Recruiting

Cystic FibrosisEnsuring Access to Optimal Therapy in CF: The ENACT Study

Sponsor:

Arkansas Children's Hospital Research Institute

Code:

NCT07148739

Conditions

Cystic Fibrosis (CF)

Eligibility Criteria

Sex: All

Age: 3+

Healthy Volunteers: Not accepted

Interventions

Elexacaftor / Ivacaftor / Tezacaftor

therapeutic drug monitoring

Study Details

Brief 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).

Conditions

Cystic Fibrosis (CF)

Study ID

NCT07148739

Start date

Jun 10, 2025

Status verified date

May, 2026

Completion date

Dec, 2030

Anticipated

Primary completion date

Dec, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 3+

Healthy Volunteers: Not accepted

Inclusion Criteria:

documentation of CF diagnosis per CFF diagnostic criteria and known CFTR genotype
age 2 years and older
ability to provide written informed consent and/or assent (by subject and/or legal guardian)
on a stable dose of triple combination CFTR modulator therapy for at least two weeks prior to Visit 1
clinically stable lung disease, defined as no documented acute decrease in FEV1 > 10%, OR use of additional antibiotics (intravenous \[IV\] or oral \[PO\]) within 4 weeks prior to screening

Exclusion Criteria:

recent significant unintentional weight loss, as determined by the investigator, in the 4 weeks prior to screening
pregnant or breastfeeding female
history of alcohol or substance abuse in the 6 months prior to screening
participation in a study involving an investigational intervention within 28 days (or 5 half-lives, whichever is longer) prior to screening
in the opinion of the Investigator, medical or psychiatric illness, or other conditions that would interfere with participation

Study Design

Enrollment

100 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

other: Single Arm

Participants may be enrolled in an observational one-visit study for association of concentration with side effects. Participants may proceed to a single arm study if they have side effects to assess the feasibility of adjusting dose to maintain concentrations within an estimated effective range. Once within the range, dosing is no longer adjusted. Side effects will be evaluated as described in the protocol.

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.

Primary outcome measure

Concentration (ng/mL) [ Time Frame: One time assessment for observational part of the study, up to 6 times (6 months or more) for the therapeutic drug monitoring pilot and feasibility study. ]

Central Contacts and Locations

Central contacts

Jennifer S Guimbellot, Medical Degree and License

501-364-5365 jguimbellot@uams.edu

Michelle Gillespie

501-364-3377 GillespieM@archildrens.org

Locations

The University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72205

Contacts

Michelle Gillespie, Research Coordinator

501-364-3377 GillespieM@archildrens.org

Bianca Johnson, Supervisor Pulmonary Research, MPH

JohnsonB2@archildrens.org

Principal Investigator:

Jennifer S Guimbellot, Medical Degree and License

University of Washington

Recruiting

Seattle, Washington, United States, 98195

More Information

Sponsor

Arkansas Children's Hospital Research Institute

Last update posted

Jun 2, 2026

Last verified

May, 2026

Keywords

Cystic Fibrosis
CF
CFTR modulator
Pediatric CF patients
Elexacaftor
Tezacaftor
Ivacaftor

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Arkansas Children's Hospital Research Institute on 2026-06-02.