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

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 主要目的是评估MYO-029在成人肌营养不良症患者中的安全性。 次要目标是(1)通过评估肌肉质量、体积、力量和组织病理学、患者报告的结果和功能测量来评估MYO-029在成年肌营养不良患者中的生物活性；(2)探索基因表达模式、试验品给药和临床参数之间的关联；以及(3)评估MYO-029在MD成人患者中的药代动力学。 这是一项随机、双盲、安慰剂对照、多次递增剂量的安全性研究。约136名受试者将被分配到1至4个序贯剂量队列，第1和第2组各有936名受试者，第3和第4组各有32至36名受试者)。 队列1-1毫克/公斤MYO-029或安慰剂 队列2-3毫克/公斤MYO-029或安慰剂 队列3-10毫克/公斤MYO-029或安慰剂 队列4-30毫克/公斤MYO-029或安慰剂 每名受试者将每两周接受一次测试物品，共13剂。同样数量的BMD、FSHD和LGMD受试者将被纳入每个队列。这项研究中的安慰剂设计元素将为给定研究人群中MYO-029的安全性、耐受性和药理活性方面的无药物影响提供参考。与安慰剂对照结合使用的双盲设计将为研究提供保护，防止研究人员或受试者在研究评估中引入偏见。

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.