1 completed. 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 1 | Completed 1 |
Follow Niemann-Pick Diseases: new trials by email Literature Review of Niemann-Pick Diseases trials Research Report on Niemann-Pick Diseases
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Miglustat in Chinese NPC Patients Miglustat - Niemann-Pick Disease, Type C | Phase 4 | Completed | Actelion | 17 | 2020-04-02 | 2025-03-30 |
Niemann-Pick Disease, Type C (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) · Hemic and Lymphatic Diseases (1) · Histiocytosis (1) · Histiocytosis, Non-Langerhans-Cell (1) · Lipid Metabolism Disorders (1) · Lipid Metabolism, Inborn Errors (1) · Lipidoses (1) · Lymphatic Diseases (1) · Lysosomal Storage Diseases (1) · Lysosomal Storage Diseases, Nervous System (1)
miglustat (1)
Actelion (1)
China (1)
Yue Wu (1)