Spironolactone Versus Prednisolone in DMD
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|ClinicalTrials.gov Identifier: NCT03777319|
Recruitment Status : Recruiting
First Posted : December 17, 2018
Last Update Posted : February 1, 2019
|Condition or disease||Intervention/treatment||Phase|
|Muscular Dystrophy, Duchenne||Drug: Spironolactone Drug: Prednisolone||Phase 1|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Subjects will be randomly assigned to either to the spironolactone treatment group or to the standard clinical dose of corticosteroids.|
|Masking:||None (Open Label)|
|Official Title:||A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMD|
|Actual Study Start Date :||December 5, 2018|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
Twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension.
Spironolactone will be prescribed for 6 months, after which the family and primary care physician will determine to either remain on spironolactone or transfer to prednisolone.
Active Comparator: Prednisolone
Twelve subjects will be prescribed a standard clinical dose of prednisolone of 0.75 mg/kg/day or weekend dosing of 5 mg/kg/day as per sites standard of care. The prednisolone will be provided will be provided as suspension.
Prednisolone will be prescribed for 6 months as the clinical standard of care.
- Efficacy: Change in time to complete a 100 meter timed test. [ Time Frame: 6 months ]The determination of whether spironolactone has similar efficacy to glucocorticoids in improving muscle strength in steroid naïve DMD patients. This will be determined by measuring the time to complete a 100 meter timed test (100M).
- Safety will be monitored through regular review of electrolytes. [ Time Frame: 6 months ]Electrolytes (Sodium, Potassium, Cloride and Carbon dioxide, mmol/L) will be measured on a monthly basis following initiation of either spironolactone or prednisolone.
- Efficacy: Dynamometry score [ Time Frame: 6 months ]Secondary outcome measures will be Dynamometry score, which is a summation of maximum voluntary isometric contraction test values for knee flexion, knee extension, elbow flexion, and elbow extension; 4-stair climb; North Star Ambulatory Assessment (NSAA); and time to arise from the floor.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03777319
|Contact: Allie Fenterfirstname.lastname@example.org|
|United States, Ohio|
|Nationwide Children's Hospital||Recruiting|
|Columbus, Ohio, United States, 43205|
|Principal Investigator:||Kevin Flanigan, MD||Nationwide Children's Hospital|
|Principal Investigator:||Megan Waldrop, MD||Nationwide Children's Hospital|