1 active not recruiting, 1 completed. 2 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Active not recruiting 1, Completed 1 |
Follow Niemann-Pick Type C Disease: new trials by email Literature Review of Niemann-Pick Type C Disease trials Research Report on Niemann-Pick Type C 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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease AZ-3102 - GM2 Gangliosidosis, Niemann-Pick Type C Disease | Phase 2 | Active not recruiting | Azafaros B.V. | 21 | 2026-02-04 | 2026-10-21 |
| Miglustat in Niemann-Pick Type C Disease miglustat - Niemann-Pick Type C Disease | Phase 2 | Completed | Actelion | 29 | 2002-01-01 | 2025-02-04 |
Niemann-Pick Disease, Type C (2) · Gangliosidoses, GM2 (1)
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) · Hemic and Lymphatic Diseases (2) · Histiocytosis (2) · Histiocytosis, Non-Langerhans-Cell (2) · Lipid Metabolism Disorders (2) · Lipid Metabolism, Inborn Errors (2) · Lipidoses (2) · Lymphatic Diseases (2) · Lysosomal Storage Diseases (2) · Lysosomal Storage Diseases, Nervous System (2)
miglustat (1)
Actelion (1) · Azafaros B.V. (1)
Brazil (1)