The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

NCT02653833 | Terminated Early Phase 1 FDA Drug

• 1 other identifier: Pro00041688
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Dystrophy

Keywords

Becker; Muscular Dystrophy; Skeletal Muscle; blood flow; Ultrasound

Outcome Measures

Primary Outcomes (1)

• 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.

  At baseline and within one hours after taking beetroot juice or Tadalafil

Secondary Outcomes (2)

• Change in Measured Functional Sympatholysis

  At baseline, and within an hour after taking beetroot juice or Tadalafil

• Change in Measured Flow Mediated Dilation (FMD)

  At baseline, and within an hour after taking beetroot juice or Tadalafil

Study Arms (2)

Healthy Controls (HC)

EXPERIMENTAL

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; Other: beetroot juice extract

Becker Muscular Dystrophy (BMD)

EXPERIMENTAL

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

Interventions

Tadalafil 20 MG 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.

Becker Muscular Dystrophy (BMD); Healthy Controls (HC)

beetroot juice extract 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); Healthy Controls (HC)

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Cedars-Sinai Medical Center: lead

Study Sites (1)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies; Muscular Dystrophy, Duchenne

Interventions

Tadalafil

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Carbolines; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Indole Alkaloids; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds, 3-Ring

Study Officials

• Ronald G Victor, MD

  Cedars-Sinai Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 14, 2015
• First Posted: January 12, 2016
• Study Start: November 1, 2017
• Primary Completion: September 1, 2018
• Study Completion: September 1, 2018
• Last Updated: January 14, 2020

Record last verified: 2019-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)