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Saving lives through education, advances in treatment and finding a cure for Barth syndrome

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CARDIOMAN Clinical Trial Results Summary Released

Researchers at the University Hospitals Bristol  in the UK completed the second-ever clinical trial in Barth syndrome: Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN) - A randomised placebo-controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service.

 

The lay summary of results can be read here.

 

Members of the Barth syndrome community in the UK participated in a 9-month trial in 2019 to assess whether bezafibrate could be successfully repurposed to improve health outcomes in Barth syndrome. Bezafibrate is an approved and widely used drug in many countries, although it is not approved for use in the US.

 

Although data from the trial did not show a statistically significant improvement in the primary clinical outcome (Peak body oxygen uptake during static bicycle exercise/Vo2 max) and quality of life scores did not change significantly, there were several interesting findings regarding heart strain values and amino acid levels.

 

Full study results will be released by the Bristol research team in the future. “As we await the full results from this trial, we are thankful for the participants and researchers who have contributed so much time, effort, and energy towards designing, executing, and accomplishing a fully-recruited clinical trial for our ultra-rare community,” says Dr. Erik Lontok, Director of Research for the Barth Syndrome Foundation.

 

Despite this outcome, CARDIOMAN marks an important milestone. “We have learned valuable lessons through this first UK-based clinical trial for our rare condition and I have no doubt that this will have a positive impact on future trials. Our community showed incredible engagement and fortitude in participating in this study. Without them, this study would never have been possible. They are the heroes we all have to thank,” says Michaela Damin, Founder and CEO of Barth Syndrome UK. “We would also like to thank the National Institute for Health Research (NIHR)’s Efficacy and Mechanism Evaluation (EME) Programme for funding the trial and the Barth Syndrome Foundation who sponsored the additional cost of resizing the tablets to make them available to more people in our community.”

 

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