| Recruiting | A Study of Patients With Fabry Disease (US Specific) Fabry Disease | — | 2026-02-13 |
| Recruiting | A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants Fabry Disease | Phase 3 | 2026-01-08 |
| Recruiting | A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Br Fabry Disease | — | 2025-03-18 |
| Recruiting | A Global Prospective Observational Registry of Patients With Pompe Disease Pompe Disease | — | 2024-02-16 |
| Recruiting | A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Glycogen Storage Disease Type II Infantile Onset | Phase 3 | 2023-07-18 |
| Completed | Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients Fabry Disease | — | 2023-03-01 |
| Active Not Recruiting | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease Fabry Disease | Phase 3 | 2022-10-31 |
| Completed | A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease Wit Fabry Disease | — | 2020-03-01 |
| Active Not Recruiting | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y Pompe Disease (Late-onset) | Phase 3 | 2020-02-13 |
| Completed | A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe D Pompe Disease (Late-onset) | Phase 3 | 2019-12-18 |
| Completed | Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease Fabry Disease | Phase 3 | 2019-10-11 |
| Completed | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Diseas Pompe Disease (Late-onset) | Phase 3 | 2018-12-04 |
| Completed | Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Y Fabry Disease | Phase 3 | 2018-09-27 |
| Terminated | STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT Late-onset Pompe Disease | — | 2017-12-08 |
| Completed | Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa Epidermolysis Bullosa | — | 2017-04-02 |
| Completed | First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administ Pompe Disease | Phase 1 / Phase 2 | 2016-04-01 |
| Completed | Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease Fabry Disease | Phase 3 | 2015-03-14 |
| Completed | A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers Fabry Disease | Phase 1 | 2014-03-01 |
| Completed | Drug-drug Interaction Study Pompe Disease | Phase 2 | 2011-10-31 |
| Terminated | Open-Label Phase 3 Long-Term Safety Study of Migalastat Fabry Disease | Phase 3 | 2011-10-14 |
| Completed | Migalastat Food Effect Study Fabry Disease | Phase 1 | 2011-10-01 |
| Completed | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Fabry Disease | Phase 3 | 2011-09-08 |
| Completed | A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Eval Fabry Disease | Phase 1 | 2011-09-01 |
| Completed | Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Fu Fabry Disease | Phase 1 | 2011-08-01 |
| Completed | A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) Fabry Disease | Phase 1 | 2011-08-01 |
| Completed | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabr Fabry Disease | Phase 2 | 2011-02-02 |
| Completed | Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease Fabry Disease | Phase 3 | 2009-10-23 |
| Completed | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1 | Phase 2 | 2009-05-11 |
| Terminated | A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101 Type 1 Gaucher Disease | Phase 1 | 2009-04-01 |
| Terminated | Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease Pompe Disease | Phase 2 | 2008-12-08 |
| Completed | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1 | Phase 2 | 2008-06-11 |
| Terminated | Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Fabry Disease | Phase 2 | 2007-09-17 |
| Completed | A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease Pompe Disease, Glycogen Storage Disease Type II | — | 2007-08-01 |
| Completed | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzym Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease | Phase 2 | 2007-03-23 |
| Completed | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fa Fabry Disease | Phase 2 | 2006-09-07 |
| Completed | Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease Gaucher Disease | — | 2006-07-01 |
| Completed | A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Dis Fabry Disease | Phase 2 | 2006-06-27 |
| Completed | A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Dis Fabry Disease | Phase 2 | 2006-05-09 |
| Completed | A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Fabry Disease | Phase 2 | 2006-01-02 |
| Available | Expanded Access for ATB200/AT2221 for the Treatment of IOPD Pompe Disease Infantile-Onset | — | — |
| Available | Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease Pompe Disease | — | — |
| Available | Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease Fabry Disease | — | — |