DMD Stakeholder Meeting

Platform Trial Stakeholder Meeting Brings Duchenne Community Together to Discuss Innovative Protocol

On Sept. 9, more than 100 stakeholders from the Duchenne muscular dystrophy (DMD) community gathered in Silver Spring, Md., to discuss the novel platform trial being proposed by I-ACT for Children, Parent Project Muscular Dystrophy and the Critical Path Institute. The goal of the platform trial is to speed the development of therapies for DMD by creating a platform in which multiple compounds can be tested at once. Potential benefits include:

• More rapid completion of testing and approval of new DMD therapies
• Reduced clinical trial start-up and execution time
• Enhanced patient experience, reduced number on placebo
• Potential for rapidly testing combination therapies

At the Sept. 9 meeting, researchers, Duchenne parents, advocacy groups, FDA representatives and drug sponsors met to learn the details of the trial protocol and provide feedback on topics such as proposed eligibility criteria, endpoints, trial structure and governance. FDA's Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research, and Dr. Billy Dunn, Director of CDER's Division of Neurology Products, spoke at the start of the meeting about FDA's interest in platform trials and their potential benefits for the DMD community. "We need to get work done and do it efficiently," Dr. Dunn told the stakeholder audience. "That's what this effort is all about."