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Phenylbutyrate for Pyruvate Dehydrogenase Complex Deficiency
NCT03734263Phase 2Completed
- Sponsor
- Fondazione Telethon
- Start
- Oct 2018
- Primary completion
- Jul 2019
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of sodium phenylbutyrate in Pyruvate dehydrogenase complex deficiency (PDCD), sponsored by Fondazione Telethon. Primary endpoint: Blood lactate concentration at 4 weeks. Started Oct 2018; status: completed.
02
Drugs
sodium phenylbutyrate
03
Conditions
Pyruvate dehydrogenase complex deficiency (PDCD)
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