Sodium Nitrate for Muscular Dystrophy

NCT ID: NCT02434627

Last Updated: 2020-01-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-30

Study Completion Date

2018-04-30

Brief Summary

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The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation. This was translated from work using the mdx mouse model of dystrophinopathy. Recent work has shown that there is a frank improvement in grip strength when mdx mice are treated with an inorganic Nitric Oxide (NO) donor. The purpose of this study is to determine if chronic treatment with an inorganic NO donor can benefit patients with muscular dystrophy beyond blood flow regulation.

Detailed Description

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Conditions

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Becker Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Sodium Nitrate (Beetroot Juice)

Sodium nitrate in the form of beetroot juice will be administered orally. Patients will be assessed with a number of functional muscle assessments.

Group Type EXPERIMENTAL

Sodium Nitrate

Intervention Type DRUG

Patients will be given sodium nitrate daily in the form of beetroot juice.

Interventions

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Sodium Nitrate

Patients will be given sodium nitrate daily in the form of beetroot juice.

Intervention Type DRUG

Eligibility Criteria

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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
* 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))
Minimum Eligible Age

15 Years

Maximum Eligible Age

45 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Cedars-Sinai Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ronald G Victor, MD

Role: PRINCIPAL_INVESTIGATOR

Cedars-Sinai Medical Center

Locations

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Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

Countries

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United States

Other Identifiers

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Pro35228

Identifier Type: -

Identifier Source: org_study_id

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