A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 86
- 试验地点
- 29
- 主要终点
- Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
研究概览
简要总结
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.
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
- 0 Months 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ivacaftor Arm: Participants From Study 124 (NCT02725567 ) Part B:
- •Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
- •As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).
- •Ivacaftor Arm: Participants Not From Study 124 Part B:
- •Confirmed diagnosis of CF, or 2 CF-causing mutations.
- •An ivacaftor- responsive CFTR mutation on at least 1 allele. Participants will be eligible in countries/regions where ivacaftor is approved for use in participants 2 years of age and older.
- •As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.
- •Observational Arm:
- •Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.
排除标准
- •Ivacaftor Arm: Participants From Study 124 Part B:
- •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 ivacaftor to the participant.
- •Participants receiving commercially available ivacaftor treatment
- •Ivacaftor Arm: Participants Not From Study 124 Part B:
- •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 ivacaftor to the participant
- •An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1
- •Abnormal liver function at screening
- •Hemoglobin <9.5 g/dL at screening
- •History of solid organ or hematological transplantation
- •Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1
- •Observational Arm:
- •Receiving ivacaftor treatment
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
Ivacaftor Arm
Participants less than (<) 24 months of age and weighing 5 to less than (<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to <14 kg received 50 mg IVA q12h, and those weighing 14 to <25 kg received 75 mg IVA q12h.
Participants more than or equal (>=) 24 months of age and weighing <14 kg received 50 mg IVA q12h, and those weighing more than or equal to (>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks.
Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight.
干预措施: 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 TEAEs
时间窗: Day 1 up to Week 120
次要结局
- Absolute Change in Sweat Chloride(From Baseline at Week 96)