Ottawa, Canada

sites_addedGeographic AtrophyOct 27thSanofiNCT07215234
Added 3 new sites: Oxnard, California, Walnut Creek, California, Stuart, Florida

Added 3 new sites: Oxnard, California, Walnut Creek, California, Stuart, Florida

eligibility_changeGastrointestinal Stromal TumorOct 27thUniversity of MiamiNCT05366816
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateGastrointestinal Stromal TumorOct 27thCentre Leon BerardNCT05152472
Target enrollment decreased from 110 to 58

Target enrollment decreased from 110 to 58

eligibility_changeFocal Segmental GlomerulosclerosisOct 26thNational Human Genome Research Institute (NHGRI)NCT06664814
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyOct 26thRapport Therapeutics Inc.NCT07563881
Added 1 new site: Birmingham, Alabama

Added 1 new site: Birmingham, Alabama

eligibility_changeFabry DiseaseOct 26thAcibadem UniversityNCT07888998
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaOct 26thNovartis PharmaceuticalsNCT07357727
Added 3 new sites: Salzburg, Austria, Palermo, Italy, Bursa, Turkey (Türkiye)

Added 3 new sites: Salzburg, Austria, Palermo, Italy, Bursa, Turkey (Türkiye)

eligibility_changeFacioscapulohumeral Muscular DystrophyOct 26thRestem, LLC.NCT07086521
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal Segmental GlomerulosclerosisOct 26thSanofiNCT06500702
Added 5 new sites: Toronto, Canada, Pécs, Hungary, Szeged, Hungary and 2 more

Added 5 new sites: Toronto, Canada, Pécs, Hungary, Szeged, Hungary and 2 more

sites_addedFocal EpilepsyOct 26thRapport Therapeutics Inc.NCT07563881
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedEssential ThrombocythemiaOct 26thNovartis PharmaceuticalsNCT07357727
Added 9 new sites: Austin, Texas, Vienna, Austria, Curitiba, Brazil and 6 more

Added 9 new sites: Austin, Texas, Vienna, Austria, Curitiba, Brazil and 6 more

sites_addedEssential ThrombocythemiaOct 26thDisc Medicine, IncNCT05320198
Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

Added 3 new sites: Rolling Meadows, Illinois, Iowa City, Iowa, Temple, Texas

sites_addedEpidermolysis BullosaOct 26thXinnate ABNCT06594393
Added 3 new sites: Modena, Italy, Gdansk, Poland, Lodz, Poland

Added 3 new sites: Modena, Italy, Gdansk, Poland, Lodz, Poland

sites_addedDuchenne Muscular DystrophyOct 26thSarepta Therapeutics, Inc.NCT06270719
Added 7 new sites: Birmingham, Alabama, Los Angeles, California, Washington D.C., District of Columbia and 4 more

Added 7 new sites: Birmingham, Alabama, Los Angeles, California, Washington D.C., District of Columbia and 4 more

sites_addedDuchenne Muscular DystrophyOct 26thWave Life Sciences USA, Inc.NCT04906460
Added 1 new site: Irbid, Jordan

Added 1 new site: Irbid, Jordan

Enrollment UpdateDravet SyndromeOct 26thEncoded TherapeuticsNCT05419492
Target enrollment increased from 47 to 113

Target enrollment increased from 47 to 113

eligibility_changeDravet SyndromeOct 26thEncoded TherapeuticsNCT05419492
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDilated CardiomyopathyOct 26thAffinia TherapeuticsNCT07426419
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedDiffuse Intrinsic Pontine GliomaOct 26thGustave Roussy, Cancer Campus, Grand ParisNCT05476939
Added 3 new sites: Brussels, Belgium, Leuven, Belgium, Athens, Greece

Added 3 new sites: Brussels, Belgium, Leuven, Belgium, Athens, Greece

eligibility_changeCystic FibrosisOct 25thVertex Pharmaceuticals IncorporatedNCT07283770
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Inflammatory Demyelinating PolyneuropathyOct 25thSanofiNCT06290141
Added 11 new sites: Scottsdale, Arizona, Los Angeles, California, Orange, California and 8 more

Added 11 new sites: Scottsdale, Arizona, Los Angeles, California, Orange, California and 8 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyOct 25thKite, A Gilead CompanyNCT07304154
Added 2 new sites: Montreal, Canada, Ottawa, Canada

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

eligibility_changeChronic Inflammatory Demyelinating PolyneuropathyOct 25thKite, A Gilead CompanyNCT07304154
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaOct 25thInstitut für Klinische Krebsforschung IKF GmbH at Krankenhaus NordwestNCT06440993
Added 2 new sites: Cologne, Germany, Düsseldorf, Germany

Added 2 new sites: Cologne, Germany, Düsseldorf, Germany

eligibility_changeCholangiocarcinomaOct 25thInstitut für Klinische Krebsforschung IKF GmbH at Krankenhaus NordwestNCT06440993
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateCholangiocarcinomaOct 25thNational Cancer Institute (NCI)NCT05564403
Target enrollment decreased from 66 to 17

Target enrollment decreased from 66 to 17

sites_addedCholangiocarcinomaOct 25thiOMEDICO AGNCT06607302
Added 3 new sites: Bamberg, Germany, Berlin, Germany, Freiburg im Breisgau, Germany

Added 3 new sites: Bamberg, Germany, Berlin, Germany, Freiburg im Breisgau, Germany

sites_addedAutoimmune HepatitisOct 24thUniversity Health Network, TorontoNCT06381453
Added 1 new site: Ottawa, Canada

Added 1 new site: Ottawa, Canada

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Oct 24thAlnylam PharmaceuticalsNCT07052903
Added 1 new site: Nangang, China

Added 1 new site: Nangang, China

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Oct 24thNovo Nordisk A/SNCT07207811
Added 3 new sites: Phoenix, Arizona, Buenos Aires, Argentina, Ottawa, Canada

Added 3 new sites: Phoenix, Arizona, Buenos Aires, Argentina, Ottawa, Canada

sites_addedAngelman SyndromeOct 24thOHB Pediatrics Ltd.NCT07605429
Added 2 new sites: Nashville, Tennessee, Taipei, Taiwan

Added 2 new sites: Nashville, Tennessee, Taipei, Taiwan

eligibility_changeAmyotrophic Lateral SclerosisOct 24thBiogenNCT04856982
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateAmyotrophic Lateral SclerosisOct 24thVector Y TherapeuticsNCT07287397
Target enrollment increased from 12 to 36

Target enrollment increased from 12 to 36

sites_addedAlpha-1 Antitrypsin DeficiencyOct 24thAIRNA CorporationNCT07431112
Added 1 new site: Hanover, Germany

Added 1 new site: Hanover, Germany

Enrollment UpdateAlpha-1 Antitrypsin DeficiencyOct 24thWave Life Sciences USA, Inc.NCT06405633
Target enrollment increased from 24 to 48

Target enrollment increased from 24 to 48

sites_addedAcromegalyOct 24thDebiopharm International SANCT06930625
Added 1 new site: Brasília, Brazil

Added 1 new site: Brasília, Brazil

eligibility_changeWaldenström MacroglobulinemiaOct 23rdBeOne MedicinesNCT05294731
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedVon Willebrand DiseaseOct 23rdUnity Health TorontoNCT06205095
Added 1 new site: Kingston, Canada

Added 1 new site: Kingston, Canada

sites_addedSystemic SclerosisOct 22ndCRISPR TherapeuticsNCT06925542
Added 1 new site: Valencia, Spain

Added 1 new site: Valencia, Spain

sites_removedSpinal Muscular AtrophyOct 22ndHoffmann-La RocheNCT05861999
Reduced from 19 to 17 sites

Reduced from 19 to 17 sites

Enrollment UpdateSpinal Muscular AtrophyOct 22ndHoffmann-La RocheNCT05861999
Target enrollment decreased from 28 to 19

Target enrollment decreased from 28 to 19

sites_addedThyroid Eye DiseaseOct 22ndSling Therapeutics, Inc.NCT07753603
Added 17 new sites: Glendale, Arizona, Beverly Hills, California, Canoga Park, California and 14 more

Added 17 new sites: Glendale, Arizona, Beverly Hills, California, Canoga Park, California and 14 more

eligibility_changeSystemic SclerosisOct 22ndAvalyn Pharma Inc.NCT06329401
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSystemic Juvenile Idiopathic ArthritisOct 22ndSanofiNCT02991469
Added 4 new sites: Athens, Greece, Chamberí, Spain, Madrid, Spain and 1 more

Added 4 new sites: Athens, Greece, Chamberí, Spain, Madrid, Spain and 1 more

sites_addedSpinal Muscular AtrophyOct 22ndBiogenNCT07221669
Added 2 new sites: São Paulo, Brazil, Ahmedabad, India

Added 2 new sites: São Paulo, Brazil, Ahmedabad, India

sites_addedSjögren's SyndromeOct 22ndVor BiopharmaNCT07404865
Added 6 new sites: Miami, Florida, Durham, North Carolina, Oklahoma City, Oklahoma and 3 more

Added 6 new sites: Miami, Florida, Durham, North Carolina, Oklahoma City, Oklahoma and 3 more

sites_addedSickle Cell DiseaseOct 22ndNovartis PharmaceuticalsNCT06546670
Added 4 new sites: New York, New York, Ribeirão Preto, Brazil, São Paulo, Brazil and 1 more

Added 4 new sites: New York, New York, Ribeirão Preto, Brazil, São Paulo, Brazil and 1 more

Enrollment UpdateSickle Cell DiseaseOct 22ndUniversity of PittsburghNCT06374238
Target enrollment decreased from 470 to 390

Target enrollment decreased from 470 to 390

eligibility_changeSickle Cell DiseaseOct 22ndUniversity of PittsburghNCT06374238
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSickle Cell DiseaseOct 22ndCSL BehringNCT06699849
Added 4 new sites: Washington D.C., District of Columbia, Yenişehir, Turkey (Türkiye), Liverpool, United Kingdom and 1 more

Added 4 new sites: Washington D.C., District of Columbia, Yenişehir, Turkey (Türkiye), Liverpool, United Kingdom and 1 more

sites_addedSickle Cell DiseaseOct 22ndMark Walters, MDNCT04774536
Added 1 new site: Davis, California

Added 1 new site: Davis, California

Enrollment UpdateSickle Cell DiseaseOct 22ndVanderbilt University Medical CenterNCT05153967
Target enrollment decreased from 750 to 377

