UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Plasma BNP
研究概览
简要总结
This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults who will receive the purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. After screening visits, participants will be enrolled in the study if they meet all inclusion criteria. They will be evaluated at screening, baseline, and weeks 4, 8, 12, 24, 16 and 48. The main criterion for success of the study will be presence of one or more biologic or strength and performance outcome measures that yield a response magnitude that allows for sufficient power in a Phase II B study with a sample size of 30 individuals.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Prior participation in UCD0113 BMD epicatechin pilot study
- •Age 18 years to 70 years
- •Average to low daily physical activity
- •Ability to ambulate for 75 meters without assistive devices
- •Diagnosis of BMD confirmed by at least one the following:
- •Dystrophin immunofluorescence and/or immunoblot showing partial dystrophin deficiency, and clinical picture consistent with typical BMD, or
- •Gene deletions test positive (missing one or more exons) of the dystrophin gene, where reading frame can be predicted as 'in-frame', and clinical picture consistent with typical BMD, or
- •Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with BMD, with a typical clinical picture of BMD, or
- •Positive family history of BMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of BMD.
- •Hematology profile within normal range
- •Baseline laboratory safety chemistry profile within normal range
- •No plan to change exercise regimen during study participation
- •Nutritional, herbal and antioxidant supplements taken with the intent of maintaining or improving skeletal muscle strength or functional mobility have been discontinued at least 2 weeks prior to screening (daily multivitamin use is acceptable).
排除标准
- •Currently enrolled in another treatment clinical trial.
- •History of significant concomitant illness or significant impairment of renal or hepatic function.
- •Use of regular daily aspirin or other medication with antiplatelet effects within 3 weeks of first dose of study medication.
- •Regular participation in vigorous exercise.
- •Symptomatic heart failure with cardiac ejection fraction <25%
研究组 & 干预措施
Treatment
(-)-epicatechin 50mg twice per day (100mg per day total dose)
干预措施: (-)-Epicatechin (Drug)
结局指标
主要结局
Plasma BNP
时间窗: 48 weeks
blood biomarker concentration
Plasma Follistatin
时间窗: 48 weeks
blood biomarker concentration
Plasma Myostatin
时间窗: 48 weeks
blood biomarker concentration
Plasma Nitrates/ SNO
时间窗: 48 weeks
blood biomarker concentration
Plasma TGF-Beta
时间窗: 48 weeks
blood biomarker concentration
Plasma Creatine Kinase
时间窗: 48 weeks
blood biomarker concentration
Plasma MMP-9
时间窗: 48 weeks
blood biomarker concentration
Plasma TNF-Alpha
时间窗: 48 weeks
blood biomarker concentration
Plasma Follistatin:Myostain Ratio
时间窗: 48 weeks
Ratio of plasma follistatin to plasma myostatin
次要结局
- Graded Exercise Test Using a Recumbent Cycle Ergometer(baseline and at 2-minute intervals)
- 6-minute Walk Test(48 weeks)
研究者
Craig McDonald, MD
Principal Investigator
University of California, Davis