2 completed, 1 active not recruiting, 1 not yet recruiting. 4 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 4 | Completed 2, Active not recruiting 1, Not yet recruiting 1 |
Follow Pompe Disease (Late-onset): new trials by email Literature Review of Pompe Disease (Late-onset) trials Research Report on Pompe Disease (Late-onset)
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 |
|---|---|---|---|---|---|---|
| Treatment Frequency Reduction in Pompe Disease Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks - Pompe Disease (Late-onset), GAA Deficiency | Phase 4 | Not yet recruiting | Erasmus Medical Center | 10 | 2024-10-01 | 2024-08-28 |
| Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years Avalglucosidase Alfa - Glycogen Storage Disease Type II Late Onset, Pompe Disease (Late-onset) | Phase 4 | Active not recruiting | Iris Plug | 6 | 2022-10-13 | 2026-06-17 |
| Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Alglucosidase Alfa - Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II) | Phase 4 | Completed | Genzyme, a Sanofi Company | 16 | 2011-06-01 | 2014-12-19 |
| Late-Onset Treatment Study Extension Protocol alglucosidase alfa - Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II) | Phase 4 | Completed | Genzyme, a Sanofi Company | 81 | 2007-03-01 | 2014-03-07 |
Glycogen Storage Disease Type II (4)
Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Carbohydrate Metabolism, Inborn Errors (4) · Central Nervous System Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Glycogen Storage Disease (4) · Lysosomal Storage Diseases (4) · Lysosomal Storage Diseases, Nervous System (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Nervous System Diseases (4) · Nutritional and Metabolic Diseases (4)
Genzyme, a Sanofi Company (2) · Erasmus Medical Center (1) · Iris Plug (1)
Netherlands (3) · United States (2) · Australia (1) · Canada (1) · France (1) · Germany (1) · United Kingdom (1)
Nadine van der Beek, MD PHD (1) · Pieter A. van Doorn, Prof. dr. (1) · Tim Preijers, Dr. (1)
2019 (1)