In June 2010, 25 representatives from Europe and the US met in Washington, DC, USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the context of clinical trial design and analysis. The workshop was organized in response to a September 2009 European Medicines Agency meeting where a clear directive was given that an international consensus needs to be developed that provides a foundation for age-appropriate clinical outcome measures for use in clinical trials of emerging therapeutics for DMD. Data were presented from eight multicenter longitudinal datasets, representing nearly 1900 patients over a 20-year time period. This experience confirmed the feasibility of repeated evaluations performed at multiple sites and addressed several core issues in drug development for DMD, such as the ‘new’ natural history in the steroid-era, reliability and sensitivity of specific outcome measures, as well as disease staging and patient selection. These data form a valuable asset for academic investigators, pharmaceutical sponsors and regulatory agencies involved in DMD therapeutics. The group
remains committed working together on a number of collaborative goals to support the therapeutics development effort in this orphan disease and to make these data available to stakeholders working in the field.
The aims of the meeting were to:
- Map the outcome measures currently used in DMD natural history studies and clinical trials, and to highlight and assess the data currently being collected;
- Establish the current expected clinical course of the disease from these contemporary datasets;
- Determine whether data exist to define the relationship between the outcome measures and milestones of disease progression;
- Determine areas where the available data clearly describe the disease and where gaps in data highlight the need for further work.
Kate Bushby & Edward Connor, For the International DMD Clinical Outcomes Working Group. A list of all members of the working group can be found at the end of this article.
Disease Category: Muscle disease
Disease Name: Duchenne muscular dystrophy
Age Range: 0 - 18
Sex: Either
Nature of Intervention: Not specified
- Academic research representatives
- Methodologists
- Study investigators
- Systematic review of outcome measures/measurement instruments
- Systematic review of outcomes measured in trials
- Literature review
- Semi structured discussion
On 28–29 June 2010, 25 participants from Europe and the US met in Washington, DC, USA, to discuss clinical outcome measures in DMD in the context of clinical trial design. The workshop was organized by TREAT-NMD and Children’s National Medical Center and was supported by Cure Duchenne, the Foundation to Eradicate Duchenne and Ryan’s Quest. A followup meeting was held in Naples on 21 July 2010. Participants included representatives of international studies of DMD natural history and clinical trials experts.
Preliminary work - To establish the international dataset on which to base the discussion, preliminary work identified eight natural history and clinical trial datasets, representing over 1900 patients who are being, or have been, prospectively followed. This work took place over the winter and spring of 2009–2010.