Phase 1 study of Spironolactone versus Prednisolone in people with Duchenne

A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid Naïve Boys with DMD

Questions and Answers

What stage is this research?

This trial is actively recruiting participants at Nationwide Children’s Hospital. Other sites may be opening up in the future.

What is the goal or purpose of this study?

The goal of this study is to determine the safety of spironolactone in boys with DMD. If it is safe, we will then conduct a larger scale trial to determine its effectiveness compared to prednisone.

Who is funding this study?

This study is funded by the Muscular Dystrophy Association.

Who is eligible to participate in this study?

To participate in this study you must be a boy with DMD between the ages of 4 and 7 who is able to cooperate with physical therapy assessments and is steroid naive. Heart function must be normal. You must not have any allergies to spironolactone, kidney problems, peptic ulcer disease, or recent gastrointestinal perforations.

What do I have to do if I decide to participate in this study?

This study will involve three clinic visits, occurring at screening, 3 months after starting study drug, and 6 months after starting study drug. This study will also involve monthly surveys that will be done online and blood draws that can be done at a lab near your home.

How long will this study last, and will I have access to the drug/treatment once the study has ended?

The anticipated length of the study is 6 months. Depending on the results of the study, you may have access to the drug/treatment while data is being analyzed and a larger scale efficacy trial is being performed.

How many visits to the study site are necessary?

3

Can any visits be done locally?

Study visits cannot be done locally, but blood draws can be done locally

Is there any funding to help pay for travel?

No, there is no funding to help pay for travel.

Will I get paid for participating in this study?

No.

Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new research treatments before they are widely available and having access to medical specialists that are normally not available to you or your child, and helping others by contributing to the better understanding of DMD.

Spironolactone is an FDA-approved drug with a long history of demonstrated safety and efficacy in children and adults with cardiomyopathy. Spironolactone is from a class of drugs also recently shown to be beneficial for heart function in older DMD patients. Numerous preclinical studies in muscular dystrophy models support that spironolactone and this class of drugs, have benefit on muscle strength and preventing muscle damage.

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