1 withdrawn. 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 1 | Withdrawn 1 |
| Phase 3 | 1 | Withdrawn 1 |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 |
|---|---|---|---|---|---|---|
| An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 |
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)
Genetic Therapy (1) · Velaglucerase alfa, human (1) · imiglucerase (1) · taliglucerase alfa (1)
AVROBIO (1)