1 active not recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Active not recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow Gaucher Disease, Type 2: new trials by email Literature Review of Gaucher Disease, Type 2 trials Research Report on Gaucher Disease, Type 2
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 |
|---|---|---|---|---|---|---|
| Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) LY3884961, Methylprednisolone, Sirolimus - Gaucher Disease, Type 2 | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 7 | 2021-06-29 | 2026-10-22 |
Gaucher Disease (1)
Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Central Nervous System Diseases (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Lipid Metabolism Disorders (1) · Lipid Metabolism, Inborn Errors (1) · Lipidoses (1) · Lysosomal Storage Diseases (1) · Lysosomal Storage Diseases, Nervous System (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1) · Nervous System Diseases (1) · Nutritional and Metabolic Diseases (1)
Methylprednisolone (1) · Prednisone (1) · Sirolimus (1)
United Kingdom (1) · United States (1)