Experimental drug aims to boost energy in rare genetic disorders
NCT ID NCT06792500
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare mitochondrial diseases that cause severe symptoms like strokes and vision loss. The trial is open-label (everyone gets the drug) and uses each person as their own control over 20 months. The goal is to check safety and see if the drug improves cellular energy production and daily function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
glycerol tributyrate
What this could lead to
If it works, this could point toward a treatment that improves energy production in cells for people with MELAS and LHON-Plus, potentially slowing disease progression.
What could go wrong
This is a very early, small trial (24 people) with no placebo group, so results may be hard to interpret. The drug may not show clear benefit, and side effects are unknown.
Disclaimer
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This is a summary of
the original study
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact