Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial

Objective: To assess the feasibility, safety, and efficacy of intracoronary allogeneic cardiosphere-derived cells (CAP-1002) in patients with Duchenne muscular dystrophy (DMD). Methods: The Halt Cardiomyopathy Progression (HOPE)-Duchenne trial is a phase I/II, randomized, controlled, open-label trial (NCT02485938). Patients with DMD >12 years old, with substantial myocardial fibrosis, were randomized (1:1) to usual care (control) or global intracoronary infusion of CAP-1002 (75 million cells). Participants were enrolled at 3 US medical centers between January and August 2016 and followed for 12 months. An independent Data and Safety Monitoring Board provided safety oversight. Cardiac function and structure were assessed by MRI, and analyzed by a blinded core laboratory. Skeletal muscle function was assessed by performance of the upper limb (PUL). Results: Twenty-five eligible patients (mean age 17.8 years; 68% wheelchair-dependent) were randomized to CAP-1002 (n = 13) or contro...研究目标：评估冠状动脉内输注同种异体心肌球来源细胞（cardiosphere-derived cells, CAP-1002）用于杜氏肌营养不良症（Duchenne muscular dystrophy, DMD）患者的可行性、安全性与有效性。 研究方法：本项「延缓心肌病进展（HOPE-Duchenne）」试验为I/II期随机对照开放标签临床试验（临床试验注册号：NCT02485938）。纳入年龄＞12岁、存在显著心肌纤维化的DMD患者，以1:1的比例随机分配至常规治疗组（对照组）或CAP-1002全域冠状动脉内输注组（细胞输注剂量为7500万个）。受试者于2016年1月至8月间在美国3家医学中心入组，随访周期为12个月。独立数据与安全监察委员会负责安全性监管。采用磁共振成像（Magnetic Resonance Imaging, MRI）评估心脏结构与功能，并由设盲的中心实验室完成数据分析。骨骼肌功能通过上肢功能测试（Performance of the Upper Limb, PUL）进行评估。 研究结果：共计25例符合入组标准的患者（平均年龄17.8岁；68%需依赖轮椅）被随机分配至CAP-1002组（n=13）或对照组（原文内容截断至此）。