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A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis

3 de abril de 2017 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 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

The purpose of this study is to evaluate the efficacy and safety of long-term treatment with lumacaftor in combination with ivacaftor in people 12 years and older with Cystic Fibrosis.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Descrição detalhada

This is a Phase 3, parallel group, multicenter, rollover study in participants with CF who are homozygous or heterozygous for the F508del CFTR mutation and who previously participated in Study 103 (Study VX12-809-103, NCT01807923), Study 104 (Study VX12-809-104, NCT01807949), or Cohort 4 of Study 102 (Study VX09-809-102, NCT01225211).

Tipo de estudo

Intervencional

Inscrição (Real)

1164

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
      • Bochum, Alemanha
      • Erlangen, Alemanha
      • Essen, Alemanha
      • Frankfurt, Alemanha
      • Giessen, Alemanha
      • Hannover, Alemanha
      • Jena, Alemanha
      • Koeln, Alemanha
      • Leipzig, Alemanha
      • Muenchen, Alemanha
      • Tuebingen, Alemanha
      • Wuerzburg, Alemanha
    • Bayem
      • Muenchen, Bayem, Alemanha
    • Bayern
      • Muenchen, Bayern, Alemanha
  • Austrália
      • Nedlands, Austrália
      • Subiaco, Austrália
    • New South Wales
      • New Lambton Heights, New South Wales, Austrália
      • Westmead, New South Wales, Austrália
    • Queensland
      • Adelaide, Queensland, Austrália
      • Chermside, Queensland, Austrália
      • Herston, Queensland, Austrália
      • South Brisbane, Queensland, Austrália
  • Bélgica
      • Bruxelles, Bélgica
      • Gent, Bélgica
      • Leuven, Bélgica
      • Liège, Bélgica
  • Canadá
    • Alberta
      • Calgary, Alberta, Canadá
      • Edmonton, Alberta, Canadá
    • British Columbia
      • Vancouver, British Columbia, Canadá
    • Nova Scotia
      • Halifax, Nova Scotia, Canadá
    • Ontario
      • Ottowa, Ontario, Canadá
      • Toronto, Ontario, Canadá
    • Quebec
      • Montreal, Quebec, Canadá
  • Dinamarca
      • Copenhagen, Dinamarca
  • Espanha
      • Barcelona, Espanha
      • Valencia, Espanha
  • Estados Unidos
    • Alabama
      • Birmingham, Alabama, Estados Unidos
    • Alaska
      • Anchorage, Alaska, Estados Unidos
    • Arizona
      • Tucson, Arizona, Estados Unidos
    • Arkansas
      • Little Rock, Arkansas, Estados Unidos
    • California
      • La Jolla, California, Estados Unidos
      • Loma Linda, California, Estados Unidos
      • Longbeach, California, Estados Unidos
      • Los Angeles, California, Estados Unidos
      • Madera, California, Estados Unidos
      • Oakland, California, Estados Unidos
      • Palo Alto, California, Estados Unidos
      • Sacramento, California, Estados Unidos
    • Colorado
      • Aurora, Colorado, Estados Unidos
      • Denver, Colorado, Estados Unidos
    • Connecticut
      • Hartford, Connecticut, Estados Unidos
      • New Haven, Connecticut, Estados Unidos
    • Florida
      • Altamonte Springs, Florida, Estados Unidos
      • Hollywood, Florida, Estados Unidos
      • Jacksonville, Florida, Estados Unidos
      • Miami, Florida, Estados Unidos
      • Orlando, Florida, Estados Unidos
      • Tampa, Florida, Estados Unidos
    • Georgia
      • Atlanta, Georgia, Estados Unidos
    • Idaho
      • Boise, Idaho, Estados Unidos
    • Illinois
      • Chicago, Illinois, Estados Unidos
      • Park Ridge, Illinois, Estados Unidos
      • Peoria, Illinois, Estados Unidos
    • Indiana
      • Indianapolis, Indiana, Estados Unidos
    • Iowa
      • Iowa City, Iowa, Estados Unidos
    • Kansas
      • Kansas City, Kansas, Estados Unidos
    • Kentucky
      • Lexington, Kentucky, Estados Unidos
    • Louisiana
      • New Orleans, Louisiana, Estados Unidos
    • Maine
      • South Portland, Maine, Estados Unidos
    • Maryland
      • Baltimore, Maryland, Estados Unidos
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos
      • Worcester, Massachusetts, Estados Unidos
    • Michigan
      • Ann Arbor, Michigan, Estados Unidos
      • Detroit, Michigan, Estados Unidos
      • Grand Rapids, Michigan, Estados Unidos
    • Minnesota
      • Minneapolis, Minnesota, Estados Unidos
    • Mississippi
      • Jackson, Mississippi, Estados Unidos
    • Missouri
      • Kansas City, Missouri, Estados Unidos
      • St Louis, Missouri, Estados Unidos
      • St. Louis, Missouri, Estados Unidos
    • Nebraska
      • Omaha, Nebraska, Estados Unidos
    • New Hampshire
      • Bedford, New Hampshire, Estados Unidos
      • Lebanon, New Hampshire, Estados Unidos
    • New Jersey
      • Long Branch, New Jersey, Estados Unidos
      • Morristown, New Jersey, Estados Unidos
      • New Brunswick, New Jersey, Estados Unidos
    • New Mexico
      • Albuquerque, New Mexico, Estados Unidos
    • New York
      • Albany, New York, Estados Unidos
      • Buffalo, New York, Estados Unidos
      • Lake Success, New York, Estados Unidos
      • New York, New York, Estados Unidos
      • Rochester, New York, Estados Unidos
      • Syracuse, New York, Estados Unidos
      • Valhalla, New York, Estados Unidos
    • North Carolina
      • Chapel Hill, North Carolina, Estados Unidos
      • Durham, North Carolina, Estados Unidos
    • Ohio
      • Akron, Ohio, Estados Unidos
      • Cincinnati, Ohio, Estados Unidos
      • Cleveland, Ohio, Estados Unidos
      • Columbus, Ohio, Estados Unidos
      • Dayton, Ohio, Estados Unidos
      • Toledo, Ohio, Estados Unidos
    • Oklahoma
      • Oklahoma City, Oklahoma, Estados Unidos
    • Oregon
      • Portland, Oregon, Estados Unidos
    • Pennsylvania
      • Hershey, Pennsylvania, Estados Unidos
      • Philadelphia, Pennsylvania, Estados Unidos
      • Pittsburgh, Pennsylvania, Estados Unidos
    • South Carolina
      • Charelston, South Carolina, Estados Unidos
    • South Dakota
      • Sioux Falls, South Dakota, Estados Unidos
    • Tennessee
      • Knoxville, Tennessee, Estados Unidos
      • Memphis, Tennessee, Estados Unidos
      • Nashville, Tennessee, Estados Unidos
    • Texas
      • Austin, Texas, Estados Unidos
      • Dallas, Texas, Estados Unidos
      • Fort Worth, Texas, Estados Unidos
      • Houston, Texas, Estados Unidos
      • San Antonio, Texas, Estados Unidos
      • Tyler, 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
      • Spokane, Washington, Estados Unidos
    • West Virginia
      • Morgantown, West Virginia, Estados Unidos
    • Wisconsin
      • Madison, Wisconsin, Estados Unidos
      • Milwaukee, Wisconsin, Estados Unidos
  • França
      • Bordeaux, França
      • Paris, França
      • Pierre Benite, França
      • Rhone, França
      • Roscoff, França
    • Bas Rhin
      • Strasbourg, Bas Rhin, França
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, França
    • Haute Garonne
      • Toulouse, Haute Garonne, França
    • Herault
      • Montpellier, Herault, França
    • Nord
      • Lille, Nord, França
    • Rhone
      • Bron Cedex, Rhone, França
  • Holanda
      • Amsterdam, Holanda
      • Den Haag, Holanda
      • Nijmegen, Holanda
      • Rotterdam, Holanda
  • Irlanda
      • Dublin, Irlanda
  • Itália
      • Ancona, Itália
      • Firenze, Itália
      • Genova, Itália
      • Milano, Itália
      • Roma, Itália
      • Verona, Itália
  • Reino Unido
      • Belfast, Reino Unido
      • Birmingham, Reino Unido
      • Bristol, Reino Unido
      • Leeds, Reino Unido
      • London, Reino Unido
      • Newcastle, Reino Unido
      • Nottingham, Reino Unido
      • Southampton, Reino Unido
    • Devon
      • Exeter, Devon, Reino Unido
  • República Checa
      • Brno, República Checa
      • Plzeň - Bory, República Checa
      • Praha 5, República Checa
  • Suécia
      • Goteborg, Suécia
      • Stockholm, Suécia
  • Áustria
      • Innsbruck, Áustria
      • Wels, Áustria

