Ensuring Access to Optimal Therapy in CF: The ENACT Study

Last updated: May 29, 2026
Sponsor: Arkansas Children's Hospital Research Institute
Overall Status: Active - Recruiting

Phase

4

Condition

Cystic Fibrosis

Lung Disease

Treatment

therapeutic drug monitoring

Elexacaftor / Ivacaftor / Tezacaftor

Clinical Study ID

NCT07148739
ENACT-NP
1R01HL171034-01A1
  • Ages > 3
  • All Genders

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).

Eligibility Criteria

Inclusion

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 legalguardian)

  • on a stable dose of triple combination CFTR modulator therapy for at least two weeksprior 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 weeksprior to screening

Exclusion

Exclusion Criteria:

  • recent significant unintentional weight loss, as determined by the investigator, inthe 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 otherconditions that would interfere with participation

Study Design

Total Participants: 100
Treatment Group(s): 2
Primary Treatment: therapeutic drug monitoring
Phase: 4
Study Start date:
June 10, 2025
Estimated Completion Date:
December 31, 2030

Study 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).

Connect with a study center

  • The University of Alabama at Birmingham

    Birmingham, Alabama 35233
    United States

    Active - Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72205
    United States

    Active - Recruiting

  • University of Washington

    Seattle, Washington 98195
    United States

    Active - Recruiting

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