Clinical Outcome Measures in DMD - Meeting Summary

On June 28 and 29, 25 participants from Europe and the US met in Washington DC to discuss Clinical Outcome Measures in Duchenne Muscular Dystrophy in the context of clinical trial design.  The meeting was organized by TREAT-NMD and the Children’s National Medical Center and supported by CureDuchenne, the Foundation to Eradicate Duchenne and Ryan’s Quest.

This meeting was planned as a follow-up to the September 2009 EMA meeting where a clear directive was given that an international consensus needed to be developed to provide guidance on age appropriate clinical outcome measures for use in clinical trials for DMD, especially as these relate to clinically meaningful events. In order to establish the international dataset on which to base such decisions, in this meeting, results from 8 natural history data sets, representing over 1500 patients, were collectively examined to create a robust and contemporary picture of disease progression. The various outcome measures used in these studies were discussed to determine their sensitivity, reliability and applicability in clinical trials as well as relationship to disease progression.  Other topics included new methods to analyze data, gaps in age/status of current datasets, and the need to standardize data collection.

The 8 data sets presented were:

• CINRG DMD Natural History Study
• UK North Star Project
• Italian North Star Project
• UDP
• MFM
• Danish DMD Dataset
• PTC 124 trial control group data
• MD-NET cyclosporine trial dataset

The meeting demonstrated that we have a strong foundation on which to base decisions around clinical trial design in DMD.  There is no doubt that trial design in DMD is challenging given the complexity and variability of the disease progression but when all the results from the prospective and retrospective datasets is put together, much is known about the current natural history of DMD.   Outcome measures are reasonably well defined making multicenter clinical trials feasible. The value of cross-collaborative efforts of clinicians, researchers, academics and advocacy together with industry cannot be overestimated in driving forward this paradigm.

A comprehensive meeting report will be published in mid-September.