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Ensuring Access to Optimal Therapy in CF: The ENACT Study

NCT ID: NCT07148739

Last Updated: 2025-08-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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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

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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

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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

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Cystic Fibrosis (CF)

Keywords

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Cystic Fibrosis CF CFTR modulator Pediatric CF patients Elexacaftor Tezacaftor Ivacaftor

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This is a pilot and feasibility study to assess the association of drug concentration with side effects, and to assess the feasibility of the use of therapeutic drug monitoring to adjust dosing in individuals with side effects.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

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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

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

Eligibility Criteria

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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

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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

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Jennifer Guimbellot

Principal Investigator - MD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jennifer S Guimbellot, Medical Degree and License

Role: PRINCIPAL_INVESTIGATOR

Arkansas Children's Hospital Research Institute

Locations

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Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status RECRUITING

University of Washington

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Jennifer S Guimbellot, Medical Degree and License

Role: CONTACT

Phone: 501-364-5365

Email: [email protected]

Michelle Gillespie

Role: CONTACT

Phone: 501-364-5365

Email: [email protected]

Facility Contacts

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Clinical Research Assistant

Role: primary

Clinical Research Regulatory Coordinator

Role: backup

Kathleen Ramos, MD

Role: primary

Other Identifiers

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1R01HL171034-01A1

Identifier Type: NIH

Identifier Source: secondary_id

View Link

ENACT-NP

Identifier Type: -

Identifier Source: org_study_id