Ignite Creation Date:
2024-05-06 @ 12:14 AM
Last Modification Date:
2024-10-26 @ 10:46 AM
Study NCT ID:
NCT01521546
Status:
COMPLETED
Last Update Posted:
2016-11-08
First Post:
2012-01-26
Brief Title:
Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
Sponsor:
Subha Raman
Organization:
Ohio State University
Study Overview
Official Title:
Early Treatment With Aldosterone Antagonism Attenuates Cardiomyopathy in Duchenne Muscular Dystrophy
Status:
COMPLETED
Status Verified Date:
2016-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
E-SCAR DMD
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 drugs 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
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 drugs 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 3500 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None