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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT02434627
Date of registration: 28/04/2015
Prospective Registration: Yes
Primary sponsor: Cedars-Sinai Medical Center
Public title: Sodium Nitrate for Muscular Dystrophy
Scientific title: Sodium Nitrate for Muscular Dystrophy
Date of first enrolment: June 2015
Target sample size: 5
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02434627
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1
Countries of recruitment
United States
Contacts
Name:     Ronald G Victor, MD
Address: 
Telephone:
Email:
Affiliation:  Cedars-Sinai Medical Center
Key inclusion & exclusion 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))



Age minimum: 15 Years
Age maximum: 45 Years
Gender: Male
Health Condition(s) or Problem(s) studied
Becker Muscular Dystrophy
Intervention(s)
Drug: Sodium Nitrate
Primary Outcome(s)
Change in maximal handgrip strength [Time Frame: Change from baseline in handgrip strength at 3 months]
Secondary Outcome(s)
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 muscle function - North Star Ambulatory Assessment [Time Frame: Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months]
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 muscle tissue markers - histology and proteomics [Time Frame: Change from baseline in muscle tissue markers at 3 monthss]
Change in systolic wall strain - imaging [Time Frame: Change from baseline in cardiac systolic wall strain at 3 months]
Secondary ID(s)
Pro35228
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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