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Ensuring Access to Optimal Therapy in CF: The ENACT Study

NCT07148739 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 95

Last updated 2025-08-29

No results posted yet for this study

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)

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.

Sponsors & Collaborators

  • National Institutes of Health (NIH)

    collaborator NIH

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Arkansas Children's Hospital Research Institute

    lead OTHER

Principal Investigators

  • Jennifer S Guimbellot, Medical Degree and License · Arkansas Children's Hospital Research Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-06-10
Primary Completion
2030-12-31
Completion
2030-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07148739 on ClinicalTrials.gov