Parent Project Muscular Dystrophy, I-ACT for Children, and Critical Path Institute Host Duchenne Muscular Dystrophy Platform Trial Community Meeting


HACKENSACK, N.J., Sept. 5, 2019 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced that they 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.

Parent Project Muscular Dystrophy, I-ACT for Children, and Critical Path Institute Host Duchenne Muscular Dystrophy Platform Trial Community Meeting

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

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.

"Many families in our Duchenne community are familiar with the complicated, long, and often frustrating clinical trial process," said Abby Bronson, PPMD Senior Vice President of Research Strategy and lead on the exploration of a platform trial design in Duchenne. "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."

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.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne—our mission is to end Duchenne.

We demand optimal care standards and strive to ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won two FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at and follow PPMD on Facebook, Twitter, and YouTube

About I-ACT for Children

I-ACT for Children is an independent non-profit organization that believes that children of all ages deserve innovative medical therapies that are developed with the same level of urgency and commitment afforded adults. I-ACT for Children works with others to ensure that studies are designed to generate sufficient data to allow safe and effective use of new medications and devices in pediatric populations.

A key factor in making this a reality is to optimize and accelerate biomedical innovation using child-centered clinical trial networks and collaboration with like-minded institutions, trial sponsors and other stakeholders. Together with parents, patients, investigators, foundations, regulators, other government agencies, biopharmaceutical sponsors and children's networks, we work 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.

About Critical Path Institute

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit and


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