Becker Muscular Dystrophy
Conditions
Keywords
Becker, Dystrophy
Brief 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.
Interventions
DRUGSodium Nitrate
Patients will be given sodium nitrate daily in the form of beetroot juice.
Sponsors
Cedars-Sinai Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
15 Years to 45 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in maximal handgrip strength | Change from baseline in handgrip strength at 3 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in muscle function - North Star Ambulatory Assessment | Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months | Change in functional muscle assessment as measured by the North Star Ambulatory Assessment |
| Change in muscle function - 6 minute walk test | Change from baseline in muscle function - 6 minute walk test at 3 months | Change in functional muscle assessment as measured by the 6 minute walk test |
| 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 functional muscle assessment as measured by the Performance of Upper Limb Scale |
| Change in muscle tissue markers - histology and proteomics | Change from baseline in muscle tissue markers at 3 monthss | Change in tissue markers such as neuronal Nitric Oxide Synthase (nNOS) content and location and nitrosative stress by histology and proteomics |
| Change in systolic wall strain - imaging | Change from baseline in cardiac systolic wall strain at 3 months | Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging |
Countries
United States
Outcome results
None listed