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Single Patient Investigational Treatment for Cree Leukoencephalopathy
NCT07300397Active, not recruiting
- Sponsor
- McGill University Health Centre
- Start
- Dec 2025
- Primary completion
- Jan 2029
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label, single-arm trial of fosigotifator in Cree leukoencephalopathy (CLE), sponsored by McGill University Health Centre. Primary endpoint: Time to death or permanent ventilation at 2 years. Started Dec 2025; status: active, not recruiting.
02
Drugs
fosigotifator
03
Conditions
Cree leukoencephalopathy (CLE)
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