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Sodium Nitrate for Muscular Dystrophy

Primary Purpose

Becker Muscular Dystrophy

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Sodium Nitrate

About this trial

This is an interventional treatment trial for Becker Muscular Dystrophy focused on measuring Becker, Dystrophy

Eligibility Criteria

15 Years - 45 Years (Child, Adult)MaleDoes not accept healthy volunteers

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
Contraindications to NO donors (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or Phosphodiesterase type 5 (PDE5) inhibitors) Glucocorticoid therapy and prophylactic use of Angiotensin Converting Enzyme (ACE) inhibitors and beta-blockers for cardiac protection will not be exclusion criteria.
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))

Sites / Locations

Cedars-Sinai Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sodium Nitrate (Beetroot Juice)

Arm Description

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

Outcomes

Primary Outcome Measures

Change in maximal handgrip strength

Secondary Outcome Measures

Change in muscle function - Performance of Upper Limb Scale

Change in functional muscle assessment as measured by the Performance of Upper Limb Scale

Change in muscle tissue markers - histology and proteomics

Change in tissue markers such as neuronal Nitric Oxide Synthase (nNOS) content and location and nitrosative stress by histology and proteomics

Change in systolic wall strain - imaging

Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging

Change in muscle function - North Star Ambulatory Assessment

Change in functional muscle assessment as measured by the North Star Ambulatory Assessment

Change in muscle function - 6 minute walk test

Change in functional muscle assessment as measured by the 6 minute walk test

Full Information

NCT ID

NCT02434627

First Posted

April 28, 2015

Last Updated

January 9, 2020

Sponsor

Cedars-Sinai Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT02434627

Brief Title

Sodium Nitrate for Muscular Dystrophy

Official Title

Sodium Nitrate for Muscular Dystrophy

Study Type

Interventional

2. Study Status

Record Verification Date

October 2016

Overall Recruitment Status

Completed

Study Start Date

June 2015 (undefined)

Primary Completion Date

April 2018 (Actual)

Study Completion Date

April 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Cedars-Sinai Medical Center

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Becker Muscular Dystrophy

Keywords

Becker, Dystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Sodium Nitrate (Beetroot Juice)

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Sodium Nitrate

Intervention Description

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

Primary Outcome Measure Information:

Title

Change in maximal handgrip strength

Time Frame

Change from baseline in handgrip strength at 3 months

Secondary Outcome Measure Information:

Title

Change in muscle function - Performance of Upper Limb Scale

Description

Change in functional muscle assessment as measured by the Performance of Upper Limb Scale

Time Frame

Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months

Title

Change in muscle tissue markers - histology and proteomics

Description

Change in tissue markers such as neuronal Nitric Oxide Synthase (nNOS) content and location and nitrosative stress by histology and proteomics

Time Frame

Change from baseline in muscle tissue markers at 3 monthss

Title

Change in systolic wall strain - imaging

Description

Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging

Time Frame

Change from baseline in cardiac systolic wall strain at 3 months

Title

Change in muscle function - North Star Ambulatory Assessment

Description

Change in functional muscle assessment as measured by the North Star Ambulatory Assessment

Time Frame

Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months

Title

Change in muscle function - 6 minute walk test

Description

Change in functional muscle assessment as measured by the 6 minute walk test

Time Frame

Change from baseline in muscle function - 6 minute walk test at 3 months

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

15 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

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 Contraindications to NO donors (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or Phosphodiesterase type 5 (PDE5) inhibitors) Glucocorticoid therapy and prophylactic use of Angiotensin Converting Enzyme (ACE) inhibitors and beta-blockers for cardiac protection will not be exclusion criteria. 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))

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ronald G Victor, MD

Organizational Affiliation

Cedars-Sinai Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cedars-Sinai Medical Center

City

Los Angeles

State/Province

California

ZIP/Postal Code

90048

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Sodium Nitrate for Muscular Dystrophy

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