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

NCT06340685 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-02-17

No results posted yet for this study

Summary

This is a medical research study to test a medication in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency. The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders. Previous research suggests that triheptanoin may also be effective in the treatment PDC Deficiency. This study will investigate the safety and efficacy (how well it works) of triheptanoin in patients with PDC Deficiency.

Conditions

  • Pyruvate Dehydrogenase Complex Deficiency

Interventions

DRUG

Triheptanoin

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

Sponsors & Collaborators

Principal Investigators

  • Jirair Bedoyan, MD, PhD · UPMC Children's Hospital of Pittsburgh

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-07-11
Primary Completion
2028-12-31
Completion
2029-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06340685 on ClinicalTrials.gov