NCT07097311View on ClinicalTrials.gov

Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

PHASE2RecruitingINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

November 30, 2025

Primary Completion Date

January 31, 2027

Study Completion Date

March 31, 2027

Conditions
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Interventions
DRUG

Triheptanoin

Open-label design with doses of triheptanoin up to 1.0 gm/kg/day. The dose will be titrated from Day 1-24.

Trial Locations (1)

15224

RECRUITING

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

All Listed Sponsors
collaborator

Ultragenyx Pharmaceutical Inc

INDUSTRY

lead

Jerry Vockley, MD, PhD

OTHER

NCT07097311 - Study to Evaluate the Use of Triheptanoin in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD) | Biotech Hunter | Biotech Hunter