Sodium Nitrate for Muscular Dystrophy

Cedars-Sinai Medical Center

Status and phase: Completed; Phase 1

Conditions: Becker Muscular Dystrophy

Treatments: Drug: Sodium Nitrate

Study type: Interventional

Funder types: Other

Identifiers

NCT02434627

Pro35228

Details and patient eligibility

About

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.

Enrollment: 5 patients

Sex: Male

Ages: 15 to 45 years old

Volunteers: No Healthy Volunteers

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))

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

5 participants in 1 patient group

Sodium Nitrate (Beetroot Juice)

Experimental group

Description:

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

Treatment:

Drug: Sodium Nitrate