Sodium Nitrate for Muscular Dystrophy

NCT02434627 | Completed Phase 1

• 1 other identifier: Pro35228
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Becker Muscular Dystrophy

Keywords

Becker; Dystrophy

Outcome Measures

Primary Outcomes (1)

• Change in maximal handgrip strength

  Change from baseline in handgrip strength at 3 months

Secondary Outcomes (5)

• 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 muscle tissue markers - histology and proteomics

  Change from baseline in muscle tissue markers at 3 monthss

• Change in systolic wall strain - imaging

  Change from baseline in cardiac systolic wall strain at 3 months

• Change in muscle function - North Star Ambulatory Assessment

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

• Change in muscle function - 6 minute walk test

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

Study Arms (1)

Sodium Nitrate (Beetroot Juice)

EXPERIMENTAL

Sodium nitrate in the form of beetroot juice will be administered orally. Patients will be assessed with a number of functional muscle assessments.

Drug: Sodium Nitrate

Interventions

Sodium Nitrate DRUG

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

Sodium Nitrate (Beetroot Juice)

Eligibility Criteria

• Age: 15 Years - 45 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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))

Sponsors & Collaborators

• Cedars-Sinai Medical Center: lead

Study Sites (1)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

sodium nitrate

Condition Hierarchy (Ancestors)

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

Study Officials

• Ronald G Victor, MD

  Cedars-Sinai Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2015
• First Posted: May 5, 2015
• Study Start: June 1, 2015
• Primary Completion: April 1, 2018
• Study Completion: April 1, 2018
• Last Updated: January 13, 2020

Record last verified: 2016-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)