2 terminated. 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Terminated 2 |
| Phase 2 | 1 | Terminated 1 |
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 |
|---|---|---|---|---|---|---|
| Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease AVR-RD-02 - Gaucher Disease | Phase 1 / Phase 2 | Terminated | AVROBIO | 8 | 2019-05-30 | 2024-01-18 |
| A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101 AT2101 - Type 1 Gaucher Disease | Phase 1 | Terminated | Amicus Therapeutics | 1 | 2009-04-01 | 2010-08-19 |
Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Lipid Metabolism Disorders (2) · Lipid Metabolism, Inborn Errors (2) · Lipidoses (2) · Lysosomal Storage Diseases (2) · Lysosomal Storage Diseases, Nervous System (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Nutritional and Metabolic Diseases (2)
AT2101 (1)
AVROBIO (1) · Amicus Therapeutics (1)
United States (2) · Canada (1) · United Kingdom (1)
Eugene Schneider, MD (1) · Milena Veselinovic, MD (1)