New shot aims to help kids with rare heart-muscle disease move easier
NCT ID NCT07531251
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug improves walking, standing up, and moving around.
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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
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building
Bristol, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Trial Not Offered in the U.S
Needham, Massachusetts, 02494, United States