14 completed. 14 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 14 | Completed 14 |
| Phase 2 | 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 |
|---|---|---|---|---|---|---|
| PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg - Fabry Disease | Phase 1 | Completed | Bio Sidus SA | 24 | 2021-10-23 | 2025-10-15 |
| 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 |
| Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease Moss-aGal (recombinant human alpha-galactosidase A produced in moss) - Fabry Disease | Phase 1 | Completed | Greenovation Biotech GmbH | 6 | 2016-11-01 | 2017-12-13 |
| Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease Lentivirus Alpha-gal A transduced stem cells - Fabry Disease | Phase 1 | Completed | University Health Network, Toronto | 5 | 2016-07-01 | 2024-04-17 |
| A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease Lucerastat, Enzyme replacement therapy (ERT) - Fabry Disease | Phase 1 | Completed | Idorsia Pharmaceuticals Ltd. | 14 | 2015-02-01 | 2018-07-10 |
| A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers IV migalastat HCl, IV placebo, oral migalastat HCl - Fabry Disease | Phase 1 | Completed | Amicus Therapeutics | 31 | 2014-03-01 | 2014-06-18 |
| 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 |
| Evaluate the Safety and Exploratory Efficacy of GC1119 GC1119 - Fabry Disease | Phase 1 | Completed | Green Cross Corporation | 8 | 2012-11-01 | 2016-06-24 |
| 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 |
| Migalastat Food Effect Study A (migalastat), B (migalastat), C (migalastat) - Fabry Disease | Phase 1 | Completed | Amicus Therapeutics | 20 | 2011-10-01 | 2013-12-18 |
| A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects GR181413A/AT1001 solution, GR181413A/AT1001 capsule, Potable water - Fabry Disease | Phase 1 | Completed | Amicus Therapeutics | 14 | 2011-09-01 | 2013-12-18 |
| A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) [14C] AT1001 - Fabry Disease | Phase 1 | Completed | Amicus Therapeutics | 6 | 2011-08-01 | 2013-12-18 |
| Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function AT1001 150 mg - Fabry Disease | Phase 1 | Completed | Amicus Therapeutics | 32 | 2011-08-01 | 2017-08-03 |
| An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease Replagal agalsidase alfa, Replagal - Fabry Disease | Phase 1 | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 3 | 2003-10-01 | 2011-08-17 |
Lysosomal Storage Diseases (3) · Peripheral Nervous System Diseases (1) · Stroke (1)
Brain Diseases (14) · Brain Diseases, Metabolic (14) · Brain Diseases, Metabolic, Inborn (14) · Cardiovascular Diseases (14) · Central Nervous System Diseases (14) · Cerebral Small Vessel Diseases (14) · Cerebrovascular Disorders (14) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (14) · Genetic Diseases, Inborn (14) · Genetic Diseases, X-Linked (14) · Lipid Metabolism Disorders (14) · Lipid Metabolism, Inborn Errors (14) · Lipidoses (14) · Lysosomal Storage Diseases, Nervous System (14) · Metabolic Diseases (14)
migalastat (5) · larazotide acetate (4) · agalsidase alfa (2) · agalsidase beta (2) · Drinking Water (1) · Enzyme Replacement Therapy (1) · Fumigant 93 (1) · Sodium Chloride (1)
Amicus Therapeutics (5) · Protalix (2) · Bio Sidus SA (1) · Green Cross Corporation (1) · Greenovation Biotech GmbH (1) · Idorsia Pharmaceuticals Ltd. (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · Sangamo Therapeutics (1) · University Health Network, Toronto (1)
United States (6) · Australia (3) · Germany (3) · United Kingdom (3) · Canada (2) · Paraguay (2) · Spain (2) · Argentina (1) · Italy (1) · Netherlands (1) · Serbia (1) · South Korea (1) · Taiwan (1)
PMID 27136905, PMID 27536442, PMID 28088251, PMID 30834538, PMID 33633114