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RECRUITING
NCT07148739
PHASE4

Ensuring Access to Optimal Therapy in CF: The ENACT Study

Sponsor: Arkansas Children's Hospital Research Institute

View on ClinicalTrials.gov

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

Interventions

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.

Locations (2)

Arkansas Children's Hospital

Little Rock, Arkansas, United States

University of Washington

Seattle, Washington, United States