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

27 de abril de 2021 actualizado 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.

Descripción general del estudio

Estado

Terminado

Condiciones

Intervención / Tratamiento

Tipo de estudio

Intervencionista

Inscripción (Actual)

246

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Alemania
      • Berlin, Alemania
      • Giessen, Alemania
      • Hanover, Alemania
      • Koeln, Alemania
  • Australia
      • Herston, Australia
      • New Lambton Heights, Australia
      • Subiaco, Australia
      • Westmead, Australia
    • Victoria
      • Parkville, Victoria, Australia
  • 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
  • Francia
      • Bordeaux Cedex, Francia
      • Paris, Francia
      • Paris Cedex 15, Francia
    • Cedex
      • Bron, Cedex, Francia
  • Reino Unido
      • Belfast, Reino Unido
      • Edinburgh, Reino Unido
      • London, Reino Unido
    • West Yorkshire
      • Leeds, West Yorkshire, Reino Unido
  • Suecia
      • Stockholm, Suecia

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

6 años y mayores (Niño, Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

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).

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Treatment Period 1: LUM/IVA to LUM/IVA
Droga: 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).

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

Otros nombres:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Droga: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Otros nombres:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Sin intervención: Treatment Period 1: Observational Cohort
Experimental: Treatment Period 2: LUM/IVA
Droga: 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).

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

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Periodo de tiempo
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)
Periodo de tiempo: Day 1 up to Week 100
Day 1 up to Week 100

Medidas de resultado secundarias

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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Vertex Pharmaceuticals Incorporated

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

1 de agosto de 2015

Finalización primaria (Actual)

1 de agosto de 2018

Finalización del estudio (Actual)

1 de abril de 2020

Fechas de registro del estudio

Enviado por primera vez

1 de septiembre de 2015

Primero enviado que cumplió con los criterios de control de calidad

4 de septiembre de 2015

Publicado por primera vez (Estimar)

9 de septiembre de 2015

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

24 de mayo de 2021

Última actualización enviada que cumplió con los criterios de control de calidad

27 de abril de 2021

Última verificación

1 de febrero de 2021

Más información

Términos relacionados con este estudio

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • VX15-809-110

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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