Ensuring Access to Optimal Therapy in CF: ENACT study
Funded at Arkansas Children's Hospital Res Inst · NIH-11352661 · Data updated 2026-11-12
A CF study to understand and improve how people with cystic fibrosis respond to a triple therapy by looking at drug levels and genetics to guide dosing.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Arkansas Children's Hospital Res Inst NIH-funded |
| Lab location | 1 site (Little Rock, United States) |
| Project start | July 17, 2024 |
| Project end | June 30, 2029 |
| Funding year | 2026 |
| Award (this year) | $569,850 |
| NIH project number | 5R01HL171034-03 |
| Project ID | NIH-11352661 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with cystic fibrosis currently treated with triple combination CFTR modulator therapy and who are willing to provide blood samples and clinical data.
✕Less likely to help
Patients not taking triple therapy or those unable to participate in serial sampling or follow-up may not benefit.
What this research studies
This study follows people with cystic fibrosis who are taking a triple combination CFTR modulator therapy. It will collect real-world drug concentration data and genetic information related to drug-metabolizing enzymes to understand why responses vary among patients. The team will explore whether adjusting doses based on concentration measurements can improve lung outcomes and reduce side effects. The goal is to develop practical guidance for optimizing therapy in routine care, not just in trials. Participants may contribute blood samples and clinical data over time at multiple visits.
Why it matters
Potential benefit: If successful, patients could receive more personalized dosing that improves lung function and tolerability of CFTR modulator therapy.
How similar studies have gone: Preliminary observations suggest concentration variability exists; this study builds on observational data to test dosing approaches, with potential for meaningful impact.
Where this research is happening
-
Arkansas Children's Hospital Res Inst
Little Rock, United States Active
Researchers
- Principal investigator: Guimbellot, Jennifer S — Arkansas Children's Hospital Res Inst
- Study coordinator: Guimbellot, Jennifer S
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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.