Target enrollment decreased from 750 to 377

sites_removedSickle Cell DiseaseOct 22ndNational Alliance for Sickle Cell CentersNCT06071377
Reduced from 10 to 8 sites

Reduced from 10 to 8 sites

sites_addedSickle Cell DiseaseOct 22ndSanofiNCT06975865
Added 19 new sites: Clovis, California, Los Angeles, California, Washington D.C., District of Columbia and 16 more

Added 19 new sites: Clovis, California, Los Angeles, California, Washington D.C., District of Columbia and 16 more

sites_removedSevere Combined ImmunodeficiencyOct 22ndCenter for International Blood and Marrow Transplant ResearchNCT03619551
Reduced from 40 to 38 sites

Reduced from 40 to 38 sites

eligibility_changeSevere Combined ImmunodeficiencyOct 22ndNational Institute of Allergy and Infectious Diseases (NIAID)NCT01306019
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSarcoidosisOct 22ndPriovant Therapeutics, Inc.NCT06978725
Added 34 new sites: Birmingham, Alabama, Phoenix, Arizona, Tucson, Arizona and 31 more

Added 34 new sites: Birmingham, Alabama, Phoenix, Arizona, Tucson, Arizona and 31 more

eligibility_changeSarcoidosisOct 22ndDuke UniversityNCT01745237
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraOct 21stOno Pharmaceutical Co., Ltd.NCT07429266
Added 15 new sites: Orange, California, Columbus, Ohio, Philadelphia, Pennsylvania and 12 more

Added 15 new sites: Orange, California, Columbus, Ohio, Philadelphia, Pennsylvania and 12 more

sites_addedPolycythemia VeraOct 21stMerck Sharp & Dohme LLCNCT07722611
Added 1 new site: Charlottesville, Virginia

Added 1 new site: Charlottesville, Virginia

eligibility_changePhenylketonuriaOct 21stOtsuka Pharmaceutical Development & Commercialization, Inc.NCT06971731
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedRett SyndromeOct 21stUCB BIOSCIENCES, Inc.NCT07503444
Added 1 new site: Tbilisi, Georgia

Added 1 new site: Tbilisi, Georgia

eligibility_changeRetinitis PigmentosaOct 21stPeking University Third HospitalNCT05805007
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPulmonary Arterial HypertensionOct 21stInsmed IncorporatedNCT07481981
Added 13 new sites: New Hyde Park, New York, New York, New York, New York, New York and 10 more

Added 13 new sites: New Hyde Park, New York, New York, New York, New York, New York and 10 more

sites_addedPulmonary Arterial HypertensionOct 21stAllRock Bio, Inc.NCT07175038
Added 1 new site: Krakow, Poland

Added 1 new site: Krakow, Poland

sites_addedProgressive Supranuclear PalsyOct 21stNovartis PharmaceuticalsNCT07498426
Added 22 new sites: Boca Raton, Florida, Lexington, Kentucky, New Orleans, Louisiana and 19 more

Added 22 new sites: Boca Raton, Florida, Lexington, Kentucky, New Orleans, Louisiana and 19 more

sites_addedProgressive Supranuclear PalsyOct 21stAdam BoxerNCT07173803
Added 7 new sites: Sun City, Arizona, Chicago, Illinois, Indianapolis, Indiana and 4 more

Added 7 new sites: Sun City, Arizona, Chicago, Illinois, Indianapolis, Indiana and 4 more

sites_addedPrimary Biliary CholangitisOct 21stGilead SciencesNCT06051617
Added 4 new sites: Roseville, California, Milwaukee, Wisconsin, San Miguel de Tucumán, Argentina and 1 more

Added 4 new sites: Roseville, California, Milwaukee, Wisconsin, San Miguel de Tucumán, Argentina and 1 more

sites_addedPrader-Willi SyndromeOct 21stSoleno Therapeutics, Inc.NCT07450664
Added 25 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 22 more

Added 25 new sites: Phoenix, Arizona, Los Angeles, California, Sacramento, California and 22 more

Enrollment UpdatePrader-Willi SyndromeOct 21stRhythm Pharmaceuticals, Inc.NCT06239116
Target enrollment decreased from 150 to 111

Target enrollment decreased from 150 to 111

eligibility_changePrader-Willi SyndromeOct 21stRhythm Pharmaceuticals, Inc.NCT06239116
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPolycythemia VeraOct 21stNovartis PharmaceuticalsNCT07357727
Added 22 new sites: Denver, Colorado, Austin, Texas, Buenos Aires, Argentina and 19 more

Added 22 new sites: Denver, Colorado, Austin, Texas, Buenos Aires, Argentina and 19 more

eligibility_changePolycythemia VeraOct 21stProtagonist Therapeutics, Inc.NCT05210790
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPheochromocytomaOct 21stPerspective TherapeuticsNCT05636618
Added 1 new site: Orlando, Florida

Added 1 new site: Orlando, Florida

sites_addedPhelan-McDermid SyndromeOct 21stNeuren Pharmaceuticals LimitedNCT07593391
Added 2 new sites: Chicago, Illinois, Charlottesville, Virginia

Added 2 new sites: Chicago, Illinois, Charlottesville, Virginia

sites_addedMyelofibrosisOct 20thNovartis PharmaceuticalsNCT07357727
Added 1 new site: Hamburg, Germany

Added 1 new site: Hamburg, Germany

sites_addedMyelodysplastic SyndromesOct 20thAscentage Pharma Group Inc.NCT06641414
Added 155 new sites: Birmingham, Alabama, Phoenix, Arizona, Los Angeles, California and 152 more

Added 155 new sites: Birmingham, Alabama, Phoenix, Arizona, Los Angeles, California and 152 more

eligibility_changeMyelodysplastic SyndromesOct 20thAscentage Pharma Group Inc.NCT06641414
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesOct 20thNational Cancer Institute (NCI)NCT05564390
Added 3 new sites: Bismarck, North Dakota, Fargo, North Dakota, Fargo, North Dakota

Added 3 new sites: Bismarck, North Dakota, Fargo, North Dakota, Fargo, North Dakota

sites_addedMultiple SclerosisOct 20thHoffmann-La RocheNCT06780150
Added 1 new site: Münster, Germany

Added 1 new site: Münster, Germany

sites_addedMultiple SclerosisOct 20thZenas BioPharma (USA), LLCNCT07067463
Added 3 new sites: Orlando, Florida, Warsaw, Poland, Ivano-Frankivsk, Ukraine

Added 3 new sites: Orlando, Florida, Warsaw, Poland, Ivano-Frankivsk, Ukraine

eligibility_changeMultiple SclerosisOct 20thBiogenNCT05688436
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedParagangliomaOct 20thPerspective TherapeuticsNCT05636618
Added 1 new site: Orlando, Florida

Added 1 new site: Orlando, Florida

Enrollment UpdateNeuromyelitis OpticaOct 20thAlexion Pharmaceuticals, Inc.NCT05966467
Target enrollment increased from 122 to 200

Target enrollment increased from 122 to 200

sites_addedMyotonic DystrophyOct 20thDyne TherapeuticsNCT07486934
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

sites_addedMyotonic DystrophyOct 20thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Westmead, Australia

Added 1 new site: Westmead, Australia

sites_addedMyotonic DystrophyOct 20thVertex Pharmaceuticals IncorporatedNCT06926621
Added 1 new site: Winston-Salem, North Carolina

Added 1 new site: Winston-Salem, North Carolina

sites_addedMyelofibrosisOct 20thNovartis PharmaceuticalsNCT07357727
Added 21 new sites: Denver, Colorado, Austin, Texas, Buenos Aires, Argentina and 18 more

Added 21 new sites: Denver, Colorado, Austin, Texas, Buenos Aires, Argentina and 18 more

eligibility_changeMyelofibrosisOct 20thUniversity of UtahNCT07521046
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelofibrosisOct 20thFred Hutchinson Cancer CenterNCT07228624
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesOct 20thTakedaNCT07422480
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMyelodysplastic SyndromesOct 20thTakedaNCT04419649
Target enrollment increased from 160 to 210

Target enrollment increased from 160 to 210

eligibility_changeMyelodysplastic SyndromesOct 20thTakedaNCT04419649
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateMyelodysplastic SyndromesOct 20thWashington University School of MedicineNCT05434598
Target enrollment increased from 60 to 100

Target enrollment increased from 60 to 100

Enrollment UpdateMyelodysplastic SyndromesOct 20thEric Stephen Winer, MDNCT05703542
Target enrollment decreased from 24 to 12

Target enrollment decreased from 24 to 12

eligibility_changeMyelodysplastic SyndromesOct 20thTakedaNCT07319845
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesOct 20thMontefiore Medical CenterNCT05986240
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisOct 20thargenxNCT07673627
Added 4 new sites: Carlsbad, California, Las Vegas, Nevada, Thessaloniki, Greece and 1 more

Added 4 new sites: Carlsbad, California, Las Vegas, Nevada, Thessaloniki, Greece and 1 more

sites_addedMyasthenia GravisOct 20thKite, A Gilead CompanyNCT07304154
Added 2 new sites: Montreal, Canada, Ottawa, Canada

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

eligibility_changeMyasthenia GravisOct 20thKite, A Gilead CompanyNCT07304154
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisOct 20thargenxNCT07294170
Added 1 new site: Liège, Belgium

Added 1 new site: Liège, Belgium

sites_addedMyasthenia GravisOct 20thVor BiopharmaNCT06456580
Added 25 new sites: Clearwater, Florida, New Brunswick, New Jersey, New York, New York and 22 more

Added 25 new sites: Clearwater, Florida, New Brunswick, New Jersey, New York, New York and 22 more

sites_addedMultiple SclerosisOct 20thZenas BioPharma (USA), LLCNCT07067463
Added 1 new site: Veliko Tarnovo, Bulgaria

Added 1 new site: Veliko Tarnovo, Bulgaria

sites_addedMultiple SclerosisOct 20thSanofiNCT07325292
Added 29 new sites: Tucson, Arizona, Lancaster, California, Aurora, Colorado and 26 more

Added 29 new sites: Tucson, Arizona, Lancaster, California, Aurora, Colorado and 26 more

sites_addedMultiple SclerosisOct 20thNovartis PharmaceuticalsNCT07225504
Added 38 new sites: Phoenix, Arizona, Newport Beach, California, Frederick, Maryland and 35 more

Added 38 new sites: Phoenix, Arizona, Newport Beach, California, Frederick, Maryland and 35 more

