|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 December 2020 |
|
Main ID: |
NCT01168908 |
|
Date of registration:
|
22/07/2010 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
REVERSE-DBMD |
|
Scientific title:
|
Phase 2 Clinical Trial of Sildenafil for Cardiac Dysfunction in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy |
|
Date of first enrolment:
|
September 2010 |
|
Target sample size:
|
20 |
|
Recruitment status: |
Terminated |
|
URL:
|
https://clinicaltrials.gov/show/NCT01168908 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Daniel P Judge, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Johns Hopkins School of Medicine |
|
|
Name:
|
Kathryn R. Wagner, M.D., Ph.D. |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
The Kennedy Krieger Institute |
| |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
1. 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
2. Male gender
3. Age greater than or equal to 18 years
4. Cardiac dysfunction with ejection fraction less than or equal to 50% as determined by
echocardiogram, cardiac MRI, or multi-gated acquisition (MUGA) scan
5. 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.
6. Ability of the subject or legal guardian to provide informed consent
7. Ability to adhere with study follow-up
8. Willingness to abstain from food and alcohol for 8 hours prior to FMD
Exclusion Criteria:
1. Use of nitrates or alpha-adrenergic receptor blockers
2. Known intolerance or allergy to sildenafil, or a history of any severe allergic or
anaphylactic reactions
3. Any medical or psychosocial condition, which, in the view of the study investigator,
makes study participation inadvisable
4. Known hereditary retinal disorder such as retinitis pigmentosa
5. History of priapism or conditions that may predispose to priapism such as sickle cell
anemia, multiple myeloma, or leukemia
6. Bleeding disorders
7. Active tobacco use
8. Chronic atrial fibrillation or frequent arrhythmia that would result in an irregular
pulse
9. Factors that would preclude obtaining an MRI study - (e.g. implantable pacemaker or
cardioverter-defibrillator; body habitus cannot fit into scanner)
10. Systolic blood pressure (SBP) less than 85 mmHg at baseline evaluation
11. 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)
12. Current use of sildenafil.
Age minimum:
18 Years
Age maximum:
50 Years
Gender:
Male
|
|
Health Condition(s) or Problem(s) studied
|
|
Duchenne Muscular Dystrophy
|
|
Becker Muscular Dystrophy
|
|
Intervention(s)
|
|
Drug: Sildenafil
|
|
Primary Outcome(s)
|
|
Change in Cardiac Left Ventricular End-systolic Volume (LVESV) by Cardiac Magnetic Resonance (CMR) Imaging.
[Time Frame: 6 months compared to baseline]
|
|
Secondary Outcome(s)
|
|
Change in Cardiac Mass
[Time Frame: 6 months and 12 months]
|
|
Change in Cardiac Systolic and Diastolic Function by CMR
[Time Frame: 6 months and 12 months]
|
|
Ejection Fraction
[Time Frame: 6 months]
|
|
Change in Skeletal Muscle Strength
[Time Frame: 6 months and 12 months]
|
|
Change in Forced Vital Capacity (FVC) by Pulmonary Function Testing
[Time Frame: 6 months and 12 months]
|
|
Secondary ID(s)
|
|
NA-00036602
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|