Sodium Nitrate for Muscular Dystrophy

Cedars-Sinai Medical Center5 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Becker Muscular Dystrophy

Interventions

Sodium NitrateDRUG

Patients will be given sodium nitrate daily in the form of beetroot juice.

Eligibility

Sex: MALEMin age: 15 YearsMax age: 45 Years

Medical Language ↔ Plain English

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))

Locations (1)

Cedars-Sinai Medical Center

Los Angeles, California, United States

Outcomes

Primary Outcomes

Change in maximal handgrip strength

Time frame: Change from baseline in handgrip strength at 3 months

Secondary Outcomes

Change in muscle function - Performance of Upper Limb Scale

Change in functional muscle assessment as measured by the Performance of Upper Limb Scale

Time frame: Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months

Change in muscle tissue markers - histology and proteomics

Change in tissue markers such as neuronal Nitric Oxide Synthase (nNOS) content and location and nitrosative stress by histology and proteomics

Time frame: Change from baseline in muscle tissue markers at 3 monthss

Change in systolic wall strain - imaging

Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging

Time frame: Change from baseline in cardiac systolic wall strain at 3 months

Change in muscle function - North Star Ambulatory Assessment

Change in functional muscle assessment as measured by the North Star Ambulatory Assessment

Time frame: Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months

Change in muscle function - 6 minute walk test

Change in functional muscle assessment as measured by the 6 minute walk test

Time frame: Change from baseline in muscle function - 6 minute walk test at 3 months

Data from ClinicalTrials.gov

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