This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.
This study is a 26 week, prospective, multicenter, randomized, double-blinded, placebo-controlled safety and efficacy study of udenafil vs. placebo in adolescent subjects who have had the Fontan procedure. The primary efficacy endpoint will be change from baseline at 26 weeks in peak minute oxygen consumption (VO2 mL/kg/min) as measured by maximal cardiopulmonary exercise test (CPET) reading laboratory who will be blinded to treatment allocation.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
436
Change in Exercise Capacity
The change in exercise capacity (as measured by maximal VO2 at maximum exercise effort) from baseline to 26 weeks (or study completion)
Time frame: Baseline to 26 Weeks
Change in VO2 at the ventilatory anaerobic threshold (VAT)
Change in peak oxygen consumption (VO2) from baseline to Week 26 as measured by maximal central cardiopulmonary exercise test (CPET) reading laboratory.
Time frame: Baseline to 26 weeks
Change in Enhanced Liver Fibrosis (ELF) Score
Change from baseline to Week 26 in the Enhanced Liver Fibrosis (ELF) Score calculated by the change in the following biomarkers: hyaluronic acid, amino-terminal propeptide of type III collagen, and tissue inhibitor of metalloproteinase-1. subjects with scores of \<7.7 were assigned to the no or mild fibrosis group; 7.7 to 9.8 to the moderate fibrosis group, and \>9.8 to the severe fibrosis group.
Time frame: Baseline to 26 weeks
Work rate (watts) at ventilatory anaerobic threshold (VAT)
Change from baseline to 26 weeks in work rate in watts at the ventilatory anaerobic threshold as measured by the central cardiopulmonary exercise test reading laboratory.
Time frame: Baseline to 26 weeks
Change in ventilatory efficiency (VE/VCO2) at ventilatory anaerobic threshold
Change from baseline to Week 26 in ventilatory efficiency calculated by ventilatory efficiency divided by minute carbon dioxide production at the ventilatory anaerobic threshold (VE/VCO2 at VAT).
Time frame: Baseline to 26 weeks
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