The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

Cedars-Sinai Medical Center

Status and phase: Terminated; Early Phase 1

Conditions: Muscular Dystrophy

Treatments: Drug: Tadalafil 20 MG; Other: beetroot juice extract

Study type: Interventional

Funder types: Other

Identifiers

NCT02653833

Pro00041688

Details and patient eligibility

About

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.

Full 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.

Enrollment: 6 patients

Sex: Male

Ages: 18 to 45 years old

Volunteers: Accepts 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

Trial design

Primary purpose: Basic Science

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

Healthy Controls (HC)

Experimental group

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.

Treatment:

Other: beetroot juice extract

Drug: Tadalafil 20 MG

Becker Muscular Dystrophy (BMD)

Experimental group

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.

Treatment:

Other: beetroot juice extract

Drug: Tadalafil 20 MG