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Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation (KONNECTION)

2014年10月23日 更新者：Vertex Pharmaceuticals Incorporated

A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation

The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).

調査の概要

状態

完了

条件

介入・治療

詳細な説明

Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in subjects with CF. Results from Phase 3 studies (VX08-770-102 [Study 102] [NCT00909532] and VX08-770-103 [Study 103] [NCT00909727]) showed that ivacaftor is effective in the treatment of subjects with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments.

Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in subjects 6 years of age and older who have a G551D mutation in the CFTR gene.

研究の種類

介入

入学 (実際)

39

段階

  • フェーズ 3

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

  • アメリカ
    • Florida
      • Tampa、Florida、アメリカ
    • Georgia
      • Atlanta、Georgia、アメリカ
    • Illinois
      • Chicago、Illinois、アメリカ
    • Massachusetts
      • Boston、Massachusetts、アメリカ
    • Michigan
      • Ann Arbor、Michigan、アメリカ
    • Minnesota
      • Minneapolis、Minnesota、アメリカ
    • Missouri
      • St. Louis、Missouri、アメリカ
    • Texas
      • Houston、Texas、アメリカ
  • フランス
      • Lyon、フランス
      • Montpellier、フランス
      • Paris、フランス
  • ベルギー
      • Leuven、ベルギー

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

6年歳以上 (子、大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

Inclusion Criteria:

  • Male or female with confirmed diagnosis of CF
  • At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
  • Percent predicted forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) predicted normal for age, sex, and height
  • 6 years of age or older
  • Minimum weight of 15 kilogram (kg) at screening
  • Females of childbearing potential must not be pregnant
  • Willing to comply with contraception requirements

Exclusion Criteria:

  • G551D-CFTR mutation on at least 1 allele
  • History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug
  • History of solid organ or hematological transplantation
  • History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug
  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening
  • Use of inhaled hypertonic saline treatment
  • Use of any inhibitors or inducers of cytochrome (CYP) P450 3A
  • Evidence of cataract or lens opacity at screening

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：ランダム化
  • 介入モデル：クロスオーバー割り当て
  • マスキング：4倍

武器と介入

参加者グループ / アーム
介入・治療
実験的：Part 1: Ivacaftor First, Then Placebo
Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
薬：Ivacaftor
150 mg tablet, oral use, administered twice a day (q12h)
他の名前：
  • VX-770
  • カリデコ
薬：Placebo
oral use, administered twice a day (q12h)
実験的：Part 1: Placebo First, Then Ivacaftor
Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
薬：Ivacaftor
150 mg tablet, oral use, administered twice a day (q12h)
他の名前：
  • VX-770
  • カリデコ
薬：Placebo
oral use, administered twice a day (q12h)
実験的：Part 2: Ivacaftor
Ivacaftor 150 mg tablet orally twice daily for 16 weeks.
薬：Ivacaftor
150 mg tablet, oral use, administered twice a day (q12h)
他の名前：
  • VX-770
  • カリデコ

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
時間枠：Part 1: Baseline (pre-dose Day 1), Week 8
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit)
時間枠：Baseline (pre-dose Week 12), Week 36
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Absolute change in percent predicted FEV1 over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2, as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36

二次結果の測定

結果測定
メジャーの説明
時間枠
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8
時間枠：Part 1: Baseline (pre-dose Day 1), Week 8
BMI was defined as weight in kilogram (kg) divided by height in meters^2 (m^2). Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Change From Baseline in Body Mass Index (BMI) at 24 Weeks of Treatment (Week 36 Visit)
時間枠：Baseline (pre-dose Week 12), Week 36
BMI was defined as weight in kg divided by height in m^2. Change in BMI over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36
Part 1: Change From Baseline in Sweat Chloride Through Week 8
時間枠：Part 1: Baseline (pre-dose Day 1), Week 8
Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Change From Baseline in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit)
時間枠：Baseline (pre-dose Week 12), Week 36
Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Change in sweat chloride over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36
Part 1: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
時間枠：Part 1: Baseline (pre-dose Day 1), Week 8
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.
Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through 24 Weeks of Treatment (Week 36 Visit)
時間枠：Baseline (pre-dose Week 12), Week 36
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Change in CFQ-R respiratory domain score over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.
Baseline (pre-dose Week 12), Week 36
Part 1: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
時間枠：Part 1: From signing of informed consent up to Week 20
AE: any adverse change from subject's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event.
Part 1: From signing of informed consent up to Week 20
Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
時間枠：Part 2: Week 20 up to Week 40
AE: any adverse change from subject's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event.
Part 2: Week 20 up to Week 40

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Vertex Pharmaceuticals Incorporated

協力者

Cystic Fibrosis Foundation

捜査官

  • 主任研究者：Christine De Boeck, MD, PhD、University of Leuven

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始

2012年7月1日

一次修了 (実際)

2013年10月1日

研究の完了 (実際)

2013年10月1日

試験登録日

最初に提出

2012年6月5日

QC基準を満たした最初の提出物

2012年6月7日

最初の投稿 (見積もり)

2012年6月8日

学習記録の更新

投稿された最後の更新 (見積もり)

2014年10月29日

QC基準を満たした最後の更新が送信されました

2014年10月23日

最終確認日

2014年10月1日

詳しくは

本研究に関する用語

追加の関連 MeSH 用語

その他の研究ID番号

  • VX12-770-111

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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