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Ensuring Access to Optimal Therapy in CF: The ENACT Study
Sponsor: Arkansas Children's Hospital Research Institute
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).
Official title: Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
Key Details
Gender
All
Age Range
3 Years - Any
Study Type
INTERVENTIONAL
Enrollment
95
Start Date
2025-06-10
Completion Date
2030-12
Last Updated
2025-08-29
Healthy Volunteers
No
Conditions
Interventions
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.
therapeutic drug monitoring
Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.
Locations (2)
Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of Washington
Seattle, Washington, United States