Ensuring Access to Optimal Therapy in Cystic Fibrosis
ENsuring ACcess to optimal Therapy in CF: ENACT study
This research looks at how a triple combination therapy for cystic fibrosis works differently in people and aims to find ways to make it more effective and reduce side effects.
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 ID | NIH-11140492 on NIH RePORTER |
What this research studies
Many people with cystic fibrosis benefit greatly from a triple combination therapy, but some don't respond as well or experience side effects. This project explores why these differences occur, focusing on how the body processes the medication and how individual genetic variations might play a role. We will look at medication levels in patients and genetic factors to understand how they affect treatment success. The goal is to learn if adjusting the medication dose based on these factors can improve lung function and overall well-being for those with CF.
Who could benefit from this research
Good fit: Ideal candidates are individuals with cystic fibrosis who are currently using or considering triple combination therapy, especially those experiencing varied responses or side effects.
Not a fit: Patients who are not affected by cystic fibrosis or are not candidates for triple combination therapy would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to personalized dosing strategies for cystic fibrosis triple combination therapy, helping more patients achieve better treatment results and fewer side effects.
How similar studies have performed: While triple combination therapy has shown success in clinical trials, this research is novel in its focus on real-world concentration variability and pharmacogenetic factors to optimize individual patient responses.
Where this research is happening
Little Rock, United States
- 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
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.