Purpose

This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.

Conditions

Eligibility

Eligible Ages
Between 2 Years and 8 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

HEMT Group: - Children with documentation of a CF diagnosis - Age 2-8 years old at first study visit - CFTR mutation consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) - Clinician intent to prescribe ivacaftor or ETI so that enrollment is before start of HEMT Non-HEMT/Control Group: - Children with documentation of a CF diagnosis - Age 2-8 years at first study visit - Ineligible for highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) based on CFTR mutation or clinical decision not to initiate HEMT if eligible

Exclusion Criteria

For Both Groups: - Use of an investigational drug within 28 days prior to the first study visit - Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 180 days prior to and including the first study visit - Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit. - Sinus surgery within 180 days prior to the first study visit

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
HEMT Group Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the non-HEMT group of this study may enroll into the HEMT cohort if they become eligible for these CFTR modulator therapies and plan to start them.
  • Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor
    HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study.
    Other names:
    • Kalydeco or Trikafta; Vertex (VX)-770 or VX-445/VX-661/VX-770
Non-HEMT/Control Group Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66160
Contact:
Michael Lewis, MD

More Details

Status
Recruiting
Sponsor
University of California, Los Angeles

Study Contact

Daniel M Beswick, MD
310-206-8457
dbeswick@mednet.ucla.edu

Detailed Description

This multi-center, prospective, observational study investigates the effects of highly effective modulator therapy (HEMT) on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). The study spans two years and includes two distinct groups of children with cystic fibrosis: children ≤ 8 years old receiving HEMT and a control group of children ≤ 8 not receiving HEMT. The study aims to assess the efficacy of HEMT in improving sinus health and olfactory capabilities in this young demographic. Key assessments include magnetic resonance imaging (MRI) sinus opacification, olfactory bulb volume measured via MRI, objective olfactory testing, and various quality (QOL) surveys. This investigation seeks to characterize the severity of CRS and OD in YCwCF, and to elucidate if early initiation of HEMT improves CRS and OD . In the HEMT group, participants will have a pre-HEMT assessment followed by 1-year and 2-year post-HEMT evaluations. In the control/non-HEMT group, participants will undergo parallel assessments at baseline, 1-year, and 2-year intervals to track the natural progression of CRS and OD without HEMT.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.