“Duchenne muscular dystrophy (DMD) is a, rare, progressive and ultimately fatal pediatric genetic disease that affects male children. Parents usually learn that their seemingly healthy son has DMD at age 4 to 5. By age 9 to 10, afflicted boys typically lose the ability to walk, eat and care for themselves, eventually becoming reliant on a ventilator. These patients commonly die in their 20s of respiratory complications or cardiomyopathy. There are currently no effective treatments.

Fortunately, there are very promising clinical candidates entering phase II and III trials. The problem is that the only validated endpoint for DMD drug approval is the Six Minute Walk Test, a measure that is most sensitive at a time when the boys are still ambulatory but beginning to lose the ability to walk. Furthermore, any time spent in the placebo arm of a trial for an effective treatment represents a lost opportunity for those participants. Some boys in the placebo arm may plateau at a much lower functional level after they are crossed over to the drug, or they may lose the ability to walk altogether. This loss of ambulation can mean that the patient is no longer eligible for future trials, and can lead to a loss of hope for the family involved.

We need to think about creative trial designs to minimize the amount of time spent in the placebo treatment, as well as different endpoints with faster, more efficient ways to validate them.”

– Sharon Hesterlee, PhD, VP Research, Parent Project Muscular Dystrophy

This piece was featured in CTTI’s 2013 Annual Report. To view this document, CLICK HERE.