Ensuring Access to Optimal Therapy in CF: The ENACT Study (NCT07148739) | Clinical Trial Compass
RecruitingPhase 4
Ensuring Access to Optimal Therapy in CF: The ENACT Study
United States100 participantsStarted 2025-06-10
Plain-language 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).
Who can participate
Age range
3 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Concentration (ng/mL)
Timeframe: One time assessment for observational part of the study, up to 6 times (6 months or more) for the therapeutic drug monitoring pilot and feasibility study.
Trial details
NCT IDNCT07148739
SponsorArkansas Children's Hospital Research Institute