Clinical trial NCT06191640: Sinus Disease in Young Children With Cystic Fibrosis
Official title: Sinus Disease in Young Children With Cystic Fibrosis
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Cystic Fibrosis in Children
Cystic Fibrosis
Chronic Rhinosinusitis (Diagnosis)
Olfactory Disorder
Olfactory Impairment
Keywords: Cystic Fibrosis
Chronic Rhinosinusitis
Olfactory Dysfunction
Lead sponsor: University of California, Los Angeles
Sponsor class: OTHER
Collaborators: name: University of Kansas Medical Center
name: Children's Hospital Medical Center, Cincinnati
name: University of Iowa
name: University of Virginia
name: Children's Hospital Colorado
name: University of Vermont
First posted: 2024-01-05
Enrollment: 80
Sex: ALL
Minimum age (years): 2
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study 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.

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in MRI sinus opacification
timeFrame: Baseline, 1-year, and 2-year follow-up
measure: Change in olfactory bulb volume
timeFrame: Baseline, 1-year, and 2-year follow-up
Secondary outcomes:
measure: Change in Pediatric Smell Wheel (PSW) Scores
timeFrame: Baseline, 1-year, and 2-year follow-up
measure: Olfactory Cleft Opacification
timeFrame: Baseline, 1-year, and 2-year follow-up
measure: Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores
timeFrame: Baseline, 1-year, and 2-year follow-up
measure: Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains
timeFrame: Baseline, 1-year, and 2-year follow-up
measure: Change in Sinus and Nasal Quality of Life Survey (SN-5) - Overall Quality of Life (QOL) Domain
timeFrame: Baseline, 1-year, and 2-year follow-up
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Colchester
country: United States
facility: University of Vermont
state: Vermont
city: Charlottesville
country: United States
facility: University of Virginia
state: Virginia
Study officials:
affiliation: University of California, Los Angeles
name: Daniel M Beswick, MD
role: PRINCIPAL_INVESTIGATOR
References: