NCT02514473已完成3 期
A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
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
- 206
- 主要终点
- Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24
研究概览
简要总结
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects who weigh ≥15 kg without shoes a the Screening Visit
- •Subjects with confirmed diagnosis of CF at the Screening Visit.
- •Subjects who are homozygous for the F508del CFTR mutation
- •Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height
- •Subjects with a screening LCI2.5 result greater than or equal to 7.5
排除标准
- •History of any comorbidity reviewed at the Screening Visit 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.
- •Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
- •Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
- •An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
- •History of solid organ or hematological transplantation at the Screening Visit
研究组 & 干预措施
LUM/IVA
Experimental
Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
干预措施: VX-809 (Drug)
LUM/IVA
Experimental
Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
干预措施: VX-770 (Drug)
Placebo
Placebo Comparator
Matching placebo q12h
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24
时间窗: Baseline, Through Week 24
Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
次要结局
- Average Absolute Change From Baseline in Sweat Chloride at Day 15 and Week 4(Baseline, Day 15 and Week 4)
- Absolute Change From Baseline in Body Mass Index (BMI) at Week 24(Baseline, Week 24)
- Absolute Change From Baseline in Sweat Chloride at Week 24(Baseline, Week 24)
- Relative Change From Baseline in ppFEV1 Through Week 24(Baseline, Through Week 24)
- Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24(Baseline, Through Week 24)
- Percentage of Participants With At Least 1 Pulmonary Exacerbation Event(Baseline through Week 24)
- Absolute Change From Baseline in Lung Clearance Index 5.0 (LCI5.0) Through Week 24(Baseline, Through Week 24)
- Absolute Change From Baseline in Weight at Week 24(Baseline, Week 24)
- Absolute Change From Baseline in Height at Week 24(Baseline, Week 24)
- Absolute Change From Baseline in Height-for-age Z-score at Week 24(Baseline, Week 24)
- Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domains Through Week 24(Baseline, Through Week 24)
- Number of Pulmonary Exacerbation Events(Baseline through Week 24)
- Time-to-first Pulmonary Exacerbation(Baseline through Week 24)
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Baseline up to Week 28)
- Average Pre-dose Concentration (Ctrough,Ave) and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Ave) For Lumacaftor and Ivacaftor(For Ctrough,ave: before morning dose on Week 4 and 24; For C3-6h,ave: 3 to 6 hours after morning dose on Day 1, 15 and Week 4)
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24(Baseline, Through Week 24)
- Absolute Change From Baseline in BMI-for-age Z-score at Week 24(Baseline, Week 24)
- Absolute Change From Baseline in Weight-for-age Z-score at Week 24(Baseline, Week 24)
研究者
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