In this study, the primary aim will be to estimate the magnitude and variability of strength
change over time that may be expected for subjects on the study treatment. This estimate of
effect will allow us to develop a rigorous statistical plan in the future randomized study.
The specific estimation technique to be applied will use a linear random effects model to
estimate average strength change during the 3-month lead-in period and then during the
twelve-month treatment period, taking into account the quantitative muscle testing (QMT)
measures for each subject. Accounting for the correlation between repeated measures from each
subject by using a random effects model will yield an unbiased estimate of variability for
the population average change in strength. We will use an analysis of pre- and post-treatment
data to inform a best estimate of treatment effect. For example, the difference in QMT trends
pre- and post-treatment would provide a straightforward measure of efficacy.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Cooperative International Neuromuscular Research Group