Enrollment UpdateMultiple SclerosisOct 20thAutolus LimitedNCT07139743
Target enrollment increased from 18 to 24

Target enrollment increased from 18 to 24

eligibility_changeMultiple SclerosisOct 20thAutolus LimitedNCT07139743
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple MyelomaOct 20thNational Cancer Institute (NCI)NCT06948084
Added 3 new sites: Cedar Rapids, Iowa, Cedar Rapids, Iowa, Great Falls, Montana

Added 3 new sites: Cedar Rapids, Iowa, Cedar Rapids, Iowa, Great Falls, Montana

eligibility_changeMultiple MyelomaOct 20thWashington University School of MedicineNCT07200102
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedLupus NephritisOct 19thHoffmann-La RocheNCT07598396
Added 2 new sites: CABA, Argentina, Buenos Aires, Argentina

Added 2 new sites: CABA, Argentina, Buenos Aires, Argentina

eligibility_changeImmune ThrombocytopeniaOct 19thargenxNCT04812925
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIgA NephropathyOct 19thVertex Pharmaceuticals IncorporatedNCT07807995
Added 1 new site: Idaho Falls, Idaho

Added 1 new site: Idaho Falls, Idaho

sites_addedIdiopathic Pulmonary FibrosisOct 19thUnited TherapeuticsNCT04905693
Added 2 new sites: Kogarah, Australia, Kaohsiung City, Taiwan

Added 2 new sites: Kogarah, Australia, Kaohsiung City, Taiwan

sites_removedMesotheliomaOct 19thInstituto do Cancer do Estado de São PauloNCT06638931
Reduced from 8 to 5 sites

Reduced from 8 to 5 sites

sites_addedMesotheliomaOct 19thMedicenna Therapeutics, Inc.NCT05086692
Added 1 new site: Zurich, Switzerland

Added 1 new site: Zurich, Switzerland

Enrollment UpdateMesotheliomaOct 19thMedicenna Therapeutics, Inc.NCT05086692
Target enrollment increased from 115 to 160

Target enrollment increased from 115 to 160

sites_addedMerkel Cell CarcinomaOct 19thMedicenna Therapeutics, Inc.NCT05086692
Added 1 new site: Zurich, Switzerland

Added 1 new site: Zurich, Switzerland

Enrollment UpdateMerkel Cell CarcinomaOct 19thMedicenna Therapeutics, Inc.NCT05086692
Target enrollment increased from 115 to 160

Target enrollment increased from 115 to 160

sites_addedKCNT1-Related EpilepsyOct 19thActio Biosciences, Inc.NCT07600736
Added 1 new site: Rochester, New York

Added 1 new site: Rochester, New York

sites_addedImmune ThrombocytopeniaOct 18thargenxNCT07194850
Added 4 new sites: La Tronche, France, Vandœuvre-lès-Nancy, France, Sapporo, Japan and 1 more

Added 4 new sites: La Tronche, France, Vandœuvre-lès-Nancy, France, Sapporo, Japan and 1 more

sites_addedImmune ThrombocytopeniaOct 18thNovartis PharmaceuticalsNCT07421167
Added 2 new sites: Torrance, California, Pittsburgh, Pennsylvania

Added 2 new sites: Torrance, California, Pittsburgh, Pennsylvania

sites_addedIgA NephropathyOct 18thNovartis PharmaceuticalsNCT07498335
Added 6 new sites: Bron, France, Nantes, France, Toulouse, France and 3 more

Added 6 new sites: Bron, France, Nantes, France, Toulouse, France and 3 more

sites_addedIgA NephropathyOct 18thBiogenNCT06935357
Added 1 new site: Hamburg, Germany

Added 1 new site: Hamburg, Germany

sites_addedIdiopathic Pulmonary FibrosisOct 18thTrevi TherapeuticsNCT07671911
Added 3 new sites: Reno, Nevada, The Bronx, New York, Murray, Utah

Added 3 new sites: Reno, Nevada, The Bronx, New York, Murray, Utah

sites_addedIdiopathic Pulmonary FibrosisOct 18thBoehringer IngelheimNCT06238622
Added 2 new sites: São Paulo, Brazil, Kaohsiung City, Taiwan

Added 2 new sites: São Paulo, Brazil, Kaohsiung City, Taiwan

sites_removedIdiopathic Pulmonary FibrosisOct 18thMediar TherapeuticsNCT06967805
Reduced from 71 to 59 sites

Reduced from 71 to 59 sites

sites_addedIdiopathic Pulmonary FibrosisOct 18thAvalyn Pharma Inc.NCT07194382
Added 25 new sites: New Lambton Heights, Australia, Sydney, Australia, Launceston, Australia and 22 more

Added 25 new sites: New Lambton Heights, Australia, Sydney, Australia, Launceston, Australia and 22 more

eligibility_changeIdiopathic Pulmonary FibrosisOct 18thInSilico Medicine Hong Kong LimitedNCT07687459
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisOct 18thRein TherapeuticsNCT06968845
Added 12 new sites: Winston-Salem, North Carolina, Melbourne, Australia, Haskovo, Bulgaria and 9 more

Added 12 new sites: Winston-Salem, North Carolina, Melbourne, Australia, Haskovo, Bulgaria and 9 more

sites_addedIdiopathic Pulmonary FibrosisOct 18thOorja Bio, Inc.NCT07869147
Added 1 new site: Doral, Florida

Added 1 new site: Doral, Florida

sites_addedIdiopathic Pulmonary FibrosisOct 18thContineum TherapeuticsNCT07284459
Added 4 new sites: Bobigny, France, Athens, Greece, Santiago de Compostela, Spain and 1 more

Added 4 new sites: Bobigny, France, Athens, Greece, Santiago de Compostela, Spain and 1 more

sites_addedIdiopathic Pulmonary FibrosisOct 18thChiesi Farmaceutici S.p.A.NCT07516951
Added 16 new sites: Dothan, Alabama, St. Petersburg, Florida, Rock Hill, South Carolina and 13 more

Added 16 new sites: Dothan, Alabama, St. Petersburg, Florida, Rock Hill, South Carolina and 13 more

sites_addedHypoparathyroidismOct 18thMBX BiosciencesNCT07699471
Added 7 new sites: Englewood, Colorado, Lawrenceville, Georgia, Jackson, Mississippi and 4 more

Added 7 new sites: Englewood, Colorado, Lawrenceville, Georgia, Jackson, Mississippi and 4 more

sites_addedHypersensitivity PneumonitisOct 18thNantes University HospitalNCT05988437
Added 6 new sites: Ancenis, France, Angers, France, Châteaubriant, France and 3 more

Added 6 new sites: Ancenis, France, Angers, France, Châteaubriant, France and 3 more

eligibility_changeHypersensitivity PneumonitisOct 18thAvalyn Pharma Inc.NCT06329401
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHuntington DiseaseOct 18thSkyhawk Therapeutics, Inc.NCT07378644
Added 7 new sites: Boston, Massachusetts, Charlestown, Massachusetts, Columbus, Ohio and 4 more

Added 7 new sites: Boston, Massachusetts, Charlestown, Massachusetts, Columbus, Ohio and 4 more

eligibility_changeHomozygous Familial HypercholesterolemiaOct 18thUniversity of Wisconsin, MadisonNCT07470723
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHemophilia BOct 18thPfizerNCT05611801
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia AOct 18thSanofiNCT06941870
Added 6 new sites: Indianapolis, Indiana, Columbus, Ohio, Seattle, Washington and 3 more

Added 6 new sites: Indianapolis, Indiana, Columbus, Ohio, Seattle, Washington and 3 more

eligibility_changeHemophilia AOct 18thSanofiNCT06941870
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHemophilia AOct 18thPfizerNCT05611801
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia AOct 18thPfizerNCT06703606
Added 3 new sites: La Jolla, California, La Jolla, California, San Diego, California

Added 3 new sites: La Jolla, California, La Jolla, California, San Diego, California

sites_addedHemophilia AOct 18thHoffmann-La RocheNCT07226206
Added 3 new sites: New Haven, Connecticut, Newark, New Jersey, New York, New York

Added 3 new sites: New Haven, Connecticut, Newark, New Jersey, New York, New York

sites_addedGraves’ DiseaseOct 18thargenxNCT07596849
Added 13 new sites: Aalst, Belgium, Ghent, Belgium, Plovdiv, Bulgaria and 10 more

Added 13 new sites: Aalst, Belgium, Ghent, Belgium, Plovdiv, Bulgaria and 10 more

sites_addedGraves’ DiseaseOct 18thargenxNCT07570316
Added 6 new sites: Pasadena, California, San Jose, California, Wichita, Kansas and 3 more

Added 6 new sites: Pasadena, California, San Jose, California, Wichita, Kansas and 3 more

sites_addedGraves’ DiseaseOct 18thBiohaven Therapeutics Ltd.NCT07661056
Added 2 new sites: Orchard Park, New York, Philadelphia, Pennsylvania

Added 2 new sites: Orchard Park, New York, Philadelphia, Pennsylvania

sites_addedGraves’ DiseaseOct 18thBiogenNCT07722546
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedGeographic AtrophyOct 18thNovartis PharmaceuticalsNCT07441642
Added 4 new sites: Caba, Argentina, Serra, Brazil, Blumenau, Brazil and 1 more

Added 4 new sites: Caba, Argentina, Serra, Brazil, Blumenau, Brazil and 1 more

sites_addedGeographic AtrophyOct 18thComplement TherapeuticsNCT07392255
Added 1 new site: London, United Kingdom

Added 1 new site: London, United Kingdom

sites_addedGeographic AtrophyOct 18thComplement TherapeuticsNCT07144137
Added 1 new site: Wake Forest, North Carolina

Added 1 new site: Wake Forest, North Carolina

sites_addedGeographic AtrophyOct 18thRegeneron PharmaceuticalsNCT06541704
Added 6 new sites: Manchester, Connecticut, Carmel, Indiana, Nashua, New Hampshire and 3 more

Added 6 new sites: Manchester, Connecticut, Carmel, Indiana, Nashua, New Hampshire and 3 more

sites_addedFocal Segmental GlomerulosclerosisOct 17thBoehringer IngelheimNCT07220083
Added 6 new sites: Granada Hills, California, Victorville, California, Grand Prairie, Texas and 3 more

Added 6 new sites: Granada Hills, California, Victorville, California, Grand Prairie, Texas and 3 more

sites_addedFocal EpilepsyOct 17thRapport Therapeutics Inc.NCT07594119
Added 2 new sites: Honolulu, Hawaii, Omaha, Nebraska

