An Update on Clinical Trial Innovation for Duchenne – a Master Protocol Share | DuchenneXchange

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An Update on Clinical Trial Innovation for Duchenne – a Master Protocol Share


PPMD is committed to making research and development of new medicines for families living with Duchenne as fast and efficient as possible.  Over the years, by listening to you, we have developed a clinical trials imperative that we strive to live up to.

Trials should not be limited to a subset of patients, but rather inclusive of young and old, ambulatory and non-ambulatory, those on steroids and those who are not.

Exposure to placebo should be limited or non-existent.

Trials should end quickly if they are not showing positive results and participants should be given another opportunity in a timely manner.

Efficiencies are needed across clinical trials, including contract negotiation, a single Institutional Review Board (IRB), standardized protocols and outcome measures, opportunities for combinations, and faster approvals.

As part of making these imperatives a reality, PPMD is evaluating a master protocol for Duchenne, along with community stakeholders, and has partnered with the Institute for Advanced Clinical Trials for Children (I-ACT) to address the complex issues that running a large trial with multiple therapies entails.

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