Cystic Fibrosis in ChildrenNCT06191640
Sinus Disease in Young Children With Cystic Fibrosis
An observational study of Cystic Fibrosis in Children and Cystic Fibrosis, run by University of California, Los Angeles at 6 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 2 to 8 years
- Conditions the study is for
- Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis)
- What is being tested?
- Ivacaftor or elexacaftor/tezacaftor/ivacaftor
- How many people
- 80 participants
- Where
- 6 sitesUnited States
- Study dates
- 12 Apr 2023to 30 Jun 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 8 years
- All sexes
- No healthy volunteers
- Child
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.
Who may be able to join
- 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:
Show all 9Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Edith Zemanick, MD
Mary Cross
- Recruiting
University of Virginia
Charlottesville, Virginia, 22903
Jaime Mata, MS, PhD
Roselove N Nunoo-Asare
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039
Jason Woods, PhD
Priyanka Desirazu
- Recruiting
University of Vermont
Colchester, Vermont, 05446
Jillian S Sullivan, MD
Julie Sweet
- Recruiting
University of Iowa
Iowa City, Iowa, 52242
Sean B Fain, PhD
Mary Teresi
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Michael Lewis, MD
Larry Scott
About this study
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.
Read the 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.
- Conditions
- Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory Disorder, Olfactory Impairment
- Being tested
- Ivacaftor or elexacaftor/tezacaftor/ivacaftor
- Keywords
- Cystic Fibrosis, Chronic Rhinosinusitis, Olfactory Dysfunction
- Medical subject headings
- Cystic Fibrosis, Disease, Olfaction Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ivacaftor or elexacaftor/tezacaftor/ivacaftorDrug
Also called Kalydeco or Trikafta; Vertex (VX)-770 or VX-445/VX-661/VX-770
HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor | |
| Non-HEMT/Control Group | Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, 1-year, and 2-year follow-up
Change in olfactory bulb volume
Assessment includes segmenting the olfactory bulb on consecutive coronal slices using MRI, then combining these into a 3-dimensional region. The volume of this region is then calculated in cubic millimeters (mm3).
Measured: Baseline, 1-year, and 2-year follow-up
Secondary outcomes (5)
Change in Pediatric Smell Wheel (PSW) Scores
The PSW test involves children identifying microencapsulated odorants. The score (Range: 0-11) is based on the number of correctly identified smells, with higher scores indicating better olfactory function.
Measured: Baseline, 1-year, and 2-year follow-up
Olfactory Cleft Opacification
Olfactory cleft opacification is quantified by segmenting the olfactory cleft on MRI and applying pixel intensity thresholds to calculate the percentage (%) of opacification.
Measured: Baseline, 1-year, and 2-year follow-up
Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores
This BQOD is a parent-completed survey for assessing olfactory-specific quality of life (QOL). The survey scores (Range: 0-21) quantify the degree of olfactory QOL impairment, with higher scores indicating greater olfactory QOL impairment.
Measured: Baseline, 1-year, and 2-year follow-up
Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains
SN-5 is a parent-completed survey assessing sinus symptoms and QOL in children. It captures sinus infections, nasal obstruction, allergy symptoms, emotional distress, activity limitations, and overall QOL. For the first five symptom domain questions, scores range 1-7 for each domain, higher scores indicating higher frequency at which symptom affected patient over the past 4 weeks.
Measured: Baseline, 1-year, and 2-year follow-up
Change in Sinus and Nasal Quality of Life Survey (SN-5) - Overall Quality of Life (QOL) Domain
SN-5 is a parent-completed survey assessing sinus symptoms and QOL in children. It captures sinus infections, nasal obstruction, allergy symptoms, emotional distress, activity limitations, and overall QOL. The final question assessing the child's overall QOL as a result of nose or sinus problems is scored on a scale 0-10, with higher scores indicating better QOL.
Measured: Baseline, 1-year, and 2-year follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start12 Apr 2023
- Primary completion30 Jun 2029 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis in ChildrenStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- University of Kansas Medical Center, Children's Hospital Medical Center, Cincinnati, University of Iowa, University of Virginia, University of Vermont, University of North Carolina, Chapel Hill, Children's Hospital Colorado
- Responsible party
- Principal investigator, Daniel M. Beswick, University of California, Los Angeles
- Investigators
- Daniel M Beswick, MD, Principal investigator, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06191640
- Sponsor's protocol ID
- 22-000594
- Status last verified
- Sep 2026
- First submitted
- 19 Dec 2023
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Participant data will be securely stored using REDCap software.
