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Tadalafil in Becker Muscular Dystrophy

NCT01070511 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2013-08-20

No results posted yet for this study

Summary

Summary for Patients: This study, funded by the Muscular Dystrophy Association, is intended to build on recent findings published in the journal Nature showing beneficial effects of tadalafil (also known as Cialis) in mice with an animal version of Duchenne and Becker muscular dystrophies. Only two doses of tadalafil improved muscle blood flow, allowing the dystrophic mice to perform more exercise with less muscle injury. This new short-term clinical trial will move the testing from animals to human patients with Becker muscular dystrophy and examine the effects of acute tadalafil dosing on muscle blood flow during a bout of exercise. Patients will take two doses of tadalafil prior to exercising. Then doctors will measure whether muscles receive increased blood flow and therefore are better protected during exercise.

Scientific Hypothesis: In patients with Becker muscular dystrophy (particularly those with dystrophin gene mutations between exons 41-46), loss of sarcolemmal nitric oxide synthase engenders functional muscle ischemia and thus muscle edema after an acute bout of exercise. The investigators further hypothesize that PDE5A inhibition, which boosts nitric oxide-cGMP signaling, constitutes an effective new countermeasure for these patients.

Conditions

  • Becker Muscular Dystrophy

Interventions

DRUG

Tadalafil

Tadalafil will be administered orally on two consecutive days. The first dose-10 mg- will be administered the afternoon before the study. The second dose -20 mg-will be administered the morning of the study.

DRUG

Placebo

A placebo pill will be administered orally on two consecutive days. The first pill will be administered the afternoon before the study. The second pill will be administered the morning of the study.

Sponsors & Collaborators

Principal Investigators

  • Ronald G Victor, MD · Cedars-Sinai Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
MALE
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2010-01-31
Primary Completion
2012-12-31
Completion
2012-12-31

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01070511 on ClinicalTrials.gov