Ensuring access to optimal therapy for children with cystic fibrosis
Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
This project will test whether genetic testing and therapeutic drug monitoring can predict which children with cystic fibrosis will respond to the triple-combination elexacaftor/tezacaftor/ivacaftor therapy.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 95 (estimated) |
| Ages | 3 Years and up |
| Sex | All |
| Sponsor | Arkansas Children's Hospital Research Institute Academic / other |
| Locations | 2 sites (Little Rock, Arkansas and 1 other locations) |
| Trial ID | NCT07148739 on ClinicalTrials.gov |
What this trial studies
ENACT is a Phase 4 interventional study enrolling children age 2 and older with confirmed CF who are already on stable triple-combination CFTR modulator therapy. Participants will provide genetic samples and undergo therapeutic drug monitoring while clinical status and lung function are tracked. The study aims to identify biomarkers and genetic factors that predict individual response or risk of side effects to elexacaftor/tezacaftor/ivacaftor. The trial is run at Arkansas Children's Hospital and the University of Washington with NIH/NHLBI collaboration.
Who should consider this trial
Good fit: Children aged 2 years and older with confirmed CF and a known CFTR genotype who are clinically stable and on a stable dose of the elexacaftor/tezacaftor/ivacaftor combination are ideal candidates.
Not a fit: Patients who are pregnant or breastfeeding, have recent significant unintentional weight loss, recent substance abuse, recent investigational drug use, or unstable/acute lung disease are unlikely to qualify and may not receive benefit from participation.
Why it matters
Potential benefit: If successful, clinicians could use genetic and drug-level information to choose CFTR modulators more precisely, reducing ineffective treatments, side effects, and costs.
How similar studies have performed: Elexacaftor/tezacaftor/ivacaftor has shown strong clinical benefit for many patients, and early pharmacogenetic and drug-monitoring work is promising, but using these tools to predict individual response to triple therapy is still relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Little Rock, Arkansas and 1 other locations
- Arkansas Children's Hospital — Little Rock, Arkansas, United States (Recruiting)
- University of Washington — Seattle, Washington, United States (Recruiting)
Study contacts
- Principal investigator: Jennifer S Guimbellot, Medical Degree and License — Arkansas Children's Hospital Research Institute
- Study coordinator: Jennifer S Guimbellot, Medical Degree and License
- Email: jguimbellot@uams.edu
- Phone: 501-364-5365
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.