Duchenne Muscular Dystrophy Clinical Trial
Official title:
Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS
The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. The data obtained from this study will be used to make recommendations for strength measurement endpoints in prospective muscular dystrophy trials and provide more reliable and accurate recommendations in the clinic for strength assessment. This study will be performed at six participating sites in the Cooperative International Neuromuscular Research Group (CINRG).
We propose to compare the reliability of CQMS vs. HHM in the DMD population. The importance
of this study is to be able to compare the results of clinical trials done by different
networks using distinct strength endpoints. If results indicate a distinct difference in
reliability of one tool over the other, a standardized tool could be established for
research groups throughout the world to interpret strength in the context of clinical
trials. If the results indicate minor differences then it would be possible to interpret and
compare/contrast strength measurements used in different studies.
Understanding the relationship between the HHM vs CQMS will help us examine other surrogate
measures capable of predicting functionality that are based on strength measurements.
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Observational Model: Case-Crossover, Time Perspective: Prospective
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