Inability to recruit participants.

This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy. The goals are to determine the safety of 6 months of treatment with spironolactone treatment int he steroid-naive DMD population as well as to determine if either spironolactone or a standard clinical dose of corticosteroids results in equivalent improvement in time to complete the 100 meter timed test (100M).

Until recently, the only treatment shown to improve strength and preserve ambulation in DMD patients was the use of glucocorticoids, which are accompanied by significant side effects including obesity, cushingoid features, osteoporosis, and behavioral disturbances. Spironolactone is an aldosterone antagonist primarily used as a potassium sparing diuretic that is widely used in the pediatric population, with limited side-effects including gynecomastia and hyperkalemia. Recent studies by Dr. Rafael-Fortney have evaluated the effect of spironolactone treatment in several different mouse models of DMD. Her results show that treatment of these mice demonstrates increased muscle membrane stabilization while reducing the negative side-effects typically associated with standard of care glucocorticoids. This pilot study is designed to determine whether this commonly used medication, spironolactone, may have similar beneficial effects with a lower side effect profile and be applicable to a wider population of DMD patients.

The hypothesis for this controlled pilot trial is that spironolactone and prednisolone are of equal efficacy in improving skeletal muscle function over a 6-month period, and that spironolactone will be well tolerated in this patient population.

One outcome is that both drugs demonstrate equal efficacy in motor function. This would then serve as pilot data for a longer term study.

Inclusion Criteria:

• Duchenne muscular dystrophy (DMD) patients ≥4 to ≤7 years of age
• Clinical features of DMD that include proximal predominant weakness and/or gait disturbance
• Presence of a truncating mutation of the DMD gene in the patient or an affected male relative OR a muscle biopsy that demonstrates <5% dystrophin in the patient or an affected male relative
• Normal left ventricular ejection fraction by screening echocardiogram
• Ability to cooperate for testing
• No prior treatment with glucocorticoids or vamorolone
• No concomitant experimental therapies

Exclusion Criteria:

• Subject amenable to or currently being treated with eteplirsen, casimersen, or viltolarsen
• Hyperkalemia at screening
• History of or ongoing renal failure (elevated creatinine, oliguria, anuria)
• Hypersensitivity to spironolactone (rash, respiratory distress, arrhythmia, numbness or tingling of extremities)
• Current treatment with an ACEi
• Severe peptic ulcer disease or recent gastrointestinal perforations

Data was collected for each participant over a 7 month duration.

Full enrollment into the study was unable to be obtained.