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Triheptanoin versus MCT in pediatric LC-FAOD
jRCT2031240472Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Mar 2025
- Modality
- Small Molecules
01
Overview
Randomised, double-blind trial in long-chain fatty acid oxidation disorder (LC-FAOD) comparing Triheptanoin with MCT oil (2 arms). Primary endpoint: Annualized event rate of major clinical events. Started Mar 2025; status: completed.
02
Drugs
triheptanoinmedium-chain triacylglycerol oil
03
Conditions
long-chain fatty acid oxidation disorder (LC-FAOD)
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