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

Conditions

• Pyruvate Dehydrogenase Complex Deficiency

Eligibility Criteria

Sex

ALL

Age

1 Year - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Triheptanoin — DRUG
  Open-label design with doses of triheptanoin up to 4.0 gm/kg triheptanoin

Study Details

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.

Key Dates

Start date

Jul 11, 2024

Status verified

Feb 2026

Primary completion

Dec 31, 2028

Completion

Jun 30, 2029

Study Design

Enrollment

6 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Triheptanoin
  Open label study

Primary Outcome Measure

Number of participants who report side-effects related to gastrointestinal (GI) distress [ Time Frame: 24 months ]

Central Contacts

• Angela Riemenschneider
  412-692-5232
  [email protected]
• Jirair Bedoyan, MD, PhD
  [email protected]

Locations (1)

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