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Nizubaglustat in GM1/GM2 Gangliosidosis
NCT07082543Phase 3Active, not recruiting
- Sponsor
- Azafaros
- Start
- Jun 2025
- Primary completion
- Feb 2028
- Modality
- Orthosteric Ligands
- Countries
- 14
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Overview
Phase 3, randomised, double-blind trial in GM1 gangliosidosis (GM1) and GM2 gangliosidosis comparing Nizubaglustat with Placebo (2 arms). Primary endpoints: Scale for the Assessment and Rating of Ataxia at 18 months and Functional Scale for the Assessment and Rating of Ataxia at 18 months. Started Jun 2025; status: active, not recruiting.
02
Drugs
nizubaglustat
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Conditions
GM1 gangliosidosis (GM1)GM2 gangliosidosis
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