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Sodium Nitrate for Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02434627
Recruitment Status : Recruiting
First Posted : May 5, 2015
Last Update Posted : October 17, 2016
Sponsor:
Information provided by (Responsible Party):
Ron Victor, 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.

Condition or disease Intervention/treatment Phase
Becker Muscular Dystrophy Drug: Sodium Nitrate Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Sodium Nitrate for Muscular Dystrophy
Study Start Date : June 2015
Estimated Primary Completion Date : October 2016
Estimated Study Completion Date : June 2017


Arm Intervention/treatment
Experimental: Sodium Nitrate (Beetroot Juice)
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
Patients will be given sodium nitrate daily in the form of beetroot juice.



Primary Outcome Measures :
  1. Change in maximal handgrip strength [ Time Frame: Change from baseline in handgrip strength at 3 months ]

Secondary Outcome Measures :
  1. Change in muscle function - Performance of Upper Limb Scale [ Time Frame: 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

  2. Change in muscle tissue markers - histology and proteomics [ Time Frame: 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

  3. Change in systolic wall strain - imaging [ Time Frame: Change from baseline in cardiac systolic wall strain at 3 months ]
    Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging

  4. Change in muscle function - North Star Ambulatory Assessment [ Time Frame: 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

  5. Change in muscle function - 6 minute walk test [ Time Frame: 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



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Ages Eligible for Study:   15 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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))

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02434627


Contacts
Contact: Mohammad Rashid Mohamad.Rashid@cshs.org

Locations
United States, California
Cedars-Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact: Mohamad Rashid, CCRC    424-315-2501    Mohamad.Rashid@cshs.org   
Contact: Matthew Hakimi, MD    310.967.3848    Matthew.Hakimi@cshs.org   
Sponsors and Collaborators
Cedars-Sinai Medical Center
Investigators
Principal Investigator: Ronald G Victor, MD Cedars-Sinai Medical Center

Responsible Party: Ron Victor, Professor of Medicine, Cedars-Sinai Medical Center
ClinicalTrials.gov Identifier: NCT02434627     History of Changes
Other Study ID Numbers: Pro35228
First Posted: May 5, 2015    Key Record Dates
Last Update Posted: October 17, 2016
Last Verified: October 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Ron Victor, Cedars-Sinai Medical Center:
Becker
Dystrophy

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked