Sodium Nitrate for Muscular Dystrophy

Becker Muscular Dystrophy Clinical Trial

Official title:

Sodium Nitrate for Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02434627
• Other study ID #: Pro35228
• Status: Completed
• Phase: Phase 1
• Start date: June 2015
• Est. completion date: April 2018

Study information

• Verified date: October 2016
• Source: Cedars-Sinai Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: April 2018
• Est. primary completion date: April 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 15 Years to 45 Years

Eligibility

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))

Related Conditions & MeSH terms

• Becker Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Sodium Nitrate
Patients will be given sodium nitrate daily in the form of beetroot juice.

Locations

Country	Name	City	State
United States	Cedars-Sinai Medical Center	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Cedars-Sinai Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in maximal handgrip strength		Change from baseline in handgrip strength at 3 months
Secondary	Change in muscle function - Performance of Upper Limb Scale	Change in functional muscle assessment as measured by the Performance of Upper Limb Scale	Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months
Secondary	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	Change from baseline in muscle tissue markers at 3 monthss
Secondary	Change in systolic wall strain - imaging	Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging	Change from baseline in cardiac systolic wall strain at 3 months
Secondary	Change in muscle function - North Star Ambulatory Assessment	Change in functional muscle assessment as measured by the North Star Ambulatory Assessment	Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months
Secondary	Change in muscle function - 6 minute walk test	Change in functional muscle assessment as measured by the 6 minute walk test	Change from baseline in muscle function - 6 minute walk test at 3 months