Added 2 new sites: Honolulu, Hawaii, Omaha, Nebraska

sites_removedFabry DiseaseOct 17thSanofiNCT05206773
Reduced from 58 to 50 sites

Reduced from 58 to 50 sites

sites_addedEssential ThrombocythemiaOct 17thNovartis PharmaceuticalsNCT07357727
Added 18 new sites: Denver, Colorado, Buenos Aires, Argentina, CABA, Argentina and 15 more

Added 18 new sites: Denver, Colorado, Buenos Aires, Argentina, CABA, Argentina and 15 more

sites_addedEpidermolysis BullosaOct 17thParadigm TherapeuticsNCT07482787
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

Enrollment UpdateEosinophilic EsophagitisOct 17thUniversity of MichiganNCT06817213
Target enrollment increased from 40 to 55

Target enrollment increased from 40 to 55

sites_addedDuchenne Muscular DystrophyOct 17thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Westmead, Australia

Added 1 new site: Westmead, Australia

sites_addedDuchenne Muscular DystrophyOct 17thSatellos Bioscience, Inc.NCT06867107
Added 1 new site: St Louis, Missouri

Added 1 new site: St Louis, Missouri

eligibility_changeDilated CardiomyopathyOct 17thAffinia TherapeuticsNCT07426419
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCongenital Adrenal HyperplasiaOct 16thCrinetics Pharmaceuticals Inc.NCT07144163
Added 3 new sites: Brasília, Brazil, Belo Horizonte, Brazil, Dammam, Saudi Arabia

Added 3 new sites: Brasília, Brazil, Belo Horizonte, Brazil, Dammam, Saudi Arabia

sites_addedCongenital Adrenal HyperplasiaOct 16thCrinetics Pharmaceuticals Inc.NCT07159841
Added 2 new sites: Riyadh, Saudi Arabia, Riyadh, Saudi Arabia

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

sites_addedChronic Inflammatory Demyelinating PolyneuropathyOct 16thargenxNCT07091630
Added 4 new sites: Vero Beach, Florida, Salvador, Brazil, Medellín, Colombia and 1 more

Added 4 new sites: Vero Beach, Florida, Salvador, Brazil, Medellín, Colombia and 1 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyOct 16thargenxNCT06920004
Added 1 new site: Barcelona, Spain

Added 1 new site: Barcelona, Spain

eligibility_changeCharcot-Marie-Tooth DiseaseOct 16thMichael ShyNCT01193075
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCharcot-Marie-Tooth DiseaseOct 16thMichael ShyNCT01193075
Added 5 new sites: Chapel Hill, North Carolina, Columbus, Ohio, Houston, Texas and 2 more

Added 5 new sites: Chapel Hill, North Carolina, Columbus, Ohio, Houston, Texas and 2 more

sites_addedCharcot-Marie-Tooth DiseaseOct 16thUniversity of IowaNCT01193088
Added 3 new sites: Columbus, Ohio, Nicosia, Cyprus, London, United Kingdom

Added 3 new sites: Columbus, Ohio, Nicosia, Cyprus, London, United Kingdom

eligibility_changeCharcot-Marie-Tooth DiseaseOct 16thUniversity of IowaNCT01193088
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedDesmoplastic Small Round Cell TumorOct 16thNational Cancer Institute (NCI)NCT04616560
Added 1 new site: Seattle, Washington

Added 1 new site: Seattle, Washington

sites_addedDermatomyositisOct 16thCabaletta BioNCT06154252
Added 3 new sites: Göttingen, Germany, Roma, Italy, London, United Kingdom

Added 3 new sites: Göttingen, Germany, Roma, Italy, London, United Kingdom

sites_addedCushing DiseaseOct 16thCorcept TherapeuticsNCT03604198
Added 4 new sites: Vienna, Austria, Ramat Gan, Israel, Messina, Italy and 1 more

Added 4 new sites: Vienna, Austria, Ramat Gan, Israel, Messina, Italy and 1 more

sites_addedChronic Inflammatory Demyelinating PolyneuropathyOct 16thGrifols Therapeutics LLCNCT07540221
Added 5 new sites: Fayetteville, North Carolina, Houston, Texas, Krakow, Poland and 2 more

Added 5 new sites: Fayetteville, North Carolina, Houston, Texas, Krakow, Poland and 2 more

sites_addedChronic Graft-versus-Host DiseaseOct 16thIncyte CorporationNCT06585774
Added 3 new sites: Homburg, Germany, Würzburg, Germany, Leeds, United Kingdom

Added 3 new sites: Homburg, Germany, Würzburg, Germany, Leeds, United Kingdom

sites_removedCholangiocarcinomaOct 16thInstituto do Cancer do Estado de São PauloNCT06638931
Reduced from 8 to 5 sites

Reduced from 8 to 5 sites

sites_added22q11.2 Deletion SyndromeOct 15thBillionToOne Inc.NCT07643896
Added 3 new sites: Riverside, California, New York, New York, Houston, Texas

Added 3 new sites: Riverside, California, New York, New York, Houston, Texas

sites_addedBecker Muscular DystrophyOct 15thAvidity Biosciences, Inc.NCT07587242
Added 1 new site: Westmead, Australia

Added 1 new site: Westmead, Australia

Enrollment UpdateBatten DiseaseOct 15thTern Therapeutics, LLCNCT05791864
Target enrollment increased from 16 to 26

Target enrollment increased from 16 to 26

eligibility_changeBatten DiseaseOct 15thTern Therapeutics, LLCNCT05791864
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Oct 15thAlnylam PharmaceuticalsNCT07358078
Added 3 new sites: La Jolla, California, Honolulu, Hawaii, Würzburg, Germany

Added 3 new sites: La Jolla, California, Honolulu, Hawaii, Würzburg, Germany

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Oct 15thBayerNCT07235462
Added 17 new sites: Multiple Locations, Austria, Multiple Locations, Belgium, Multiple Locations, Croatia and 14 more

Added 17 new sites: Multiple Locations, Austria, Multiple Locations, Belgium, Multiple Locations, Croatia and 14 more

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Oct 15thAlnylam PharmaceuticalsNCT07223203
Added 4 new sites: Belgrano, Argentina, Salvador, Brazil, Guadalajara, Mexico and 1 more

Added 4 new sites: Belgrano, Argentina, Salvador, Brazil, Guadalajara, Mexico and 1 more

sites_addedAngelman SyndromeOct 15thOHB Pediatrics Ltd.NCT07605429
Added 1 new site: Miami, Florida

Added 1 new site: Miami, Florida

sites_addedAmyotrophic Lateral SclerosisOct 15thTrace Neuroscience, Inc.NCT07674667
Added 2 new sites: Lübeck, Germany, London, United Kingdom

Added 2 new sites: Lübeck, Germany, London, United Kingdom

Enrollment UpdateAmyotrophic Lateral SclerosisOct 15thOliver BlanchardNCT06249867
Target enrollment increased from 30 to 60

Target enrollment increased from 30 to 60

eligibility_changeAmyotrophic Lateral SclerosisOct 15thOliver BlanchardNCT06249867
Eligibility criteria have been updated

Eligibility criteria have been updated

Enrollment UpdateAchondroplasiaOct 15thKyowa Kirin Co., Ltd.NCT06926491
Target enrollment increased from 6 to 13

Target enrollment increased from 6 to 13

sites_removedSystemic SclerosisOct 13thNovartis PharmaceuticalsNCT06470048
Reduced from 128 to 106 sites

Reduced from 128 to 106 sites

sites_addedSjögren's SyndromeOct 13thNovartis PharmaceuticalsNCT07621809
Added 7 new sites: Voorhees Township, New Jersey, Wilmington, North Carolina, Sydney, Canada and 4 more

Added 7 new sites: Voorhees Township, New Jersey, Wilmington, North Carolina, Sydney, Canada and 4 more

sites_addedSjögren's SyndromeOct 13thNovartis PharmaceuticalsNCT07737743
Added 2 new sites: Clearwater, Florida, Trois-Rivières, Canada

Added 2 new sites: Clearwater, Florida, Trois-Rivières, Canada

sites_addedWild-Type ATTR Cardiac AmyloidosisOct 13thBayerNCT07235462
Added 17 new sites: Multiple Locations, Austria, Multiple Locations, Belgium, Multiple Locations, Croatia and 14 more

Added 17 new sites: Multiple Locations, Austria, Multiple Locations, Belgium, Multiple Locations, Croatia and 14 more

sites_addedSystemic SclerosisOct 13thProlium Bioscience, IncNCT07641634
Added 2 new sites: Henan, China, Seoul, South Korea

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

eligibility_changeSystemic SclerosisOct 13thUniversity of GaziantepNCT07842133
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedSystemic MastocytosisOct 13thBlueprint Medicines CorporationNCT04910685
Added 15 new sites: La Jolla, California, Garran, Australia, Leuven, Belgium and 12 more

Added 15 new sites: La Jolla, California, Garran, Australia, Leuven, Belgium and 12 more

sites_addedSystemic MastocytosisOct 13thBlueprint Medicines CorporationNCT07255638
Added 5 new sites: Berlin, Germany, Freiburg im Breisgau, Germany, Leipzig, Germany and 2 more

Added 5 new sites: Berlin, Germany, Freiburg im Breisgau, Germany, Leipzig, Germany and 2 more

sites_addedStargardt DiseaseOct 13thAscidian Therapeutics, IncNCT06467344
Added 1 new site: Philadelphia, Pennsylvania

Added 1 new site: Philadelphia, Pennsylvania

sites_addedStargardt DiseaseOct 13thAscidian Therapeutics, IncNCT06445322
Added 1 new site: Boston, Massachusetts

Added 1 new site: Boston, Massachusetts

sites_addedSpinal Muscular AtrophyOct 13thargenxNCT07287982
Added 5 new sites: Randwick, Australia, Parkville, Australia, Calgary, Canada and 2 more

Added 5 new sites: Randwick, Australia, Parkville, Australia, Calgary, Canada and 2 more

sites_addedPolycythemia VeraOct 12thOno Pharmaceutical Co., Ltd.NCT07429266
Added 7 new sites: Osaka, Japan, Tokyo, Japan, Tokyo, Japan and 4 more

Added 7 new sites: Osaka, Japan, Tokyo, Japan, Tokyo, Japan and 4 more

sites_addedPolycythemia VeraOct 12thNovartis PharmaceuticalsNCT07357727
Added 14 new sites: Chicago, Illinois, New York, New York, Austin, Texas and 11 more

