NCT04235140已完成3 期
A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 23
- 主要终点
- Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This is a Phase 3, multicenter, open-label and roll-over study in subjects who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.
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
- 12 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects From Study VX16-809-122 Part B (Study 122)
- •Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B
- •Subjects Not From Study 122
- •Subjects will be 1 to less than 2 years of age
- •Homozygous for the F508del mutation (F/F)
排除标准
- •Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject
- •Solid organ or hematological transplantation
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
LUM/IVA
Experimental
Participants weighing 7 to less than (<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks.
干预措施: LUM/IVA (Drug)
结局指标
主要结局
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 120
次要结局
- Absolute Change in Sweat Chloride (SwCl)(From Baseline at Week 96)
研究者
研究点 (23)
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