The Fontan Dapagliflozin Pilot Study (FonDap)

JOHNS HOPKINS MEDICAL INSTITUTION IN COLLABORATION WITH UCLA

Principal Investigator: Ari Cedars MD, Associate Professor in the Division of Cardiology at the Johns Hopkins Medical Institution (JHMI) and the Director for the Adult Congenital Heart Disease Clinic in the Blalock-Taussig-Thomas Pediatric and Congenital Heart Center at Johns Hopkins Children's Center.

Abstract: Congenital heart disease is the most common form of birth defect, impacting nearly 40,000 live births each year. Medical and surgical advances have permitted the great majority of these defects to be effectively repaired in childhood. However, individuals with the most complex forms of congenital heart disease, those with only one functional pumping chamber, are an exception. Due to a lack of other options, these patients frequently end up undergoing a palliative procedure called a Fontan repair instead of a complete repair of their heart. The Fontan repair creates a very abnormal circulatory system that is prone to failing when patients are in their 30s and 40s1. Currently, there are no therapies proven to prolong survival in patients with a Fontan repair. In our study, we plan to investigate how a new treatment for Fontan patients helps improve the abnormalities in their circulatory system. For this study, we will be using a class of drugs called SGLT2 inhibitors that are very beneficial in patients with heart failure without congenital heart disease but have not been tested in patients with a Fontan repair. Because of the unique circulatory abnormalities resulting in Fontan heart failure, we believe that patients with a Fontan repair will benefit from therapy with SGLT2 inhibitors.

We hypothesize that the SGLT2 inhibitor dapagliflozin will decrease the physiologic stress imposed by the Fontan circulation.