Questions?
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 27 clinical trials and has been dosed in nearly 1,400 people including healthy volunteers. There are two ongoing clinical trials evaluating ifetroban in other rare diseases. Animal studies mimicking Duchenne demonstrated ifetroban prevents heart muscle disease and prolongs survival. Ifetroban was also shown to be cardioprotective in animal models of non-DMD heart disease. Recruitment started in 2020.
what is the goal or purpose of this study?
The goal of the FIGHT DMD trial is to slow or prevent the heart disease associated with Duchenne muscular dystrophy.
who is funding this study?
This study is funded by the FDA Orphan Product Development (OPD) program and Cumberland Pharmaceuticals. The FIGHT DMD trial is the first DMD trial awarded FDA OPD funding to study an orphan product for DMD. Cumberland Pharmaceuticals is the grant recipient and responsible for the Ifetroban DMD clinical program.
Who is eligible to participate in the fight dmd study?
To participate in this study you must be male, 7 years of age or older, diagnosed with Duchenne and with stable cardiac function. You must have LVEF 35% - 45% or historically documented LVEF 35%-45% and baseline LVEF less than 50%.
What is involved if I decide to participate?
This FIGHT DMD trial involves taking an oral medication once a day for 12 months. You will visit the nearest participating medical center 3 times in 1 year:
Visit 1. the first day you start treatment,
Visit 2. 6 months after starting treatment and
Visit 3. 12 months after starting treatment.
You will have your finger pricked on the first day of treatment and the 7th day of treatment to measure the levels of the oral medication in two drops of your blood. You will do some of these finger pricks at home. We will follow the health of your heart using an MRI before you start treatment and after receiving oral medication for 6 months (optional) and 12 months. Before you have 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 study drug when the trial has ended?
The anticipated length of the study is 2 years: 1 year to enroll 48 participants and 1 year to complete treatment. After completing 12 months of treatment, all participants have the option of participating in the open-label extension. All participants of the open-label extension will have access to ifetroban.
Where does this study take place?
This multicenter phase 2 study is currently open and enrolling at Children’s National Hospital in Washington DC, Riley Children’s Hospital in Indiana, Mattel Children’s Hospital in Los Angeles, California, Emory’s Children’s Healthcare of Atlanta in Georgia, Arkansas Children’s Hospital, Lurie’s Children’s Hospital in Chicago, IL, Washington University in St. Louis, MO, Cincinnati Children’s Hospital in Ohio and Krieger Research Institute in Baltimore, MD. Vanderbilt Children’s Hospital in Nashville, TN is closed to enrollment.
is there funding to help with travel?
We understand there are costs to you and your family. Cumberland can help defray some of these costs for participation.
who can i contact to learn more about participating on this study?
You can contact your doctor or the nearest participating center near you. For further assistance, please call 615-564-2188 or email at fightdmd@cumberlandpharma.com .