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Special oil shows promise for rare brain energy disorder

NCT ID NCT02014883

First seen Oct 31, 2025 · Last updated Jun 21, 2026 · Updated 35 times

Summary

This phase 2 trial tested triheptanoin oil in 20 people with GLUT1 deficiency syndrome, a rare genetic disorder that prevents sugar from fueling the brain. The goal was to see if this special oil could reduce seizures and abnormal movements. Researchers monitored participants for side effects and adjusted doses as needed.

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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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Contacts and locations

Locations

  • Brain and Spine Institute

    Paris, 75013, France

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

triheptanoin oil

What this could lead to

If it works, this could provide a new treatment option to reduce seizures and abnormal movements in people with GLUT1 deficiency syndrome.

What could go wrong

This is a small, early-phase trial with only 20 participants, so results may not apply to everyone. The treatment may cause side effects like elevated blood levels of certain metabolites, requiring dose adjustments.

Conditions

The condition(s) this trial relates to.

encephalopathy due to GLUT1 deficiency GLUT1 deficiency syndrome

As listed by the trial registrant

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