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Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02147639
Recruitment Status : Unknown
Verified May 2014 by Ron Victor, Cedars-Sinai Medical Center.
Recruitment status was:  Recruiting
First Posted : May 28, 2014
Last Update Posted : May 28, 2014
Sponsor:
Information provided by (Responsible Party):
Ron Victor, Cedars-Sinai Medical Center

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).

Condition or disease Intervention/treatment Phase
Becker Muscular Dystrophy Dietary Supplement: Sodium Nitrate Dietary Supplement: Sodium Nitrate - double dose Dietary Supplement: Placebo Procedure: Increased exercise intensity Phase 2 Phase 3

Detailed Description:

There are 2 phases to this research project: (1) an initial baseline study to confirm the blood flow abnormality in Becker muscular dystrophy, and (2) a subsequent brief treatment trial. There are also 3 optional study protocols: (A) dose-escalation trial, (B) placebo trial, and (C) increased exercise intensity trial.

The baseline study involves an intake history, physical examination, and phlebotomy for blood chemistries and DNA followed by non-invasive forearm blood flow studies (Near Infrared Spectroscopy, Doppler Ultrasound) before and after a brief bout of handgrip exercise (approximately 3-5 hours to complete). Blood flow studies will be performed with the subject's lower body enclosed in an airtight chamber. Blood flow and oxygen delivery to the forearm muscles will be measured before and during application of lower body negative pressure at rest and during handgrip exercise. Lower body negative pressure simulates the blood flow changes that normally occur when a person sits up after lying down.

The results of the baseline study will determine which patients meet preset eligibility criteria to participate in the medication phase of the study. These criteria include (1) normal kidney and liver function tests and normal BNP levels (the latter to exclude heart failure), and (2) abnormal blood flow responses to handgrip exercise.

Eligible patients will be asked to repeat the above laboratory procedures on a subsequent study day after receiving a single dose of sodium nitrate. Eligible patients will also be offered the option to repeat the above laboratory procedures on three subsequent study visits, where upon: (A) the dose of sodium nitrate is increased, (B) a placebo is ingested, and/or (C) the level of exercise in increased.

The investigators plan to enroll 20 adult men with Becker muscular dystrophy.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Study Start Date : September 2013
Estimated Primary Completion Date : May 2014
Estimated Study Completion Date : May 2014


Arm Intervention/treatment
Experimental: Sodium Nitrate
Patients will ingest a single oral dose of sodium nitrate (~8.4 mmol)
Dietary Supplement: Sodium Nitrate
No Intervention: Baseline
This 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.
Experimental: Dose-escalation trial
This is an optional study visit, where subjects will ingest twice the dose of sodium nitrate (~16.8 mmol).
Dietary Supplement: Sodium Nitrate - double dose
Placebo Comparator: Placebo-control trial
This is an optional study visit, where patients will ingest a placebo.
Dietary Supplement: Placebo
Experimental: Increased exercise intensity
This 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.
Procedure: Increased exercise intensity



Primary Outcome Measures :
  1. Skeletal muscle blood flow [ Time Frame: 24 hours after initial visit ]
    Skeletal muscle blood flow regulation will be assessed at each visit by near infrared spectroscopy and Doppler ultrasound.



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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:

  • 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).

Exclusion Criteria:

  • 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

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): NCT02147639


Contacts
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Contact: Michael D Nelson, PhD 310-967-3844 michael.nelson@cshs.org

Locations
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United States, California
Cedars-Sinai Heart Institute Recruiting
Los Angeles, California, United States, 90048
Contact: Michael D Nelson, PhD    310-967-3844    michael.nelson@cshs.org   
Sponsors and Collaborators
Cedars-Sinai Medical Center
Investigators
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Principal Investigator: Ronald G Victor, MD Cedars-Sinai Medical Center

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Responsible Party: Ron Victor, Director, Hypertension Center of Exercellance; Associate Director, Heart Institute, Cedars-Sinai Medical Center
ClinicalTrials.gov Identifier: NCT02147639     History of Changes
Other Study ID Numbers: NANO3
First Posted: May 28, 2014    Key Record Dates
Last Update Posted: May 28, 2014
Last Verified: May 2014

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