NCT02742519终止3 期
A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 14
- 主要终点
- Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)
研究概览
简要总结
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 3 Years 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female with confirmed diagnosis of CF.
- •Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
- •Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator.
排除标准
- •An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
- •Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
- •Abnormal liver function, at Screening, defined as ≥3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
- •History of solid organ or hematological transplantation
- •Any clinically significant "non-CF-related" illness within 2 weeks before Day 1
- •Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
- •Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening
研究组 & 干预措施
Part 1-Sequence 1
Experimental
ivacaftor in Treatment Period 1 →washout→placebo in Treatment Period 2
干预措施: ivacaftor (Drug)
Part 1-Sequence 1
Experimental
ivacaftor in Treatment Period 1 →washout→placebo in Treatment Period 2
干预措施: Placebo (Drug)
Part 1 - Sequence 2
Experimental
placebo in Treatment Period 1→washout→ivacaftor in Treatment Period 2
干预措施: ivacaftor (Drug)
Part 1 - Sequence 2
Experimental
placebo in Treatment Period 1→washout→ivacaftor in Treatment Period 2
干预措施: Placebo (Drug)
Part 2: ivacaftor
Experimental
open label period
干预措施: ivacaftor (Drug)
结局指标
主要结局
Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)
时间窗: Baseline Through Week 8 for each treatment period, Up to 24 Weeks
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
- Absolute Change From Baseline in Fecal Elastase-1 Levels at Week 8(Baseline and Week 8 of each treatment period, Up to 24 Weeks)
- Absolute Change From Baseline in Immunoreactive Trypsinogen Levels at Week 8(Baseline and Week 8 of each treatment period, Up to 24 Weeks)
- Absolute Change From Baseline in Body Mass Index (BMI) at Week 8(Baseline and Week 8 of each treatment period, Up to 24 Weeks)
- Absolute Change From Baseline in Weight at Week 8(Baseline and Week 8 of each treatment period, Up to 24 Weeks)
- Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Baseline up to Month 15)
研究者
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