Status:
TERMINATED
Spironolactone Versus Prednisolone in DMD
Lead Sponsor:
Kevin Flanigan
Collaborating Sponsors:
Muscular Dystrophy Association
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
4-7 years
Phase:
PHASE1
Brief 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 o...
Detailed Description
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 5 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 30 2021
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT03777319
Start Date
December 5 2018
End Date
November 30 2021
Last Update
October 23 2023
Active Locations (4)
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1
University of Iowa
Iowa City, Iowa, United States, 52242
2
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
3
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
4
University of Utah
Salt Lake City, Utah, United States, 84108