CNMC0301: AN OPEN-LABEL PILOT STUDY OF COENZYME Q10

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This study evaluated the effect of Coenzyme Q10 on muscle strength, at a therapeutic level of 2.5 ug/ml or higher, in an open-label pilot study. The population included ambulant children aged 5 to 11 years with an established DMD diagnosis with a stable steroids regimen. Enrolled subjects were placed on oral CoQ10 supplements, after which a dose escalation period occurred to assure blood levels were in the desired range. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoint is percentage change in muscle strength as measured by Quantitative Muscle Testing (QMT) performance for specific muscle groups. Secondary outcome variables are manual muscle testing by Medical Research Council (MRC) score, timed function tests, functional grades for arms and legs and LVEF as assessed by 2-D echocardiogram (LVEF testing only at CNMC and selected institutions). If the drug shows an improvement in muscle strength following six (6) 28-day treatment cycles, a randomized placebo-controlled study will be developed to provide solid evidence of safety and efficacy.