Skip to main content
OpenTrials
Completed

NCT Number: NCT02434627

Sodium Nitrate for Muscular Dystrophy

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Becker Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

15 year–45 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1

Primary location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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))

Treatment and study plan

Sodium Nitrate

Drug

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

Primary outcomes

  1. Change in maximal handgrip strength

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

Secondary outcomes

  1. Change in muscle function - Performance of Upper Limb Scale

    Time frame: 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

  2. Change in muscle tissue markers - histology and proteomics

    Time frame: 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

  3. Change in systolic wall strain - imaging

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

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

  4. Change in muscle function - North Star Ambulatory Assessment

    Time frame: 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

  5. Change in muscle function - 6 minute walk test

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Cedars-Sinai Medical Center

Other

Registry information

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
May 5, 2015
Registry last updated
Jan 13, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.