18 completed, 4 active not recruiting, 3 recruiting, 2 terminated. 30 Phase 3, 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 30 | Completed 18, Active not recruiting 4, Recruiting 3, Terminated 2, Not yet recruiting 1, Unknown 1, Withdrawn 1 |
| Phase 2 | 2 | Recruiting 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 |
|---|---|---|---|---|---|---|
| A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease Lucerastat - Fabry Disease | Phase 3 | Not yet recruiting | Idorsia Pharmaceuticals Ltd. | 16 | 2026-09-01 | 2026-08-21 |
| A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants Migalastat HCl 20 mg - Fabry Disease | Phase 3 | Recruiting | Amicus Therapeutics | 8 | 2026-01-08 | 2026-10-22 |
| 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 |
| 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 |
| Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease Fabagal® (Agalsidase beta), Active comparator (Agalsidase beta) - Fabry Disease | Phase 3 | Unknown | ISU Abxis Co., Ltd. | 24 | 2023-04-14 | 2024-07-29 |
| 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 | Bio Sidus SA | 20 | 2022-12-13 | 2026-02-18 |
| A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Active not recruiting | Amicus Therapeutics | 14 | 2022-10-31 | 2026-07-06 |
| A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828) - Fabry Disease | Phase 3 | Active not recruiting | Sanofi | 104 | 2022-05-03 | 2026-06-24 |
| A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease REPLAGAL - Fabry Disease | Phase 3 | Completed | Takeda | 20 | 2022-05-01 | 2024-10-08 |
| A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Venglustat (GZ402671), Placebo - Fabry Disease | Phase 3 | Active not recruiting | Sanofi | 122 | 2022-03-11 | 2026-10-13 |
| A Study of Replagal in Treatment-naïve Adults With Fabry Disease REPLAGAL - Fabry Disease | Phase 3 | Terminated | Shire | 17 | 2021-12-28 | 2024-06-17 |
| Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | Amicus Therapeutics | 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 | Active not recruiting | Idorsia Pharmaceuticals Ltd. | 107 | 2018-12-18 | 2026-08-31 |
| 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 | Chiesi Farmaceutici S.p.A. | 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 | Amicus Therapeutics | 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 | Chiesi Farmaceutici S.p.A. | 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 | Idorsia Pharmaceuticals Ltd. | 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 | Protalix | 30 | 2017-07-10 | 2023-09-13 |
| Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease PRX-102, Placebo - Fabry Disease | Phase 3 | Withdrawn | Protalix | 2017-07-01 | 2018-01-08 | |
| 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 | Protalix | 22 | 2017-05-17 | 2023-09-13 |
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Proteinuria (3) · Abdominal Pain (1) · Diarrhea (1) · Kidney Diseases (1) · Kidney Failure, Chronic (1) · Lysosomal Storage Diseases (1) · Renal Insufficiency (1)
Brain Diseases (30) · Brain Diseases, Metabolic (30) · Brain Diseases, Metabolic, Inborn (30) · Cardiovascular Diseases (30) · Central Nervous System Diseases (30) · Cerebral Small Vessel Diseases (30) · Cerebrovascular Disorders (30) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (30) · Genetic Diseases, Inborn (30) · Genetic Diseases, X-Linked (30) · Lipid Metabolism Disorders (30) · Lipid Metabolism, Inborn Errors (30) · Lipidoses (30) · Lysosomal Storage Diseases, Nervous System (30) · Metabolic Diseases (30)
migalastat (12) · larazotide acetate (8) · agalsidase alfa (7) · agalsidase beta (7) · venglustat (2) · alpha-Galactosidase (1)
Amicus Therapeutics (8) · Chiesi Farmaceutici S.p.A. (4) · Protalix (4) · Shire (4) · Idorsia Pharmaceuticals Ltd. (3) · Genzyme, a Sanofi Company (2) · Sanofi (2) · Bio Sidus SA (1) · ISU Abxis Co., Ltd. (1) · Takeda (1)
United Kingdom (22) · United States (21) · Australia (12) · Canada (12) · Spain (12) · France (11) · Italy (11) · Norway (11) · Czechia (9) · Poland (9) · Belgium (8) · Denmark (8) · Netherlands (8) · Austria (7) · Japan (7)
PMID 39847314, PMID 27509102, PMID 27657681, PMID 31889231, PMID 32994552, PMID 20730727, PMID 23531228, PMID 25795794, PMID 25955246, PMID 26185417, PMID 27834756, PMID 29703262, PMID 30723321, PMID 31934472, PMID 33012654, PMID 34542871, PMID 35782623, PMID 37865771, PMID 37940383, PMID 41862950
2009 (2), 2018 (2), 2014 (1), 2017 (1), 2019 (1), 2023 (1)