Clinical Research Study Viewer

Research Study for Males 10 Years and Older with Duchenne Muscular Dystrophy

What is the purpose of the study?

The purpose of this study is to see if both spironolactone and eplerenone (Inspra), generic drugs used in other types of heart conditions, help maintain heart and lung function in boys with Duchenne Muscular Dystrophy (DMD).

Who is eligible for the study?

If you are a male with DMD, at least 10 years old, AND:

  • Non-ambulatory
  • Do not have kidney problems
  • Not taking eplerenone or spironolactone
  • Can undergo a cardiac magnetic resonance imaging (MRI) scan without sedation
  • Have preserved heart function (left ventricular ejection fraction ≥45%±5%)

How long will this study last and how many study visits are there?

  • The study lasts for 12 months.
  • After the initial (baseline) screening visit, there are five (5) study visits during the 12-month period.
  • You will receive a follow-up phone call two weeks after enrollment. 
  • The study visits after the baseline visit will happen during the following time periods: 1 month, 2 months, 3 months, 6 months, 9 months and 12 months.

Where do the study visits take place?

  • The baseline visit will take place at one of the following:
    • The Ohio State University (OSU) in Columbus, OH 
    • Mattel Children’s Hospital in Los Angeles, CA
    • University of Colorado Hospital in Aurora, CO
    • University of Utah Hospital in Salt Lake City, UT
  • Blood draw at 1, 2, 3, 6, and 9 months can be done at a local laboratory where you reside.
  • The 12-month visit will be done at the same site where you had your baseline visit.

What will happen during this study?

  • Participants will be randomly assigned to receive either eplerenone or spironolactone.
  • Participants will take one tablet of the assigned drug once daily for duration of the study.
  • All participants will have blood drawn at the baseline visit as well as at months 1, 2, 3, 6, 9 and 12.
  • All participants will have a cardiac MRI scan and pulmonary function tests at baseline and 12 months.
There is no payment for participating in this study, but you will be reimbursed for travel, food and lodging expenses at the baseline and 12-month visit. The study drug will be provided by the study, and the blood draws will be paid for by the study. The cardiac MRI scan and pulmonary functional test at baseline and 12-month visit will be paid for by the study, if it is not part of your regular clinical visit.

What are the benefits of participating?

If the results are promising, this medication may be available to you or your child after the study has ended. While no personal benefit can ever be guaranteed, there are other benefits to participating in a research study. You may be able to:

  • Play an active role in your own health care (or that of your child)
  • Gain access to new research treatments before they are widely available
  • Have access to medical specialists who may not normally be available
  • Help others by contributing to the better understanding and treatment of DMD-associated heart disease

How do I learn more about this study?

You can learn more about this study at ClinicalTrials.gov. You may also contact:

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000