Studies how cystic fibrosis drugs work in children and genetic factors
Tests elexacaftor, ivacaftor and tezacaftor in children with cystic fibrosis.
Plain-language title generated from the registry record
Registry title: Ensuring Access to Optimal Therapy in CF: The ENACT Study
Key facts
- Where
- Birmingham, Little Rock, and Seattle, United StatesSee the 3 sites
- Phase
- Phase 4
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children and adults with cystic fibrosis who already take a stable triple combination CFTR modulator treatment and whose lung disease is stable.
- Age
- The inclusion criteria say 2 years and older; the registry lists a minimum age of 3 Years.
- Sex
- All sexes can join.
- Disease stage
- Cystic fibrosis confirmed by CFF diagnostic criteria. Lung disease must be clinically stable: no documented acute drop in FEV1 of more than 10%, and no extra antibiotics (IV or oral) within 4 weeks before screening.
- Earlier treatment
- Must have been on a stable dose of triple combination CFTR modulator therapy for at least two weeks before Visit 1.
- Biomarker or test result
- Known CFTR genotype is required.
Main reasons for not taking part
- Recent significant unintentional weight loss in the 4 weeks before screening
- Pregnant or breastfeeding
- Alcohol or substance abuse in the 6 months before screening
- Took part in a study with an investigational treatment within 28 days (or 5 half-lives, whichever is longer) before screening
- Medical or psychiatric illness or other conditions that would interfere with taking part, in the investigator's opinion
Read by machine on 9 November 2026.
Where in United States
- The University of Alabama at BirminghamBirmingham, United StatesTaking people
- Arkansas Children's HospitalLittle Rock, United StatesTaking people
- University of WashingtonSeattle, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Arkansas Children's Hospital Research Institute
- Trial code
- NCT07148739
- Record updated
- 2 June 2026
- Original description
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).Read full original descriptionShow less
Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT 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).
Inclusion: 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: recent significant unintentional weight loss, as determined by the investigator, in the 4 weeks prior to screening; 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; pregnant or breastfeeding female.