Sinus Disease in Young Children With Cystic Fibrosis

ConditionsCystic Fibrosis in Children Cystic Fibrosis Chronic Rhinosinusitis (Diagnosis)Olfactory Disorder Olfactory Impairment

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age2-8

SponsorUniversity of California, Los Angeles

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial population

Participants for this study will be enrolled into one of two groups. The first group (HEMT Group) will enroll children between 2-8 years of age who have a clinical intent to initiate HEMT, which is defined as ivacaftor or elexacaftor/tezacaftor/ivacaftor based on underlying CFTR mutation status. The second group (non-HEMT Group) will consist of children under the age of 8 without intent to clinically initiate HEMT. Children for both groups will be identified and recruited from cystic fibrosis clinics.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Ivacaftor or elexacaftor/tezacaftor/ivacaftor
Drug
HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study.

Treatment groups

80 Participants

are divided into 2 treatment groups

Group A: HEMT Group1 intervention

Group B: Non-HEMT/Control Group0 interventions

Trial outcomes

Primary outcomes

Change in MRI sinus opacification

Measurement involves calculating percent total sinus opacification (%) using MRI, where sinus contents are differentiated into air and soft tissue/fluid.

Time frame