NCT07148739

Ensuring Access to Optimal Therapy in CF: The ENACT Study

Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 95 (estimated)
• Sponsor: Arkansas Children's Hospital Research Institute · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial is examining the action and effects of several new drugs in the treatment of cystic fibrosis in children. In addition, several genetic factors are examined. The hope is that the ability to determine prior to treatment those individuals who will or will not respond to existing therapies will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen. Understanding the way these drugs work in the body and the best way to study them is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).

Detailed description

Understanding variation in genetic response to pharmacological treatments and personalized CFTR modulator response is crucial to the optimization of the use of these novel compounds; expansion to all patients who might benefit from them; and development of predictive biomarkers. In addition, the ability to determine prior to treatment those individuals who will or will not respond to existing therapies will avoid needless risk of side effects and the high cost of a potentially ineffective treatment regimen. Understanding the way these drugs work in the body and the best way to study them and the downstream effects is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).

Conditions

• Cystic Fibrosis (CF)

Interventions

Type	Name	Description
DRUG	Elexacaftor / Ivacaftor / Tezacaftor	This study will examine different dosing strategies and outcomes for triple combination CFTR modulator therapy using the drug(s) elexacaftor, tezacaftor, and/or ivacaftor in patients with cystic fibrosis.
OTHER	therapeutic drug monitoring	Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.

Timeline

• Start date: 2025-06-10
• Primary completion: 2030-12-01
• Completion: 2030-12-01
• First posted: 2025-08-29
• Last updated: 2025-08-29

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study