FUEL Pediatric Drug Trial

Child Health Research Institute (CHRI) investigators Angela Yetman, MD (site principal investigator) and Sara Warta, BSN, RN (research nurse coordinator), Ling Li, MD, PhD, APCA, FASE (research associate) and Mary Craft, RDCS, FASE (lead research sonographer) participated in an NIH-funded pediatric drug trial that showed promising results for teenagers born with a single working ventricle of the heart. Teenagers who participated in the trial, conducted in partnership with the Pediatric Heart Network (funded by the National Heart, Lung, and Blood Institute, part of the National Institutes of Health) and Mezzion Pharma, showed significant improvement in their ability to sustain moderate exercise after treatment with udenafil, a drug that helps improve blood flow.

To improve flow of oxygenated blood to the rest of the body, babies born with single ventricle defects undergo a series of three palliative procedures, concluding in the Fontan operation. Babies that have undergone the Fontan operation see satisfactory blood and oxygen distribution during childhood, but function will worsen through adolescence and early adulthood. This results in a decline in exercise capacity and an increase in likelihood of heart failure symptoms, hospitalization and death.

The purpose of the Fontan Udenafil Exercise Longitudinal (FUEL) trial was to determine the impact of udenafil on exercise performance over a six-month period in adolescents who have undergone the Fontan operation. Half of the participants were randomly selected to receive udenafil twice daily, while the other half was given a placebo. To measure exercise capacity, researchers had participants pedal on a special exercise bike programmed to increase resistance while wearing a mask that measured oxygen consumption.

After six months, participants in the udenafil group showed significant improvement to sustain sub-maximal exercise measured by oxygen consumption (+33 mL/min), compared to the placebo group (-9 mL/min). These levels are near the normal exertion levels of daily living, a resounding success in terms of improvement.

The FUEL trial is a prime example of CHRI living its mission to improve the health of children through innovative research, collaborative discovery, community engagement and advocacy. With the involvement of researchers at 30 sites and 400 participants, collaboration proved a key ingredient for success in determining the positive impact of udenafil on exercise capacity in adolescents who have undergone the Fontan operation.

CHRI investigators and personnel received an award for enrolling a high number of participants in a short amount of time – a testament to the relentless efforts to improve the health of children. Warta recognizes the significance of this study and the need for future research projects of its nature. “It was a privilege to be able to work on such an important study. There are limited treatment options for this population. I believe that the results from this research will help guide future research and efforts to providing improved care and wellness for patients who have undergone the Fontan procedure in the future. I am excited to see what is next!”

NIH published a news release on the FUEL trial, and the full study can be found here. Follow the Pediatric Heart Network @PHNresearch.


by Ilyssa Schaming | CHRI Marketing Coordinator | January 16, 2020