Could a Fatty-Acid drug help kids with rare energy disorder?
NCT ID NCT06340685
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early study is testing a drug called triheptanoin (Dojolvi) in children with pyruvate dehydrogenase complex (PDC) deficiency, a rare genetic condition that affects energy production. The trial will enroll 6 children aged 1 to 18 and look at safety, side effects, and changes in blood markers like lactate. It is an open-label, proof-of-concept study, meaning everyone gets the drug and the main goal is to see if it is safe and worth studying further.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- triheptanoin (Dojolvi)
- What this could lead to
- If it works, this could point toward a treatment option for PDC deficiency, a rare metabolic disorder.
- What could go wrong
- This is a very small, early-phase study with only 6 participants. It is designed to test safety and biological signals, not to prove effectiveness. The drug may not improve symptoms or could cause side effects like stomach upset.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.