Ensuring Access to Optimal Therapy in CF: The ENACT Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Elexacaftor / Ivacaftor / Tezacaftor, therapeutic drug monitoring.
- Who it may be relevant to
- Registry conditions: Cystic Fibrosis (CF). Basic parameters: from 3 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
Overview
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).
Detailed description
Understanding variation in genetic response to pharmacological treatments and personalized CFTR modulator response is crucial to the optimization of the use of these novel compounds; expansion to all patients who might benefit from them; and development of predictive biomarkers. In addition, 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 and the downstream effects is critical to expanding the use of these drugs to all patients with cystic fibrosis (CF).
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.]
Secondary outcome measures (3)
- Participant Mental and Neuropsychological Health [Time frame: From enrollment to the end of treatment at 6-12 months.]
- Investigators will evaluate the feasibility of reducing dose to manage Neuropsychological Side Effects (NPSE). [Time frame: From enrollment to study conclusion at 6-12 months (after all visits are completed).]
- Response to dosing adjustments [Time frame: From enrollment to study conclusion at 6-12 months, after all visits are completed.]
Eligibility 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- The University of Alabama at Birmingham — Birmingham
- Arkansas Children's Hospital — Little Rock
- University of Washington — Seattle
Identifiers
NCT: NCT07148739 · ENACT-NP · 1R01HL171034-01A1