CFTR Biomarker Studie Bei Patient*Innen Mit Mukoviszidose Und CFTR-Modulatortherapie
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 500
- 试验地点
- 4
- 主要终点
- Intestinal current measurement (ICM)
研究概览
简要总结
This observational study evaluates the effect of therapy with cystic fibrosis transmembrane regulator (CFTR) modulators on CFTR function measured by the CFTR biomarker intestinal current measurement (ICM), nasal potential difference (NPD) and sweat chloride in a post-approval setting in patients with cystic fibrosis (CF).
详细描述
Cystic fibrosis transmembrane regulator (CFTR) biomarker (intestinal current measurement (ICM), nasal potential difference (NPD), sweat chloride) before the start of therapy and 12 and 52 weeks after initiation of therapy Clinical parameters (anthropometry, lung function, lung magnetic resonance imaging (MRI), lung computer tomography (CT)) before the start of therapy and after initiation of therapy Assessment of airway secretion specimens before the start of therapy and after initiation of therapy
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
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Decision for cystic fibrosis (CF) transmembrane regulator (CFTR)-modulator therapy by the patient and the caring CF physician
- •Signed informed consent form (ICF) and, where appropriate, signed assent form.
排除标准
- •Ongoing participation in an investigational drug study (including studies investigating lumacaftor, tezacaftor or ivacaftor)
结局指标
主要结局
Intestinal current measurement (ICM)
时间窗: 12 weeks
Absolute change from baseline of the chloride secretory ion current induced by cyclic adenosine monophosphate (cAMP) stimulation (forskolin/3-isobutyl-1-methylxanthine (IBMX)) in rectal tissue determined by intestinal current measurement (ICM) as a cystic fibrosis transmembrane conductance regulator (CFTR) biomarker
次要结局
- Nasal potential Difference (NPD)(12 weeks)
- Lung computer tomography(52, 104 weeks)
- Forced expiratory volume in 1 second (FEV1)(quaterly in the first year, yearly thereafter up to 5 years)
- Sweat chloride(12, 52 weeks, yearly thereafter up to 5 years)
- Lung clearance index (LCI)(quaterly in the first year, yearly thereafter up to 5 years)
- Lung magnetic resonance imaging (MRI)(12, 52 weeks, yearly thereafter up to 5 years)
- Paranasal sinus magnetic resonance imaging (MRI)(12, 52 weeks, yearly thereafter up to 5 years)
- Fecal elastase(12, 52 weeks, yearly thereafter up to 5 years)
- Weight(quaterly in the first year, yearly thereafter up to 5 years)
- Airway Microbiome(4, 12, 52 weeks, yearly thereafter up to 5 years)
- Sputum Elasticity(4, 12, 52 weeks, yearly thereafter up to 5 years)
- Sputum Viscocity(4, 12, 52 weeks, yearly thereafter up to 5 years)
- Cystic Fibrosis Questionnaire-Revised (CFQ-R)(quarterly in the first year, yearly thereafter up to 5 years)
- Patient Health Questionnaire-9 (PHQ-9)(quarterly in the first year, yearly thereafter up to 5 years)
- Generalized Anxiety Disorder-7 (GAD-7)(quarterly in the first year, yearly thereafter up to 5 years)
- Child Behavior Checklist for Ages 1½-5 (CBCL/1½-5)(quarterly in the first year, yearly thereafter up to 5 years)
- Child Behavior Checklist for Ages 6-18 (CBCL/6-18)(quarterly in the first year, yearly thereafter up to 5 years)
- Youth Self-Report for Ages 11-18 (YSR/11-18)(quarterly in the first year, yearly thereafter up to 5 years)
- Strengths and Difficulties Questionnaire (SDQ)(quarterly in the first year, yearly thereafter up to 5 years)
- 22-Item Sino-Nasal Outcome Test (SNOT-22)(quarterly in the first year, yearly thereafter up to 5 years)
研究者
Simon Graeber
Clinician Scientist
Charite University, Berlin, Germany