Status:
TERMINATED
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
Lead Sponsor:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Collaborating Sponsors:
Johns Hopkins University
Conditions:
Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Eligibility:
MALE
18-50 years
Phase:
PHASE2
Brief 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, ...
Detailed Description
This clinical trial is focused on cardiovascular disease due to dystrophin deficiency. Dystrophin is normally localized to the muscle cell membrane where it interacts with a complex of proteins includ...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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.
Key Trial Info
Start Date :
September 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2014
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT01168908
Start Date
September 1 2010
End Date
January 1 2014
Last Update
February 26 2019
Active Locations (1)
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1
Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205