Added 14 new sites: Chicago, Illinois, New York, New York, Austin, Texas and 11 more

sites_addedPhenylketonuriaOct 12thMaze TherapeuticsNCT07694440
Added 1 new site: Chicago, Illinois

Added 1 new site: Chicago, Illinois

eligibility_changePhenylketonuriaOct 12thMaze TherapeuticsNCT07694440
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1Oct 12thAlkermes, Inc.NCT06767683
Added 1 new site: Dallas, Texas

Added 1 new site: Dallas, Texas

Enrollment UpdateNarcolepsy Type 1Oct 12thAlkermes, Inc.NCT06767683
Target enrollment increased from 256 to 786

Target enrollment increased from 256 to 786

eligibility_changeNarcolepsy Type 1Oct 12thAlkermes, Inc.NCT06767683
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNarcolepsy Type 1Oct 12thAlkermes, Inc.NCT07455383
Added 2 new sites: Ghent, Belgium, Kaohsiung City, Taiwan

Added 2 new sites: Ghent, Belgium, Kaohsiung City, Taiwan

sites_addedNarcolepsy Type 1Oct 12thAlkermes, Inc.NCT07540897
Added 3 new sites: Spokane, Washington, Diest, Belgium, Kumamoto, Japan

Added 3 new sites: Spokane, Washington, Diest, Belgium, Kumamoto, Japan

sites_addedMyotonic DystrophyOct 12thDyne TherapeuticsNCT07486934
Added 2 new sites: Gainesville, Florida, Baltimore, Maryland

Added 2 new sites: Gainesville, Florida, Baltimore, Maryland

sites_addedMyelofibrosisOct 12thNovartis PharmaceuticalsNCT07357727
Added 14 new sites: Chicago, Illinois, New York, New York, Austin, Texas and 11 more

Added 14 new sites: Chicago, Illinois, New York, New York, Austin, Texas and 11 more

eligibility_changeMyelodysplastic SyndromesOct 12thNational Cancer Institute (NCI)NCT06904066
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesOct 12thNational Cancer Institute (NCI)NCT05564390
Added 5 new sites: Ruskin, Florida, Tampa, Florida, Tampa, Florida and 2 more

Added 5 new sites: Ruskin, Florida, Tampa, Florida, Tampa, Florida and 2 more

sites_addedMyelodysplastic SyndromesOct 12thNational Cancer Institute (NCI)NCT06577441
Added 2 new sites: Minneapolis, Minnesota, Sturgeon Bay, Wisconsin

Added 2 new sites: Minneapolis, Minnesota, Sturgeon Bay, Wisconsin

sites_addedMyelodysplastic SyndromesOct 12thTakedaNCT06499285
Added 6 new sites: Monroe, Louisiana, Houston, Texas, Red Oak, Texas and 3 more

Added 6 new sites: Monroe, Louisiana, Houston, Texas, Red Oak, Texas and 3 more

eligibility_changeMyelodysplastic SyndromesOct 12thTakedaNCT06499285
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesOct 12thTakedaNCT07319845
Added 1 new site: Yamagata, Japan

Added 1 new site: Yamagata, Japan

sites_addedMyelodysplastic SyndromesOct 12thTaiho Oncology, Inc.NCT04256317
Added 2 new sites: Maidstone, United Kingdom, Oxford, United Kingdom

Added 2 new sites: Maidstone, United Kingdom, Oxford, United Kingdom

eligibility_changeMyasthenia GravisOct 12thUCB Biopharma SRLNCT07465289
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyasthenia GravisOct 12thAmgenNCT06987539
Added 6 new sites: Seattle, Washington, CABA, Argentina, São Paulo, Brazil and 3 more

Added 6 new sites: Seattle, Washington, CABA, Argentina, São Paulo, Brazil and 3 more

sites_addedMyasthenia GravisOct 12thUCB Biopharma SRLNCT07463521
Added 1 new site: Málaga, Spain

Added 1 new site: Málaga, Spain

eligibility_changeMultiple SclerosisOct 12thUniversity of LiegeNCT07316764
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisOct 12thZenas BioPharma (USA), LLCNCT07299019
Added 3 new sites: Worcester, Massachusetts, Albuquerque, New Mexico, New York, New York

Added 3 new sites: Worcester, Massachusetts, Albuquerque, New Mexico, New York, New York

sites_addedMultiple SclerosisOct 12thImmunic AGNCT05201638
Added 30 new sites: Mostar, Bosnia and Herzegovina, Haryāna, India, Raipur, India and 27 more

Added 30 new sites: Mostar, Bosnia and Herzegovina, Haryāna, India, Raipur, India and 27 more

sites_addedMultiple SclerosisOct 12thZenas BioPharma (USA), LLCNCT07067463
Added 2 new sites: Chieti, Italy, Lviv, Ukraine

Added 2 new sites: Chieti, Italy, Lviv, Ukraine

sites_removedMultiple SclerosisOct 12thNovartis PharmaceuticalsNCT06551519
Reduced from 89 to 82 sites

Reduced from 89 to 82 sites

sites_addedMultiple SclerosisOct 12thHoffmann-La RocheNCT07282574
Added 6 new sites: São Paulo, Brazil, Frankfurt am Main, Germany, Bonn, Germany and 3 more

Added 6 new sites: São Paulo, Brazil, Frankfurt am Main, Germany, Bonn, Germany and 3 more

eligibility_changeMultiple SclerosisOct 12thHoffmann-La RocheNCT07282574
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaOct 11thPerspective TherapeuticsNCT06710756
Added 5 new sites: Los Angeles, California, Los Angeles, California, New York, New York and 2 more

Added 5 new sites: Los Angeles, California, Los Angeles, California, New York, New York and 2 more

Enrollment UpdateMesotheliomaOct 11thGenmabNCT05579366
Target enrollment increased from 884 to 952

Target enrollment increased from 884 to 952

eligibility_changeMesotheliomaOct 11thGenmabNCT05579366
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMembranous NephropathyOct 11thClimb Bio, Inc.NCT07096843
Reduced from 43 to 38 sites

Reduced from 43 to 38 sites

sites_addedMastocytosisOct 11thBlueprint Medicines CorporationNCT07143669
Added 4 new sites: Scottsdale, Arizona, Tucson, Arizona, La Jolla, California and 1 more

Added 4 new sites: Scottsdale, Arizona, Tucson, Arizona, La Jolla, California and 1 more

sites_addedMastocytosisOct 11thBlueprint Medicines CorporationNCT04910685
Added 15 new sites: La Jolla, California, Garran, Australia, Leuven, Belgium and 12 more

Added 15 new sites: La Jolla, California, Garran, Australia, Leuven, Belgium and 12 more

sites_addedMastocytosisOct 11thBlueprint Medicines CorporationNCT07255638
Added 5 new sites: Berlin, Germany, Freiburg im Breisgau, Germany, Leipzig, Germany and 2 more

Added 5 new sites: Berlin, Germany, Freiburg im Breisgau, Germany, Leipzig, Germany and 2 more

sites_addedLennox-Gastaut SyndromeOct 10thEpygenixNCT05066217
Added 10 new sites: Changsha, China, Shanghai, China, Debrecen, Hungary and 7 more

Added 10 new sites: Changsha, China, Shanghai, China, Debrecen, Hungary and 7 more

sites_addedIgA NephropathyOct 10thCentre Hospitalier Universitaire de Saint EtienneNCT06350630
Added 1 new site: Metz, France

Added 1 new site: Metz, France

sites_removedIgA NephropathyOct 10thHoffmann-La RocheNCT05797610
Reduced from 178 to 171 sites

Reduced from 178 to 171 sites

sites_addedIgA NephropathyOct 10thNovartis PharmaceuticalsNCT04557462
Added 8 new sites: Newark, Delaware, Houston, Texas, Roeselare, Belgium and 5 more

Added 8 new sites: Newark, Delaware, Houston, Texas, Roeselare, Belgium and 5 more

Enrollment UpdateIgA NephropathyOct 10thNovartis PharmaceuticalsNCT04557462
Target enrollment increased from 448 to 540

Target enrollment increased from 448 to 540

sites_addedIdiopathic Pulmonary FibrosisOct 10thContineum TherapeuticsNCT07284459
Added 3 new sites: Alexandroupoli, Greece, Heraklion, Greece, Taipei, Taiwan

Added 3 new sites: Alexandroupoli, Greece, Heraklion, Greece, Taipei, Taiwan

sites_removedIdiopathic Pulmonary FibrosisOct 10thUnited TherapeuticsNCT04905693
Reduced from 193 to 190 sites

Reduced from 193 to 190 sites

sites_addedHodgkin LymphomaOct 10thNational Cancer Institute (NCI)NCT05675410
Added 2 new sites: Phoenix, Arizona, Macomb, Michigan

Added 2 new sites: Phoenix, Arizona, Macomb, Michigan

sites_addedHereditary Hemorrhagic TelangiectasiaOct 10thAtavistik Bio, IncNCT07601425
Added 3 new sites: Augusta, Georgia, Indianapolis, Indiana, Salt Lake City, Utah

Added 3 new sites: Augusta, Georgia, Indianapolis, Indiana, Salt Lake City, Utah

sites_addedHemophilia BOct 10thCSL BehringNCT07080905
Added 2 new sites: Westmead, Australia, Lille, France

Added 2 new sites: Westmead, Australia, Lille, France

eligibility_changeHemophilia BOct 10thCSL BehringNCT07080905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BOct 10thRegeneron PharmaceuticalsNCT06379789
Added 1 new site: Phoenix, Arizona

Added 1 new site: Phoenix, Arizona

sites_addedHemophilia AOct 10thHoffmann-La RocheNCT07416604
Added 17 new sites: Miami, Florida, Ann Arbor, Michigan, Minneapolis, Minnesota and 14 more

Added 17 new sites: Miami, Florida, Ann Arbor, Michigan, Minneapolis, Minnesota and 14 more

sites_addedFriedreich AtaxiaOct 9thLarimar Therapeutics, Inc.NCT07778836
Added 1 new site: Tampa, Florida

Added 1 new site: Tampa, Florida

sites_addedFriedreich AtaxiaOct 9thLarimar Therapeutics, Inc.NCT06447025
Added 6 new sites: San Francisco, California, Iowa City, Iowa, Columbus, Ohio and 3 more

