Actively Recruiting
Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
Led by Arkansas Children's Hospital Research Institute · Updated on 2026-06-02
100
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
Sponsors
A
Arkansas Children's Hospital Research Institute
Lead Sponsor
N
National Institutes of Health (NIH)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying how several new drugs affect children with cystic fibrosis (CF) and how genetic differences influence their response to treatments. The study aims to identify who is most likely to benefit from existing therapies, potentially reducing side effects and high costs associated with ineffective treatments. Understanding how these drugs work and how best to study them can help expand their use to all patients with CF. The study involves participants who may join an observational one-visit study to examine drug concentration and side effects. Those experiencing side effects can join a single arm study focused on adjusting their dose to keep drug levels within an effective range. Once the optimal dose is reached, no further adjustments occur. The study evaluates side effects and dosing strategies specifically for the triple combination CFTR modulator therapy consisting of elexacaftor, tezacaftor, and ivacaftor. Participants will undergo assessments of drug concentration, mental and neuropsychological health, and treatment responses over 6 to 12 months. The study monitors side effects and the feasibility of dose adjustments to manage neuropsychological side effects. Data collection includes up to six concentration assessments and ongoing evaluation throughout the treatment period. The total involvement duration varies but includes careful follow-up to understand treatment effects and safety.
CONDITIONS
Brief Title
Ensuring Access to Optimal Therapy in CF: The ENACT Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documentation of cystic fibrosis diagnosis according to CFF criteria and known CFTR genotype
- Age 2 years and older
- Ability to provide written informed consent and/or assent by subject or legal guardian
- On a stable dose of triple combination CFTR modulator therapy for at least two weeks before Visit 1
- Clinically stable lung disease without recent acute decrease in lung function greater than 10% or recent antibiotic use within 4 weeks prior to screening
You will not qualify if you...
- Recent significant unintentional weight loss within 4 weeks prior to screening
- Pregnant or breastfeeding female
- History of alcohol or substance abuse within 6 months prior to screening
- Participation in another investigational intervention study within 28 days or 5 half-lives before screening
- Any medical or psychiatric illness or condition that would interfere with participation as determined by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 6 to 12 months
Participants receive triple combination CFTR modulator therapy with elexacaftor, tezacaftor, and ivacaftor, with possible dosing adjustments to maintain effective drug concentrations and manage side effects.
Up to 6 visits over 6 months or more for therapeutic drug monitoring and dosing adjustments
Trial Site Locations
Total: 3 locations
1
The University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72205
Actively Recruiting
3
University of Washington
Seattle, Washington, United States, 98195
Actively Recruiting
Research Team
J
Jennifer S Guimbellot, Medical Degree and License
M
Michelle Gillespie
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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