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PDH-RAVICTI trial of Ravicti in pyruvate dehydrogenase deficiency
2024-516410-38-00Phase 2Authorised
- Sponsor
- Assistance publique - Hôpitaux de Paris
- Start
- Oct 2025
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sodium phenylbutyrate in pyruvate dehydrogenase deficiency (PDH), sponsored by Assistance publique - Hôpitaux de Paris. Primary endpoint: Change in fatigue score at 6 months. Planned start Oct 2025; status: authorised.
02
Drugs
sodium phenylbutyrate
03
Conditions
pyruvate dehydrogenase deficiency (PDH)
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