19 completed. 19 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 19 | Completed 19 |
| Phase 1 | 3 | Completed 3 |
Follow Fabry Disease: new trials by email Literature Review of Fabry Disease trials Research Report on Fabry Disease
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 |
|---|---|---|---|---|---|---|
| Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) ST-920 - Fabry Disease | Phase 1 / Phase 2 | Completed | Sangamo Therapeutics | 36 | 2019-07-23 | 2026-10-27 |
| Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease GZ/SAR402671 - Fabry Disease | Phase 2 | Completed | Genzyme, a Sanofi Company | 8 | 2015-07-07 | 2021-03-23 |
| Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease GZ/SAR402671 - Fabry Disease | Phase 2 | Completed | Genzyme, a Sanofi Company | 11 | 2014-11-01 | 2019-12-17 |
| Extension Study of PRX-102 for up to 60 Months PRX-102 (pegunigalsidase alfa) - Fabry Disease | Phase 1 / Phase 2 | Completed | Protalix | 15 | 2014-01-16 | 2023-09-13 |
| Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients PRX-102 - Fabry Disease | Phase 1 / Phase 2 | Completed | Protalix | 18 | 2012-10-01 | 2023-09-13 |
| This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. agalsidase alfa - Fabry Disease | Phase 2 | Completed | Shire | 17 | 2011-11-17 | 2021-07-19 |
| Safety Study of Replagal® Therapy in Children With Fabry Disease Replagal (agalsidase alfa) - Fabry Disease | Phase 2 | Completed | Shire | 15 | 2011-05-12 | 2021-06-09 |
| Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease Migalastat HCl, Agalsidase Beta, Agalsidase Alfa - Fabry Disease | Phase 2 | Completed | Amicus Therapeutics | 20 | 2011-02-02 | 2018-12-19 |
| A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease migalastat HCl - Fabry Disease | Phase 2 | Completed | Amicus Therapeutics | 9 | 2006-09-07 | 2018-10-03 |
| A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease migalastat HCl - Fabry Disease | Phase 2 | Completed | Amicus Therapeutics | 4 | 2006-06-27 | 2018-10-31 |
| A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease migalastat HCl - Fabry Disease | Phase 2 | Completed | Amicus Therapeutics | 5 | 2006-05-09 | 2018-09-07 |
| A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease migalastat HCl - Fabry Disease | Phase 2 | Completed | Amicus Therapeutics | 9 | 2006-01-02 | 2018-10-30 |
| Replagal Enzyme Replacement Therapy for Children With Fabry Disease Agalsidase alfa - Fabry Disease | Phase 2 | Completed | Shire | 17 | 2004-06-10 | 2021-07-30 |
| Alternative Dosing and Regimen of Replagal to Treat Fabry Disease Replagal - Fabry Disease | Phase 2 | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 25 | 2004-01-01 | 2008-03-04 |
| An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease Replagal - Fabry Disease | Phase 2 | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 25 | 2003-10-01 | 2008-03-04 |
| Dosing Study of Replagal in Patients With Fabry Disease Replagal - Fabry Disease | Phase 2 | Completed | Baylor Research Institute | 13 | 2003-09-01 | 2015-03-31 |
| A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease Fabrazyme (agalsidase beta) - Fabry Disease | Phase 2 | Completed | Genzyme, a Sanofi Company | 21 | 2003-06-01 | 2015-04-03 |
| Alpha-Galactosidase A Replacement Therapy for Fabry Disease DRX005B - Fabry Disease | Phase 2 | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 3 | 2002-11-01 | 2008-03-04 |
| A Study of Fabrazyme in Pediatric Patients With Fabry Disease Fabrazyme (agalsidase beta) - Fabry Disease | Phase 2 | Completed | Genzyme, a Sanofi Company | 16 | 2002-10-01 | 2015-04-02 |
Stroke (4) · Hypohidrosis (2) · Lysosomal Storage Diseases (1) · Pain (1) · Peripheral Nervous System Diseases (1) · Renal Insufficiency (1)
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)
agalsidase alfa (7) · larazotide acetate (5) · migalastat (5) · agalsidase beta (3) · venglustat (2)
Amicus Therapeutics (5) · Genzyme, a Sanofi Company (4) · National Institute of Neurological Disorders and Stroke (NINDS) (3) · Shire (3) · Protalix (2) · Baylor Research Institute (1) · Sangamo Therapeutics (1)
United States (15) · United Kingdom (8) · Australia (5) · Canada (5) · France (5) · Poland (4) · Brazil (2) · Paraguay (2) · Russia (2) · Spain (2) · Belgium (1) · Czechia (1) · Estonia (1) · Germany (1) · Italy (1)
PMID 27657681, PMID 23176611, PMID 36481125, PMID 18346516, PMID 23474038, PMID 25425121, PMID 26252393, PMID 27307708, PMID 30834538
2014 (1)