Ensuring access to optimal therapy for children with cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• The University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• 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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.