First-of-its-Kind trial offers hope for devastating genetic disorders
NCT ID NCT06792500
Summary
This study is testing a new oral drug called glycerol tributyrate in adults with two rare and serious genetic disorders, MELAS and LHON-Plus. The main goals are to find a safe dose for each person and to see if the drug can improve how their cells produce energy. Each participant will take the drug for up to 20 months, and researchers will closely monitor their health and symptoms.
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
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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: •••••@•••••
Contact
Conditions
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