Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
Status:
Terminated
Trial end date:
2014-01-01
Target enrollment:
Participant gender:
Summary
This study, supported by Charley's Fund, Inc., is being done to determine if the drug
Revatio®(also known as Sildenafil), as compared to placebo (an inactive substance that looks
like the study drug, but contains no medication), improves heart function in people with
Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (DBMD).
In people with DBMD, dystrophin is not present or lacking in heart and muscle. This is
associated with abnormalities in an enzyme called "neuronal nitric oxide synthase" or nNOS,
and leads to decreases in "cyclic GMP," which is necessary for proper function of those
muscles. Revatio blocks an enzyme called phosphodiesterase 5 (PDE5), and helps to restore the
normal amounts of cyclic GMP. The purpose of this research is to determine if Revatio is safe
for people with DBMD and if it can improve heart function.
Hypothesis : PDE5 inhibition, with the use of Revatio, will improve cardiac function in
patients with DBMD.
Phase:
Phase 2
Details
Lead Sponsor:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.