5 active not recruiting. 5 Phase 2, 4 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Active not recruiting 5 |
| Phase 1 | 4 | Active not recruiting 4 |
Follow Gaucher Disease: new trials by email Literature Review of Gaucher Disease trials Research Report on Gaucher 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 |
|---|---|---|---|---|---|---|
| Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) FLT201 - Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Spur Therapeutics | 75 | 2024-05-13 | 2025-11-04 |
| A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) LY3884961 - Gaucher Disease, Gaucher Disease, Type 1 | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 8 | 2022-12-20 | 2026-08-17 |
| 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 |
| Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) LY3884961, Methylprednisolone, Sirolimus - Parkinson Disease | Phase 1 / Phase 2 | Active not recruiting | Prevail Therapeutics | 32 | 2020-01-03 | 2026-10-13 |
| Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension venglustat (GZ402671), imiglucerase - Gaucher Disease Type 1, Gaucher Disease Type 3 | Phase 2 | Active not recruiting | Genzyme, a Sanofi Company | 12 | 2017-01-04 | 2026-07-10 |
Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Lipid Metabolism Disorders (5) · Lipid Metabolism, Inborn Errors (5) · Lipidoses (5) · Lysosomal Storage Diseases (5) · Lysosomal Storage Diseases, Nervous System (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (5) · Nervous System Diseases (5) · Nutritional and Metabolic Diseases (5)
Methylprednisolone (2) · Sirolimus (2) · Prednisone (1) · imiglucerase (1) · venglustat (1)
Prevail Therapeutics (3) · Genzyme, a Sanofi Company (1) · Spur Therapeutics (1)
United States (5) · United Kingdom (3) · Germany (2) · Spain (2) · Australia (1) · Brazil (1) · Israel (1) · Japan (1)
Aaron Tward, MD, PhD (1) · Hamzeh Migdadi, M.D. (1) · Travis B. Lewis, MD, PhD (1)
2014 (1), 2023 (1)