19 completed, 5 terminated, 4 recruiting, 2 unknown. 34 Phase 2, 11 Phase 1, 2 Phase 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 |
|---|---|---|---|---|---|---|
| A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease PRX-102 1 mg/kg every two weeks - Fabry Disease | Phase 2 / Phase 3 | Recruiting | Chiesi Farmaceutici S.p.A. | 22 | 2025-07-29 | 2026-08-10 |
| 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 |
| Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease AL01211 - Fabry Disease | Phase 2 | Unknown | AceLink Therapeutics, Inc. | 16 | 2023-10-25 | 2024-07-30 |
| Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease PRX-102 1 mg/kg every 2 weeks, PRX-102 2 mg/kg every 4 weeks - Fabry Disease | Phase 2 / Phase 3 | Recruiting | Chiesi Farmaceutici S.p.A. | 16 | 2023-08-01 | 2026-03-18 |
| Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients Dapagliflozin 10mg Tab, Placebo - Fabry Disease | Phase 2 | Unknown | Albina Nowak, MD | 46 | 2023-08-01 | 2023-02-02 |
| Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease RVX000222 - Fabry Disease | Phase 1 / Phase 2 | Withdrawn | Resverlogix Corp | 2022-11-22 | 2023-11-18 | |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 |
| 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 |
| 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 |
| Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease Gabapentin, placebo - Fabry Disease, Neuropathic Pain | Phase 2 | Withdrawn | University of Minnesota | 2012-04-01 | 2019-05-29 | |
| 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 |
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Lysosomal Storage Diseases (6) · Stroke (4) · Cardiovascular Diseases (3) · Central Nervous System Diseases (2) · Cerebral Small Vessel Diseases (2) · Cerebrovascular Disorders (2) · Genetic Diseases, Inborn (2) · Hypohidrosis (2) · Lipid Metabolism Disorders (2) · Lipidoses (2) · Metabolic Diseases (2) · Nervous System Diseases (2) · Sphingolipidoses (2) · Vascular Diseases (2)
Brain Diseases (34) · Brain Diseases, Metabolic, Inborn (34) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (34) · Genetic Diseases, X-Linked (34) · Lipid Metabolism, Inborn Errors (34) · Lysosomal Storage Diseases, Nervous System (34) · Metabolism, Inborn Errors (34) · Nutritional and Metabolic Diseases (34) · Brain Diseases, Metabolic (33) · Central Nervous System Diseases (32) · Cerebral Small Vessel Diseases (32) · Cerebrovascular Disorders (32) · Genetic Diseases, Inborn (32) · Lipid Metabolism Disorders (32) · Lipidoses (32)
agalsidase alfa (7) · larazotide acetate (6) · migalastat (6) · agalsidase beta (4) · venglustat (2) · Gabapentin (1) · apabetalone (1) · dapagliflozin (1)
Amicus Therapeutics (6) · Genzyme, a Sanofi Company (5) · National Institute of Neurological Disorders and Stroke (NINDS) (3) · Shire (3) · 4D Molecular Therapeutics (2) · Chiesi Farmaceutici S.p.A. (2) · Protalix (2) · Spur Therapeutics (2) · AVROBIO (1) · AceLink Therapeutics, Inc. (1) · Albina Nowak, MD (1) · Baylor Research Institute (1) · Guangzhou Jiayin Biotech Ltd (1) · Resverlogix Corp (1) · Sangamo Therapeutics (1)
United States (23) · United Kingdom (12) · Australia (8) · Canada (7) · France (7) · Brazil (4) · Poland (4) · Germany (3) · Spain (3) · Austria (2) · China (2) · Italy (2) · Norway (2) · Paraguay (2) · Russia (2)
Alan H Cohen, MD (2) · Arian Pano, MD, MPH (1) · Inderpal Panesar, MRPharmS (1) · Jeanine R. Jarnes, PharmD (1) · Michael West, MD (1) · Nan Chen, Dr. (1) · Zhihong Liu, MD (1)
PMID 27657681, PMID 23176611, PMID 36481125, PMID 18346516, PMID 23474038, PMID 25425121, PMID 26252393, PMID 27307708, PMID 30834538, PMID 36631545
2014 (1)