New hope for rare metabolic disease patients: expanded access to triheptanoin

NCT ID NCT03773770

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program provides expanded access to triheptanoin for people with long-chain fatty acid oxidation disorders (LC-FAOD) who have few treatment options and cannot join a clinical trial. The goal is to help manage the disease by providing an alternative energy source for the body. It is not a cure, but aims to control the condition and improve quality of life.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

carnitine-acylcarnitine translocase deficiency inherited fatty acid metabolism disorder mitochondrial trifunctional protein deficiency VLCAD deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••