14 completed, 4 terminated, 2 recruiting, 1 active not recruiting. 23 Phase 1, 11 Phase 2.
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 |
|---|---|---|---|---|---|---|
| The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease EXG110 Injection - Fabry Disease | Phase 1 / Phase 2 | Not yet recruiting | Guangzhou Jiayin Biotech Ltd | 16 | 2025-03-01 | 2025-02-11 |
| Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease AMT-191 - Fabry Disease | Phase 1 / Phase 2 | Recruiting | UniQure Biopharma B.V. | 20 | 2024-06-05 | 2026-09-24 |
| Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease RVX000222 - Fabry Disease | Phase 1 / Phase 2 | Withdrawn | Resverlogix Corp | 2022-11-22 | 2023-11-18 | |
| 4D-310 in Adults With Fabry Disease and Cardiac Involvement 4D-310 - Fabry Disease | Phase 1 / Phase 2 | Recruiting | 4D Molecular Therapeutics | 18 | 2022-10-16 | 2025-02-21 |
| 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 |
| A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 FLT190 - Fabry Disease, Lysosomal Storage Diseases | Phase 1 / Phase 2 | Terminated | Spur Therapeutics | 3 | 2020-09-08 | 2026-09-03 |
| An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease 4D-310 - Fabry Disease | Phase 1 / Phase 2 | Active not recruiting | 4D Molecular Therapeutics | 18 | 2020-09-01 | 2024-04-08 |
| 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 |
| A Fabry Disease Gene Therapy Study FLT190 - Fabry Disease, Lysosomal Storage Diseases | Phase 1 / Phase 2 | Terminated | Spur Therapeutics | 3 | 2019-07-08 | 2026-09-03 |
| Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease AVR-RD-01 - Fabry Disease | Phase 1 / Phase 2 | Terminated | AVROBIO | 15 | 2018-02-21 | 2024-01-05 |
| 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 |
| A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease Iohexol - Fabry Disease | Phase 1 | Terminated | Genzyme, a Sanofi Company | 39 | 2013-05-01 | 2020-12-16 |
| 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 |
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Lysosomal Storage Diseases (8) · Cardiovascular Diseases (3) · Central Nervous System Diseases (2) · Cerebral Small Vessel Diseases (2) · Cerebrovascular Disorders (2) · Genetic Diseases, Inborn (2) · Lipid Metabolism Disorders (2) · Lipidoses (2) · Metabolic Diseases (2) · Nervous System Diseases (2) · Sphingolipidoses (2) · Vascular Diseases (2) · Brain Diseases, Metabolic (1) · Kidney Diseases (1)
Brain Diseases (23) · Brain Diseases, Metabolic, Inborn (23) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (23) · Genetic Diseases, X-Linked (23) · Lipid Metabolism, Inborn Errors (23) · Lysosomal Storage Diseases, Nervous System (23) · Metabolism, Inborn Errors (23) · Nutritional and Metabolic Diseases (23) · Brain Diseases, Metabolic (22) · Central Nervous System Diseases (21) · Cerebral Small Vessel Diseases (21) · Cerebrovascular Disorders (21) · Genetic Diseases, Inborn (21) · Lipid Metabolism Disorders (21) · Lipidoses (21)
migalastat (5) · larazotide acetate (4) · agalsidase alfa (2) · agalsidase beta (2) · Drinking Water (1) · Enzyme Replacement Therapy (1) · Fumigant 93 (1) · Iohexol (1) · Sodium Chloride (1) · apabetalone (1)
Amicus Therapeutics (5) · 4D Molecular Therapeutics (2) · Protalix (2) · Spur Therapeutics (2) · AVROBIO (1) · Bio Sidus SA (1) · Genzyme, a Sanofi Company (1) · Green Cross Corporation (1) · Greenovation Biotech GmbH (1) · Guangzhou Jiayin Biotech Ltd (1) · Idorsia Pharmaceuticals Ltd. (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · Resverlogix Corp (1) · Sangamo Therapeutics (1) · UniQure Biopharma B.V. (1)
United States (11) · United Kingdom (6) · Australia (5) · Canada (5) · Germany (5) · Spain (3) · Taiwan (3) · Argentina (2) · Austria (2) · Brazil (2) · Italy (2) · Norway (2) · Paraguay (2) · Belgium (1) · China (1)
Alan H Cohen, MD (2) · Arian Pano, MD, MPH (1) · Eduardo Pirotzky, MD (1) · Inderpal Panesar, MRPharmS (1) · Michael West, MD (1) · Zhihong Liu, MD (1)
PMID 27136905, PMID 27536442, PMID 28088251, PMID 30834538, PMID 33633114, PMID 36631545