Revatio for heart disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (REVERSE DBMD)
This study is currently recruiting participants.
Verified on may well 2011 by Hugo W. Moser research Institute at Kennedy Krieger, Inc.
First received on July 22, 2010. last Updated on may well 6, 2011 history of changes
Sponsor: Hugo W. Moser research Institute at Kennedy Krieger, Inc.
Collaborator: Johns Hopkins University
Information provided by: Hugo W. Moser research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT01168908
This study, supported by Charley’s Fund, Inc., is being done to determine if the medication 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 people muscles. Revatio blocks an enzyme called phosphodiesterase 5 (PDE5), and helps to restore the normal amounts of cyclic GMP. The objective of this research is to determine if Revatio is safe for people with DBMD and if it can enhance heart function.
Hypothesis : PDE5 inhibition, with the use of Revatio, will enhance cardiac function in patients with DBMD.