Spironolactone Versus Prednisolone in DMD
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 4 - 7 |
| Updated: | 2/3/2019 |
| Start Date: | December 5, 2018 |
| End Date: | December 2021 |
| Contact: | Allie Fenter |
| Email: | allie.fenter@nationwidechildrens.org |
| Phone: | 614-355-2759 |
A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMD
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).
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.
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 OR a muscle biopsy that demonstrates
<5% dystrophin in the patient or an affected relative
- Normal left ventricular ejection fraction by screening echocardiogram
- Ability to cooperate for testing
- No prior glucocorticoid treatment
- No concomitant experimental therapies
Exclusion Criteria:
- Subject amenable to or currently being treated with eteplirsen
- 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
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