Upcoming trial to test diabetes drug for right heart failure in PAH

2 federal grants totaling over $6M will fund research into empagliflozin

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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An upcoming clinical trial will test whether empagliflozin, a drug widely approved to treat diabetes and heart failure, might be able to improve heart health in people with pulmonary arterial hypertension (PAH).

The trial will be led by scientists at Cleveland Clinic, with study sites planned at two other U.S. medical centers.

The National Heart, Lung, and Blood Institute of the National Institutes of Health (NIH) has awarded two grants, totaling more than $6 million, to help fund the research, the nonprofit medical center said in a Cleveland Clinic press release.

“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 Gustavo Heresi, MD, a pulmonologist and researcher at Cleveland Clinic who will be helping to lead the trial.

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Unlike available PAH therapies that target lungs, diabetes drug acts on heart

PAH is characterized by unusually high pressure in the blood vessels of the lungs. This pressure puts strain on the right side of the heart, which is responsible for pumping blood to the lungs to pick up oxygen. The left side of the heart then pumps the oxygen-rich blood out to the body.

Over time, people with PAH typically develop right heart failure, in which the right side of the heart cannot adequately perform its job. This can contribute to symptoms like shortness of breath, and it’s a major cause of death in PAH.

While several treatments for PAH are available, all of them target the lungs; there are no current PAH therapy options that act on the heart.

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.

Empagliflozin, a drug sold under the brand name Jardiance among others, is approved as a treatment for type 2 diabetes and chronic heart failure. It acts to reduce blood sugar by increasing the amount of sugar excreted into the urine. The medication also has other effects on metabolism and heart health that aren’t fully understood.

“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,” Heresi said.

The newly funded clinical trial will enroll people with PAH who have right heart failure and are already taking available PAH drugs that target the lungs. Participants will be randomly assigned to take empagliflozin or a placebo for about six months, at which point their right heart health will be assessed via an MRI scan.

In addition to Cleveland Clinic, the upcoming study will take place at Vanderbilt University Medical Center in Tennessee and the Johns Hopkins Medical Center in Maryland. Cleveland Clinic did not specify when the trial is expected to begin.