RecruitingPhase 4NCT07148739

Ensuring Access to Optimal Therapy in CF: The ENACT Study

Ensuring Access to Optimal Therapy in Cystic Fibrosis: The ENACT Study

Sponsor

Arkansas Children's Hospital Research Institute

Enrollment

95 participants

Start Date

Jun 10, 2025

Study Type

INTERVENTIONAL

Conditions

Cystic Fibrosis (CF)

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).

Eligibility

Min Age: 3 Years

Inclusion Criteria5

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 Criteria5

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

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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.