Parent Project Muscular Dystrophy (PPMD) will co-host the first Duchenne Muscular Dystrophy Platform Trial Community Meeting, along with collaborators I-ACT for Children and Critical Path Institute (C-Path) on September 9, 2019 at the Sheraton Silver Spring in Silver Spring, Maryland.  This meeting will be an opportunity for stakeholders to provide input into the design of the platform trial and gain consensus on critical design elements.

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.

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

Join the Livestream

This meeting will be streamed live on our website for those unable to attend the event in person starting at 8:30 AM eastern time. Click here to join the livestream. 

About the Meeting

Since 2016, PPMD has been closely examining the potential of a platform trial for Duchenne, including studying the history of this concept in other diseases and having in-depth discussions with clinical trial experts who have developed platform trials. At every step of the way, the Duchenne community – including clinicians, drug developers, and patients – have been invited to participate, so that all stakeholder questions and concerns could be included in this exploration.

Last year, PPMD launched a collaborative effort with community volunteers, I-ACT for Children, and C-Path to explore the idea of a Duchenne platform trial because of a shared belief that ending Duchenne will require creative thinking and innovative options. A platform trial is intended to make the clinical trial process more efficient. This is especially important with a rare disease such as Duchenne, which has a small population of patients to recruit.

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,” said 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.”

PPMD has a long history with C-Path, and is a member of the Duchenne Regulatory Science Consortium (D-RSC).

“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,” said I-ACT for Children CEO Laura Gordon. “Simply put, children are waiting. We must shorten the time it takes to bring innovative medicines and devices to them.”

To learn more about the potential of a platform trial design in Duchenne, including PPMD’s exploration of successful designs in other diseases, watch last year’s introductory webinar. Registration for the September 9 meeting in Silver Spring is closed. The meeting will be streamed online, click here for more information.