2 recruiting, 1 terminated. 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Recruiting 2, Terminated 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 Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis AZ-3102, Placebo - Niemann-Pick Type C Disease, GM1 Gangliosidosis | Phase 3 | Recruiting | Azafaros B.V. | 147 | 2025-06-30 | 2026-10-27 |
| A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) Nizubaglustat, Placebo - Niemann-Pick Type C Disease | Phase 3 | Recruiting | Azafaros B.V. | 72 | 2025-06-30 | 2026-10-28 |
| Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System Adrabetadex - Niemann-Pick Type C Disease | Phase 3 | Terminated | Mandos LLC | 27 | 2018-06-18 | 2024-01-17 |
Niemann-Pick Disease, Type C (3) · Gangliosidoses, GM2 (1) · Gangliosidosis, GM1 (1)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Hemic and Lymphatic Diseases (3) · Histiocytosis (3) · Histiocytosis, Non-Langerhans-Cell (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Lipidoses (3) · Lymphatic Diseases (3) · Lysosomal Storage Diseases (3) · Lysosomal Storage Diseases, Nervous System (3)
2-Hydroxypropyl-beta-cyclodextrin (1)
Azafaros B.V. (2) · Mandos LLC (1)
United States (3) · Argentina (2) · Australia (2) · Brazil (2) · Canada (2) · Germany (2) · India (2) · Italy (2) · Mexico (2) · Pakistan (2) · Portugal (2) · Spain (2) · Switzerland (2) · Turkey (Türkiye) (2) · United Kingdom (2)