The National Heart, Lung, and Blood Institute of the National Institutes of Health (NIH) has awarded two separate grants, totaling $6.1 million, to Cleveland Clinic to support testing the effects of a common drug to treat diabetes on patients with pulmonary arterial hypertension (PAH). Researchers plan to lead a future national multi-site Phase 2 clinical trial that will be the first time the drug, empafliflozin, an SGLT2 inhibitor, will be tested to target the right heart.
Pulmonary arterial hypertension (PAH) is a rare and deadly condition that kills 40% of patients within five years of diagnosis, mostly due to right heart failure. All approved PAH drugs target the lungs, not the heart. Empagliflozin is currently used as a treatment for diabetes and for left heart failure.
“We chose to investigate empagliflozin because of its proven success as a treatment for managing left heart failure and its ability to modulate key metabolic pathways dysregulated in heart failure,” said Gustavo Heresi, M.D., Cleveland Clinic pulmonologist and researcher. The planned trial will be led by Dr. Heresi and Jennifer Gassman, Ph.D., biostatistician and trialist at Cleveland Clinic Lerner Research Institute. Together, their teams secured the research funding from the NIH.
Dr. Heresi’s previous work has shown that metabolic abnormalities are very common in PAH, worsening heart function and severely impacting an individual’s quality of life and survival. He believes empagliflozin may help lessen the common symptoms of PAH, including shortness of breath and fluid retention — symptoms which are caused by the gradual failure of the right heart.
To test this hypothesis, a planned randomized, triple-masked, parallel arm Phase 2 clinical trial of empagliflozin versus placebo will include PAH patients experiencing right heart failure and already on existing PAH therapeutic drugs that target the lungs. After each patient has 24 weeks on the study medication, researchers will determine via cardiac MRI the patient’s change in right heart function.
“If this trial shows empagliflozin is effective, I expect significant improvements in PAH patients’ symptoms, quality of life and, critically, the function of their right heart,” said Dr. Heresi.
Several multidisciplinary teams at Cleveland Clinic will collaborate on the trial, which is supported by two separate grants comprising the total funding. Vanderbilt University Medical Center and Johns Hopkins Medical Center are other planned sites for the trial.
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