PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 2: Optimizing Clinical Trials

This week, PPMD convened Duchenne industry partners, experts in adaptive trial design and biostatistics, and innovative partners from outside of the Duchenne world who’ve revolutionized their clinical trial spaces for the second meeting of our Duchenne Drug Development Roundtable's 3-part meeting series, focusing on Clinical Trial Optimization.

According to ClinicalTrials.gov, there are over 50 interventional trials open for Duchenne. And while we couldn’t be happier that there are so many “shots on goal”, as a community we are committed to minimizing the length of these trials, the number of patients required, and the exposure to placebo  while maximizing the robustness of the results.

In short, we want those shots on goal to yield “wins”.

Discussion Topics

The 1st meeting of the 3-Part meeting series took place on February 1st. That meeting included much thoughtful discussion of some of the issues clinical trial participants and families when participating in clinical trials. Several themes emerged focusing on bringing the trial sites to the patients via mobile devices and other technology advancements, increasing awareness among treating clinicians of available trial options, and creating mobile apps that can collect real world information more efficiently. The planning team for the first small meeting is eager to stay engaged and move initiatives forward.

During this 2nd meeting held on March 1st, we explored adaptive trial design using a Bayesian approach and platform trials. The Bayesian approach is a statistical methodology built with dynamic elements so that aspects of the trials can evolve as the trial is in process. These have to be thought through upfront, but the result can be smaller, shorter trials. We also heard from partners in the cancer community who presented on an alternative model of clinical trial design, the platform trial. While the applicability to Duchenne needs to be thoroughly examined, our industry partners are incredibly committed to exploring innovative paths forward.

Additionally, we discussed novel endpoints in trials and the day ended with a discussion of natural history data being used as control data in clinical trial and some examples from Duchenne.

From this meeting, we hope to have some direction as to the best path to pursue for optimizing clinical trials in the Duchenne space. With so much learned over the past several years, we hope this meeting is a chance for our industry partners to incorporate what we've learned into current trial design and to come up with new ideas.


About PPMD's Duchenne Drug Development Roundtable

Established more than seven years ago, PPMD's Duchenne Drug Development Roundtable (DDDR) is a group of committed innovators representing Industry and relevant stakeholders that has the goal of accelerating the development of meaningful treatments for Duchenne muscular dystrophy through open discussion to minimize duplication and to pool resources in pre-competitive space.

PPMD appreciates the support of our partners which enables the DDDR to continue driving the field forward.


We are extremely grateful to the DDDR Steering Committee and the company representatives who have served as planning committee members for each of the three meetings. Their guidance and leadership has impacted our Duchenne community in immeasurable ways.


The DDDR Steering Committee includes:

  • Cliff Bechtold (BMS)
  • Michael Binks (Pfizer)
  • David Cox (Eli Lilly)
  • Joanne Donovan (Catabasis)
  • Cartier Esham (BIO)
  • Ed Kaye (Sarepta)
  • Kim McCleary (FasterCures)
  • Thomas Meier (Santhera)
  • Jeff Watkins (patient community representative, parent)


This Roundtable series summary will be disseminated to all DDDR members in time for discussion at our annual DDDR meeting in June. We hope that all of our Duchenne partner organizations will continue to join us in this annual discussion and planning meeting.

It is our hope that this DDDR meeting series will assist PPMD and the Duchenne community to work together to drive forward collaborations and projects that will benefit all partners working in this space – and yield effective therapies that are accessible to all who need them.

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