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Trial record 31 of 1002 for:    BMD

Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy (E-SCAR DMD)

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ClinicalTrials.gov Identifier: NCT01521546
Recruitment Status : Completed
First Posted : January 30, 2012
Results First Posted : May 27, 2015
Last Update Posted : November 8, 2016
Sponsor:
Collaborator:
Ballou Skies
Information provided by (Responsible Party):
Subha Raman, Ohio State University

Tracking Information
First Submitted Date  ICMJE January 26, 2012
First Posted Date  ICMJE January 30, 2012
Results First Submitted Date  ICMJE April 21, 2015
Results First Posted Date  ICMJE May 27, 2015
Last Update Posted Date November 8, 2016
Study Start Date  ICMJE February 2012
Actual Primary Completion Date June 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 28, 2015)
12-month Change in Myocardial Strain [ Time Frame: baseline and 12 months ]
a sensitive measurement of heart function using cardiac MRI, change was 12 months minus baseline.
Original Primary Outcome Measures  ICMJE
 (submitted: January 26, 2012)
myocardial strain [ Time Frame: 1 year ]
a sensitive measurement of heart function using cardiac MRI
Change History Complete list of historical versions of study NCT01521546 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE
 (submitted: January 26, 2012)
collagen turnover [ Time Frame: 1 yr ]
a measurement of collagen turnover in the blood
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Official Title  ICMJE Early Treatment With Aldosterone Antagonism Attenuates Cardiomyopathy in Duchenne Muscular Dystrophy
Brief Summary

Duchenne muscular dystrophy (DMD), the most common muscular dystrophy, leads to skeletal and cardiac muscle damage. Treatment of pulmonary complications has improved survival; however, heart muscle disease or cardiomyopathy has emerged as a leading cause of death, typically by the third decade. Although myocardial changes begin early, clinically significant heart disease is rarely detected in the first decade of life. Consequently, DMD cardiomyopathy frequently goes unrecognized (and untreated) until advanced (and irreversible).

Current DMD cardiovascular care guidelines recommend beta-blockers and angiotensin converting enzyme inhibitors (ACEIs) when decreased ejection fraction (EF) is noted by echocardiography (echo); however, this strategy has not significantly improved outcomes. Our team has recently made a breakthrough in a mouse study, showing in a model that causes the same heart muscle disease in humans with DMD adding an old medicine traditionally used for high blood pressure and late-stage heart failure can actually prevent heart muscle damage. Because of this drug's proven safety in both children and adults, it is ready to be studied immediately in an RCT in patients with DMD to hopefully show, as we did in mice, that we can prevent the devastating consequences of heart muscle damage.

Detailed Description Duchenne Muscular dystrophy (DMD) is a deadly X-linked disease affecting 1 in 3,500 males. DMD patients suffer significant disability due to skeletal myopathy and excess death due to cardiomyopathy. Current guidelines advocate initiating cardioprotective treatment with evident global cardiac dysfunction, yet this treatment paradigm has not improved survival much beyond the third decade of life. Potentially promising approaches like gene therapy will take considerable time to improve outcomes. Recently completed studies in a DMD mouse model at our institution indicate that existing drugs known as aldosterone antagonists, typically reserved for advanced heart failure patients, preserve skeletal and cardiac muscle function at 80% of normal. Clinical studies at many centers including ours have shown that high-resolution, noninvasive cardiac magnetic resonance (CMR) detects subclinical myocardial fibrosis and abnormal regional function prior to global functional abnormalities. Combining findings from these preclinical and clinical studies, we plan to execute a randomized, controlled clinical trial (RCT) of eplerenone plus background therapy vs. background therapy alone in patients with DMD. We expect that the aldosterone antagonist eplerenone compared to standard therapy significantly delays progressive cardiomyopathy and skeletal myopathy using highly reproducible imaging biomarkers selected for efficient sample size design, to ultimately reduce disability and death.
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: eplerenone
    25mg tablet, once daily by mouth for 12 months
  • Drug: placebo
    one tablet by mouth daily for 12 months
Study Arms  ICMJE
  • Active Comparator: eplerenone
    active study drug
    Intervention: Drug: eplerenone
  • Placebo Comparator: sugar pill
    placebo
    Intervention: Drug: placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 28, 2014)
42
Original Estimated Enrollment  ICMJE
 (submitted: January 26, 2012)
40
Actual Study Completion Date  ICMJE June 2016
Actual Primary Completion Date June 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • DMD patients age 7 years and older (and able to complete cardiac MRI without sedation) with preserved left ventricular (LV) systolic function and abnormal heart muscle by late post-gadolinium imaging (LGE)

Exclusion Criteria:

  • renal insufficiency (GFR <40 mL/min/m2)
  • non-MR compatible implants (e.g. neurostimulator, AICD)
  • severe claustrophobia
  • allergy to gadolinium contrast
  • prior use of or known allergy to epleronone
  • use of potassium-sparing diuretics
  • serum potassium level of >5.0 mmol/L
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 7 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01521546
Other Study ID Numbers  ICMJE 2011H0251
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Subha Raman, Ohio State University
Study Sponsor  ICMJE Subha Raman
Collaborators  ICMJE Ballou Skies
Investigators  ICMJE
Principal Investigator: Subha V Raman, MD, MSEE Ohio State University
PRS Account Ohio State University
Verification Date October 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP