NCT07148739 · Arkansas Children's Hospital Research Institute
Ensuring Access to Optimal Therapy in CF: The ENACT Study
(ENACT)
What this study is about
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.
View original scientific 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.
Interventions
DRUG
Elexacaftor / Ivacaftor / Tezacaftor
This study will examine different dosing strategies and outcomes for triple combination CFTR modulator therapy using the drug(s) elexacaftor, tezacaftor, and/or ivacaftor in patients with cystic fibrosis.
OTHER
therapeutic drug monitoring
Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.
Primary outcome measures
Concentration (ng/mL)
Time frame: 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.
Drug concentration of CFTR modulators
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 condition
Where
- Birmingham, Alabama
- Little Rock, Arkansas
- Seattle, Washington
Collaborators
National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI)
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 2, 2026 · Source of record for eligibility and locations