Sinus Disease in Young Children With Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 80
- 试验地点
- 12
- 主要终点
- Change in olfactory bulb volume
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 8 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Non-HEMT/Control Group
Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
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.
干预措施: Ivacaftor or elexacaftor/tezacaftor/ivacaftor (Drug)
结局指标
主要结局
Change in olfactory bulb volume
时间窗: 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).
Change in MRI sinus opacification
时间窗: 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.
次要结局
- Change in Pediatric Smell Wheel (PSW) Scores(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(Baseline, 1-year, and 2-year follow-up)
- Olfactory Cleft Opacification(Baseline, 1-year, and 2-year follow-up)
- Change in Sinus and Nasal Quality of Life Survey (SN-5) - Symptom Frequency Domains(Baseline, 1-year, and 2-year follow-up)
- Change in Brief Questionnaire of Olfactory Disorders (BQOD) Scores(Baseline, 1-year, and 2-year follow-up)
研究者
Daniel M. Beswick
MD; Associate Professor-in-Residence; Associate Residency Program Director; Co-Chair, Equity, Diversity, and Inclusion Committee Department of Head and Neck Surgery
University of California, Los Angeles