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Phase 3 randomized double-blind placebo-controlled study of nizubaglustat in late-infantile/juvenile Niemann-Pick type C disease and GM1/GM2 gangliosidoses
2024-515778-28-00Phase 3Authorised
- Sponsor
- Azafaros
- Start
- Jun 2025
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
- Countries
- 16
01
Overview
Phase 3, randomised, double-blind trial in Niemann-Pick disease type C (NPC), GM1 gangliosidosis (GM1) and GM2 gangliosidosis comparing Nizubaglustat with Placebo (2 arms). Primary endpoint: Change in SARA score at Week 72. Planned start Jun 2025; status: authorised.
02
Drugs
nizubaglustat
03
Conditions
Niemann-Pick disease type C (NPC)GM1 gangliosidosis (GM1)GM2 gangliosidosis
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