Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study
Arkansas Children's Hospital Research Institute
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).
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 t…
Eligibility
- Age range
- 3+ years
- Sex
- All
- Healthy volunteers
- No
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 determ…
Interventions
- DrugElexacaftor / 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.
- Othertherapeutic drug monitoring
Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.
Locations (2)
- Arkansas Children's HospitalLittle Rock, Arkansas
- University of WashingtonSeattle, Washington