RecruitingPhase 4NCT07148739

Ensuring Access to Optimal Therapy in CF: The ENACT Study

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


Sponsor

Arkansas Children's Hospital Research Institute

Enrollment

100 participants

Start Date

Jun 10, 2025

Study Type

INTERVENTIONAL

Conditions

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).


Eligibility

Min Age: 3 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Elexacaftor / Ivacaftor / Tezacaftor and therapeutic drug monitoring for people with cystic fibrosis (cf). The study is currently recruiting participants at 3 locations. People eligible for this study include aged 3 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGElexacaftor / 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.

OTHERtherapeutic drug monitoring

Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.


Locations(3)

The University of Alabama at Birmingham

Birmingham, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

University of Washington

Seattle, Washington, United States

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NCT07148739


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