Sodium Nitrate for Muscular Dystrophy

• Status: Completed
• Trial end date: 2018-04-01
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cedars-Sinai Medical Center

Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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))