Added 6 new sites: San Francisco, California, Iowa City, Iowa, Columbus, Ohio and 3 more

sites_addedFamilial Hypertrophic CardiomyopathyOct 9thMontreal Heart InstituteNCT05100420
Added 4 new sites: Winnipeg, Canada, Kingston, Canada, Montreal, Canada and 1 more

Added 4 new sites: Winnipeg, Canada, Kingston, Canada, Montreal, Canada and 1 more

sites_addedEssential ThrombocythemiaOct 9thNovartis PharmaceuticalsNCT07357727
Added 6 new sites: Chicago, Illinois, New York, New York, Montreal, Canada and 3 more

Added 6 new sites: Chicago, Illinois, New York, New York, Montreal, Canada and 3 more

sites_addedGraves’ DiseaseOct 9thBiohaven Therapeutics Ltd.NCT07661056
Added 1 new site: Washington D.C., District of Columbia

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

sites_addedGraves’ DiseaseOct 9thBiogenNCT07722546
Added 3 new sites: Lutherville, Maryland, Hamilton, New Jersey, Richmond, Virginia

Added 3 new sites: Lutherville, Maryland, Hamilton, New Jersey, Richmond, Virginia

sites_addedGraves’ DiseaseOct 9thUniversity Children's Hospital BaselNCT06864351
Added 5 new sites: Yerevan, Armenia, Lausanne, Switzerland, Lucerne, Switzerland and 2 more

Added 5 new sites: Yerevan, Armenia, Lausanne, Switzerland, Lucerne, Switzerland and 2 more

sites_addedGeographic AtrophyOct 9thComplement TherapeuticsNCT07392255
Added 2 new sites: Gloucester, United Kingdom, Sunderland, United Kingdom

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

sites_addedGeographic AtrophyOct 9thComplement TherapeuticsNCT07144137
Added 1 new site: Springfield, Illinois

Added 1 new site: Springfield, Illinois

sites_addedFocal EpilepsyOct 9thRapport Therapeutics Inc.NCT07563881
Added 1 new site: New York, New York

Added 1 new site: New York, New York

sites_addedFocal EpilepsyOct 9thXenon Pharmaceuticals Inc.NCT05716100
Added 3 new sites: Itami, Japan, Hamamatsu, Japan, Kagoshima, Japan

Added 3 new sites: Itami, Japan, Hamamatsu, Japan, Kagoshima, Japan

sites_addedFamilial Chylomicronemia SyndromeOct 9thIonis Pharmaceuticals, Inc.NCT07727538
Added 1 new site: Tacoma, Washington

Added 1 new site: Tacoma, Washington

sites_addedEssential ThrombocythemiaOct 9thIncyte CorporationNCT07623200
Added 5 new sites: Birmingham, Alabama, Baton Rouge, Louisiana, Dallas, Texas and 2 more

Added 5 new sites: Birmingham, Alabama, Baton Rouge, Louisiana, Dallas, Texas and 2 more

sites_addedEssential ThrombocythemiaOct 9thNovartis PharmaceuticalsNCT07357727
Added 9 new sites: Austin, Texas, Graz, Austria, Lens, France and 6 more

Added 9 new sites: Austin, Texas, Graz, Austria, Lens, France and 6 more

sites_removedEssential ThrombocythemiaOct 9thMerck Sharp & Dohme LLCNCT06079879
Reduced from 163 to 161 sites

Reduced from 163 to 161 sites

phase_changeDuchenne Muscular DystrophyOct 8thUniversity of FloridaNCT07766980
Moved from N/A to Phase 2

Moved from N/A to Phase 2

eligibility_changeCystic FibrosisOct 8thGhady HaidarNCT07275905
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedChronic Graft-versus-Host DiseaseOct 8thIncyte CorporationNCT05919511
Added 5 new sites: Denver, Colorado, Detroit, Michigan, Nashville, Tennessee and 2 more

Added 5 new sites: Denver, Colorado, Detroit, Michigan, Nashville, Tennessee and 2 more

sites_addedChronic Graft-versus-Host DiseaseOct 8thSanofiNCT07116031
Added 5 new sites: Atlanta, Georgia, Boston, Massachusetts, Toronto, Canada and 2 more

Added 5 new sites: Atlanta, Georgia, Boston, Massachusetts, Toronto, Canada and 2 more

sites_addedChronic Graft-versus-Host DiseaseOct 8thIncyte CorporationNCT06585774
Added 2 new sites: Pittsburgh, Pennsylvania, Salamanca, Spain

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

Enrollment UpdateChronic Graft-versus-Host DiseaseOct 8thJanssen Research & Development, LLCNCT01804686
Target enrollment decreased from 700 to 539

Target enrollment decreased from 700 to 539

eligibility_changeChronic Graft-versus-Host DiseaseOct 8thIncyte CorporationNCT07124078
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeCystic FibrosisOct 8thIndiana UniversityNCT06512064
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCommon Variable ImmunodeficiencyOct 8thMemorial Sloan Kettering Cancer CenterNCT06976476
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

eligibility_changeChronic Granulomatous DiseaseOct 8thNational Institute of Allergy and Infectious Diseases (NIAID)NCT05463133
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeChronic Graft-versus-Host DiseaseOct 8thNational Cancer Institute (NCI)NCT05531786
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedCholangiocarcinomaOct 8thMehmet AkceNCT06728410
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

phase_changeCholangiocarcinomaOct 8thMinia UniversityNCT07155525
Moved from Phase 3 to N/A

Moved from Phase 3 to N/A

Enrollment UpdateCholangiocarcinomaOct 8thPheast TherapeuticsNCT06840886
Target enrollment increased from 272 to 426

Target enrollment increased from 272 to 426

eligibility_changeCholangiocarcinomaOct 8thPheast TherapeuticsNCT06840886
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedC3 GlomerulopathyOct 8thAkebia TherapeuticsNCT06419205
Added 2 new sites: Hollywood, Florida, Orangeburg, South Carolina

Added 2 new sites: Hollywood, Florida, Orangeburg, South Carolina

sites_addedATTR Amyloidosis (Transthyretin Amyloidosis)Oct 7thNovo Nordisk A/SNCT06260709
Added 5 new sites: Toulouse, France, Hiroshima, Japan, Utrecht, Netherlands and 2 more

Added 5 new sites: Toulouse, France, Hiroshima, Japan, Utrecht, Netherlands and 2 more

eligibility_changeAplastic AnemiaOct 7thNational Heart, Lung, and Blood Institute (NHLBI)NCT02979873
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeAlport SyndromeOct 7thEloxx Pharmaceuticals, Inc.NCT07523581
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAlpha-1 Antitrypsin DeficiencyOct 7thBeam Therapeutics Inc.NCT06389877
Added 1 new site: Birmingham, United Kingdom

Added 1 new site: Birmingham, United Kingdom

Enrollment UpdateAlpha-1 Antitrypsin DeficiencyOct 7thBeam Therapeutics Inc.NCT06389877
Target enrollment increased from 106 to 192

Target enrollment increased from 106 to 192

eligibility_changeAlpha-1 Antitrypsin DeficiencyOct 7thBeam Therapeutics Inc.NCT06389877
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedAlpha-1 Antitrypsin DeficiencyOct 7thAIRNA CorporationNCT07431112
Added 4 new sites: Tübingen, Germany, Leiden, Netherlands, Auckland, New Zealand and 1 more

Added 4 new sites: Tübingen, Germany, Leiden, Netherlands, Auckland, New Zealand and 1 more

sites_addedWilson DiseaseOct 6thPrime Medicine, Inc.NCT07748403
Added 1 new site: New Haven, Connecticut

Added 1 new site: New Haven, Connecticut

eligibility_changeWaldenström MacroglobulinemiaOct 6thBeOne MedicinesNCT05006716
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedTurner SyndromeOct 6thAscendis Pharma A/SNCT07221851
Added 7 new sites: Washington D.C., District of Columbia, Cincinnati, Ohio, Salt Lake City, Utah and 4 more

Added 7 new sites: Washington D.C., District of Columbia, Cincinnati, Ohio, Salt Lake City, Utah and 4 more

sites_addedSickle Cell DiseaseOct 5thDisc Medicine, IncNCT07187973
Added 1 new site: San Francisco, California

Added 1 new site: San Francisco, California

Enrollment UpdateProgressive Supranuclear PalsyOct 5thVanderbilt University Medical CenterNCT06588673
Target enrollment increased from 10 to 19

Target enrollment increased from 10 to 19

sites_addedThalassemiaOct 5thAgios Pharmaceuticals, Inc.NCT07506863
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

sites_addedThalassemiaOct 5thAgios Pharmaceuticals, Inc.NCT07517133
Added 1 new site: Atlanta, Georgia

Added 1 new site: Atlanta, Georgia

sites_addedSystemic SclerosisOct 5thKite, A Gilead CompanyNCT07038447
Added 2 new sites: Seoul, South Korea, Seoul, South Korea

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

eligibility_changeSystemic SclerosisOct 5thKite, A Gilead CompanyNCT07038447
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedStargardt DiseaseOct 5thAAVantgarde Bio SrlNCT07161544
Added 2 new sites: Los Angeles, California, Oslo, Norway

Added 2 new sites: Los Angeles, California, Oslo, Norway

sites_addedSpinal Muscular AtrophyOct 5thBiogenNCT07444476
Added 2 new sites: Palo Alto, California, Beijing, China

Added 2 new sites: Palo Alto, California, Beijing, China

sites_addedSjögren's SyndromeOct 5thNovartis PharmaceuticalsNCT07621809
Added 7 new sites: Boston, Massachusetts, Potsdam, New York, Sherbrooke, Canada and 4 more

Added 7 new sites: Boston, Massachusetts, Potsdam, New York, Sherbrooke, Canada and 4 more

sites_addedSickle Cell DiseaseOct 5thTheraviaNCT06578507
Added 1 new site: Saint-Denis, France

Added 1 new site: Saint-Denis, France

sites_addedSickle Cell DiseaseOct 5thNovartis PharmaceuticalsNCT06439082
Added 1 new site: Eldoret, Kenya

Added 1 new site: Eldoret, Kenya

sites_addedShort Bowel SyndromeOct 5thZealand PharmaNCT07197944
Added 2 new sites: Pittsburgh, Pennsylvania, Skawina, Poland

Added 2 new sites: Pittsburgh, Pennsylvania, Skawina, Poland

Enrollment UpdateSarcoidosisOct 5thXentria, Inc.NCT05890729
Target enrollment decreased from 94 to 32

