Ensuring Access to Optimal Therapy in CF: The ENACT Study

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Arkansas Children's Hospital Research Institute
Study ID
NCT07148739
Phase
PHASE4
Status
Recruiting
Apply to this trial

Conditions

  • Cystic Fibrosis (CF)

Eligibility Criteria

Sex
ALL
Age
3 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Elexacaftor / Ivacaftor / Tezacaftor — DRUG
    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.
  • therapeutic drug monitoring — OTHER
    Participants who consent to the therapeutic drug monitoring study will have their dose adjusted to remain within estimated effective concentrations.

Study Details

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).

Key Dates

Start date
Jun 10, 2025
Status verified
May 2026
Primary completion
Dec 31, 2030
Completion
Dec 31, 2030

Study Design

Enrollment
100 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Other: Single Arm
    Participants may be enrolled in an observational one-visit study for association of concentration with side effects. Participants may proceed to a single arm study if they have side effects to assess the feasibility of adjusting dose to maintain concentrations within an estimated effective range. Once within the range, dosing is no longer adjusted. Side effects will be evaluated as described in the protocol.

Primary Outcome Measure

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. ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
The University of Alabama at BirminghamBirminghamAlabama35233
Stefanie Krick, MD
Arkansas Children's HospitalLittle RockArkansas72205
Michelle Gillespie, Research Coordinator
[email protected]
501-364-3377
Bianca Johnson, Supervisor Pulmonary Research, MPH
[email protected]
Jennifer S Guimbellot, Medical Degree and License (PRINCIPAL_INVESTIGATOR)
University of WashingtonSeattleWashington98195
Kathleen Ramos, MD

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The University of Alabama at Birmingham· Birmingham, ALArkansas Children's Hospital· Little Rock, ARUniversity of Washington· Seattle, WA

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