DMD: Vasodilator Drug Study Needs Four Boys

A 12-participant study of the acute effects of two vasodilating drugs on blood flow to exercising muscles needs four more boys with Duchenne muscular dystrophy (DMD) who meet study criteria and are able to travel to Los Angeles. Vasodilators increase the diameter of blood vessels.

"We’ve made some wonderful progress with our latest Duchenne MD blood flow studies investigating the effects of tadalafil," said Sharon Tang, clinical research coordinator for the study. "In order to complete this exciting project, we only need four more boys. The study only requires one week of study visits in sunny Los Angeles. All travel expenses are covered by the study. Get away from your winter blues and help make us make a difference!"

The study involves taking two doses of each of two drugs — tadalafil and sildenafil — both of which may improve the ability of muscle fibers to increase blood flow during exercise despite the presence of a DMD-causing mutation in the dystrophin gene.

Participants must:

  • have a diagnosis of DMD;
  • be 7 to 15 years old;
  • be able to walk; and
  • be without clinical signs of heart failure.

The trial will involve measuring blood flow in exercising forearm muscles and blood medication levels during two sets of visits to Cedars-Sinai Medical Center in Los Angeles, approximately one week apart.

For details, see PDE Inhibitors in DMD (Acute Dosing Study); or enter NCT01580501 in the search box at ClinicalTrials.gov.

Prospective participants should contact Sharon Tang in Los Angeles at (310) 248-8080 or Sharon.Tang@cshs.org.

About Clinical Trials

About Clinical Trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.

MDA has no ability to influence who is chosen to participate in a clinical trial.

To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.

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