Impact of Elexacaftor-tezacaftor-ivacaftor Triple Combination CFTR Therapy on Sinus Disease: Quantitative Sinus Computed Tomography, Patient Reported Outcomes and Cellular and Molecular Changes
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 31
- 试验地点
- 1
- 主要终点
- Change in Sinus CT opacification.
研究概览
简要总结
The study's main goal is to observe how effective elexacaftor-tezacaftor-ivacaftor is for improving the symptoms and signs of CF-related sinus disease.
详细描述
To determine if elexacfator-tezacaftor-ivacaftor improves the signs and symptoms of CF-related sinus disease, before and after initiation of triple combination therapy, each subject will undergo sinus CT scan and complete questionnaires related to signs and symptoms of sinus disease.
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
- 18 Years 至 89 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects from 18 to 89 years old.
- •Subjects with cystic fibrosis and comorbid chronic sinus disease (historic diagnosis based on physician assessment of signs and symptoms and medical record).
- •Subjects who clinically elect to initiate elexacaftor-tezacaftor-ivacaftor therapy and/or that are ineligible for CFTR modulator therapy based on their genotype, but have class I/II mutations based on sweat chloride > 90 mmol/L.
排除标准
- •Subjects under the age of 18 or over the age of
- •Subjects who do not elect to initiate elexacaftor-tezacaftor-ivacaftor for clinical reasons.
- •Subjects who had sinus surgery within the last 6 months or will have sinus surgery during the study period.
- •Subjects who have had a recent pulmonary exacerbation or viral infection within two weeks of initial visit.
研究组 & 干预措施
Prescribed triple combination
A group of 60 people with CF who are clinically prescribed elexacaftor-tezacaftor-ivacaftor and have chronic sinusitis
干预措施: Elexacaftor-tezacaftor-ivacaftor exposure (Drug)
结局指标
主要结局
Change in Sinus CT opacification.
时间窗: Change in Sinus CT opacification between the initial and 6 month visits.
Calculated from an automated quantification of the size of sinuses.
次要结局
- Change in 22-item Sino-Nasal Outcome Test (SNOT-22) score.(Change in SNOT-22 score between the initial and 6 month visits.)
- Change in Questionnaire for Olfactory Disorders (QOD) score(Change in QOD score between the initial and 6 month visits.)
研究者
Jennifer Taylor-Cousar
Professor of Medicine
National Jewish Health