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The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

Primary Purpose

Muscular Dystrophy

Status
Terminated
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Tadalafil 20 MG
beetroot juice extract
Sponsored by
Cedars-Sinai Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional basic science trial for Muscular Dystrophy focused on measuring Becker, Muscular Dystrophy, Skeletal Muscle, blood flow, Ultrasound

Eligibility Criteria

18 Years - 45 Years (Adult)MaleAccepts Healthy Volunteers

Inclusion Criteria:

  • Healthy man, aged 18 to 45 years, currently taking no medication. OR
  • Clinical diagnosis of Becker Muscular Dystrophy (BMD), aged 18 to 45 years, and currently taking no medication.

Exclusion Criteria:

  • Hypertension, diabetes, heart failure, liver disease
  • ECG evidence of prolonged QT interval

Sites / Locations

  • Cedars-Sinai Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Healthy Controls (HC)

Becker Muscular Dystrophy (BMD)

Arm Description

Healthy men currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. This is repeated at baseline and shortly after taking beetroot juice extract.

currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. Using a lower body negative pressure (LBNP) chamber, the subjects are tested for impaired exercise induced vasodilation to the skeletal muscle or "functional sympatholysis". This is repeated at baseline and shortly after taking beetroot juice extract.

Outcomes

Primary Outcome Measures

Change in Skeletal Muscle Blood Flow
Exercise induced skeletal muscle perfusion is measured by contrast-enhanced ultrasound (CEU) during graded voluntary contraction on a hand grip dynamometer.

Secondary Outcome Measures

Change in Measured Functional Sympatholysis
Functional sympatholysis (attenuation of exercise induced skeletal muscle vasoconstriction) is assessed using a lower body negative pressure chamber.
Change in Measured Flow Mediated Dilation (FMD)
Endothelial function is assessed by measuring flow-mediated dilation (FMD) by Doppler ultrasound.

Full Information

First Posted
December 14, 2015
Last Updated
January 10, 2020
Sponsor
Cedars-Sinai Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT02653833
Brief Title
The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
Official Title
Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
June 2019
Overall Recruitment Status
Terminated
Why Stopped
PI passed away
Study Start Date
November 1, 2017 (Actual)
Primary Completion Date
September 1, 2018 (Actual)
Study Completion Date
September 1, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cedars-Sinai Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise. The investigators will use cutting edge technology with contrast enhanced ultrasound to visualize the microvascular blood supply to the forearm. Animal studies have shown reversal of muscle damage with improved delivery of blood to the exercising muscle. This research aims to understand the mechanism of action of this medication in a way it has never been studied before. The results may help benefit individuals with muscular Dystrophy in the future.
Detailed Description
The subjects are asked to perform a graded hand-grip exercise while blood flow to the skeletal muscle is visualized by contrast enhanced ultrasound. This is done at baseline and after taking the study agent.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy
Keywords
Becker, Muscular Dystrophy, Skeletal Muscle, blood flow, Ultrasound

7. Study Design

Primary Purpose
Basic Science
Study Phase
Early Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Healthy Controls (HC)
Arm Type
Experimental
Arm Description
Healthy men currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. This is repeated at baseline and shortly after taking beetroot juice extract.
Arm Title
Becker Muscular Dystrophy (BMD)
Arm Type
Experimental
Arm Description
currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. Using a lower body negative pressure (LBNP) chamber, the subjects are tested for impaired exercise induced vasodilation to the skeletal muscle or "functional sympatholysis". This is repeated at baseline and shortly after taking beetroot juice extract.
Intervention Type
Drug
Intervention Name(s)
Tadalafil 20 MG
Intervention Description
Blood flow to the forearm is visualized via an ultrasound technique at rest and with graded exercise. The experiment is then repeated shortly after the subject takes the study agent, beetroot juice extract.
Intervention Type
Other
Intervention Name(s)
beetroot juice extract
Intervention Description
Blood flow to the forearm is visualized via an ultrasound technique at rest and with graded exercise. The experiment is then repeated shortly after the subject takes the study agent, beetroot juice extract.
Primary Outcome Measure Information:
Title
Change in Skeletal Muscle Blood Flow
Description
Exercise induced skeletal muscle perfusion is measured by contrast-enhanced ultrasound (CEU) during graded voluntary contraction on a hand grip dynamometer.
Time Frame
At baseline and within one hours after taking beetroot juice or Tadalafil
Secondary Outcome Measure Information:
Title
Change in Measured Functional Sympatholysis
Description
Functional sympatholysis (attenuation of exercise induced skeletal muscle vasoconstriction) is assessed using a lower body negative pressure chamber.
Time Frame
At baseline, and within an hour after taking beetroot juice or Tadalafil
Title
Change in Measured Flow Mediated Dilation (FMD)
Description
Endothelial function is assessed by measuring flow-mediated dilation (FMD) by Doppler ultrasound.
Time Frame
At baseline, and within an hour after taking beetroot juice or Tadalafil

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria: Healthy man, aged 18 to 45 years, currently taking no medication. OR Clinical diagnosis of Becker Muscular Dystrophy (BMD), aged 18 to 45 years, and currently taking no medication. Exclusion Criteria: Hypertension, diabetes, heart failure, liver disease ECG evidence of prolonged QT interval
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
No

Learn more about this trial

The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

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