Pediatric drug trial marks milestone for treatment of congenital heart defect

Teens with a single ventricle heart defect significantly improved their capacity to sustain moderate exercise by taking an oral medication. The trial results, researchers say, represent a milestone in the care for those who have undergone the Fontan procedure, a palliative operation for this patient population.

The Fontan Udenafil Exercise Longitudinal (FUEL) trial enrolled 400 male and female participants aged 12 to 18 from 30 Pediatric Heart Network centers and auxiliary sites across the United States, Canada, and South Korea.

Participants in the udenafil group had significant improvement in oxygen consumption and other measures of exercise capacity during moderate activity when compared to participants who took a placebo. Peak exercise performance numerically improved, although this measure did not reach statistical significance.

Udenafil was well tolerated by the participants. The study, funded by NHLBI, published in the journal Circulation.