A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
2019年3月18日 更新者：Vertex Pharmaceuticals Incorporated
A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise Tolerance in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
This study is designed to evaluate the effect of LUM/IVA on exercise tolerance in subjects with CF, homozygous for the F508del-CFTR mutation.
調査の概要
研究の種類
介入
入学 (実際)
70
段階
- フェーズ 4
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。
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
12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:
- Homozygous for the F508del-CFTR mutation
- Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
- Stable CF disease as judged by the investigator
- Forced expiratory volume in 1 second (FEV1) at least 40% and not greater than 90% of predicted
Exclusion Criteria:
- History of any comorbidity that might confound the results of the study, interfere with the use of cardiopulmonary exercise tests (CPETs) as an assessment, or pose an additional risk in administering study drug to the subject
- Any previous exposure to LUM or IVA
- History of cardiac arrhythmia, ischemic heart disease, congestive heart failure, or other clinically significant cardiac condition, or medical condition requiring chronic use of a beta blocker, non-dihydropyridine calcium channel blocker, or other cardiac medication known to affect exercise tolerance
- History of solid organ or hematological transplantation
- For subjects under 18 years of age at Screening, except those who have had bilateral lens removal, selected findings on a screening ophthalmologic examination will be exclusionary
- Using or expected to require any concomitant medication that is prohibited in this study
- History of alcohol or drug abuse, as deemed by the investigator, in the past year, including but not limited to cannabis, cocaine, and opiates
- Participation in an investigational drug study within 30 days before the Screening Visit
- Pregnant or nursing females; males with a female partner who is pregnant or nursing
- Colonization with organisms associated with a more rapid decline in pulmonary status
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：他の
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：トリプル
武器と介入
参加者グループ / アーム |
介入・治療 |
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プラセボコンパレーター：Placebo
Placebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
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実験的：LUM/IVA
LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
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他の名前：
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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 |
|---|---|---|
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Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
時間枠：Baseline, Week 24
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CPET was used to assess change in exercise tolerance, as measured by VO2max.
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Baseline, Week 24
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
時間枠：Baseline, Week 24
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Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
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Baseline, Week 24
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Absolute Change From Baseline in Exercise Duration During CPET at Week 24
時間枠：Baseline, Week 24
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Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
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Baseline, Week 24
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Absolute Change From Baseline in VO2max During CPET at Week 24
時間枠：Baseline, Week 24
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CPET was used to assess change in exercise tolerance, as measured by VO2max.
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Baseline, Week 24
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Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
時間枠：Baseline, Week 24
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Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
時間枠：Baseline, Week 24
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Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
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Baseline, Week 24
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Absolute Change From Baseline in Functional VO2 Gain at Week 24
時間枠：Baseline, Week 24
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24
時間枠：Baseline, Week 24
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Baseline, Week 24
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Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
時間枠：Baseline, Week 24
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
時間枠：Baseline, Week 24
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Baseline, Week 24
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Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
時間枠：Baseline, Week 24
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
時間枠：Baseline, Week 24
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Baseline, Week 24
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Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
時間枠：Baseline, Week 24
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BMI was defined as weight in kilograms (kg) divided by height in square meter (m^2).
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Baseline, Week 24
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Relative (Percent) Change From Baseline in BMI at Week 24
時間枠：Baseline, Week 24
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BMI was defined as weight in kg divided by height in m^2.
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Baseline, Week 24
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Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
時間枠：Baseline, Week 24
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The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
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Baseline, Week 24
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Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
時間枠：Baseline, Week 24
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The PHQ-8 is an eight item self-reported measure of depression.
Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day).
Total score is the sum of individual eight items and ranges from 0 to 24, with higher scores indicating more severe depression symptoms.
Total score of 0 to 5 indicates none to minimal depression, 6 to 10 indicates mild depression, 11 to 15 indicates moderate depression, 16 to 20 indicates moderately severe depression and 21 to 24 indicates severe depression.
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Baseline, Week 24
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Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
時間枠：Baseline, Week 24
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The GAD-7 is a seven item, self-reported measurement of GAD severity.
Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day).
Total score is the sum of individual seven items and ranges from 0 to 21, with higher scores indicating more severe anxiety symptoms.
Total score of 0 to 5 indicates none to minimal anxiety, 6 to 10 indicates mild anxiety, 11 to 15 indicates moderate anxiety, 16 to 21 indicates severe anxiety.
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Baseline, Week 24
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Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24
時間枠：Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24
時間枠：Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.
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Baseline, Week 24
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Absolute Change From Baseline in Duration of Sleep Time at Week 24
時間枠：Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24
時間枠：Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
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Baseline, Week 24
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Absolute Change From Baseline in Time Above Sedentary Duration at Week 24
時間枠：Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
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Baseline, Week 24
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Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24
時間枠：Baseline, Week 24
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Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
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Baseline, Week 24
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Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
時間枠：Day 1 up to Week 28
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Day 1 up to Week 28
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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。
出版物と役立つリンク
研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。
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 (実際)
2016年9月1日
一次修了 (実際)
2017年9月1日
研究の完了 (実際)
2017年10月1日
試験登録日
最初に提出
2016年8月15日
QC基準を満たした最初の提出物
2016年8月22日
最初の投稿 (見積もり)
2016年8月23日
学習記録の更新
投稿された最後の更新 (実際)
2019年6月17日
QC基準を満たした最後の更新が送信されました
2019年3月18日
最終確認日
2019年3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
- VX15-809-112
- 2016-000066-34 (EudraCT番号)
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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