Fabry Disease: Clinical Trials

19 trials for Fabry Disease, newest first

ClinicalTrials Daily  ·  Areas  ·  Conditions  ·  Drugs  ·  Sponsors  ·  

19 completed. 19 Phase 3.

By phase and status

PhaseTrialsStatus
Phase 319Completed 19

Follow Fabry Disease: new trials by email Literature Review of Fabry Disease trials Research Report on Fabry Disease

Trials (filtered) newest first · largest first

All 19 trials

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta) - Fabry Disease
Phase 3CompletedBio Sidus SA202022-12-132026-02-18
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
REPLAGAL - Fabry Disease
Phase 3CompletedTakeda202022-05-012024-10-08
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
migalastat HCl 150 mg - Fabry Disease
Phase 3CompletedAmicus Therapeutics162019-10-112026-02-05
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Lucerastat - Fabry Disease
Phase 3CompletedIdorsia Pharmaceuticals Ltd.1072018-12-182026-11-02
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
pegunigalsidase alfa - Fabry Disease
Phase 3CompletedChiesi Farmaceutici S.p.A.292018-11-132026-05-22
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Migalastat HCl 150 mg - Fabry Disease
Phase 3CompletedAmicus Therapeutics222018-09-272021-11-30
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
pegunigalsidase alfa - Fabry Disease
Phase 3CompletedChiesi Farmaceutici S.p.A.972018-09-162026-03-24
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Lucerastat, Placebo - Fabry Disease
Phase 3CompletedIdorsia Pharmaceuticals Ltd.1182018-06-212024-08-09
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
Pegunigalsidase alfa - Fabry Disease
Phase 3CompletedProtalix302017-07-102023-09-13
Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
PRX-102 (pegunigalsidase alfa) - Fabry Disease
Phase 3CompletedProtalix222017-05-172023-09-13
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
PRX-102 (pegunigalsidase alfa), agalsidase beta - Fabry Disease
Phase 3CompletedProtalix782016-06-012023-09-13
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
migalastat HCl 150 mg - Fabry Disease
Phase 3CompletedAmicus Therapeutics842015-03-142020-12-21
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
migalastat hydrochloride, agalsidase - Fabry Disease
Phase 3CompletedAmicus Therapeutics682011-09-082018-11-01
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
agalsidase alfa - Fabry Disease
Phase 3CompletedShire1712011-08-102021-06-08
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
Replagal - Fabry Disease
Phase 3CompletedShire352010-04-132021-06-08
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
migalastat hydrochloride, Placebo - Fabry Disease
Phase 3CompletedAmicus Therapeutics672009-10-232018-10-30
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
Replagal - Fabry Disease
Phase 3CompletedShire442008-12-292021-06-09
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
Agalsidase beta - Fabry Disease
Phase 3CompletedGenzyme, a Sanofi Company312008-09-012016-06-29
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Fabrazyme (agalsidase beta) - Fabry Disease
Phase 3CompletedGenzyme, a Sanofi Company581999-10-012013-12-04

Narrower conditions

Proteinuria (3)  ·  Lysosomal Storage Diseases (1)

Broader conditions

Brain Diseases (19)  ·  Brain Diseases, Metabolic (19)  ·  Brain Diseases, Metabolic, Inborn (19)  ·  Cardiovascular Diseases (19)  ·  Central Nervous System Diseases (19)  ·  Cerebral Small Vessel Diseases (19)  ·  Cerebrovascular Disorders (19)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (19)  ·  Genetic Diseases, Inborn (19)  ·  Genetic Diseases, X-Linked (19)  ·  Lipid Metabolism Disorders (19)  ·  Lipid Metabolism, Inborn Errors (19)  ·  Lipidoses (19)  ·  Lysosomal Storage Diseases, Nervous System (19)  ·  Metabolic Diseases (19)

Interventions

migalastat (7)  ·  agalsidase alfa (5)  ·  agalsidase beta (5)  ·  larazotide acetate (5)  ·  alpha-Galactosidase (1)

Sponsors

Amicus Therapeutics (5)  ·  Protalix (3)  ·  Shire (3)  ·  Chiesi Farmaceutici S.p.A. (2)  ·  Genzyme, a Sanofi Company (2)  ·  Idorsia Pharmaceuticals Ltd. (2)  ·  Bio Sidus SA (1)  ·  Takeda (1)

Countries

United Kingdom (16)  ·  United States (15)  ·  Australia (9)  ·  Canada (8)  ·  Czechia (8)  ·  Italy (8)  ·  Norway (8)  ·  France (7)  ·  Netherlands (7)  ·  Belgium (6)  ·  Poland (6)  ·  Spain (6)  ·  Denmark (5)  ·  Slovenia (5)  ·  Argentina (4)

Results publications

PMID 39847314, PMID 27657681, PMID 31889231, PMID 32994552, PMID 20730727, PMID 23531228, PMID 25795794, PMID 25955246, PMID 26185417, PMID 27509102, PMID 27834756, PMID 29703262, PMID 30723321, PMID 31934472, PMID 33012654, PMID 34542871, PMID 35782623, PMID 37865771, PMID 37940383, PMID 41862950

NIH funding

2009 (2), 2014 (1), 2017 (1), 2018 (1), 2019 (1)