Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.

PHASE2CompletedINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

October 1, 2018

Primary Completion Date

July 30, 2019

Study Completion Date

December 30, 2020

Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
DRUG

sodium phenylbutyrate

Enrolled subjects will receive a four-week period of treatment with sodium phenylbutyrate (oral use)

Trial Locations (1)

80131

Federico II University, Napoli

All Listed Sponsors
lead

Fondazione Telethon

OTHER

NCT03734263 - Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. | Biotech Hunter | Biotech Hunter