How cystic fibrosis modulator medicine affects young children's lungs and pancreas
Official title: Pulmonary and pancreatic response to cystic fibrosis modulator therapy in young children
Funded at Emory University · NIH-11306644 · Data updated 2026-11-12
This project looks at whether the triple-drug CF medicine ETI helps protect and improve lung health and pancreatic function in young children with cystic fibrosis.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project start | May 15, 2024 |
| Project end | April 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $686,183 |
| NIH project number | 5R01HL173340-03 |
| Project ID | NIH-11306644 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with cystic fibrosis aged about 2–5 years who are starting or already taking ETI, especially those with genetic mutations that make them eligible for the drug.
✕Less likely to help
Children or adults whose pancreas is already largely replaced by scar tissue, or people with CF mutations not helped by ETI, are unlikely to gain pancreatic benefit from this approach.
What this research studies
This project follows young children with cystic fibrosis who are taking the modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) to track lung and pancreas changes over time. Clinicians will use breathing tests, lung imaging, blood and stool markers, and clinical exams to watch for bronchiectasis, exocrine pancreatic function, and early signs of diabetes. The team will compare results before and after ETI and follow children for several years to see whether early treatment prevents damage or allows repair. Findings aim to guide care and monitoring for children started on ETI.
Why it matters
Potential benefit: If successful, this work could show that starting ETI in early childhood preserves lung structure and pancreatic function and reduces the risk of cystic fibrosis–related diabetes.
How similar studies have gone: ETI has already produced major lung benefits in teens and adults, and early reports suggest pancreatic improvements in younger children, but long-term prevention of bronchiectasis and diabetes remains unproven.
Where this research is happening
-
Emory University
Atlanta, United States Active
Researchers
- Principal investigator: Stecenko, Arlene a — Emory University
- Study coordinator: Stecenko, Arlene a
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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.