Clinical trial NCT02434627: Sodium Nitrate for Muscular Dystrophy
Official title: Sodium Nitrate for Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Becker Muscular Dystrophy
Keywords: Becker
Dystrophy
Lead sponsor: Cedars-Sinai Medical Center
Sponsor class: OTHER
First posted: 2015-05-05
Enrollment: 5
Sex: MALE
Minimum age (years): 15
Maximum age (years): 45
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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))

Primary outcomes:
measure: Change in maximal handgrip strength
timeFrame: Change from baseline in handgrip strength at 3 months
Secondary outcomes:
measure: Change in muscle function - Performance of Upper Limb Scale
timeFrame: Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months
measure: Change in muscle tissue markers - histology and proteomics
timeFrame: Change from baseline in muscle tissue markers at 3 monthss
measure: Change in systolic wall strain - imaging
timeFrame: Change from baseline in cardiac systolic wall strain at 3 months
measure: Change in muscle function - North Star Ambulatory Assessment
timeFrame: Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months
measure: Change in muscle function - 6 minute walk test
timeFrame: Change from baseline in muscle function - 6 minute walk test at 3 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
Study officials:
affiliation: Cedars-Sinai Medical Center
name: Ronald G Victor, MD
role: PRINCIPAL_INVESTIGATOR
References: