Ensuring Access to Optimal Therapy in CF: The ENACT Study

NCT ID: NCT07148739

Last Updated: 2025-08-29

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 95 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-10
• Study Completion Date: 2030-12-31

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).

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)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: OTHER

Elexacaftor / Ivacaftor / Tezacaftor

Intervention Type: DRUG

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

Intervention Type: OTHER

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

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.

Intervention Type: DRUG

therapeutic drug monitoring

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

Intervention Type: OTHER

Other Intervention Names

Triple Combination CFTR Modulator Therapy; ETI - Elexacaftor/Tezacaftor/Ivacaftor

Eligibility Criteria

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

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Arkansas Children's Hospital Research Institute

OTHER

Sponsor Role: lead

Responsible Party

Jennifer Guimbellot

Principal Investigator - MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jennifer S Guimbellot, Medical Degree and License

Role: PRINCIPAL_INVESTIGATOR

Arkansas Children's Hospital Research Institute

Locations

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status: RECRUITING

University of Washington

Seattle, Washington, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Jennifer S Guimbellot, Medical Degree and License

Role: CONTACT

jguimbellot@uams.edu

501-364-5365

Michelle Gillespie

Role: CONTACT

GillespieM@archildrens.org

501-364-5365

Facility Contacts

Clinical Research Assistant

Role: primary

pintiliuca@archildrens.org

501-364-5954

Clinical Research Regulatory Coordinator

Role: backup

FowlerML@archildrens.org

501-364-7376

Kathleen Ramos, MD

Role: primary

Other Identifiers

1R01HL171034-01A1

Identifier Type: NIH

Identifier Source: secondary_id

View Link

ENACT-NP

Identifier Type: -

Identifier Source: org_study_id