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Ensuring Access to Optimal Therapy in CF: The ENACT Study
Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
Who can join
Ages 3 and older · All sexes
Full 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
About the study
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).
What is being tested
- Elexacaftor / Ivacaftor / Tezacaftor (drug)
- therapeutic drug monitoring (other)
Sponsor: Arkansas Children's Hospital Research Institute · Participants: 100 · Started: Jun 10, 2025
Contact the study team
- Jennifer S Guimbellot, Medical Degree and License · Phone: 501-364-5365
- Michelle Gillespie · Phone: 501-364-3377
Official record on ClinicalTrials.gov — NCT07148739
Locations in the U.S.
| Alabama | The University of Alabama at Birmingham, Birmingham |
| Arkansas | Arkansas Children's Hospital, Little Rock |
| Washington | University of Washington, Seattle |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.