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.
ClinicalTrials.gov 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:
- WSPH PAH groups 1 or 3
- 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
Sponsor/Funding
NIH National Heart, Lung, and Blood Institute (NHLBI)
Learn More
Clinical Coordinating Center PL: Eric Christensen