A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 33
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will further explore the efficacy of long-term ivacaftor treatment in this population of patients with CF.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •(Observational Arm)
- •Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.
研究组 & 干预措施
Ivacaftor
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:
- 50 mg q12h for subjects 2 to <6 years of age and <14 kg,
- 75 mg q12h for subjects 2 to <6 years of age and ≥ 14 kg, or
- 150 mg q12h for subjects ≥ 6 years of age.
干预措施: Ivacaftor (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing)
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation was considered treatment-emergent.
次要结局
- Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84(Baseline (study 108), Week 24, 48, 72 and 84 (study 109))
- Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84(Baseline (study 109), Week 24, 48, 72 and 84 (study 109))
- Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84(Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109))
- Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84(Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109))
- Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84(Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109))
- Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84(Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109))
- Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84(Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109))
- Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84(Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109))