Esta página foi traduzida automaticamente e a precisão da tradução não é garantida. Por favor, consulte o versão em inglês para um texto fonte.

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

27 de abril de 2021 atualizado por: 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.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

246

Estágio

  • Fase 3

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

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

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

6 anos e mais velhos (Filho, Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

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).

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Não randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Treatment Period 1: LUM/IVA to LUM/IVA
Medicamento: 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).

Outros nomes:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Medicamento: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Outros nomes:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Experimental: Treatment Period 1: Placebo (PBO) to LUM/IVA
Medicamento: 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).

Outros nomes:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Medicamento: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Outros nomes:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Sem intervenção: Treatment Period 1: Observational Cohort
Experimental: Treatment Period 2: LUM/IVA
Medicamento: 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).

Outros nomes:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Medicamento: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Outros nomes:
  • lumacaftor/ivacaftor
  • VX-809/VX-770

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Prazo
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)
Prazo: Day 1 up to Week 100
Day 1 up to Week 100

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Absolute Change in Lung Clearance Index (LCI) 2.5
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
Prazo: 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
Prazo: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: Day 1 up to Week 168
Day 1 up to Week 168

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Vertex Pharmaceuticals Incorporated

Publicações e links úteis

A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.

Publicações Gerais

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

1 de agosto de 2015

Conclusão Primária (Real)

1 de agosto de 2018

Conclusão do estudo (Real)

1 de abril de 2020

Datas de inscrição no estudo

Enviado pela primeira vez

1 de setembro de 2015

Enviado pela primeira vez que atendeu aos critérios de CQ

4 de setembro de 2015

Primeira postagem (Estimativa)

9 de setembro de 2015

Atualizações de registro de estudo

Última Atualização Postada (Real)

24 de maio de 2021

Última atualização enviada que atendeu aos critérios de controle de qualidade

27 de abril de 2021

Última verificação

1 de fevereiro de 2021

Mais Informações

Termos relacionados a este estudo

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • VX15-809-110

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

Ensaios clínicos em LUM/IVA

Pesquisar ensaios semelhantes

Patrocinadores e Colaboradores

Condições médicas

Intervenções de drogas

CROs by country

CROs in South Africa

Condições

Doenças Raras

Intervenções de drogas

Suplementos Alimentares

Patrocinador / Colaboradores

Localizações

3
Se inscrever