Low-level Mechanical Vibration, Bone Density, Bone Resorption and Muscular Strength in Ambulant Children Affected by Duchenne Muscular Dystrophy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 20
- 主要终点
- Change in bone mineral density at lumbar spine.
研究概览
简要总结
Duchenne muscular dystrophy (DMD) is a X-linked recessive disorder due to a mutation of the dystrophin gene (Xp21). Dystrophin is a sarcolemmal protein of skeletal and cardiac muscle, and its absence causes progressive muscle degeneration and substitution with fat and connective tissue. The progressive muscle degeneration leads to loss of autonomous walking before the age of 15 years and death for cardiac and/or respiratory failure. There are no specific treatment for DMD, and the standard of care is now based on long-term corticosteroid (CS) use. The studies on bone mass in DMD are very few, but they agree in reporting the presence of a reduced bone mass and an increased rate of fractures probably due to long-term steroid therapy and disuse-osteopenia. The aim of this study, involving 20 ambulant DMD boys (age 7-10 years) has been the evaluation of the effects of low-level mechanical vibrations on bone in a group of ambulant DMD children for 1 year, with RDA-adjusted dietary calcium intake and 25OH vitamin D supplementation.
详细描述
All children were instructed to have a daily intake of calcium equal to the 100% of the RDA and were supplemented with calcifediol (0.7 mcg/kg/die).
The 20 boys were randomly assigned to two groups:
group 1 (mechanical intervention group) = a mechanical device (a small platform designed to induce vertical, sinusoidal acceleration) was installed in the home of each boy of group 1.
group 2 (placebo control group) = a placebo device was installed in the home of each boy of group 2
All boys were instructed to stand on the platform for 10 minutes each day for 12 months.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 7 Years 至 10 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of DMD
- •Ability to stand up and walk (some balance assistance allowed, but full weight-bearing necessary)
- •All the children must already be on glucocorticoid therapy for at least 6 months before the start of the study.
排除标准
- •Presence of other diseases interfering with bone density and bone turnover
- •The inability to regularly use the vibratory platform.
结局指标
主要结局
Change in bone mineral density at lumbar spine.
时间窗: baseline and 12th month
Bone mineral density evaluated by DXA. Bone mineral apparent density calculated to correct for bone size (growing subjects). Z-score calculated. Measurements: baseline and 12 months.
次要结局
- 25-OH vitamin D(baseline and 12th month)
- 1,25(OH)2 vitamin D(baseline and 12th month)
- Magnesium(baseline and 12th month)
- Parathyroid Hormone(baseline and 12th month)
- Calcium(baseline and 12th month)
- Phosphate(baseline and 12th month)
- Bone Alkaline Phosphatase(baseline and 12th month)
- Osteocalcin(baseline and 12th month)
- Creatinine(baseline and 12th month)