The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

NCT ID: NCT02653833

Last Updated: 2020-01-14

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-11-01
• Study Completion Date: 2018-09-01

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.

Conditions

Muscular Dystrophy

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Tadalafil 20 MG

Intervention Type: DRUG

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.

beetroot juice extract

Intervention Type: OTHER

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.

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.

Group Type: EXPERIMENTAL

Tadalafil 20 MG

Intervention Type: DRUG

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.

beetroot juice extract

Intervention Type: OTHER

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.

Interventions

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.

Intervention Type: DRUG

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.

Intervention Type: OTHER

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

Cedars-Sinai Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ronald G Victor, MD

Role: PRINCIPAL_INVESTIGATOR

Cedars-Sinai Medical Center

Locations

Cedars-Sinai Medical Center

Los Angeles, California, United States

Countries

United States

Other Identifiers

Pro00041688

Identifier Type: -

Identifier Source: org_study_id