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Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation (STRIVE)

2013年1月14日 更新者：Vertex Pharmaceuticals Incorporated

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects With Cystic Fibrosis and the G551D Mutation

The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.

研究概览

地位

完全的

条件

干预/治疗

详细说明

This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.

Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.

This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.

研究类型

介入性

注册 (实际的)

167

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

  • 加拿大
    • Nova Scotia
      • Halifax、Nova Scotia、加拿大、B3H 3A7
        • Queen Elizabeth Ii Health Sciences Centre
    • Ontario
      • Toronto、Ontario、加拿大、M5B 1W8
        • St. Michael's Hospital
      • Toronto、Ontario、加拿大、M5G 1X8
        • CF Center, Hospital for Sick Children
    • Quebec
      • Montreal、Quebec、加拿大、H3H 1P3
        • Montreal Children's Hospital - MUHC
  • 德国
      • Erlangen、德国、91054
        • Kinder- und Jugendklinik Universitätsklinikum Erlangen
      • Jena、德国、07740
        • Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin
      • Munich、德国、80337
        • Klinikum der LMU München, Dr. von Haunersches Kinderspital (CHA)
      • Wurzburg、德国、97080
        • Universitäts-Kinderklinik Würzburg
  • 捷克共和国
      • Prague、捷克共和国、15006
        • FN Motol
  • 法国
      • Paris、法国、75014
        • Hôpital Cochin
      • Paris、法国、75015
        • Hopital Necker
      • Roscoff、法国、29684
        • Centre de Perharidy
  • 澳大利亚
    • New South Wales
      • Westmead、New South Wales、澳大利亚、2145
        • The Children's Hospital Westmead
    • Queensland
      • Chermside、Queensland、澳大利亚、4032
        • The Prince Charles Hospital
      • Herston、Queensland、澳大利亚、4026
        • Royal Children's Hospital Brisbane
      • South Brisbane、Queensland、澳大利亚、4101
        • Mater Adult Hospital
    • Victoria
      • Parkville、Victoria、澳大利亚、3052
        • Royal Children's Hospital Melbourne
    • Western Australia
      • Nedlands、Western Australia、澳大利亚、6009
        • Lung Institute of Western Australia
      • Subiaco、Western Australia、澳大利亚、6008
        • Princess Margaret Hospital for Children
  • 爱尔兰
      • Cork、爱尔兰
        • Cork University Hospital
      • Dublin、爱尔兰、9
        • Beaumont Hospital
      • Dublin、爱尔兰、4
        • St. Vincent'S University Hospital
      • Dublin、爱尔兰、12
        • Our Lady's Children's Hospital
      • Dublin、爱尔兰、24
        • The National Children's Hospital
  • 美国
    • Alabama
      • Birmingham、Alabama、美国、35233-1711
        • University of Alabama
    • California
      • Oakland、California、美国、94611
        • Kaiser Permanente Medical Care Program
      • Palo Alto、California、美国、94304
        • Cystic Fibrosis Research Office, Stanford University
      • San Diego、California、美国、92123-5070
        • Rady Children's Hospital
    • Colorado
      • Denver、Colorado、美国、80206
        • National Jewish Medical and Research Center
    • Georgia
      • Atlanta、Georgia、美国、30322
        • Emory Cystic Fibrosis Center
    • Idaho
      • Boise、Idaho、美国、83712
        • St. Luke's CF Clinic
    • Illinois
      • Chicago、Illinois、美国、60614
        • Children's Memorial Hospital
    • Indiana
      • Indianapolis、Indiana、美国、46202
        • Indiana University
    • Iowa
      • Iowa City、Iowa、美国、52242
        • University of Iowa
    • Maryland
      • Baltimore、Maryland、美国、21205
        • Johns Hopkins University
    • Massachusetts
      • Boston、Massachusetts、美国、02114
        • Massachusetts General Hospital
      • Boston、Massachusetts、美国、02115
        • Children's Hospital Boston
    • Michigan
      • Ann Arbor、Michigan、美国、48109
        • University of Michigan
    • Minnesota
      • Minneapolis、Minnesota、美国、55455
        • Pulmonary, Allergy & Critical Care Medicine, University of Minnesota
    • Missouri
      • Kansas City、Missouri、美国、64108
        • The Children's Mercy Hospital
      • St. Louis、Missouri、美国、63110
        • Washington University
    • Nebraska
      • Omaha、Nebraska、美国、68198-5300
        • Adult Pulmonary/ CF, University of Nebraska Medical Center
    • New Jersey
      • Long Branch、New Jersey、美国、07740
        • Monmouth Medical Center
    • New York
      • Buffalo、New York、美国、14222
        • Women and Children's Hospital of Buffalo
      • New Hyde Park、New York、美国、11042
        • Long Island Jewish Medical Center
      • Syracuse、New York、美国、13210
        • SUNY Upstate Medical University
    • North Carolina
      • Chapel Hill、North Carolina、美国、27599
        • University of North Carolina at Chapel Hill
    • Ohio
      • Cincinnati、Ohio、美国、45229
        • Cincinnati Children's Hospital Medical Center
      • Cleveland、Ohio、美国、44106
        • Pediatric & Pulmonary Division, Rainbow Babies/Case Western
      • Columbus、Ohio、美国、43205
        • Nationwide Children's Hospital
      • Toledo、Ohio、美国、43606
        • Toledo Children's Hospital
    • Oregon
      • Portland、Oregon、美国、97239-3098
        • Oregon Health & Sciences University
    • Pennsylvania
      • Hershey、Pennsylvania、美国、17033
        • Hershey Medical Center
      • Philadelphia、Pennsylvania、美国、19104
        • Children's Hospital of Philadelphia
      • Pittsburgh、Pennsylvania、美国、15224
        • Children's Hospital of Pittsburgh of UPMC
    • Tennessee
      • Knoxville、Tennessee、美国、37916
        • East Tennessee Children's Hospital
      • Nashville、Tennessee、美国、37232-5735
        • Vanderbilt University Medical Center
    • Utah
      • Salt Lake City、Utah、美国、84132
        • University of Utah
    • Virginia
      • Charlottesville、Virginia、美国、22908
        • University of Virginia
    • Washington
      • Seattle、Washington、美国、98105
        • Seattle Children's Hospital
      • Seattle、Washington、美国、98195-6522
        • Division of Pulmonary and CCM, University of Washington
    • West Virginia
      • Morgantown、West Virginia、美国、26506
        • West Virginia University
    • Wisconsin
      • Milwaukee、Wisconsin、美国、53226
        • Medical College of Wisconsin
  • 英国
      • London、英国、SW3 6LR
        • Imperial College London
    • Northern Ireland
      • Belfast、Northern Ireland、英国、BT9 7AB
        • Belfast City Hospital

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

12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:

  • Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
  • Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
  • No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
  • Willing to use highly effective birth control methods during the study

Exclusion Criteria:

  • History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
  • Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
  • History of alcohol, medication or illicit drug abuse within one year prior to Day 1
  • Abnormal liver function ≥ 3x the upper limit of normal
  • Abnormal renal function at Screening
  • History of solid organ or hematological transplantation
  • Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
  • Use of inhaled hypertonic saline treatment
  • Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)

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：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
安慰剂比较：安慰剂
每 12 小时 (q12h) 接受一次安慰剂治疗长达 48 周的受试者。
药品：安慰剂
口服片剂 q12h 长达 48 周
实验性的：150 毫克依伐卡托 q12h
接受 150 mg ivacaftor q12h 长达 48 周的受试者。
药品：Ivacaftor
150-mg tablets given orally q12h for up to 48 weeks
其他名称：
  • VX-770

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24
大体时间：baseline through 24 weeks
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
baseline through 24 weeks

次要结果测量

结果测量
措施说明
大体时间
第 24 周和第 48 周期间汗液氯化物浓度相对于基线的绝对变化
大体时间：基线至 24 周和 48 周
汗液氯化物（定量毛果芸香碱离子电渗疗法）试验是囊性纤维化 (CF) 的标准诊断工具，可作为囊性纤维化跨膜电导调节剂 (CFTR) 活性的指标。
基线至 24 周和 48 周
第 24 周和第 48 周体重相对于基线的绝对变化
大体时间：基线至 24 周和 48 周
由于肺部疾病和脂肪吸收不良导致能量消耗增加，因此囊性纤维化 (CF) 患者营养不良很常见，因此体重是衡量营养状况的重要临床指标。
基线至 24 周和 48 周
Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48
大体时间：baseline through 48 weeks
Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies.
baseline through 48 weeks
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled)
大体时间：baseline through 24 weeks and 48 weeks
The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID).
baseline through 24 weeks and 48 weeks
Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48
大体时间：baseline through 24 weeks and 48 weeks
Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection.
baseline through 24 weeks and 48 weeks

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

合作者

Cystic Fibrosis Foundation

调查人员

  • 首席研究员：Bonnie W. Ramsey, MD、Children's Hospital and Regional Medical Center, Seattle, Washington, USA
  • 首席研究员：Stuart Elborn, MD、Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK

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

有用的网址

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

2009年6月1日

初级完成 (实际的)

2010年7月1日

研究完成 (实际的)

2012年11月1日

研究注册日期

首次提交

2009年5月26日

首先提交符合 QC 标准的

2009年5月26日

首次发布 (估计)

2009年5月28日

研究记录更新

最后更新发布 (估计)

2013年1月18日

上次提交的符合 QC 标准的更新

2013年1月14日

最后验证

2013年1月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • VX08-770-102

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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