PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 63
- 试验地点
- 7
- 主要终点
- myocardial performance index (MPI)
研究概览
简要总结
The study will include 120 participants aged 8 and up with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies that have no clinical cardiac symptoms. Participants will be randomized to one of four arms: Arm 1 CoQ10 alone, Arm 2 Lisinopril alone, Arm 3 CoQ10 and Lisinopril or Arm 4 No study medication. Randomization will be stratified by ambulatory status and corticosteroid use. The primary outcome for the study is the myocardial performance index (MPI), measured by standard Doppler echocardiography. The study will last 24 months with visits at Months 0.5,1.5, 6, 12, 18 and 24.
Following completion of the Clinical Trial of Coenzyme Q10 and Lisinopril, participants will be offered participation in a companion protocol: PITT1215 A Natural History Companion Study to PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies. The objective of this study is to evaluate the longitudinal natural history of DMD, BMD, and LGMD2I and to evaluate the effects of Coenzyme Q10 and/or Lisinopril on prevention of cardiac dysfunction in these disorders.This will be an 18-month longitudinal natural history study designed to accompany the Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Factorial
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •8 years of age or older
- •Confirmed genetic diagnosis of Duchenne, Becker, or Limb Girdle muscular dystrophy
- •Beta-blocker naïve
- •Screening Doppler echocardiographic MPI measurement greater than or equal to 0.40 for the highest MPI value (spectral and tissue) or circumferential strain measured by STE that is less negative than or equal to - 23
- •Normal left ventricular fractional shortening (≥28%) and no clinical cardiac symptoms
- •Has not participated in other therapeutic research protocol within the last 6 months prior to screening
- •Ability to swallow tablets
排除标准
- •Spine curvature greater than 30% (based on the x-ray performed at screening)
- •History of significant concomitant illness or significant impairment of renal or hepatic function
- •History of hypersensitivity to ACE inhibitors
- •History of idiopathic or hereditary angioedema or a history of angioedema with prior ACE inhibitor use
- •Use of carnitine, creatine, glutamine, or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) in the 3-months prior to enrollment
- •CoQ10 and/or ACE inhibitor use for a duration greater than 6 months
- •CoQ10 and/or ACE inhibitor use in the 3-months prior to enrollment
- •CoQ10 serum level of 2.5 ug/ml or higher
- •Investigator assessment of inability to comply with protocol
研究组 & 干预措施
Lisinopril
干预措施: Coenzyme Q10 and Lisinopril (Drug)
Coenzyme Q10
干预措施: Coenzyme Q10 and Lisinopril (Drug)
Coenzyme Q10 and Lisinopril
干预措施: Coenzyme Q10 and Lisinopril (Drug)
结局指标
主要结局
myocardial performance index (MPI)
时间窗: every 6 months
The MPI is a sensitive, quantifiable, noninvasive measure of global ventricular function that is independent of cardiac geometry and heart rate. MPI is collected through standard echocardiogram assessment. MPI is a ratio of the total time spent in isovolumic activity (isovolumic contraction time and isovolumic relaxation time) to the time spent in ventricular ejection.
次要结局
未报告次要终点