Design and rationale of the Fontan Udenafil Exercise Longitudinal (FUEL) trial

Pediatric Heart Network Investigators

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

The Fontan operation creates a circulation characterized by elevated central venous pressure and low cardiac output. Over time, these characteristics result in a predictable and persistent decline in exercise performance that is associated with an increase in morbidity and mortality. A medical therapy that targets the abnormalities of the Fontan circulation might, therefore, be associated with improved outcomes. Udenafil, a phosphodiesterase type 5 inhibitor, has undergone phase I/II testing in adolescents who have had the Fontan operation and has been shown to be safe and well tolerated in the short term. However, there are no data regarding the long-term efficacy of udenafil in this population. The Fontan Udenafil Exercise Longitudinal (FUEL) Trial is a randomized, double-blind, placebo-controlled phase III clinical trial being conducted by the Pediatric Heart Network in collaboration with Mezzion Pharma Co, Ltd. This trial is designed to test the hypothesis that treatment with udenafil will lead to an improvement in exercise capacity in adolescents who have undergone the Fontan operation. A safety extension trial, the FUEL Open-Label Extension Trial (FUEL OLE), offers the opportunity for all FUEL subjects to obtain open-label udenafil for an additional 12 months following completion of FUEL, and evaluates the long-term safety and tolerability of this medication. This manuscript describes the rationale and study design for FUEL and FUEL OLE. Together, these trials provide an opportunity to better understand the role of medical management in the care of those who have undergone the Fontan operation.

Original languageEnglish (US)
Pages (from-to)1-8
Number of pages8
JournalAmerican Heart Journal
Volume201
DOIs
StatePublished - Jul 1 2018

Fingerprint

udenafil
Exercise
Fontan Procedure
Safety
Low Cardiac Output
Phosphodiesterase 5 Inhibitors
Phase III Clinical Trials
Central Venous Pressure

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Design and rationale of the Fontan Udenafil Exercise Longitudinal (FUEL) trial. / Pediatric Heart Network Investigators.

In: American Heart Journal, Vol. 201, 01.07.2018, p. 1-8.

Research output: Contribution to journalArticle

Pediatric Heart Network Investigators. / Design and rationale of the Fontan Udenafil Exercise Longitudinal (FUEL) trial. In: American Heart Journal. 2018 ; Vol. 201. pp. 1-8.
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abstract = "The Fontan operation creates a circulation characterized by elevated central venous pressure and low cardiac output. Over time, these characteristics result in a predictable and persistent decline in exercise performance that is associated with an increase in morbidity and mortality. A medical therapy that targets the abnormalities of the Fontan circulation might, therefore, be associated with improved outcomes. Udenafil, a phosphodiesterase type 5 inhibitor, has undergone phase I/II testing in adolescents who have had the Fontan operation and has been shown to be safe and well tolerated in the short term. However, there are no data regarding the long-term efficacy of udenafil in this population. The Fontan Udenafil Exercise Longitudinal (FUEL) Trial is a randomized, double-blind, placebo-controlled phase III clinical trial being conducted by the Pediatric Heart Network in collaboration with Mezzion Pharma Co, Ltd. This trial is designed to test the hypothesis that treatment with udenafil will lead to an improvement in exercise capacity in adolescents who have undergone the Fontan operation. A safety extension trial, the FUEL Open-Label Extension Trial (FUEL OLE), offers the opportunity for all FUEL subjects to obtain open-label udenafil for an additional 12 months following completion of FUEL, and evaluates the long-term safety and tolerability of this medication. This manuscript describes the rationale and study design for FUEL and FUEL OLE. Together, these trials provide an opportunity to better understand the role of medical management in the care of those who have undergone the Fontan operation.",
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