Could a fat compound help fight rare energy disorders?
NCT ID NCT06792500
First seen Dec 09, 2025 · Last updated Apr 30, 2026 · Updated 17 times
Summary
This study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare genetic diseases that affect energy production in cells. The goal is to see if the drug is safe and can improve energy metabolism. Each participant serves as their own control, receiving the drug for 20 months with close monitoring.
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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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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
RECRUITINGWashington D.C., District of Columbia, 20010, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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