IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
研究概览
简要总结
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
详细描述
Detailed Description:
DMD is a progressive degenerative muscle disorder for which there is no current cure. Glucocorticoids (GC) are often used to improve motor function and survival but have significant side effects such as growth failure, weight gain, insulin resistance and osteoporosis. IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy
- •Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
- •Age > 5 years of age
- •Bone maturation (assess by bone age x-ray): </= 11 years of age
- •Daily GC (prednisone or deflazacort) therapy for > 12 months
- •Ambulatory
- •Informed consent
- •Willingness and ability to comply with all protocol requirements and procedures
排除标准
- •Current or prior treatment with growth hormone or IGF-1 therapy
- •Non-ambulatory
- •Pubertal (based on clinical Tanner staging examination)
- •Congestive cardiac failure
- •History of intracranial hypertension
- •Daytime ventilatory dependence (non-invasive or tracheostomy)
- •Concomitant therapy - any other medications/supplements that would be considered, in the opinion of the investigators, to affect muscle function, need to have been started 3 months prior to enrollment
- •Patients enrolled in other clinical drug trials
- •Any physical or mental conditions which may, in the investigators'opinions, render the subject unable to complete the tasks of the study appropriately
- •There will be no selection by ethnicity
研究组 & 干预措施
IGF-1
IGF-1 plus standard steroid treatment
干预措施: IGF-1 (Drug)
结局指标
主要结局
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
时间窗: 6 months
Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
次要结局
- Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline(6 months)
- Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline(6 months)