Target enrollment decreased from 94 to 32

eligibility_changeSarcoidosisOct 5thXentria, Inc.NCT05890729
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedRett SyndromeOct 5thUCB BIOSCIENCES, Inc.NCT07503444
Added 9 new sites: Philadelphia, Pennsylvania, Seattle, Washington, Bron, France and 6 more

Added 9 new sites: Philadelphia, Pennsylvania, Seattle, Washington, Bron, France and 6 more

sites_addedProgressive Familial Intrahepatic CholestasisOct 4thIpsenNCT07185919
Added 1 new site: Seoul, South Korea

Added 1 new site: Seoul, South Korea

sites_addedPrimary Sclerosing CholangitisOct 4thIpsenNCT07387549
Added 218 new sites: Phoenix, Arizona, La Jolla, California, Los Angeles, California and 215 more

Added 218 new sites: Phoenix, Arizona, La Jolla, California, Los Angeles, California and 215 more

sites_removedPrimary Biliary CholangitisOct 4thIpsenNCT06447168
Reduced from 74 to 72 sites

Reduced from 74 to 72 sites

sites_addedPrimary Biliary CholangitisOct 4thIpsenNCT06016842
Added 12 new sites: San Antonio, Texas, Barranquilla, Colombia, Cali, Colombia and 9 more

Added 12 new sites: San Antonio, Texas, Barranquilla, Colombia, Cali, Colombia and 9 more

sites_addedPolycythemia VeraOct 4thPrelude Therapeutics IncorporatedNCT07469891
Added 9 new sites: Los Angeles, California, Adelaide, Australia, Clayton, Australia and 6 more

Added 9 new sites: Los Angeles, California, Adelaide, Australia, Clayton, Australia and 6 more

sites_addedPolycythemia VeraOct 4thNovartis PharmaceuticalsNCT07357727
Added 15 new sites: Stuart, Florida, Bethesda, Maryland, Tyler, Texas and 12 more

Added 15 new sites: Stuart, Florida, Bethesda, Maryland, Tyler, Texas and 12 more

eligibility_changePheochromocytomaOct 4thNational Cancer Institute (NCI)NCT06479811
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPheochromocytomaOct 4thCancer Research UKNCT05944237
Added 1 new site: Dundee, United Kingdom

Added 1 new site: Dundee, United Kingdom

eligibility_changePheochromocytomaOct 4thCancer Research UKNCT05944237
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedPhenylketonuriaOct 4thAgios Pharmaceuticals, Inc.NCT07241234
Added 1 new site: Gdansk, Poland

Added 1 new site: Gdansk, Poland

sites_addedPhelan-McDermid SyndromeOct 4thNeuren Pharmaceuticals LimitedNCT07281079
Added 1 new site: Columbus, Ohio

Added 1 new site: Columbus, Ohio

sites_addedParoxysmal Nocturnal HemoglobinuriaOct 4thRegeneron PharmaceuticalsNCT05744921
Added 1 new site: São Paulo, Brazil

Added 1 new site: São Paulo, Brazil

eligibility_changeParagangliomaOct 4thNational Cancer Institute (NCI)NCT06479811
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNoonan SyndromeOct 4thAscendis Pharma A/SNCT07221851
Added 7 new sites: Washington D.C., District of Columbia, Cincinnati, Ohio, Salt Lake City, Utah and 4 more

Added 7 new sites: Washington D.C., District of Columbia, Cincinnati, Ohio, Salt Lake City, Utah and 4 more

sites_addedNeuromyelitis OpticaOct 4thHoffmann-La RocheNCT05199688
Added 2 new sites: Mexico City, Mexico, Gdansk, Poland

Added 2 new sites: Mexico City, Mexico, Gdansk, Poland

eligibility_changeNeuromyelitis OpticaOct 4thHoffmann-La RocheNCT05199688
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedNeurofibromatosis Type 1Oct 4thGirish Dhall, MDNCT06502171
Added 2 new sites: Los Angeles, California, Indianapolis, Indiana

Added 2 new sites: Los Angeles, California, Indianapolis, Indiana

sites_addedNarcolepsy Type 1Oct 4thCentessa Pharmaceuticals (UK) LimitedNCT07598708
Added 3 new sites: St. Petersburg, Florida, Rincon, Georgia, Houston, Texas

Added 3 new sites: St. Petersburg, Florida, Rincon, Georgia, Houston, Texas

sites_addedMyotonic DystrophyOct 3rdDyne TherapeuticsNCT07486934
Added 1 new site: Houston, Texas

Added 1 new site: Houston, Texas

sites_addedMyotonic DystrophyOct 3rdAvidity Biosciences, Inc.NCT07587242
Added 2 new sites: Milan, Italy, Leiden, Netherlands

Added 2 new sites: Milan, Italy, Leiden, Netherlands

Enrollment UpdateMyelofibrosisOct 3rdMarina KremyanskayaNCT05025488
Target enrollment decreased from 10 to 7

Target enrollment decreased from 10 to 7

sites_addedMyelofibrosisOct 3rdPrelude Therapeutics IncorporatedNCT07469891
Added 9 new sites: Los Angeles, California, Adelaide, Australia, Clayton, Australia and 6 more

Added 9 new sites: Los Angeles, California, Adelaide, Australia, Clayton, Australia and 6 more

sites_addedMyelofibrosisOct 3rdNovartis PharmaceuticalsNCT07357727
Added 15 new sites: Stuart, Florida, Bethesda, Maryland, Tyler, Texas and 12 more

Added 15 new sites: Stuart, Florida, Bethesda, Maryland, Tyler, Texas and 12 more

eligibility_changeMyelodysplastic SyndromesOct 3rdKite, A Gilead CompanyNCT05457010
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMyelodysplastic SyndromesOct 3rdNational Cancer Institute (NCI)NCT06484062
Added 1 new site: Baltimore, Maryland

Added 1 new site: Baltimore, Maryland

sites_addedMyelodysplastic SyndromesOct 3rdGroupe Francophone des MyelodysplasiesNCT05181735
Added 1 new site: Compiègne, France

Added 1 new site: Compiègne, France

sites_addedMyelodysplastic SyndromesOct 3rdNational Cancer Institute (NCI)NCT06577441
Added 1 new site: Saint Paul, Minnesota

Added 1 new site: Saint Paul, Minnesota

eligibility_changeMyelodysplastic SyndromesOct 3rdM.D. Anderson Cancer CenterNCT03661307
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeMyelodysplastic SyndromesOct 3rdAmphista Therapeutics LtdNCT07723703
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_removedMyasthenia GravisOct 3rdNovartis PharmaceuticalsNCT06744920
Reduced from 130 to 120 sites

Reduced from 130 to 120 sites

sites_addedMultiple System AtrophyOct 3rdTeva Branded Pharmaceutical Products R&D LLCNCT07197866
Added 5 new sites: Chicago, Illinois, Durham, North Carolina, Padova, Italy and 2 more

Added 5 new sites: Chicago, Illinois, Durham, North Carolina, Padova, Italy and 2 more

sites_addedMultiple SclerosisOct 3rdImmunic AGNCT05134441
Added 15 new sites: Tbilisi, Georgia, Shimla, India, Mexico City, Mexico and 12 more

Added 15 new sites: Tbilisi, Georgia, Shimla, India, Mexico City, Mexico and 12 more

sites_addedMultiple SclerosisOct 3rdCelgeneNCT06782490
Added 4 new sites: Lewisville, Texas, Zlín, Czechia, Siegen, Germany and 1 more

Added 4 new sites: Lewisville, Texas, Zlín, Czechia, Siegen, Germany and 1 more

eligibility_changeMultiple SclerosisOct 3rdIcahn School of Medicine at Mount SinaiNCT06992115
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMultiple SclerosisOct 3rdZenas BioPharma (USA), LLCNCT07299019
Added 3 new sites: Tbilisi, Georgia, Esztergom, Hungary, Niš, Serbia

Added 3 new sites: Tbilisi, Georgia, Esztergom, Hungary, Niš, Serbia

sites_addedMultiple SclerosisOct 3rdGenentech, Inc.NCT07609719
Added 2 new sites: Scottsdale, Arizona, Maitland, Florida

Added 2 new sites: Scottsdale, Arizona, Maitland, Florida

sites_addedSanfilippo SyndromeOct 3rdJCR Pharmaceuticals Co., Ltd.NCT06488924
Added 1 new site: Tsu, Japan

Added 1 new site: Tsu, Japan

Enrollment UpdateMesotheliomaOct 3rdNational Cancer Institute (NCI)NCT06885697
Target enrollment increased from 100 to 120

Target enrollment increased from 100 to 120

eligibility_changeMesotheliomaOct 3rdNational Cancer Institute (NCI)NCT06885697
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaOct 3rdCancer Research UKNCT05944237
Added 1 new site: Dundee, United Kingdom

Added 1 new site: Dundee, United Kingdom

eligibility_changeMesotheliomaOct 3rdCancer Research UKNCT05944237
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedMesotheliomaOct 3rdIDEAYA BiosciencesNCT07277413
Added 2 new sites: Duarte, California, Zion, Illinois

Added 2 new sites: Duarte, California, Zion, Illinois

sites_addedMembranous NephropathyOct 3rdBiogenNCT06962800
Added 2 new sites: Ningbo, China, Suita-Shi, Japan

Added 2 new sites: Ningbo, China, Suita-Shi, Japan

sites_addedMarfan SyndromeOct 3rdHospices Civils de LyonNCT04194619
Added 1 new site: Bordeaux, France

Added 1 new site: Bordeaux, France

sites_addedLennox-Gastaut SyndromeOct 2ndEpygenixNCT05066217
Added 3 new sites: Hyderabad, India, Hyderabad, India, Surat, India

Added 3 new sites: Hyderabad, India, Hyderabad, India, Surat, India

sites_addedImmune ThrombocytopeniaOct 2ndNovartis PharmaceuticalsNCT07421167
Added 1 new site: Kansas City, Missouri

Added 1 new site: Kansas City, Missouri

sites_addedIgG4-Related DiseaseOct 2ndAcepodia Biotech, Inc.NCT07061938
Added 8 new sites: Beijing, China, Beijing, China, Chengdu, China and 5 more

Added 8 new sites: Beijing, China, Beijing, China, Chengdu, China and 5 more

eligibility_changeIgG4-Related DiseaseOct 2ndAcepodia Biotech, Inc.NCT07061938
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedIdiopathic Pulmonary FibrosisOct 2ndContineum TherapeuticsNCT07284459
Added 2 new sites: Chaïdári, Greece, Ioannina, Greece

