Kids MoD PAH (Mono- vs. Duo Therapy for Pediatric Pulmonary Arterial Hypertension) will be the first investigator-initiated multicenter randomized clinical trial in North America to study the best initial therapy for children with pulmonary hypertension. Pulmonary hypertension is a rare kind of high blood pressure that affects the heart and lungs.
This trial will compare the safety and efficacy of initial combination therapy (sildenafil and bosentan) to initial monotherapy (sildenafil alone) in infants, children, and adolescents with pulmonary arterial hypertension (PAH). PAH is a form of pulmonary hypertension where the lungs’ blood vessels are narrowed, blocked, or destroyed altogether.
Sildenafil and bosentan target different intracellular pathways, and a large study in adults with PAH indicated that combination therapy with agents that have the same mechanisms of action but longer durations led to improved outcomes.
Kids MoD PAH is a phase 3, randomized, open-label, pragmatic trial funded by the National Heart, Lung, and Blood Institute. This study plans to enroll 100 participants at 12-15 sites across the US and Canada. This study was launched via a comprehensive consultation from the Trial Innovation Network (TIN), a collaborative initiative within the National Center for Advancing Translational Sciences. BIOS CTCC, in its role as the Johns Hopkins University-Tufts Trial Innovation Center within the TIN, will make up part of the clinical coordinating center. BIOS CTCC is collaborating with the Johns Hopkins University Department of Anesthesiology and Critical Care Medicine, the Columbia University and University of Colorado Departments of Pediatrics, and the Pediatric Pulmonary Hypertension Network of North America. Duke Clinical Research Institute is serving as the data coordinating center.
ClinicalTrials.gov Identifier: NCT04039464