Single versus combination oral treatment for children with pulmonary arterial hypertension
Official title: 1/2 Kids MoD PAH Trial: Mono- vs. Duo-Therapy In Pediatric Pulmonary Arterial Hypertension
Funded at Johns Hopkins University · NIH-11364094 · Data updated 2026-11-12
This project compares starting one oral PAH medicine versus starting two (sildenafil plus bosentan) right away in children with pulmonary arterial hypertension to see which approach better helps breathing, heart function, and daily life.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | September 15, 2021 |
| Project end | May 31, 2026 |
| Funding year | 2026 |
| Award (this year) | $491,662 |
| NIH project number | 5UH3HL151458-05 |
| Project ID | NIH-11364094 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children diagnosed with pulmonary arterial hypertension who are eligible for oral PAH medications and not already on long-term combination PAH therapy would be the best candidates.
✕Less likely to help
Children who cannot take sildenafil or bosentan due to medical contraindications, drug interactions, or whom these drugs are not appropriate for are unlikely to benefit from this approach.
What this research studies
If your child is diagnosed with pulmonary arterial hypertension, doctors at multiple pediatric centers will randomly assign them to take either a single oral PAH drug (sildenafil) or a combination of sildenafil plus bosentan from the beginning. Your child will have scheduled visits with tests like heart imaging, exercise or walk tests, symptom and quality-of-life questionnaires, and some specialized measurements of lung pressures. The team will use age-appropriate measures and follow children over time to learn about short- and long-term outcomes. The goal is to determine whether starting both medicines right away improves health and daily functioning compared with starting one medicine.
Why it matters
Potential benefit: If successful, this could help doctors pick a better first-line treatment for children with PAH and improve symptoms, heart and lung function, and quality of life.
How similar studies have gone: Randomized adult trials of upfront combination therapy have shown improved outcomes, but randomized multicenter trials in children are rare, so this pediatric approach is promising but less tested.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Romer, Lewis H — Johns Hopkins University
- Study coordinator: Romer, Lewis H
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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.