On November 8, 2021, Dr. Colin Steward (Emeritus, U. Bristol), Dr. Guido Pieles (U. Bristol), Dr. Barney Reeves and Lucy Dabner (Bristol Trials Centre) discussed the research basis, findings, challenges, and successes for the UK-based CARDIOMAN trial. Sponsored by the UK National Institute for Health Research, co-funded by BSF, informed, designed, and led by long-time academic research leaders in our field, and ultimately made possible by the initial participation of eleven affected individuals - this nearly 8-year effort encapsulates the adage that it takes a village.
During this informal discussion of results, the presenters, SMAB members, and long-time researchers in our community joined participants and members of the BSF community to learn firsthand of how the trial did not meet its primary endpoint of improved peak oxygen consumption (VO2 max). Extended discussions regarding study design and secondary cardiac monitoring endpoints indicate the need for further analysis, with the potential to impact Barth syndrome clinical care.
Although CARDIOMAN is the second trial in our ultra-rare space to not have met its primary endpoints, this effort further demonstrates that ours is a clinical trial ready and capable population. And as we identify the scientific and programmatic learnings of both trials in our field, we look forward to applying them towards our next research study, trial, and effort – because we are a village that will never ever give up. To watch the full recording, click here.