Single versus combination oral treatment for children with pulmonary arterial hypertension
1/2 Kids MoD PAH Trial: Mono- vs. Duo-Therapy In Pediatric Pulmonary Arterial Hypertension
['FUNDING_OTHER'] · JOHNS HOPKINS UNIVERSITY · NIH-11364094
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
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | JOHNS HOPKINS UNIVERSITY (nih funded) |
| Locations | 1 site (BALTIMORE, UNITED STATES) |
| Trial ID | NIH-11364094 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
BALTIMORE, UNITED STATES
- JOHNS HOPKINS UNIVERSITY — BALTIMORE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ROMER, LEWIS H — JOHNS HOPKINS UNIVERSITY
- Study coordinator: ROMER, LEWIS H
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 below.
Conditions: Acquired brain injury, Cardiac Diseases, Cardiac Disorders