Niemann-Pick Type C Disease: Clinical Trials

2 recruiting, 1 active not recruiting, 1 completed, 1 terminated. 3 Phase 3, 2 Phase 2.

By phase and status

Phase	Trials	Status
Phase 3	3	Recruiting 2, Terminated 1
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

Trials newest first · largest first

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
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
Miglustat in Niemann-Pick Type C Disease miglustat - Niemann-Pick Type C Disease	Phase 2	Completed	Actelion	29	2002-01-01	2025-02-04

Narrower conditions

Niemann-Pick Disease, Type C (5)  ·  Gangliosidoses, GM2 (2)  ·  Gangliosidosis, GM1 (1)

Broader conditions

Brain Diseases (5)  ·  Brain Diseases, Metabolic (5)  ·  Brain Diseases, Metabolic, Inborn (5)  ·  Central Nervous System Diseases (5)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5)  ·  Genetic Diseases, Inborn (5)  ·  Hemic and Lymphatic Diseases (5)  ·  Histiocytosis (5)  ·  Histiocytosis, Non-Langerhans-Cell (5)  ·  Lipid Metabolism Disorders (5)  ·  Lipid Metabolism, Inborn Errors (5)  ·  Lipidoses (5)  ·  Lymphatic Diseases (5)  ·  Lysosomal Storage Diseases (5)  ·  Lysosomal Storage Diseases, Nervous System (5)

Interventions

2-Hydroxypropyl-beta-cyclodextrin (1)  ·  miglustat (1)

Sponsors

Azafaros B.V. (3)  ·  Actelion (1)  ·  Mandos LLC (1)

Countries

Brazil (3)  ·  United States (3)  ·  Argentina (2)  ·  Australia (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)

Top investigators

Claudia Chiriboga, Assoc. Prof. MD (1)