Parent Project Muscular Dystrophy (PPMD), along with collaborators I-ACT for Children and Critical Path Institute (C-Path), host the first Duchenne Muscular Dystrophy Platform Trial Community Meeting on September 9, 2019.

This meeting, taking place at the Sheraton Silver Spring in Silver Spring, Maryland, will be an opportunity for stakeholders to provide input into the design of the platform trial and gain consensus on critical design elements.

(PPMD) is a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne).

The Duchenne Platform Trial is being designed to accelerate the development of effective therapies to treat Duchenne and will allow patients with Duchenne, ages five and over regardless of mobility, to have better access to clinical trials. It is a method to study multiple therapies at once, using a common placebo-controlled arm, which can accelerate the trial process, according to a media release from PPMD.

Specific goals are:

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

“Many families in our Duchenne community are familiar with the complicated, long, and often frustrating clinical trial process,” says Abby Bronson, PPMD senior vice president of research strategy and lead on the exploration of a platform trial design in Duchenne, in the release.

“PPMD has been thinking about how to make clinical trials faster, less burdensome, and open to all people with Duchenne, no matter the stage of disease. We hope to leverage some of the innovations that have catalyzed the cancer world. This is why we are interested in the platform trial idea — a trial designed to study multiple, different drugs in multiple patient types with one common placebo-controlled arm,” she adds.

To help develop the concept of a platform trial for Duchenne, PPMD partnered with organizations that have complementary skill sets.

“Developing a platform trial is a complex effort,” states C-Path Executive Director of the Duchenne Regulatory Science Consortium Jane Larkindale. “It requires expertise in a myriad of areas. The Duchenne platform team includes PPMD, which has vast expertise in the disease and the needs of patients; I-ACT for Children, a nonprofit set up through C-Path to improve the planning and development of pediatric clinical trials in Duchenne; and C-Path, which provides regulatory expertise and access to analytical tools to help develop the trial protocol.

“Additional Duchenne experts have also been invaluable in getting the trial to this point, and will be needed to turn it into a reality.”

“The Duchenne Platform Trial aligns perfectly with I-ACT for Children’s goal to catalyze improvements in the quality and timely completion of global pediatric studies to address the gap in evidence for best use of therapeutics in children,” I-ACT for Children CEO Laura Gordon comments. “Simply put, children are waiting. We must shorten the time it takes to bring innovative medicines and devices to them.”

Registration for the September 9 meeting in Silver Spring is closed, so the meeting will be streamed online.

[Source(s): Parent Project Muscular Dystrophy, PR Newswire]