Sodium Nitrate for Muscular Dystrophy

Phase 1

Completed

• Conditions: Becker Muscular Dystrophy
• Interventions: Drug: Sodium Nitrate

• Registration Number: NCT02434627
• Lead Sponsor: Cedars-Sinai Medical Center

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-04-28
• Study First Submitted QC: 2015-05-04
• Study First Posted: 2015-05-05
• Study Start: 2015-06
• Status Verified: 2016-10
• Primary Completion: 2018-04
• Study Completion: 2018-04
• Last Update Submitted: 2020-01-09
• Last Update Posted: 2020-01-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 5

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))

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Sodium Nitrate (Beetroot Juice)	Sodium Nitrate	Sodium nitrate in the form of beetroot juice will be administered orally. Patients will be assessed with a number of functional muscle assessments.

Primary Outcome Measures

Name	Time	Method
Change in maximal handgrip strength	Change from baseline in handgrip strength at 3 months

Secondary Outcome Measures

Name	Time	Method
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 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 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
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 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

Trial Locations

Locations (1)

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States