Could a simple fat compound help treat rare energy disorders?
NCT ID NCT06792500
First seen Dec 09, 2025 · Last updated May 22, 2026 · Updated 18 times
Summary
This study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare diseases that affect how cells produce energy. The goal is to find a safe dose and see if it improves daily activity, walking, and reduces hospital visits. Each participant serves as their own control, and the study lasts about 20 months.
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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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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Locations
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Conditions
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