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Phase 3 crossover study of N-acetyl-L-leucine in Niemann-Pick disease type C
2023-510278-14-00Phase 3Authorised
- Sponsor
- IntraBio
- Start
- Jun 2022
- Modality
- Small Molecules
- Countries
- 8
01
Overview
Phase 3, randomised, double-blind trial of levacetylleucine in Niemann-Pick disease type C (NPC) (2 arms), sponsored by IntraBio. Primary endpoint: Scale for the Assessment and Rating of Ataxia. Planned start Jun 2022; status: authorised.
02
Drugs
levacetylleucine
03
Conditions
Niemann-Pick disease type C (NPC)
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