1 completed, 1 not yet recruiting. 2 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Completed 1, Not yet recruiting 1 |
| Phase 2 | 1 | Not yet recruiting 1 |
Follow Niemann-Pick Disease Type C: new trials by email Literature Review of Niemann-Pick Disease Type C trials Research Report on Niemann-Pick Disease Type C
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 Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders AZ-3102 - Niemann-Pick Disease Type C, GM2 Gangliosidosis | Phase 2 / Phase 3 | Not yet recruiting | Azafaros B.V. | 218 | 2026-12-10 | 2026-10-21 |
| Application of Miglustat in Patients With Niemann-Pick Type C Miglustat - Niemann-Pick Disease Type C | Phase 3 | Completed | National Taiwan University Hospital | 5 | 2008-01-01 | 2013-01-04 |
Niemann-Pick Disease, Type C (2) · Gangliosidoses, GM2 (1) · Gangliosidosis, GM1 (1) · Gaucher Disease (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)
Azafaros B.V. (1) · National Taiwan University Hospital (1)
Taiwan (1)