Kids MoD PAH Trial

Mono- vs. Duo-Therapy for Pediatric Pulmonary Arterial Hypertension

About the Study

Currently Recruiting: Participants

Study Drug/Intervention: Study subjects will be randomized to receive combination up-front therapy sildenafil and bosentan or sildenafil alone and will undergo study procedures for monitoring function, safety, and clinical worsening.

Study Timeline: This study is expected to conclude in September 2026. ID: NCT04039464


What is Kids MoD PAH?

A Phase III, randomized, open-label, pragmatic trial to compare the safety and efficacy of first-line combination therapy (sildenafil and bosentan) to first-line monotherapy (sildenafil alone) in pediatric subjects with WHO Functional Classes II or III and precapillary pulmonary hypertension of World Symposium on Pulmonary Hypertension (WSPH) Group 1 (PAH caused by idiopathic, heritable, drugs or toxins, congenital heart disease, or connective tissue disease) or Group 3 (PAH caused by lung disease or hypoxemia) according to the WSPH classification system.


Study Objectives

The primary objective of this study is to compare two treatment strategies – first-line combination therapy (sildenafil and bosentan) versus first-line monotherapy (sildenafil alone) in pediatric subjects with PAH. Subjects will also meet the following 2 criteria:

  1. WSPH PAH groups 1 or 3
  2. Current WHO Functional Classes II or III

Patient Population

Subjects aged 4 months to < 18 years with a diagnosis of precapillary pulmonary hypertension by cardiac catheterization within the previous 4 weeks prior to screening as defined by the standard WSPH definition of PAH. PAH is defined as the presence of mean pulmonary artery pressure > 25mmHg, pulmonary capillary wedge pressure (or left atrial or left ventricular end diastolic pressure) 15 mmHg, and pulmonary vascular resistance index (PVRI) > 3 woods units.

Study Timeline

Duration of Study Participation

24 months (endpoints will be analyzed at 12 and 24 months)

Study Follow-Up

Study subjects will be followed pragmatically with the current standard of care assessments and diagnostics, including longitudinal clinical evaluations, determinations of functional class (FC), serial NT-pro-Brain Natriuretic Peptide (NT-proBNP) levels, and echocardiography at recommended intervals.

Additional Information

Current Sites

  • Baylor College of Medicine; Texas/USA
  • Boston Children’s Hospital; Massachusetts/USA
  • Children’s Hospital Colorado; Colorado/USA
  • Children’s Hospital of Philadelphia; Pennsylvania/USA
  • Columbia University Medical Center; New York/USA
  • Johns Hopkins All Children’s Hospital; Maryland/USA
  • Johns Hopkins Medical Institutions; Maryland/USA
  • Medical College of Wisconsin; Wisconsin/USA
  • Seattle Children’s Hospital; Washington/USA
  • Stollery Children’s Hospital; Alberta/Canada
  • The Regents of the University of California, San Francisco; California/USA
  • Vanderbilt University Medical Center; Tennessee/USA


NIH National Heart, Lung, and Blood Institute (NHLBI)

Learn More

Clinical Coordinating Center PL: Eric Christensen