Status:

RECRUITING

Ensuring Access to Optimal Therapy in CF: The ENACT Study

Lead Sponsor:

Arkansas Children's Hospital Research Institute

Collaborating Sponsors:

National Institutes of Health (NIH)

National Heart, Lung, and Blood Institute (NHLBI)

Conditions:

Cystic Fibrosis (CF)

Eligibility:

All Genders

3+ years

Phase:

PHASE4

Brief 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 ab...

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 ...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

June 10 2025

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 1 2030

Estimated Enrollment :

95 Patients enrolled

Trial Details

Trial ID

NCT07148739

Start Date

June 10 2025

End Date

December 1 2030

Last Update

August 29 2025

Active Locations (2)

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Page 1 of 1 (2 locations)

1

Arkansas Children's Hospital

Little Rock, Arkansas, United States, 72205

2

University of Washington

Seattle, Washington, United States, 72205