A Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of Orally Administered (+)-Epicatechin in Patients With Becker or Becker-like Muscular Dystrophy With Continued Ambulation Past 16 Years of Age
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 3
- 主要终点
- Number of participants with treatment-emergent adverse events (TEAEs)
研究概览
简要总结
This is a Phase 1, open-label, dose escalation study aimed at evaluating the safety, early efficacy and potential biomarkers of (+)-epicatechin in patients with Becker or Becker-like Muscular Dystrophy (BMD).
详细描述
The safety and tolerability of three escalating doses of (+)-epicatechin will be assessed and early effectiveness measured by changes in plasma biomarkers, tissue biomarkers from muscle biopsies, cardiac imaging, and on clinical function assessments of participants' muscle strength. All patients will receive oral (+)-epicatechin for a total duration of approximately 52 weeks. Three doses of (+)-epicatechin will be tested in sequential 2 month periods with total daily doses of 75, 150, and 225 mg/day (+)-epicatechin. Doses will be escalated every 2 months, if tolerated, for the first 6 months of the study. Participants will then continue to receive the highest does they tolerated for an additional 6 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 59 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Dose escalation of (+)-epicatechin
Subjects will receive escalating doses of (+)-epicatechin starting at 75 mg/day and progressing to 150 mg/day and 225 mg/day with 2 months treatment duration for each dose. Subjects will continue treatment on the individual's maximum tolerated dose for another 6 months.
干预措施: (+)-Epicatechin (Drug)
结局指标
主要结局
Number of participants with treatment-emergent adverse events (TEAEs)
时间窗: Through study completion, up to 1 year
The TEAEs will be graded using the adult National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 5.0).
次要结局
- Change in cardiac function as assessed by cardiac magnetic resonance imaging (MRI)(Through study completion, up to 1 year)
- Change in cardiac function as assessed by plasma biomarkers [e.g. pro-B-type natriuretic peptide (pro-BNP), nitrates].(Through study completion, up to 1 year)
- Change in muscle function as assessed by 6-minute walk test (6MWT)(Through study completion, up to 1 year)
- Change in muscle function as assessed by Time to Run/Walk 10-meter Test (TTRW10)(Through study completion, up to 1 year)
- Change in muscle function as assessed by Time to 4-stair Climb Test (TT4SC)(Through study completion, up to 1 year)
- Change in muscle function as assessed by Time to Run/Walk 100-meter Test (TTRW100)(Through study completion, up to 1 year)
- Change in muscle structure and function as assessed by Western blot analysis of biopsy specimens (e.g. dystrophin expression)(Through study completion, up to 1 year)
- Change in muscle biomarkers of regeneration in biopsy specimens (e.g. follistatin)(Through study completion, up to 1 year)
- Change in plasma biomarkers of muscle regeneration (e.g. follistatin, myostatin)(Through study completion, up to 1 year)