2 completed, 1 terminated. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Terminated 1 |
| Phase 2 | 1 | Terminated 1 |
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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 |
|---|---|---|---|---|---|---|
| VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease VAL-1221, RhGAA - Pompe Disease | Phase 1 / Phase 2 | Terminated | Valerion Therapeutics, LLC | 12 | 2017-06-21 | 2020-06-02 |
| Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) Myotubular Myopathy | Completed | Valerion Therapeutics, LLC | 48 | 2014-02-01 | 2018-06-07 | |
| Myotubular Myopathy Genetic Testing Study Genetic Testing - Myotubular Myopathy | Completed | Cure CMD | 23 | 2013-03-01 | 2018-03-07 |
Myopathies, Structural, Congenital (2) · Glycogen Storage Disease (1) · Glycogen Storage Disease Type II (1) · Muscle Hypotonia (1) · Myotonia Congenita (1) · Neuromuscular Diseases (1)
Base Sequence (1) · GAA protein, human (1) · Genetic Testing (1)
United States (3) · Belgium (1) · Canada (1) · France (1) · Germany (1) · Italy (1) · Spain (1) · United Kingdom (1)
Hal Landy, MD (2) · Elizabeth DeChene, MS, GCG (1) · Sabine de Chastonay, PhD (1) · Soma Das, PhD (1)