3147K2-101-WW: A DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED, MULTIPLE ASCENDY

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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. The primary objective is to evaluate the safety of MYO-029 in adult subjects with muscular dystrophy. The secondary objectives are to (1) evaluate the biological activity of MYO-029 in adult subjects with muscular dystrophy by evaluating muscle mass, volume, strength, and histopathology; patient-reported outcome; and functional measurements; (2) explore associations between gene expression patterns, test article administration, and clinical parameters, and (3) evaluate the pharmacokinetics of MYO-029 in adult subjects with MD. This is a randomized, double blind, placebo-controlled, multiple ascending dose, safety study. Approximately 136 subjects will be assigned to 1 to 4 sequential dose cohorts 936 subjects each in Cohort 1 and 2 and 32-36 subjects each in cohort 3 and 4). Cohort 1- 1 mg/kg MYO-029 or placebo Cohort 2- 3 mg/kg MYO-029 or placebo Cohort 3- 10 mg/kg MYO-029 or placebo Cohort 4- 30 mg/kg MYO-029 or placebo Each subject will receive test article once every 2 weeks for a total of 13 doses. Equal numbers of subjects with BMD, FSHD, and LGMD will be enrolled in each cohort. The placebo design element in this study will provide a reference for no drug effects with respect to the safety, tolerability, and pharmacological activity of MYO-029 for the given study population. A double-blind design employed in conjunction with a placebo control will provide the study with protection against introduction of a bias into the study evaluation either by the investigatory or the subjects.