An observational study in Cystic Fibrosis in Children, Cystic Fibrosis and Chronic Rhinosinusitis (Diagnosis), sponsored by University of California, Los Angeles. Recruiting at 6 sites in United States. Open to participants aged 2 Years to 8 Years. Per ClinicalTrials.gov, last updated 2026-09-10.
Sponsored by University of California, Los Angeles · Observational
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.
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.
1,619 studies on the registry are indexed under Cystic Fibrosis; 199 are open to participants now.
This study's planned enrollment of 80 is close to the median of 85 across 503 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →University of California, Los Angeles is the lead sponsor of 1,170 studies on the registry; 195 are open to participants now.
Of its 95 completed or terminated interventional studies of FDA-regulated products, 70 (74%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
HEMT Group:
Non-HEMT/Control Group:
Exclusion Criteria:
For Both Groups:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
HEMT's are prescribed at the discretion of the treating physician and is not dictated by the principal investigator of this study.
Also known as: Kalydeco or Trikafta; Vertex (VX)-770 or VX-445/VX-661/VX-770
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: 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).
Time frame: Baseline, 1-year, and 2-year follow-up
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
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.
Time frame: Baseline, 1-year, and 2-year follow-up
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