PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
研究概览
简要总结
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greater than 2.5 ug/mL) and a combination arm (prednisone and CoQ10) with an enhanced standard of care arm in wheelchair confined males age 10 to 18 years with an established DMD diagnosis.
详细描述
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting 1:3500 male births worldwide. Despite an increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Improvement in the treatment of DMD will depend upon the development of better therapies. Affected boys become symptomatic at 3 to 5 years of age with proximal leg weakness that impairs mobility, ability to get up from a squat, and precludes a normal ability to run. By 8 years of age, some affected boys begin to lose the ability to walk and resort to a wheelchair for mobility. This shift from the ambulant to non-ambulant phase occurs in all boys with a diagnosis of DMD by age 12 years. In this study, participants will be randomized into groups after being screened to determine eligibility. Participants will then be followed for a 12-month investigation period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Age 10-18 years
- •Non-ambulatory (primary mode of transportation is via wheelchair for 3 years or less)
- •Confirmed DMD diagnosis
- •Steroid-naive for the 6 months prior to screening
- •Stable dose of b-blocker or ACE inhibitor medication for the 6 months prior to screening, if taking either of these medications
- •Ability to provide reproducible repeat QMT grip score within 15% of first assessment score
- •Has not participated in other therapeutic research protocol within the last 6 months prior to screening
- •Ability to swallow tablets
排除标准
- •Failure to achieve one or more of the diagnostic inclusion criteria cited above
- •Symptomatic DMD carrier
- •Use of carnitine, other amino acids, creatine, glutamine, CoQ10 or any herbal medicines (this would not include herbal teas unless they are consumed daily with intended medicinal effect) within the last 3 months
- •History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy
- •Positive PPD
- •No prior exposure to chickenpox and no immunization against chicken pox
- •Baseline serum CoQ10 level of 5.0mg/ml or greater
研究组 & 干预措施
1
CoenzymeQ10 taken once a day each morning by mouth.
干预措施: Coenzyme Q10 (Dietary Supplement)
2
Prednisone taken once a day each morning by mouth
干预措施: Prednisone (Drug)
3
CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
干预措施: Prednisone (Drug)
3
CoenzymeQ10 and prednisone each taken once a day in the morning by mouth.
干预措施: Coenzyme Q10 (Dietary Supplement)
结局指标
主要结局
One Year Change of Left Ventricular Mean Systolic Wall Stress/Rate-corrected Velocity of Fiber Shortening Relation.
时间窗: 12 months
Comparing change from baseline of mean systolic wall stress and rate-corrected mean velocity of circumferential shortening in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year. The values are obtained via an echocardiogram read locally at each site.
One Year Change in Pulmonary Function (Forced Expiratory Volume, FEV1 and Forced Vital Capacity, FVC)
时间窗: 12 months
Comparing change from baseline levels in pulmonary function (FEV1 and FVC) in the three treatment groups relative to the enhanced standard of care group and relative to each other at one year.
次要结局
- Compare Side Effect Profiles of the Three Study Groups(12 months)