The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

Sponsor

Cedars-Sinai Medical Center (Other)

Overall Status

Terminated

CT.gov ID

NCT02653833

Collaborator

(none)

Enrollment

Location

Arms

Actual Duration (Months)

0.6

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Muscular Dystrophy	Drug: Tadalafil 20 MG Other: beetroot juice extract	Early Phase 1

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

6 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Basic Science

Official Title:

Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

Actual Study Start Date :

Nov 1, 2017

Actual Primary Completion Date :

Sep 1, 2018

Actual Study Completion Date :

Sep 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Healthy Controls (HC) 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.	Drug: Tadalafil 20 MG 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. Other: beetroot juice extract 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.
Experimental: Becker Muscular Dystrophy (BMD) 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.	Drug: Tadalafil 20 MG 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. Other: beetroot juice extract 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.

Arm

Intervention/Treatment

Experimental: Healthy Controls (HC)

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.

Drug: Tadalafil 20 MG

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.

Other: beetroot juice extract

Experimental: Becker Muscular Dystrophy (BMD)

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.

Drug: Tadalafil 20 MG

Other: beetroot juice extract

Outcome Measures

Primary Outcome Measures

Change in Skeletal Muscle Blood Flow [At baseline and within one hours after taking beetroot juice or Tadalafil]
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 [At baseline, and within an hour after taking beetroot juice or Tadalafil]
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) [At baseline, and within an hour after taking beetroot juice or Tadalafil]
Endothelial function is assessed by measuring flow-mediated dilation (FMD) by Doppler ultrasound.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 45 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Yes

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.