21 completed, 4 recruiting, 2 active not recruiting, 2 available. 12 Phase 3, 9 Phase 1, 6 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 12 | Completed 7, Active not recruiting 2, Recruiting 2, Terminated 1 |
| Phase 1 | 9 | Completed 9 |
| Phase 2 | 6 | Completed 5, Terminated 1 |
Follow migalastat: new trials by email Literature Review of migalastat trials Research Report on migalastat
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 | Recruiting | Idorsia Pharmaceuticals Ltd. | 16 | 2026-12-01 | 2026-11-03 |
| A Study of Patients With Fabry Disease (US Specific) migalastat HCl, ERT - Fabry Disease | Recruiting | Amicus Therapeutics | 450 | 2026-02-13 | 2026-10-28 | |
| 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 Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding migalastat - Fabry Disease | Recruiting | Amicus Therapeutics | 20 | 2025-03-18 | 2026-10-28 | |
| 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 |
| 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 of Migalastat in Fabry Disease Migalastat - Fabry Disease | Unknown | Manchester University NHS Foundation Trust | 21 | 2019-05-16 | 2023-05-31 | |
| A Study in Healthy Subjects to Investigate Whether Administration of Lucerastat Can Affect Normal Heart Function Lucerastat (Treatment A), Placebo, Moxifloxacin - Healthy | Phase 1 | Completed | Idorsia Pharmaceuticals Ltd. | 44 | 2019-02-14 | 2019-10-31 |
| A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease Lucerastat - Fabry Disease | Phase 3 | Completed | Idorsia Pharmaceuticals Ltd. | 107 | 2018-12-18 | 2026-11-02 |
| 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 |
| 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 |
| Effect of Cimetidine on the Pharmacokinetics of Lucerastat in Healthy Subjects Lucerastat, Cimetidine - Healthy Subjects | Phase 1 | Completed | Idorsia Pharmaceuticals Ltd. | 14 | 2018-01-09 | 2022-06-02 |
| Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease migalastat HCl 150 mg - Fabry Disease | Phase 3 | Completed | Amicus Therapeutics | 84 | 2015-03-14 | 2020-12-21 |
| 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 |
| Open-Label Phase 3 Long-Term Safety Study of Migalastat migalastat hydrochloride - Fabry Disease | Phase 3 | Terminated | Amicus Therapeutics | 85 | 2011-10-14 | 2018-10-02 |
| Migalastat Food Effect Study A (migalastat), B (migalastat), C (migalastat) - Fabry Disease | Phase 1 | Completed | Amicus Therapeutics | 20 | 2011-10-01 | 2013-12-18 |
| Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease migalastat hydrochloride, agalsidase - Fabry Disease | Phase 3 | Completed | Amicus Therapeutics | 68 | 2011-09-08 | 2018-11-01 |
| 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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Fabry Disease (28) · Lysosomal Storage Diseases (3) · Kidney Diseases (1) · Kidney Failure, Chronic (1) · Renal Insufficiency (1)
Amicus Therapeutics (21) · Idorsia Pharmaceuticals Ltd. (9) · Manchester University NHS Foundation Trust (1) · Sanofi (1)
United States (20) · United Kingdom (18) · Australia (12) · France (10) · Canada (9) · Spain (8) · Belgium (7) · Brazil (7) · Germany (7) · Italy (6) · Netherlands (6) · Austria (5) · Denmark (5) · Japan (5) · Poland (4)
PMID 27657681, PMID 28088251, PMID 23176611, PMID 27509102, PMID 31889231, PMID 32994552, PMID 23474038, PMID 26252393, PMID 27136905, PMID 27536442, PMID 27834756, PMID 29703262, PMID 30723321, PMID 31836927, PMID 31934472, PMID 33012654, PMID 33109218, PMID 35782623
2018 (2), 2009 (1), 2014 (1), 2017 (1), 2019 (1), 2023 (1)