Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
| Status: | Completed |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 15 - 45 |
| Updated: | 4/2/2016 |
| Start Date: | September 2013 |
| End Date: | May 2014 |
| Contact: | Michael D Nelson, PhD |
| Email: | michael.nelson@cshs.org |
| Phone: | 310-967-3844 |
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).
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).
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.
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.
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
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