NCT02544451已完成3 期
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older 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
- 246
- 主要终点
- Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects entering the Treatment Cohort must meet both of the following criteria:
- •Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
- •Willing to remain on a stable CF medication through the Safety Follow-up Visit.
- •Subjects entering the Observational Cohort must meet 1 of the following criteria:
- •Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
- •Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.
- •Exclusion Criteria (Treatment Cohort Only):
- •History of any comorbidity or laboratory abnormality 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 (e.g., cirrhosis with portal hypertension).
- •Pregnant and nursing females.
- •Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
- •History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
- •History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
- •Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).
排除标准
- 未提供
研究组 & 干预措施
Treatment Period 1: LUM/IVA to LUM/IVA
Experimental
干预措施: LUM/IVA (Drug)
Treatment Period 1: Placebo (PBO) to LUM/IVA
Experimental
干预措施: LUM/IVA (Drug)
Treatment Period 2: LUM/IVA
Experimental
干预措施: LUM/IVA (Drug)
结局指标
主要结局
Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 100
次要结局
- Absolute Change in Weight(From Parent Study Baseline at Week 96)
- Absolute Change in Height-for-age Z-score(From Parent Study Baseline at Week 96)
- Time-to-first Pulmonary Exacerbation(From Parent Study Baseline through Week 96)
- Number of Pulmonary Exacerbation Events Per Patient-year(From Parent Study Baseline through Week 96)
- Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)(Day 1 up to Week 100)
- Absolute Change in Body Mass Index (BMI)(From Parent Study Baseline at Week 96)
- Rate of Change in ppFEV1(Day 15 after first dose of LUM/IVA through Week 96)
- Absolute Change in Sweat Chloride(From Parent Study Baseline at Week 96)
- Absolute Change in BMI-for-age Z-score(From Parent Study Baseline at Week 96)
- Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score(From Parent Study Baseline at Week 96)
- Rate of Change in LCI 2.5(Day 15 after first dose of LUM/IVA through Week 96)
- Rate of Change in LCI 5.0(Day 15 after first dose of LUM/IVA through Week 96)
- Absolute Change in Lung Clearance Index (LCI) 2.5(From Parent Study Baseline at Week 96)
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score(From Parent Study Baseline at Week 96)
- Absolute Change in LCI 5.0(From Parent Study Baseline at Week 96)
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)(From Parent Study Baseline at Week 96)
- Relative Change in ppFEV1(From Parent Study Baseline at Week 96)
- Absolute Change in Height(From Parent Study Baseline at Week 96)
- Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Day 1 up to Week 168)
- Absolute Change in Weight-for-age Z-score(From Parent Study Baseline at Week 96)
- Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event(From Parent Study Baseline through Week 96)
研究者
相似试验
已完成
3 期
Safety of AZLI in Children With Cystic Fibrosis (CF) and Chronic Pseudomonas Aeruginosa in the Lower AirwaysCystic FibrosisPseudomonas AeruginosaNCT01404234Gilead Sciences61
已完成
3 期
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR MutationCystic FibrosisNCT03125395Vertex Pharmaceuticals Incorporated57
已完成
3 期
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR MutationCystic FibrosisNCT02565914Vertex Pharmaceuticals Incorporated1,131
已完成
3 期
Second Open Label Extension to Bridging Study CTBM100C2303Pulmonary InfectionsPseudomonas AeruginosaNCT01069705Novartis Pharmaceuticals49
已完成
3 期
Open Label Extension to Bridging Study CTBM100C2303Pseudomonas AeruginosaCystic FibrosisNCT00982930Novartis Pharmaceuticals55