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Nizubaglustat in Niemann-Pick Type C and GM1/GM2 Gangliosidosis
NCT07054515Phase 3Recruiting
- Sponsor
- Azafaros
- Start
- Jun 2025
- Primary completion
- May 2028
- Modality
- Orthosteric Ligands
- Countries
- 17
01
Overview
Phase 3, randomised, double-blind trial of nizubaglustat in Niemann-Pick disease type C (NPC), GM1 gangliosidosis (GM1) and GM2 gangliosidosis (2 arms), sponsored by Azafaros. Primary endpoints: Number of participants allocated to subprotocol AZA-001-301-NPC at 18 months and Number of participants allocated to subprotocol AZA-001-301-GMx at 18 months. Started Jun 2025; status: recruiting.
02
Drugs
nizubaglustat
03
Conditions
Niemann-Pick disease type C (NPC)GM1 gangliosidosis (GM1)GM2 gangliosidosis
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