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Phase 3 Completed N=52 Randomized Quadruple-blind Treatment

Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

Source: ClinicalTrials.gov NCT02354352 ↗
Enrolled (actual)
52
Serious AEs
9.6%
Results posted
Sep 2019
Primary outcomePrimary: Left Ventricular Strain — 0.2; 0.4 Percent change in circumference — p=0.5867
◆ Published Evidence
Established
66citations · ~9 / year
Stabilization of Early Duchenne Cardiomyopathy With Aldosterone Inhibition: Results of the Multicenter AIDMD Trial.
Journal of the American Heart Association · 2019 · Open access · Likely link

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.

Linked Publications

  • Stabilization of Early Duchenne Cardiomyopathy With Aldosterone Inhibition: Results of the Multicenter AIDMD Trial.
    Journal of the American Heart Association · 2019 · 66 citations · Open access · Likely link

Outcome Measures

OutcomeResultp-value
PRIMARY
Left Ventricular Strain
0.2; 0.4 0.5867

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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02354352) and the linked publication. 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. Informational only — not medical advice.

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