Added 2 new sites: Chaïdári, Greece, Ioannina, Greece

sites_addedIdiopathic Pulmonary FibrosisOct 2ndAvalyn Pharma Inc.NCT06951217
Added 14 new sites: Newport Beach, California, New Haven, Connecticut, New York, New York and 11 more

Added 14 new sites: Newport Beach, California, New Haven, Connecticut, New York, New York and 11 more

sites_addedHunter SyndromeOct 2ndJCR Pharmaceuticals Co., Ltd.NCT04573023
Added 3 new sites: New York, New York, Afula, Israel, Ramat Gan, Israel

Added 3 new sites: New York, New York, Afula, Israel, Ramat Gan, Israel

sites_addedHodgkin LymphomaOct 2ndECOG-ACRIN Cancer Research GroupNCT06002828
Added 111 new sites: Lewes, Delaware, Millville, Delaware, Newark, Delaware and 108 more

Added 111 new sites: Lewes, Delaware, Millville, Delaware, Newark, Delaware and 108 more

eligibility_changeHodgkin LymphomaOct 2ndECOG-ACRIN Cancer Research GroupNCT06002828
Eligibility criteria have been updated

Eligibility criteria have been updated

eligibility_changeHodgkin LymphomaOct 2ndUniversity of WashingtonNCT06745076
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia BOct 2ndPfizerNCT05568719
Added 16 new sites: Indianapolis, Indiana, Melbourne, Australia, Bron, France and 13 more

Added 16 new sites: Indianapolis, Indiana, Melbourne, Australia, Bron, France and 13 more

sites_addedHemophilia AOct 2ndPfizerNCT05568719
Added 16 new sites: Indianapolis, Indiana, Melbourne, Australia, Bron, France and 13 more

Added 16 new sites: Indianapolis, Indiana, Melbourne, Australia, Bron, France and 13 more

sites_addedImmune ThrombocytopeniaOct 2ndNovartis PharmaceuticalsNCT07421167
Added 2 new sites: New Haven, Connecticut, East Brunswick, New Jersey

Added 2 new sites: New Haven, Connecticut, East Brunswick, New Jersey

sites_addedIgA NephropathyOct 2ndAkebia TherapeuticsNCT06419205
Added 2 new sites: Hollywood, Florida, Orangeburg, South Carolina

Added 2 new sites: Hollywood, Florida, Orangeburg, South Carolina

sites_addedIgA NephropathyOct 2ndJade Biosciences, Inc.NCT07541287
Added 6 new sites: Liverpool, Australia, Parkville, Australia, Taichung, Taiwan and 3 more

Added 6 new sites: Liverpool, Australia, Parkville, Australia, Taichung, Taiwan and 3 more

Enrollment UpdateIgA NephropathyOct 2ndVera Therapeutics, Inc.NCT04716231
Target enrollment increased from 376 to 428

Target enrollment increased from 376 to 428

sites_addedIgA NephropathyOct 2ndBiogenNCT06935357
Added 3 new sites: Milwaukee, Wisconsin, Stuttgart, Germany, Braunschweig, Germany

Added 3 new sites: Milwaukee, Wisconsin, Stuttgart, Germany, Braunschweig, Germany

sites_addedIdiopathic Pulmonary FibrosisOct 2ndContineum TherapeuticsNCT07284459
Added 11 new sites: Exochi, Greece, Lima, Peru, Surco, Peru and 8 more

Added 11 new sites: Exochi, Greece, Lima, Peru, Surco, Peru and 8 more

sites_addedHypoparathyroidismOct 2ndAscendis Pharma Bone Diseases A/SNCT07081997
Added 1 new site: Greeley, Colorado

Added 1 new site: Greeley, Colorado

eligibility_changeHodgkin LymphomaOct 2ndMayo ClinicNCT03418038
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedHemophilia AOct 2ndSwedish Orphan BiovitrumNCT06940830
Added 2 new sites: Salerno, Italy, Salamanca, Spain

Added 2 new sites: Salerno, Italy, Salamanca, Spain

Enrollment UpdateHemophilia AOct 2ndSanofiNCT06684314
Target enrollment decreased from 100 to 79

Target enrollment decreased from 100 to 79

sites_addedGraves’ DiseaseOct 1stSanofiNCT06984627
Added 10 new sites: Buenos Aires, Argentina, Buenos Aires, Argentina, Córdoba, Argentina and 7 more

Added 10 new sites: Buenos Aires, Argentina, Buenos Aires, Argentina, Córdoba, Argentina and 7 more

sites_addedGraves’ DiseaseOct 1stBiohaven Therapeutics Ltd.NCT07661056
Added 2 new sites: South Gate, California, Winnipeg, Canada

Added 2 new sites: South Gate, California, Winnipeg, Canada

sites_addedFocal Segmental GlomerulosclerosisOct 1stAkebia TherapeuticsNCT07268638
Added 5 new sites: Garden Grove, California, Augusta, Georgia, Valhalla, New York and 2 more

Added 5 new sites: Garden Grove, California, Augusta, Georgia, Valhalla, New York and 2 more

eligibility_changeFocal Segmental GlomerulosclerosisOct 1stAkebia TherapeuticsNCT07268638
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFocal EpilepsyOct 1stSupernus Pharmaceuticals, Inc.NCT06798896
Added 62 new sites: Phoenix, Arizona, Phoenix, Arizona, Tucson, Arizona and 59 more

Added 62 new sites: Phoenix, Arizona, Phoenix, Arizona, Tucson, Arizona and 59 more

sites_addedFocal EpilepsyOct 1stRapport Therapeutics Inc.NCT07563881
Added 2 new sites: Livingston, New Jersey, Busan, South Korea

Added 2 new sites: Livingston, New Jersey, Busan, South Korea

sites_addedEssential ThrombocythemiaOct 1stPrelude Therapeutics IncorporatedNCT07469891
Added 9 new sites: Los Angeles, California, Adelaide, Australia, Clayton, Australia and 6 more

Added 9 new sites: Los Angeles, California, Adelaide, Australia, Clayton, Australia and 6 more

sites_addedEssential ThrombocythemiaOct 1stNovartis PharmaceuticalsNCT07357727
Added 7 new sites: Bethesda, Maryland, Nîmes, France, Mannheim, Germany and 4 more

Added 7 new sites: Bethesda, Maryland, Nîmes, France, Mannheim, Germany and 4 more

sites_addedGraves’ DiseaseOct 1stMerida BiosciencesNCT07305818
Added 1 new site: Santander, Spain

Added 1 new site: Santander, Spain

sites_addedGraves’ DiseaseOct 1stImmunovant Sciences GmbHNCT07286006
Added 26 new sites: Perth, Australia, St Leonards, Australia, Sydney, Australia and 23 more

Added 26 new sites: Perth, Australia, St Leonards, Australia, Sydney, Australia and 23 more

sites_removedGeographic AtrophyOct 1stAstellas Pharma IncNCT06970665
Reduced from 16 to 14 sites

Reduced from 16 to 14 sites

eligibility_changeFriedreich AtaxiaOct 1stWeill Medical College of Cornell UniversityNCT05302271
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFragile X SyndromeOct 1stMirum Pharmaceuticals, Inc.NCT07209462
Added 2 new sites: Barcelona, Spain, Málaga, Spain

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

sites_addedFocal Segmental GlomerulosclerosisOct 1stBoehringer IngelheimNCT07220083
Added 7 new sites: Exton, Pennsylvania, Westmead, Australia, AT Albans, Australia and 4 more

Added 7 new sites: Exton, Pennsylvania, Westmead, Australia, AT Albans, Australia and 4 more

sites_removedFocal EpilepsyOct 1stBiohaven Therapeutics Ltd.NCT06132893
Reduced from 124 to 104 sites

Reduced from 124 to 104 sites

sites_addedFocal EpilepsyOct 1stRapport Therapeutics Inc.NCT07563881
Added 2 new sites: The Bronx, New York, Seoul, South Korea

Added 2 new sites: The Bronx, New York, Seoul, South Korea

Enrollment UpdateFocal EpilepsyOct 1stInstitut National de la Santé Et de la Recherche Médicale, FranceNCT04653012
Target enrollment increased from 50 to 80

Target enrollment increased from 50 to 80

phase_changeFocal EpilepsyOct 1stMayo ClinicNCT07012148
Moved from N/A to Early Phase 1

Moved from N/A to Early Phase 1

sites_addedFocal EpilepsyOct 1stMayo ClinicNCT07012148
Added 2 new sites: Scottsdale, Arizona, Jacksonville, Florida

Added 2 new sites: Scottsdale, Arizona, Jacksonville, Florida

eligibility_changeFocal EpilepsyOct 1stMayo ClinicNCT07012148
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedFabry DiseaseOct 1stUniQure Biopharma B.V.NCT06270316
Added 3 new sites: Birmingham, Alabama, Los Angeles, California, Cincinnati, Ohio

Added 3 new sites: Birmingham, Alabama, Los Angeles, California, Cincinnati, Ohio

Enrollment UpdateFabry DiseaseOct 1stUniQure Biopharma B.V.NCT06270316
Target enrollment increased from 12 to 20

Target enrollment increased from 12 to 20

eligibility_changeFabry DiseaseOct 1stUniQure Biopharma B.V.NCT06270316
Eligibility criteria have been updated

Eligibility criteria have been updated

sites_addedEssential ThrombocythemiaOct 1stNovartis PharmaceuticalsNCT07357727
Added 15 new sites: Fort Myers, Florida, Stuart, Florida, Dallas, Texas and 12 more

Added 15 new sites: Fort Myers, Florida, Stuart, Florida, Dallas, Texas and 12 more

Enrollment UpdateEssential ThrombocythemiaOct 1stMarina KremyanskayaNCT05025488
Target enrollment decreased from 10 to 7

Target enrollment decreased from 10 to 7

sites_addedEpidermolysis BullosaOct 1stChiesi Farmaceutici S.p.A.NCT06423573
Added 7 new sites: Palo Alto, California, Aurora, Colorado, Gainesville, Florida and 4 more

Added 7 new sites: Palo Alto, California, Aurora, Colorado, Gainesville, Florida and 4 more

Enrollment UpdateEpidermolysis BullosaOct 1stChiesi Farmaceutici S.p.A.NCT06423573
Target enrollment decreased from 580 to 170

Target enrollment decreased from 580 to 170