Groups Says New Measure Will Improve DMD Trial Design, Provide Predictable Results

September 26, 2019

The group published its findings about the NSAA, a 17-point rating scale to measure functional motor abilities in children with DMD who are able to walk, in the journal PLOS ONE.

The NSAA outcome measures DMD patients engaging in a variety of activities including walking, hopping, running, and others, expressed as a total score. The group said the NSAA provides quantitative insights that can assist academics, drug developers, and regulators in the design and analysis of clinical trials in patients with Duchenne muscular dystrophy. NSAA better characterizes the heterogeneity of rate of disease progression in DMD than other measures used today. It also provides a quantitative risk assessment of the likelihood that a patient will gain, remain stable, or lose function on each activity in the new assessment over time.

“The comprehensive assessment of the NSAA in this study will be invaluable for DMD clinical trial design and interpretation of findings,” said Francesco Muntoni, director of the Dubowitz Neuromuscular Centre, University College London. “Although the NSAA is being used as a primary clinical endpoint, disease progression using this endpoint has not previously been fully characterized for use in clinical trials.

The study involved the analysis of clinical data from 395 patients with DMD curated by the UK’s North Star Network.

Susan Ward, executive director of cTAP, said the article shows how pooling real-world patient data can lead to improvements in trial design, and ultimately new, effective treatments for Duchenne muscular dystrophy.

“This study is an excellent example of different stakeholders in the community pulling together to accelerate insights that advance the development of treatments for patients with Duchenne,” she said.

Photo: Susan Ward, executive director of cTAP  

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