New shot aims to help kids with rare heart-muscle disease move easier
NCT ID NCT07531251
First seen Jun 27, 2026 · Last updated Jul 14, 2026 · Updated 2 times
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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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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Locations
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Bristol Royal Hospital for Children Upper Maudlin Street Paul O'Gorman Building
RECRUITINGBristol, United Kingdom
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Metabolics and Genetics in Canada (MAGIC)
NOT_YET_RECRUITINGCalgary, Alberta, Canada
Contact Email: •••••@•••••
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Trial Not Offered in the U.S
TERMINATEDNeedham, Massachusetts, 02494, United States
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