Pharmaceutical company Eli Lilly is conducting a trial of tadalafil, which may help regulate blood flow to muscles, in some 300 boys with Duchenne MD
Multinational pharmaceutical company Eli Lilly is conducting a phase 3 trial of the drug tadalafil in approximately 300 boys with Duchenne muscular dystrophy (DMD) who are 7-14 years old, able to walk, have adequate cardiac function and meet other study criteria.
The rationale for using tadalafil to treat DMD or the related Becker muscular dystrophy (BMD) is that blood flow to exercising muscles appears to be disrupted in these disorders as a downstream effect of any of several mutations in the gene for the muscle protein dystrophin.
Drugs such as tadalafil and sildenafil (Viagra) are known to prolong the action of a substance called nitric oxide, and in so doing, may improve blood flow to exercising muscles despite the presence of dystrophin gene mutations that cause DMD and BMD. MDA has supported research in this area previously and continues to do so (see Joseph Beavo's Winter 2013 grant).
Tadalafil is being studied in patients with BMD at Cedars-Sinai Medical Center in Los Angeles.
Under the Eli Lilly brand name Cialis, tadalafil is on the market for erectile dysfunction and a type of prostate gland enlargement. Under the Eli Lilly brand name Adcirca, tadalafil is available for the treatment of pulmonary arterial hypertension.
Patients with DMD or BMD are advised not to seek an off-label prescription for tadalafil, as its effects and safety in these disorders is not yet well understood. ("Off label" refers to a physician's use of a drug or therapy to treat conditions other than the ones for which it received U.S. Food and Drug Administration approval.)
About the tadalafil in DMD study
The Lilly-sponsored study will determine whether orally administered tadalafil can slow the decline in walking ability in boys with DMD and will also assess the safety of tadalafil and any side effects that might be associated with it in DMD-affected boys.
Participants will be randomly assigned to receive tadalafil at one of two daily dosage levels or to receive a placebo for the first 48 weeks of the study. Participants will then have an option to move into a 48-week extension period during which everyone will receive tadalafil.
There are more than 60 study sites, including 22 in the United States, as well as locations in Argentina, Belgium, Canada, France, Germany, Italy, Japan, Korea, the Netherlands, Puerto Rico, the Russian Federation, Spain, Taiwan, Turkey and the United Kingdom.
Prospective participants must:
Prospective participants must not:
For details and contact information, see A Study of Tadalafil for Duchenne Muscular Dystrophy; or enter NCT01865084 in the search box at ClinicalTrials.gov. You also can call Eli Lilly toll-free at (877) 285-4559 Monday through Friday, 9 a.m. to 5 p.m. Eastern time.
And to learn more, read Enhancing Blood Flow to Exercising Muscles (April 2011).
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.