Becker Muscular Dystrophy
Conditions
Keywords
Becker, dystrophy, sympatholysis
Brief summary
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD). If tadalafil restores functional sympatholysis in BMD via the NO-cyclic guanosine monophosphate pathway, then functional sympatholysis should also be restored by sodium nitrite- which is an indirect nitric oxide donor.
Interventions
DIETARY_SUPPLEMENTSodium nitrate
Sodium nitrate will be ingested orally
Sponsors
Cedars-Sinai Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
15 Years to 55 Years
Healthy volunteers
Yes
Inclusion criteria
* Becker muscular dystrophy * age 15-55 years of age * ambulatory
Exclusion criteria
* hypertension, diabetes, or heart failure by standard clinical criteria * elevated brain natriuretic peptide level (\>100 pg/ml) * Left ventricular ejection fraction \< 50% * cardiac rhythm disorder, specifically: rhythm other than sinus, supraventricular tachycardia, atrial fibrillation, ventricular tachycardia, heart block * continuous ventilatory support * liver disease * renal impairment * history of asthma or bronchospasm * use of any medications other than common supplements * unable to perform handgrip exercise
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| change in muscle tissue oxygenation | change from baseline to post treatment (3-4 hours) | The pre-specified primary outcome is the pre vs. post treatment change in functional sympatholysis measured by muscle oxygenation. |
Countries
United States
Outcome results
None listed