Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2018-05-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ohio State University
• Collaborators: University of California, Los Angeles; University of Colorado, Denver; University of Kansas Medical Center; University of Utah; Vanderbilt University; Vanderbilt University Medical Center
• Treatments: Diuretics, Potassium Sparing; Eplerenone; Mineralocorticoid Receptor Antagonists; Spironolactone

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.