NCT06887777View on ClinicalTrials.gov

Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)

PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

May 31, 2025

Primary Completion Date

November 30, 2025

Study Completion Date

December 31, 2026

Conditions
Pyruvate Dehydrogenase Complex Deficiency Disease
Interventions
DRUG

Glycerol Phenylbutyrate 1100 MG/ML [Ravicti]

The patients will orally take a dose of 200 mg/kg/day three times a day during meals: breakfast, lunch or afternoon snack and diner for 6 months.

Trial Locations (1)

75015

Hôpital Universitaire Necker - Enfants Malades, Paris

All Listed Sponsors
lead

Assistance Publique - Hôpitaux de Paris

OTHER