Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

In brief

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.

Key facts

Study ID: NCT01168908
Run by: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
People needed: 20
Starts: 2010-09-01
Expected to finish: 2014-01-01
Last updated by the study team: 2019-02-26

Who can join

Age: 18 and older, up to 50. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

DBMD as determined by either a skeletal muscle biopsy demonstrating absence or lack of dystrophin, and/or genetic testing showing a mutation in the dystrophin gene predictive of DBMD, as well as a consistent physical examination
Male gender
Age greater than or equal to 18 years
Cardiac dysfunction with ejection fraction less than or equal to 50% as determined by echocardiogram, cardiac MRI, or multi-gated acquisition (MUGA) scan
On a stable dose of ACE-inhibitor or angiotensin receptor blocker (ARB) for at least 3 months; beta-adrenergic receptor blockers and glucocorticosteroids are not required but if used, a stable dose for at least 3 months is required.
Ability of the subject or legal guardian to provide informed consent
Ability to adhere with study follow-up
Willingness to abstain from food and alcohol for 8 hours prior to FMD

You may not qualify if…

Use of nitrates or alpha-adrenergic receptor blockers
Known intolerance or allergy to sildenafil, or a history of any severe allergic or anaphylactic reactions
Any medical or psychosocial condition, which, in the view of the study investigator, makes study participation inadvisable
Known hereditary retinal disorder such as retinitis pigmentosa
History of priapism or conditions that may predispose to priapism such as sickle cell anemia, multiple myeloma, or leukemia
Bleeding disorders
Active tobacco use
Chronic atrial fibrillation or frequent arrhythmia that would result in an irregular pulse
Factors that would preclude obtaining an MRI study - (e.g. implantable pacemaker or cardioverter-defibrillator; body habitus cannot fit into scanner)
Systolic blood pressure (SBP) less than 85 mmHg at baseline evaluation
Chronic kidney disease stages 4 and 5: GFR< 30 mL/min/1.73 m2 as determined by serum cystatin C level and the equation eGFRcys = 76.7 x (serum cystatin C-1.18)
Current use of sildenafil.

Where it is running

Kennedy Krieger Institute, Johns Hopkins School of Medicine — Baltimore, Maryland, United States

Full record on ClinicalTrials.gov

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