Spironolactone Versus Prednisolone in DMD

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Brief Title

Spironolactone Versus Prednisolone in DMD

Official Title

A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMD

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 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).
    

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, 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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

Efficacy: Change in time to complete a 100 meter timed test.

Secondary Outcome

 Efficacy: Dynamometry score

Condition

Muscular Dystrophy, Duchenne

Intervention

Spironolactone

Study Arms / Comparison Groups

 Spironolactone
Description:  Twelve subjects will be prescribed a standard clinical dose of spironolactone of 1 mg/kg/day. The spironolactone will be provided as suspension.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

2

Start Date

December 5, 2018

Completion Date

November 30, 2021

Primary Completion Date

September 27, 2021

Eligibility 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
      

Gender

Male

Ages

4 Years - 7 Years

Accepts Healthy Volunteers

No

Contacts

Kevin Flanigan, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03777319

Organization ID

IRB17-00240


Responsible Party

Sponsor-Investigator

Study Sponsor

Kevin Flanigan

Collaborators

 Muscular Dystrophy Association

Study Sponsor

Kevin Flanigan, MD, Principal Investigator, Nationwide Children's Hospital


Verification Date

January 2022