A Phase 2 study of daily, oral anti-fibrotic therapy to prevent heart muscle disease and improve heart muscle function in ambulatory and non-ambulatory Duchenne patients
Questions and Answers
What stage is this research?
Ifetroban research is at the clinical stage, meaning it has advanced to clinical trials involving people. Ifetroban has been studied in over 26 clinical trials and dosed in over 1,300 people including healthy volunteers. There are three active clinical trials evaluating ifetroban in other rare diseases. Animal studies mimicking Duchenne demonstrated ifetroban prevents heart muscle disease and prolongs survival. Recruitment is anticipated to begin in October 2019.
What is the goal or purpose of this study?
The goal of this clinical trial is to see if ifetroban, a novel targeted drug product, can be safely and effectively used for the treatment of Duchenne heart muscle disease.
Who is funding this study?
This study is funded by Cumberland Pharmaceuticals and the FDA Orphan Product Development program.
Who is eligible to participate in this study?
To participate in this study you must be male, 7 years of age or older, diagnosed with Duchenne and with stable cardiac function.
What do I have to do if I decide to participate in this study?
This study involves taking an oral medication once a day for 12 months, having your finger pricked on the first day of treatment and the 7th day of treatment to measure the oral medication levels in two drops of your blood. We will follow the health of your heart using an MRI before you start treatment and after receiving oral medication for 6 months and 12 months. As you are having your MRI, blood will be drawn to make sure your blood cells and chemistry levels are normal. Your lung function, daily activity, quality-of-life and muscle strength will be checked before you start treatment and after receiving oral medication for 6 months and 12 months. We will call you periodically to see how you are feeling between visits.
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 2 years. Treatment duration is 12 months. There is an optional open-label extension to all participants that complete 12 months of treatment.
Where does this study take place?
This multicenter phase 2 study takes place at several U.S. sites including Riley Children’s Hospital (IN), Vanderbilt’s Children’s Hospital (TN), Children’s National Medical Center (DC), University of California Los Angeles (CA), Emory’s Children’s Healthcare of Atlanta (GA), and Nationwide Children’s Hospital (OH).
How many visits to the study site are necessary?
3 visits over 12 months
Can any visits be done locally?
All 3 visits must be done at one of the participating centers. Some procedures can be completed at home.
Is there any funding to help pay for travel?
Yes. Cumberland will provide patients a stipend for their participation, time and effort in the study. If an overnight stay is needed, Cumberland will provide a travel stipend to cover meals, hotel and mileage.
Why should I consider participating in this study?
Participation will help determine whether ifetroban is an effective therapy for Duchenne heart muscle disease and whether quality-of-life, daily activity and muscle strength improve. 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 may not normally be available to you or your child, and helping others by contributing to the better understanding of Duchenne heart disease.
Where can I learn more about this study?
You can learn more about this study at www.ClinicalTrials.gov (NCT# NCT03340675) or by contacting Cumberland Pharmaceuticals at 615-564-2188 or email@example.com.