Spironolactone Versus Prednisolone in DMD

• Status: Active, not recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: Male

Summary

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).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Kevin Flanigan
• Collaborator: Muscular Dystrophy Association
• Treatments: Methylprednisolone; Methylprednisolone acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Spironolactone

Criteria

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