One versus two medicines for children with pulmonary arterial hypertension
Official title: 2/2 Kids MoD PAH Trial: Mono- vs. Duo-Therapy for Pediatric Pulmonary Arterial Hypertension-DCC
Funded at Duke University · NIH-11364147 · Data updated 2026-11-12
This compares starting one medicine versus starting two medicines for children with pulmonary arterial hypertension to find which approach helps symptoms, exercise ability, and heart health.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project start | September 15, 2021 |
| Project end | November 30, 2026 |
| Funding year | 2026 |
| Award (this year) | $465,712 |
| NIH project number | 5U24HL151457-05 |
| Project ID | NIH-11364147 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents newly diagnosed with pulmonary arterial hypertension who can attend regular visits at a participating pediatric center are ideal candidates.
✕Less likely to help
Children already on long-term combination therapy, those who cannot take sildenafil or bosentan, or those with other severe comorbid conditions may not receive benefit from this protocol.
What this research studies
If my child joins, they would be enrolled across multiple pediatric hospitals and randomly start either a single PAH medicine (like sildenafil) or two medicines together (sildenafil plus bosentan). A central data-coordinating center will collect age-appropriate outcomes such as exercise ability, heart and lung pressures, quality of life, and long-term follow-up. The approach uses lessons from adult combination therapy but adapts measurements and safety monitoring for infants, children, and adolescents. The goal is to define the best initial treatment approach and standardize outcome measures for pediatric PAH care.
Why it matters
Potential benefit: If successful, this could establish a clearer first-line treatment for children with PAH that improves symptoms, exercise capacity, and long-term heart health.
How similar studies have gone: Randomized adult trials have shown that initial combination therapy improves pulmonary pressures, exercise tolerance, and quality of life, but randomized data in children are limited.
Where this research is happening
-
Duke University
Durham, United States Active
Researchers
- Principal investigator: Chakraborty, Hrishikesh — Duke University
- Study coordinator: Chakraborty, Hrishikesh
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.