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

2021년 4월 27일 업데이트: 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.

연구 개요

상태

완전한

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

246

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

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

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

6년 이상 (어린이, 성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

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).

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위화되지 않음
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: Treatment Period 1: LUM/IVA to LUM/IVA
의약품: 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).

다른 이름들:
  • 루마카프터/이바카프터
  • VX-809/VX-770
의약품: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
다른 이름들:
  • 루마카프터/이바카프터
  • VX-809/VX-770
실험적: Treatment Period 1: Placebo (PBO) to LUM/IVA
의약품: 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).

다른 이름들:
  • 루마카프터/이바카프터
  • VX-809/VX-770
의약품: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
다른 이름들:
  • 루마카프터/이바카프터
  • VX-809/VX-770
간섭 없음: Treatment Period 1: Observational Cohort
실험적: Treatment Period 2: LUM/IVA
의약품: 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).

다른 이름들:
  • 루마카프터/이바카프터
  • VX-809/VX-770
의약품: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
다른 이름들:
  • 루마카프터/이바카프터
  • VX-809/VX-770

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
기간
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)
기간: Day 1 up to Week 100
Day 1 up to Week 100

2차 결과 측정

결과 측정
측정값 설명
기간
Absolute Change in Lung Clearance Index (LCI) 2.5
기간: 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
기간: 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)
기간: 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
기간: 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)
기간: Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
기간: 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)
기간: 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
기간: 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
기간: 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
기간: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
기간: 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
기간: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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)
기간: Day 1 up to Week 168
Day 1 up to Week 168

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Vertex Pharmaceuticals Incorporated

간행물 및 유용한 링크

연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.

일반 간행물

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2015년 8월 1일

기본 완료 (실제)

2018년 8월 1일

연구 완료 (실제)

2020년 4월 1일

연구 등록 날짜

최초 제출

2015년 9월 1일

QC 기준을 충족하는 최초 제출

2015년 9월 4일

처음 게시됨 (추정)

2015년 9월 9일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2021년 5월 24일

QC 기준을 충족하는 마지막 업데이트 제출

2021년 4월 27일

마지막으로 확인됨

2021년 2월 1일

추가 정보

이 연구와 관련된 용어

추가 관련 MeSH 약관

기타 연구 ID 번호

  • VX15-809-110

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

LUM/IVA에 대한 임상 시험

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스폰서 및 공동 작업자

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정황

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약물 개입

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스폰서 / 협력자

위치

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