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Clinical trial

Pilot Clinical Trial to Investigate the Safety and Efficacy of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.

NCT03734263 is registered in ClinicalTrials.gov with Fondazione Telethon as lead sponsor.

NCT NCT03734263
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Registry record

As filed in ClinicalTrials.gov.

Title
Pilot Clinical Trial to Investigate the Safety and Efficacy of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
Lead sponsor
Fondazione Telethon
Phase
Phase 2
Overall status
Completed
Start date
October 1, 2018
Completion date
December 30, 2020
Enrollment
1 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

pyruvate dehydrogenase complex deficiency