RECRUITING

Ensuring Access to Optimal Therapy in CF: The ENACT Study

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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. 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).

Official Title

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

Quick Facts

Study Start:2025-06-10
Study Completion:2030-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT07148739

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * documentation of CF diagnosis per CFF diagnostic criteria and known CFTR genotype
  2. * age 2 years and older
  3. * ability to provide written informed consent and/or assent (by subject and/or legal guardian)
  4. * on a stable dose of triple combination CFTR modulator therapy for at least two weeks prior to Visit 1
  5. * 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
  1. * recent significant unintentional weight loss, as determined by the investigator, in the 4 weeks prior to screening
  2. * pregnant or breastfeeding female
  3. * history of alcohol or substance abuse in the 6 months prior to screening
  4. * participation in a study involving an investigational intervention within 28 days (or 5 half-lives, whichever is longer) prior to screening
  5. * in the opinion of the Investigator, medical or psychiatric illness, or other conditions that would interfere with participation

Contacts and Locations

Study Contact

Jennifer S Guimbellot, Medical Degree and License
CONTACT
501-364-5365
jguimbellot@uams.edu
Michelle Gillespie
CONTACT
501-364-5365
GillespieM@archildrens.org

Principal Investigator

Jennifer S Guimbellot, Medical Degree and License
PRINCIPAL_INVESTIGATOR
Arkansas Children's Hospital Research Institute

Study Locations (Sites)

Arkansas Children's Hospital
Little Rock, Arkansas, 72205
United States
University of Washington
Seattle, Washington, 72205
United States

Collaborators and Investigators

Sponsor: Arkansas Children's Hospital Research Institute

  • Jennifer S Guimbellot, Medical Degree and License, PRINCIPAL_INVESTIGATOR, Arkansas Children's Hospital Research Institute

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06-10
Study Completion Date2030-12

Study Record Updates

Study Start Date2025-06-10
Study Completion Date2030-12

Terms related to this study

Keywords Provided by Researchers

  • Cystic Fibrosis
  • CF
  • CFTR modulator
  • Pediatric CF patients
  • Elexacaftor
  • Tezacaftor
  • Ivacaftor

Additional Relevant MeSH Terms

  • Cystic Fibrosis (CF)