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Sodium Nitrate for Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02434627
Recruitment Status : Completed
First Posted : May 5, 2015
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
Cedars-Sinai Medical Center

Tracking Information
First Submitted Date  ICMJE April 28, 2015
First Posted Date  ICMJE May 5, 2015
Last Update Posted Date January 13, 2020
Study Start Date  ICMJE June 2015
Actual Primary Completion Date April 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 4, 2015)
Change in maximal handgrip strength [ Time Frame: Change from baseline in handgrip strength at 3 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 4, 2015)
  • 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
  • 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
  • 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
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Sodium Nitrate for Muscular Dystrophy
Official Title  ICMJE Sodium Nitrate for Muscular Dystrophy
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Becker Muscular Dystrophy
Intervention  ICMJE Drug: Sodium Nitrate
Patients will be given sodium nitrate daily in the form of beetroot juice.
Study Arms  ICMJE 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.
Intervention: Drug: Sodium Nitrate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 9, 2020)
5
Original Estimated Enrollment  ICMJE
 (submitted: May 4, 2015)
12
Actual Study Completion Date  ICMJE April 2018
Actual Primary Completion Date April 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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))
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 15 Years to 45 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02434627
Other Study ID Numbers  ICMJE Pro35228
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Cedars-Sinai Medical Center
Study Sponsor  ICMJE Cedars-Sinai Medical Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Ronald G Victor, MD Cedars-Sinai Medical Center
PRS Account Cedars-Sinai Medical Center
Verification Date October 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP