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Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

2021年4月27日 更新者：Vertex Pharmaceuticals Incorporated

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

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.

研究概览

地位

完全的

条件

干预/治疗

研究类型

介入性

注册 (实际的)

246

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 丹麦
      • Copenhagen、丹麦
  • 加拿大
    • British Columbia
      • Vancouver、British Columbia、加拿大
    • Ontario
      • Toronto、Ontario、加拿大
    • Quebec
      • Montreal、Quebec、加拿大
  • 德国
      • Berlin、德国
      • Giessen、德国
      • Hanover、德国
      • Koeln、德国
  • 比利时
      • Brussels、比利时
      • Leuven、比利时
  • 法国
      • Bordeaux Cedex、法国
      • Paris、法国
      • Paris Cedex 15、法国
    • Cedex
      • Bron、Cedex、法国
  • 澳大利亚
      • Herston、澳大利亚
      • New Lambton Heights、澳大利亚
      • Subiaco、澳大利亚
      • Westmead、澳大利亚
    • Victoria
      • Parkville、Victoria、澳大利亚
  • 瑞典
      • Stockholm、瑞典
  • 美国
    • Alabama
      • Birmingham、Alabama、美国
    • Arizona
      • Tucson、Arizona、美国
    • California
      • Long Beach、California、美国
      • Palo Alto、California、美国
    • Colorado
      • Aurora、Colorado、美国
    • Delaware
      • Wilmington、Delaware、美国
    • Florida
      • Jacksonville、Florida、美国
      • Orlando、Florida、美国
    • Georgia
      • Atlanta、Georgia、美国
    • Illinois
      • Chicago、Illinois、美国
    • Indiana
      • Indianapolis、Indiana、美国
    • Iowa
      • Iowa City、Iowa、美国
    • Massachusetts
      • Boston、Massachusetts、美国
    • Minnesota
      • Minneapolis、Minnesota、美国
    • Missouri
      • Kansas City、Missouri、美国
      • Saint Louis、Missouri、美国
    • Nebraska
      • Omaha、Nebraska、美国
    • New Hampshire
      • Lebanon、New Hampshire、美国
    • New York
      • Buffalo、New York、美国
      • Syracuse、New York、美国
    • North Carolina
      • Chapel Hill、North Carolina、美国
    • Ohio
      • Cincinnati、Ohio、美国
      • Cleveland、Ohio、美国
      • Dayton、Ohio、美国
    • Oregon
      • Portland、Oregon、美国
    • Pennsylvania
      • Pittsburgh、Pennsylvania、美国
    • South Carolina
      • Charleston、South Carolina、美国
    • Texas
      • Austin、Texas、美国
      • Houston、Texas、美国
    • Utah
      • Salt Lake City、Utah、美国
    • Vermont
      • Colchester、Vermont、美国
    • Virginia
      • Charlottesville、Virginia、美国
      • Norfolk、Virginia、美国
      • Richmond、Virginia、美国
    • Washington
      • Seattle、Washington、美国
    • Wisconsin
      • Madison、Wisconsin、美国
      • Milwaukee、Wisconsin、美国
  • 英国
      • Belfast、英国
      • Edinburgh、英国
      • London、英国
    • West Yorkshire
      • Leeds、West Yorkshire、英国

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

6年 及以上 (孩子、成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

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).

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：非随机化
  • 介入模型：并行分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：Treatment Period 1: LUM/IVA to LUM/IVA
药品：LUM/IVA

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

其他名称：
  • lumacaftor/ivacaftor
  • VX-809/VX-770
药品：LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
其他名称：
  • lumacaftor/ivacaftor
  • VX-809/VX-770
实验性的：Treatment Period 1: Placebo (PBO) to LUM/IVA
药品：LUM/IVA

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

其他名称：
  • lumacaftor/ivacaftor
  • VX-809/VX-770
药品：LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
其他名称：
  • lumacaftor/ivacaftor
  • VX-809/VX-770
无干预：Treatment Period 1: Observational Cohort
实验性的：Treatment Period 2: LUM/IVA
药品：LUM/IVA

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

其他名称：
  • lumacaftor/ivacaftor
  • VX-809/VX-770
药品：LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
其他名称：
  • lumacaftor/ivacaftor
  • VX-809/VX-770

研究衡量的是什么？

主要结果指标

结果测量
大体时间
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)
大体时间：Day 1 up to Week 100
Day 1 up to Week 100

次要结果测量

结果测量
措施说明
大体时间
Absolute Change in Lung Clearance Index (LCI) 2.5
大体时间：From Parent Study Baseline at Week 96
LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
From Parent Study Baseline at Week 96
Absolute Change in Sweat Chloride
大体时间：From Parent Study Baseline at Week 96
Sweat samples were collected using an approved collection device.
From Parent Study Baseline at Week 96
Absolute Change in Body Mass Index (BMI)
大体时间：From Parent Study Baseline at Week 96
BMI was defined as weight in kilograms divided by height in square meter (m^2).
From Parent Study Baseline at Week 96
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
大体时间：From Parent Study Baseline at Week 96
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
From Parent Study Baseline at Week 96
Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
大体时间：Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
大体时间：From Parent Study Baseline at Week 96
LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.
From Parent Study Baseline at Week 96
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
大体时间：From Parent Study Baseline at Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Parent Study Baseline at Week 96
Relative Change in ppFEV1
大体时间：From Parent Study Baseline at Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Parent Study Baseline at Week 96
Absolute Change in BMI-for-age Z-score
大体时间：From Parent Study Baseline at Week 96
BMI was defined as weight in kilograms divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Parent Study Baseline at Week 96
Absolute Change in Weight
大体时间：From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
大体时间：From Parent Study Baseline at Week 96
z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Parent Study Baseline at Week 96
Absolute Change in Height
大体时间：From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
大体时间：From Parent Study Baseline at Week 96
z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Parent Study Baseline at Week 96
Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score
大体时间：From Parent Study Baseline at Week 96
The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction.
From Parent Study Baseline at Week 96
Time-to-first Pulmonary Exacerbation
大体时间：From Parent Study Baseline through Week 96
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Parent Study Baseline through Week 96
Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event
大体时间：From Parent Study Baseline through Week 96
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Parent Study Baseline through Week 96
Number of Pulmonary Exacerbation Events Per Patient-year
大体时间：From Parent Study Baseline through Week 96
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Parent Study Baseline through Week 96
Rate of Change in LCI 2.5
大体时间：Day 15 after first dose of LUM/IVA through Week 96
Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in LCI 5.0
大体时间：Day 15 after first dose of LUM/IVA through Week 96
Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in ppFEV1
大体时间：Day 15 after first dose of LUM/IVA through Week 96
Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Day 15 after first dose of LUM/IVA through 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)
大体时间：Day 1 up to Week 168
Day 1 up to Week 168

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Vertex Pharmaceuticals Incorporated

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2015年8月1日

初级完成 (实际的)

2018年8月1日

研究完成 (实际的)

2020年4月1日

研究注册日期

首次提交

2015年9月1日

首先提交符合 QC 标准的

2015年9月4日

首次发布 (估计)

2015年9月9日

研究记录更新

最后更新发布 (实际的)

2021年5月24日

上次提交的符合 QC 标准的更新

2021年4月27日

最后验证

2021年2月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • VX15-809-110

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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赞助者和合作者

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条件

罕见疾病

药物干预

膳食补充剂

赞助者/合作者

地点

3
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