33 completed, 8 terminated, 5 recruiting, 3 available. 14 Phase 2, 14 Phase 3, 8 Phase 1, 2 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 14 | Completed 11, Terminated 3 |
| Phase 3 | 14 | Completed 8, Active not recruiting 2, Recruiting 2, Terminated 2 |
| Phase 1 | 8 | Completed 6, Terminated 2 |
| Not applicable | 2 | Completed 2 |
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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 of Patients With Fabry Disease (US Specific) migalastat HCl, ERT - Fabry Disease | Recruiting | Amicus Therapeutics | 450 | 2026-02-13 | 2026-09-09 | |
| 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 | 2025-07-08 | |
| A Global Prospective Observational Registry of Patients With Pompe Disease Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa - Pompe Disease | Recruiting | Amicus Therapeutics | 500 | 2024-02-16 | 2026-03-10 | |
| Real World Evidence Study of Danish Fabry Patients Fabry Disease | Unknown | Caroline Michaela Kistorp | 115 | 2023-08-01 | 2024-03-12 | |
| A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 Cipaglucosidase alfa, Miglustat - Glycogen Storage Disease Type II Infantile Onset | Phase 3 | Recruiting | Amicus Therapeutics | 36 | 2023-07-18 | 2026-01-22 |
| Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients This is a non-interventional study - Fabry Disease | Completed | Amicus Therapeutics | 44 | 2023-03-01 | 2024-04-03 | |
| 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 Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. Enzyme Replacement Therapy (ERT) - Fabry Disease | Completed | Amicus Therapeutics | 82 | 2020-03-01 | 2026-02-05 | |
| ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Cipaglucosidase Alfa, Miglustat - Pompe Disease (Late-onset) | Phase 3 | Active not recruiting | Amicus Therapeutics | 21 | 2020-02-13 | 2026-09-17 |
| A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) AT2221, ATB200 - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 119 | 2019-12-18 | 2026-03-05 |
| Assessment of the Relations Between Endothelial and Venous Dysfunctions and Left Ventricular Obstruction in Genetic Hypertrophic Cardiomyopathies BNP blood sample test, Electrocardiogram, Holter ECG - Hypertrophic Cardiomyopathy, Endothelial Dysfunction | Not applicable | Completed | University Hospital, Bordeaux | 40 | 2019-10-22 | 2026-06-01 |
| 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 |
| Screening of Fabry Disease in Patients With GI Symptoms dry blood test - Fabry Disease | Unknown | General University Hospital, Prague | 200 | 2019-09-01 | 2020-02-06 | |
| A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa - Pompe Disease (Late-onset) | Phase 3 | Completed | Amicus Therapeutics | 125 | 2018-12-04 | 2025-09-11 |
| 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 |
| Effect of Migalastat on Cardiac Involvement in Fabry Disease Cardiological evaluation - Fabry Disease, Heart Diseases | Completed | Ospedale San Donato | 18 | 2018-01-10 | 2021-03-18 | |
| STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT Late-onset Pompe Disease | Terminated | Amicus Therapeutics | 12 | 2017-12-08 | 2025-07-08 | |
| Diet and Exercise in Pompe Disease Diet and Exercise - Pompe Disease, Glycogen Storage Disease Type II | Not applicable | Completed | University of Florida | 2 | 2017-11-06 | 2021-05-14 |
| German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. Fabry Disease | Completed | University Hospital Muenster | 75 | 2017-06-08 | 2021-01-05 |
Showing 20 of 53. Sign in to see all of them.
Fabry Disease (28) · Glycogen Storage Disease Type II (11) · Epidermolysis Bullosa (5) · Gaucher Disease (5) · Lysosomal Storage Diseases (5) · Kidney Diseases (2) · Angiokeratoma (1) · Cardiomyopathies (1) · Cardiomyopathy, Hypertrophic (1) · Cardiovascular Diseases (1) · Cerebrovascular Disorders (1) · Genetic Diseases, Inborn (1) · Heart Diseases (1) · Hypersensitivity (1) · Hypohidrosis (1)
migalastat (21) · larazotide acetate (19) · miglustat (7) · AT2101 (4) · GAA protein, human (3) · agalsidase beta (3) · 1-Deoxynojirimycin (2) · agalsidase alfa (2) · Blood Specimen Collection (1) · Diet (1) · Drinking Water (1) · Echocardiography (1) · Electrocardiography (1) · Electrocardiography, Ambulatory (1) · Enzyme Replacement Therapy (1)
United States (40) · United Kingdom (22) · Australia (17) · France (13) · Italy (12) · Canada (10) · Germany (10) · Belgium (9) · Netherlands (9) · Spain (9) · Brazil (8) · Denmark (8) · Japan (8) · Austria (7) · Poland (6)
Ales Linhart, prof., MD. (1) · Antonia Camporeale, MD, PhD (1) · Barry J. Byrne, MD, PhD (1) · Caroline M Kistorp, MD, Ph.D (1) · Eugene Schneider, MD (1) · Eva Brand, Prof. (1) · Irina Kline, MD (1) · Karin Ludwig, M.D. (1) · Manuela Corti, PT, PhD (1) · Niloofar Nobakht, MD (1)
PMID 27657681, PMID 41631150, PMID 23176611, PMID 27509102, PMID 31889231, PMID 32994552, PMID 23474038, PMID 26252393, PMID 27136905, PMID 27536442, PMID 27834756, PMID 29703262, PMID 30723321, PMID 31934472, PMID 32576219, PMID 32693833, PMID 33012654, PMID 34800400, PMID 36669872, PMID 38057636, PMID 38294575, PMID 38418563, PMID 39318718, PMID 39535661, PMID 40342075, PMID 40790487, PMID 40920287, PMID 41769220
2014 (3), 2009 (1), 2017 (1), 2019 (1)