Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

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Brief Title

Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

Official Title

Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

Brief Summary

      The study is to demonstrate non-inferiority of spironolactone vs. eplerenone in preserving
      cardiac and pulmonary function in patients with preserved LV ejection fraction. Males with
      Duchenne muscular dystrophy (DMD) confirmed clinically and by mutation analysis will be
      enrolled. Subjects will be randomized to either eplerenone or spironolactone. Subjects will
      use a drug diary to record daily compliance of taking the study medication as well as any
      concerns they may have during the study period. Subjects will undergo cardiac magnetic
      resonance imaging (CMR) and pulmonary function tests (PFT) at baseline and then again at 12
      months post enrollment. Subjects will also complete a quality of life questionnaire at
      baseline and 12 months. Degree of elbow contracture will be measured using a goniometer at
      baseline and 12 months.
    

Detailed Description

      DMD is an X-linked disorder in which the sarcolemmal protein dystrophin is effectively
      absent. Males with DMD typically die in the third and fourth decades of life of
      cardiopulmonary disease. Mouse models of DMD, autopsy data, and in vivo human studies using
      magnetic resonance-based late gadolinium enhancement imaging (LGE) have shown that
      progressive myocardial damage is well underway before left ventricular ejection fraction (LV
      EF) becomes abnormal.

      Exertional symptoms and signs of myocardial disease are typically absent as skeletal muscle
      disease progressively limits functional capacity in affected boys. Thus, cardiac involvement
      can go undetected until LV dysfunction and myocardial fibrosis are advanced. While
      echocardiography remains a useful tool to evaluate LV dysfunction, CMR with LGE is
      advantageous for DMD patients since it identifies myocardial injury before decline in EF is
      apparent by echocardiography. Further, greater reproducibility affords efficient sample sizes
      for cardiomyopathy clinical trials in patients with rare diseases. CMR's increasing
      availability at DMD clinical centers has afforded earlier cardiomyopathy detection, and has
      helped refine current management to typically include agents such as angiotensin converting
      enzyme inhibitors (ACEI) once damage is evident. This strategy, however, may not be
      sufficient, with prior studies showing decline in systolic function with or without ACEI or
      angiotensin receptor blocker (ARB) therapy.

      The investigators previously tested mineralocorticoid receptor antagonism (MRA) added to ACEI
      while EF was still normal in a mouse model that mimics the myocardial damage seen in DMD
      patients. This combination significantly reduced myocardial injury and improved (made more
      negative) LV circumferential strain (Ecc), a sensitive and early marker of LV systolic
      dysfunction. Additionally, preliminary findings from a recently completed clinical trial
      suggests efficacy of eplerenone vs. placebo, while further preclinical data suggests greater
      benefit without concomitant steroid use. Thus, a non-inferiority trial comparing MRAs is
      needed.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Left Ventricular Strain


Condition

Duchenne Muscular Dystrophy

Intervention

Eplerenone

Study Arms / Comparison Groups

 Eplerenone
Description:  Eplerenone is an aldosterone antagonist used as an adjunct in the management of chronic heart failure. It is marketed under the trade name Inspra. Eplerenone is a potassium-sparing diuretic.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

52

Start Date

March 20, 2015

Completion Date

May 2018

Primary Completion Date

May 2018

Eligibility Criteria

        Inclusion Criteria:

          -  Boys age ≥7 years with DMD confirmed clinically and by mutation analysis able to
             undergo cardiac magnetic resonance (CMR) without sedation

          -  LV EF ≥45% (+/-5%) by clinically-acquired echocardiography, nuclear scan or cardiac
             MRI done within 2 weeks of enrollment

        Exclusion Criteria:

          -  Non-MR compatible implants

          -  Severe claustrophobia

          -  Gadolinium contrast allergy

          -  Kidney disease

          -  Prior use of or allergy to aldosterone antagonist

          -  Use of other investigational therapy.
      

Gender

Male

Ages

7 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Subha V Raman, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02354352

Organization ID

2014H0387


Responsible Party

Principal Investigator

Study Sponsor

Ohio State University

Collaborators

 University of California, Los Angeles

Study Sponsor

Subha V Raman, MD, Principal Investigator, Ohio State University


Verification Date

September 2019