Publications
Liu CM, Fischer JL, Zemanick ET, Woods JC, Markarian KK, Fain SB, Froh D, Heltshe SL, Hoffman LR, Humphries SM, Kramer EL, Ode KL, Lewis M, Li DA, Mata J, Milla SS, Niedbalski PJ, Sawatzky BD, Sim MS, Sullivan JS, Trout AT, Goss CH, Taylor-Cousar JL, Beswick DM. The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol. ERJ Open Res. 2025 Jan 13;11(1):00137-2024. doi: 10.1183/23120541.00137-2024. eCollection 2025 Jan.
PubMed 39811548
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sinus Disease in Young Children With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT06191640
- Acronym / sponsor ID
- 22-000594
- Conditions
- Cystic Fibrosis in Children, Cystic Fibrosis, Chronic Rhinosinusitis (Diagnosis), Olfactory Disorder, Olfactory Impairment
- Keywords
- Cystic Fibrosis, Chronic Rhinosinusitis, Olfactory Dysfunction
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Collaborators
- University of Kansas Medical Center, Children's Hospital Medical Center, Cincinnati, University of Iowa, University of Virginia, University of Vermont, University of North Carolina, Chapel Hill, Children's Hospital Colorado
- Study start
- 12 Apr 2023 (actual)
- Primary completion
- 30 Jun 2029 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 5 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HEMT Group | – | Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftorChildren 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. |
| Non-HEMT/Control Group | – | –Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. |
Who may qualify
- Ages
- 2 to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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.
Key inclusion criteria (9 listed)
- 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
- Children with documentation of a CF diagnosis
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ivacaftor or elexacaftor/tezacaftor/ivacaftorAlso: Kalydeco or Trikafta; Vertex (VX)-770 or VX-445/VX-661/VX-770HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study. | Drug | HEMT Group |
Primary outcome
Change in MRI sinus opacification
Time frame: Baseline, 1-year, and 2-year follow-up
Measurement involves calculating percent total sinus opacification (%) using MRI, where sinus contents are differentiated into air and soft tissue/fluid.
Change in olfactory bulb volume
Time frame: Baseline, 1-year, and 2-year follow-up
Assessment includes segmenting the olfactory bulb on consecutive coronal slices using MRI, then combining these into a 3-dimensional region. The volume of this region is then calculated in cubic millimeters (mm3).
5 secondary outcomes
Change in Pediatric Smell Wheel (PSW) Scores
Time frame: Baseline, 1-year, and 2-year follow-up
Olfactory Cleft Opacification
Time frame: Baseline, 1-year, and 2-year follow-up
Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores
Time frame: Baseline, 1-year, and 2-year follow-up
Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains
Time frame: Baseline, 1-year, and 2-year follow-up
Change in Sinus and Nasal Quality of Life Survey (SN-5) - Overall Quality of Life (QOL) Domain
Time frame: Baseline, 1-year, and 2-year follow-up
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- Derived from this study: PubMed 39811548Liu CM, Fischer JL, Zemanick ET, Woods JC, Markarian KK, Fain SB, Froh D, Heltshe SL, Hoffman LR, Humphries SM, Kramer EL, Ode KL, Lewis M, Li DA, Mata J, Milla SS, Niedbalski PJ, Sawatzky BD, Sim MS, Sullivan JS, Trout AT, Goss CH, Taylor-Cousar JL, Beswick DM. The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol. ERJ Open Res. 2025 Jan 13;11(1):00137-2024. doi: 10.1183/23120541.00137-2024. eCollection 2025 Jan.
- Individual participant dataSharing planned: No. Participant data will be securely stored using REDCap software.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Daniel M Beswick, MD principal investigator
University of California, Los Angeles
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis in Children and Cystic Fibrosis, run by University of California, Los Angeles at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Daniel M Beswick, MD (310-206-8457, dbeswick@mednet.ucla.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in MRI sinus opacification (time frame: Baseline, 1-year, and 2-year follow-up). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06191640, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.