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Nizubaglustat in Niemann-Pick Type C Disease
NCT07082725Phase 3Recruiting
- Sponsor
- Azafaros
- Start
- Jun 2025
- Primary completion
- May 2028
- Modality
- Orthosteric Ligands
- Countries
- 15
01
Overview
Phase 3, randomised, double-blind trial in Niemann-Pick disease type C (NPC) comparing Nizubaglustat with Placebo (2 arms). Primary endpoints: Total 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: recruiting.
02
Drugs
nizubaglustat
03
Conditions
Niemann-Pick disease type C (NPC)
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