Sodium Nitrate for Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Change in maximal handgrip strength
研究概览
简要总结
The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation. This was translated from work using the mdx mouse model of dystrophinopathy. Recent work has shown that there is a frank improvement in grip strength when mdx mice are treated with an inorganic Nitric Oxide (NO) donor. The purpose of this study is to determine if chronic treatment with an inorganic NO donor can benefit patients with muscular dystrophy beyond blood flow regulation.
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
- 15 Years 至 45 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of muscular dystrophy
- •Age 15-45 years of age
- •Ambulatory
- •No clinical evidence of heart failure
- •Maximum voluntary contraction, measured by hand grip dynamometer, of 20-40 kg
排除标准
- •Hypertension, diabetes, or heart failure by standard clinical criteria
- •Elevated B-type Natruiretic Peptide level (>100 pg/ml)
- •Left Ventricular Ejection Fraction < 50%
- •Wheelchair bound
- •Cardiac rhythm disorder, specifically: rhythm other than sinus, Supraventricular Tachycardia, atrial fibrillation, ventricular tachycardia
- •Continuous ventilatory support
- •Liver disease
- •Renal impairment
- •Contraindications to NO donors (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or Phosphodiesterase type 5 (PDE5) inhibitors) Glucocorticoid therapy and prophylactic use of Angiotensin Converting Enzyme (ACE) inhibitors and beta-blockers for cardiac protection will not be exclusion criteria.
- •Maximum voluntary contraction of less than 20 kg or greater than 40 kg
- •Ventilation Devices (Continuous Positive Airway Pressure (CPAP), Bilevel Positive Airway Pressure (BiPAP))
研究组 & 干预措施
Sodium Nitrate (Beetroot Juice)
Sodium nitrate in the form of beetroot juice will be administered orally. Patients will be assessed with a number of functional muscle assessments.
干预措施: Sodium Nitrate (Drug)
结局指标
主要结局
Change in maximal handgrip strength
时间窗: Change from baseline in handgrip strength at 3 months
次要结局
- Change in muscle function - Performance of Upper Limb Scale(Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months)
- Change in muscle function - 6 minute walk test(Change from baseline in muscle function - 6 minute walk test at 3 months)
- Change in systolic wall strain - imaging(Change from baseline in cardiac systolic wall strain at 3 months)
- Change in muscle tissue markers - histology and proteomics(Change from baseline in muscle tissue markers at 3 monthss)
- Change in muscle function - North Star Ambulatory Assessment(Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months)