A Study in US Cystic Fibrosis Patients With the R117H-CFTR Mutation to Confirm the Long-term Safety and Effectiveness of Kalydeco, Including Patients <18 Years of Age, Combining Data Captured in the Cystic Fibrosis Foundation Registry From an Interventional Cohort and a Non-Interventional Cohort
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 368
- 主要终点
- Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])
研究概览
简要总结
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation <18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.
详细描述
Patient follow-up (i.e., collection of outcomes data after treatment initiation) in the Non-Interventional Cohort will be at least 36 months. The study also includes retrieval of retrospective data entered into the registry for 36 months before the initiation of Kalydeco treatment, from patients matched for Non-Interventional Cohorts. This will permit a within-group comparison of outcomes before and after Kalydeco treatment for effectiveness and safety. The interventional cohort will not be utilized.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Non Interventional Cohort
- •Male or female with confirmed diagnosis of CF
- •Must have at least 1 allele of the R117H-CFTR mutation
- •Enrolled in the US CFF Patient Registry
- •With a record of Kalydeco treatment initiation from 01 January 2015 through 31 December 2016
- •Historical Cohort
- •Patients with CF in the CFF Patient Registry as of 01 January 2009
- •Must have at least 1 allele of the R117H-CFTR mutation
- •Patients with no evidence of any prior Kalydeco exposure
排除标准
- 未提供
结局指标
主要结局
Lung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC])
时间窗: 36 Months
Spirometry will be performed according to the standard procedure at each site, and FEV1 values as recorded in the registry will be evaluated. All descriptive and summary data collected for FEV1 will be repeated for FVC
Pulmonary exacerbations, use of IV antibiotics
时间窗: 36 Months
Pulmonary exacerbation data will be collected as recorded in the registry.
Nutritional parameters (body mass index [BMI], BMI-for-age z-score, weight, and weight-for-age z-score)
时间窗: 36 Months
Height and weight measurements as recorded in the registry will be evaluated. BMI, BMI-for-age z-score, and weight-for-age z-score will be derived
Death or transplantation
时间窗: 36 months
Death will be collected from the registry database.
Hospitalizations
时间窗: 36 Months
Hospitalizations will be collected from the registry database.
Selected Complications (Symptomatic sinus disease, Pulmonary complications, CF-related diabetes (CFRD) and distal intestinal obstruction syndrome (DIOS), Hepatobiliary complications, Pancreatitis)
时间窗: 36 Months
Information for the above shown CF-related complications as recorded in the registry will be evaluated
Select pulmonary microorganisms (e.g., P. aeruginosa, S. aureus)
时间窗: 36 Months
Data on microorganisms as recorded in the registry will be evaluated
次要结局
未报告次要终点