Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
1 other identifier
interventional
19
1 country
1
Brief Summary
This study is intended to build on a growing body of literature showing a blood flow abnormality in patients with Becker muscular dystrophy. The investigators' laboratory recently showed that this blood flow abnormality could be corrected by a single oral dose of the drug Tadalafil (also known as Cialis). The investigators now wish to replicate these exciting results using a common nitric oxide donor (sodium nitrate).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2013
Shorter than P25 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2013
CompletedFirst Submitted
Initial submission to the registry
March 25, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2014
CompletedFirst Posted
Study publicly available on registry
May 28, 2014
CompletedJanuary 14, 2020
May 1, 2014
7 months
March 25, 2014
January 10, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Skeletal muscle blood flow
Skeletal muscle blood flow regulation will be assessed at each visit by near infrared spectroscopy and Doppler ultrasound.
24 hours after initial visit
Study Arms (5)
Sodium Nitrate
EXPERIMENTALPatients will ingest a single oral dose of sodium nitrate (\~8.4 mmol)
Baseline
NO INTERVENTIONThis is a baseline study visit, which will serve to assess inclusion and exclusion criteria, as well as provide untreated measurments of skeletal muscle blood flow and perfusion.
Dose-escalation trial
EXPERIMENTALThis is an optional study visit, where subjects will ingest twice the dose of sodium nitrate (\~16.8 mmol).
Placebo-control trial
PLACEBO COMPARATORThis is an optional study visit, where patients will ingest a placebo.
Increased exercise intensity
EXPERIMENTALThis is an optional study visit, where patients will be asked to repeat all of the blood flow assessments, but the exercise intensity will be increased.
Interventions
Eligibility Criteria
You may qualify if:
- Men 15-55 years of age with a pre-existing diagnosis of Becker Muscular Dystrophy by a clinical neurologist (based on clinical criteria plus previous muscle biopsy analysis and/or DNA analysis).
You may not qualify if:
- Any evidence of cardiopulmonary disease by history or by physical examination
- History of hypertension or blood pressure averaging ≥140/90 mmHg
- Diabetes mellitus or other systemic illness
- Heart failure by clinical exam, elevated BNP, or heart failure medication
- Serum creatinine ≥ 1.5 mg/dL
- Any history of substance abuse (including alcohol)
- Any history of psychiatric illness
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Cedars-Sinai Heart Institute
Los Angeles, California, 90048, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Ronald G Victor, MD
Cedars-Sinai Medical Center
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- OTHER
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 25, 2014
First Posted
May 28, 2014
Study Start
October 1, 2013
Primary Completion
May 1, 2014
Study Completion
May 1, 2014
Last Updated
January 14, 2020
Record last verified: 2014-05