The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

Early Phase 1

Terminated

• Conditions: Muscular Dystrophy
• Interventions: Drug: Tadalafil 20 MG; Other: beetroot juice extract

• Registration Number: NCT02653833
• Lead Sponsor: Cedars-Sinai Medical Center

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.

Study Timeline

• Study First Submitted: 2015-12-14
• Study First Submitted QC: 2016-01-10
• Study First Posted: 2016-01-12
• Study Start: 2017-11-01
• Primary Completion: 2018-09-01
• Study Completion: 2018-09-01
• Status Verified: 2019-06
• Last Update Submitted: 2020-01-10
• Last Update Posted: 2020-01-14

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 6

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Healthy Controls (HC)	Tadalafil 20 MG	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.
Healthy Controls (HC)	beetroot juice extract	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.
Becker Muscular Dystrophy (BMD)	Tadalafil 20 MG	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.
Becker Muscular Dystrophy (BMD)	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.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.

Trial Locations

Locations (1)

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States