19 completed. 19 Phase 3.
By phase and status
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.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
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 3 | Completed | 20 | 2022-12-13 | 2026-02-18 |
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease REPLAGAL - Fabry Disease | Phase 3 | Completed | 20 | 2022-05-01 | 2024-10-08 |
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | 16 | 2019-10-11 | 2026-02-05 |
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease Lucerastat - Fabry Disease | Phase 3 | Completed | 107 | 2018-12-18 | 2026-11-02 |
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients pegunigalsidase alfa - Fabry Disease | Phase 3 | Completed | 29 | 2018-11-13 | 2026-05-22 |
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) Migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | 22 | 2018-09-27 | 2021-11-30 |
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease pegunigalsidase alfa - Fabry Disease | Phase 3 | Completed | 97 | 2018-09-16 | 2026-03-24 |
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease Lucerastat, Placebo - Fabry Disease | Phase 3 | Completed | 118 | 2018-06-21 | 2024-08-09 |
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks Pegunigalsidase alfa - Fabry Disease | Phase 3 | Completed | 30 | 2017-07-10 | 2023-09-13 |
Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) PRX-102 (pegunigalsidase alfa) - Fabry Disease | Phase 3 | Completed | 22 | 2017-05-17 | 2023-09-13 |
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 3 | Completed | 78 | 2016-06-01 | 2023-09-13 |
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | 84 | 2015-03-14 | 2020-12-21 |
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 3 | Completed | 68 | 2011-09-08 | 2018-11-01 |
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 3 | Completed | 171 | 2011-08-10 | 2021-06-08 |
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease Replagal - Fabry Disease | Phase 3 | Completed | 35 | 2010-04-13 | 2021-06-08 |
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease migalastat hydrochloride, Placebo - Fabry Disease | Phase 3 | Completed | 67 | 2009-10-23 | 2018-10-30 |
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease Replagal - Fabry Disease | Phase 3 | Completed | 44 | 2008-12-29 | 2021-06-09 |
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms Agalsidase beta - Fabry Disease | Phase 3 | Completed | 31 | 2008-09-01 | 2016-06-29 |
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease Fabrazyme (agalsidase beta) - Fabry Disease | Phase 3 | Completed | 58 | 1999-10-01 | 2013-12-04 |
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)