An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 192
- 主要终点
- Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.
详细描述
This open-label, rollover study of orally administered VX-770 was conducted in participants with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Participants who were previously enrolled in Study 102 (VX08-770-102/NCT00909532) and Study 103 (VX08-770-103/NCT00909727), and met certain criteria were eligible to enroll in this study (Study 105/VX08-770-105/NCT01117012).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Participants who have completed the assigned study treatment in Study 102 or Study 103
- •Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)
- •Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
- •Participants of child bearing potential and who are sexually active must meet the contraception requirements
- •Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained
排除标准
- •Participants with a history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
- •Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
- •Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements
- •Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice
研究组 & 干预措施
VX-770
VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h).
干预措施: Ivacaftor (Drug)
结局指标
主要结局
Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Study 105: Day 1 up to Week 168
Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.
次要结局
- Annualized Pulmonary Exacerbation Event Rate(Study 105: Day 1 through Week 96)
- Annualized Duration of Pulmonary Exacerbation Events(Study 105: Day 1 through Week 96)
- Absolute Change From Study 105 Baseline in Weight Through Week 96(Study 105: Baseline through Week 96)
- Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96(Study 105: Baseline through Week 96)
- Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96(Study 105: Baseline through Week 96)
- Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96(Study 105: Baseline through Week 96)