Phase 3
N=52
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT02354352 ↗Enrolled (actual)
52
Serious AEs
9.6%
Results posted
Sep 2019
Primary outcome: Primary: Left Ventricular Strain — 0.2; 0.4 Percent change in circumference — p=0.5867
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Eplerenone (Drug); Spironolactone (Drug)
- Age
- Pediatric, Adult, Older Adult · 7+ yrs
- Sex
- Male
- Sponsor
- Ohio State University
- Primary completion
- May 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Left Ventricular Strain |
0.2; 0.4 | 0.5867 |
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.
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.
Data sourced from ClinicalTrials.gov (NCT02354352). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.