4 completed, 1 recruiting, 1 unknown. 2 Phase 4, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 2 | Completed 1, Recruiting 1 |
| Not applicable | 1 | Completed 1 |
Follow Pompe's Disease: new trials by email Literature Review of Pompe's Disease trials Research Report on Pompe's 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 |
|---|---|---|---|---|---|---|
| China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD Avalglucosidase alfa - Glycogen Storage Disease Type II, Pompe's Disease | Phase 4 | Recruiting | Genzyme, a Sanofi Company | 13 | 2025-05-07 | 2026-03-23 |
| Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen ALGLUCOSIDASE ALFA - Pompe's Disease | Phase 4 | Completed | Genzyme, a Sanofi Company | 41 | 2021-03-10 | 2025-02-10 |
| Prevalence of Pompe's Disease in Respiratory Clinics Prospective observational study - Pompe's Disease | Unknown | Royal Brompton & Harefield NHS Foundation Trust | 400 | 2015-09-01 | 2015-08-18 | |
| Prevalence of Heterozygote Mothers for Pompe's Disease Among Mothers Having Delivered in French Guiana observational - Pompe's Disease | Completed | Centre Hospitalier de Cayenne | 925 | 2014-04-01 | 2016-09-16 | |
| Feasability and Interest of Screening for Infantile Pompe's Diseases at Birth observational (no intervention) - Pompe's Disease | Completed | Centre Hospitalier de Cayenne | 3413 | 2014-04-01 | 2016-09-19 | |
| Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis blood test - Pompe's Disease | Not applicable | Completed | Centre Hospitalier Universitaire de Nice | 400 | 2009-01-01 | 2011-12-08 |
Glycogen Storage Disease Type II (6)
Brain Diseases (6) · Brain Diseases, Metabolic (6) · Brain Diseases, Metabolic, Inborn (6) · Carbohydrate Metabolism, Inborn Errors (6) · Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Glycogen Storage Disease (6) · Lysosomal Storage Diseases (6) · Lysosomal Storage Diseases, Nervous System (6) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Nervous System Diseases (6) · Nutritional and Metabolic Diseases (6)
GAA protein, human (2) · Hematologic Tests (1) · Watchful Waiting (1)
Centre Hospitalier de Cayenne (2) · Genzyme, a Sanofi Company (2) · Centre Hospitalier Universitaire de Nice (1) · Royal Brompton & Harefield NHS Foundation Trust (1)
China (2) · France (1)
Claude Desnuelle (1)