NCT06340685View on ClinicalTrials.gov

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

PHASE1RecruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

July 11, 2024

Primary Completion Date

December 31, 2026

Study Completion Date

June 30, 2027

Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
DRUG

Triheptanoin

Open-label design with doses of triheptanoin up to 4.0 gm/kg triheptanoin

Trial Locations (1)

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

All Listed Sponsors
collaborator

Ultragenyx Pharmaceutical Inc

INDUSTRY

lead

Jirair Krikor Bedoyan

OTHER