Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 75
- 主要终点
- Mortality: Incidence and cause of deaths
研究概览
简要总结
To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).
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 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female with confirmed diagnosis of CF16
- •At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D
- •Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent
- •Signed ICFs and, where appropriate, signed Assent Form
- •Able to understand the study requirements and comply with study data collection procedures
排除标准
- •Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment
- •Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation
- •History of organ transplantation
结局指标
主要结局
Mortality: Incidence and cause of deaths
时间窗: 48 Months
Organ transplantation: Incidence and reason for organ transplantations
时间窗: 48 Months
Pulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment
时间窗: 48 Months
Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment
时间窗: 48 Months
Respiratory Microbiology - Percentage of patients with cultures positive for bacteria other than Pseudomonas aeruginosa and for fungi during Kalydeco treatment compared to the period before Kalydeco treatment
时间窗: 48 Months
FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment
时间窗: 48 Months
Measures of nutritional status: Absolute change in weight, weight-for-age Z score, body mass index (BMI), and BMI-for-age Z-score during Kalydeco treatment
时间窗: 48 Months
Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment
时间窗: 48 Months
次要结局
未报告次要终点