1st FDA-funded DMD Study Now Recruiting Patients to Test Ifetroban
Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy — a disease of the heart muscle — linked to Duchenne muscular dystrophy (DMD).
The study, called FIGHT DMD (NCT03340675), is looking to enroll 48 Duchenne patients, ages 7 and older, from various locations across the U.S. Their heart and lung function, muscle strength, daily activity, and quality of life will be assessed during the trial.
Those interested in participating, or wishing to know more, may visit the trial’s website or contact the study coordinator, Ines Macias-Perez, PhD, by phone at (615) 979-5778; a full list of trial locations can be found here.
The U.S. Food and Drug Administration awarded Cumberland $1 million in funding under its orphan products grants program to support this trial. According to Cumberland, this was the first DMD trial awarded such funding, which is intended to advance the development of therapies, devices, or medical foods that show promise for the diagnosis and/or treatment of rare diseases or conditions.
“Studies in animal models of DMD showed oral ifetroban can prevent the scarring in the heart associated with Duchenne and improve survival compared to placebo,” Cumberland said on its website. “Ifetroban research is at the clinical stage, meaning it has advanced to clinical trials involving people.”
“The goal of the FIGHT DMD trial is to slow or prevent the heart disease associated with Duchenne muscular dystrophy,” the company said.
At the time of the 2019 award, Cumberland said it appreciated the FDA’s support of its new therapy for patients it described as critically ill. The study is being done in collaboration with Vanderbilt University Medical Center, in Tennessee.
Heart problems are common in people with DMD. Cardiomyopathy is progressive, resulting in congestive heart failure, cardiac insufficiency, and altered heartbeat. One of the signs of cardiomyopathy is fibrosis, or the thickening and scarring of the heart tissue.
Ifetroban, an inhibitor of the thromboxane receptor — a protein that binds thromboxane, which induces blood clotting and narrows blood vessels — works by reducing fibrosis and fat deposits in the heart. A study has shown that ifetroban prevents scarring of the heart tissue and improves survival in mouse models of muscular dystrophy.
In the double-blind Phase 2 study — in which neither participants nor researchers know which patients receive the medicine and which a placebo — the boys and men will be assigned randomly to receive an oral capsule containing either low-dose or high-dose ifetroban, or a placebo once daily for one year (12 months). Following this period, the participants may join an open-label extension study during which all will have access to ifetroban.
To be eligible, participants must be on a stable dose of oral corticosteroids for at least eight weeks (about two months) or have not received corticosteroids for at least 30 days.
They also must have stable cardiac function. This means that changes in left ventricle ejection fraction (LVEF) — a measure of how well the heart pumps blood with each heartbeat — should be lower than 15%, and their LVEF should be 35% or better. Those who have been admitted to a hospital for heart failure in the last year will be excluded. Patients with evidence of damage to the left ventricular myocardium — the heart muscle — are allowed to join.
To understand whether ifetroban’s effect may change depending on the degree of heart involvement, the researchers plan to test each dose in eight patients with early stage cardiomyopathy (LVEF higher than 45%) and eight others with more advanced disease, and LVEF between 35% and 45%.
Over the course of the study, participants will be required to visit their trial location three times: at the beginning of the study, and at six and 12 months after starting treatment. Blood and urine samples will be collected for testing. The trial, already underway, is expected to conclude in July 2023.