Research Study for Males with Duchenne Muscular Dystrophy – August 8th, 2012

Early Treatment of Cardiomyopathy in DMD

 

Frequently Asked Questions (FAQs) About   This Study

 

What is the purpose of the study?

  • To determine if eplerenone, a type of medication typically used for advanced heart failure patients, in combination with another medicine (ACE-I or ARB) can prevent heart muscle damage in people with DMD.

Who is eligible for the study?

  • If you are a male with DMD, at least 7 years old and:
    • no kidney problems
    • not on eplerenone or other drugs like it
    • can undergo a cardiac MRI scan without sedation and follow breathhold instructions
    • have preserved left ventricular systolic function

How long will this study last?

  • 12 months

  How many study visits are there?

  • After the initial (baseline) screening visit, there are five study visits during a 12-month period.
  • The study visits will happen during months one, three, six, nine and 12.

Where do the study visits take place?

  • The baseline visit will take place either at The Ohio State University (OSU) in Columbus, OH or Cincinnati Children’s in Cincinnati, OH.
  • Blood draw at 1, 3 and 9 months can be done at a local laboratory where you reside.
  • Visits 6 and 12 months are done at the same site where you did your baseline visit.

What will happen during this study?

  • Participants will be randomly assigned to receive either eplerenone (the study drug) or a placebo. A placebo is an inactive substance that looks like the study drug, but contains no medication.
  • Participants will take assigned drug every other day for one month.
  • If after one month, levels of potassium and creatinine (marker of kidney function) are normal, participants will continue to take the drug once daily for the remainder of the study.
  • All participants will have blood drawn at the baseline visit as well as during months 1, 3, 6, 9 and 12.
  • All participants will have a cardiac MRI scan at the baseline, 6 and 12 months.

 Is there payment for participation?

  • There is no payment for participating in the study but you will be reimbursed for travel, food and lodging expenses upon completion of the 6 month visit.
  • The blood draws during months 1, 3, 6 and 9 will be paid for by the study.
  • The cardiac MRI scan at the 6 and 12 month visit will be paid for by the study.
  • The study drug will be provided for free.

Will there be access to the drug once the study has ended?

  • If the results are promising, your physician may choose to prescribe this medication to you or your child after the study has ended.

 Why should I consider participating in this study?

  • 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

and/or

  • help others by contributing to the better understanding and treatment of DMD-associated heart disease.

Where can I learn more about this study?

  • You can learn more about this study at PPMD.com and/or ClinicalTrials.gov.
  •  You may also contact:

Dr. Subha Raman or Beth McCarthy at The Ohio State University, (614) 688-8020 or raman.1@osu.edu

Dr. Kan Hor or Lani Relucio at Cincinnati Children’s, (513)803-1910 or kan.hor@cchmc.org

 

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