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

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

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

Inclusion Criteria:

  • Signed informed consent form (ICF), and where appropriate, signed assent form.
  • Participants entering the Part A Treatment Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104 and elect to enroll in Part A treatment cohort.
  • Participants entering the Part B Treatment Cohort: Completed 56 days of study drug treatment in Cohort 4 of Study 102 and elect to enroll in Part B treatment cohort.
  • Participants entering the Part A Observational Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104, but do not elect to enroll in the Part A Treatment Cohort or do not qualify to enroll in Part A treatment cohort.
  • Willing to remain on a stable CF medication regimen through the end of study (Part A and Part B Treatment Cohorts only).

Exclusion Criteria:

  • 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 participant (e.g., cirrhosis with portal hypertension).
  • Pregnant and nursing females. Females of childbearing potential must have a negative pregnancy test at the Day 1 Visit.
  • History of drug intolerance in the prior study that would pose an additional risk to the participant in the opinion of investigator or Vertex.
  • History of poor compliance with study drug and/or procedures in the previous study as deemed by the investigator.
  • Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor, or studies requiring blood collections with or without administration of study drug)

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: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Triplo

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h
Participants who received lumacaftor (LUM, VX-809) 600 milligram (mg) plus ivacaftor (IVA, VX-770) 250 mg fixed-dose combination (FDC) tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening, in the previous study VX12-809-103 or VX12-809-104, and will receive the same treatment in this study VX12-809-105 up to Week 96.
Medicamento: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Medicamento: Ivacaftor
Film-coated tablet, oral use
Experimental: Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h
Participants who received placebo matched to LUM and IVA tablet in the previous study VX12-809-103 or VX12-809-104, and will receive LUM 600 mg plus IVA 250 mg FDC tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening in this study VX12-809-105 up to Week 96.
Medicamento: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Medicamento: Ivacaftor
Film-coated tablet, oral use
Experimental: Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h
Participants who received LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in the previous study VX12-809-103 or VX12-809-104, and will receive the same treatment in this study VX12-809-105 up to Week 96.
Medicamento: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Experimental: Arm 4 Part A: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h
Participants who received placebo matched to LUM and IVA tablet in the previous study VX12-809-103 or VX12-809-104, and will receive LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in this study VX12-809-105 up to Week 96.
Medicamento: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Sem intervenção: Arm 5 Part A: Observational Cohort
Participants who received either LUM 600 mg plus IVA 250 mg FDC tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening OR LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening OR placebo matched to LUM and IVA in the morning and evening, in the previous study VX12-809-103 or VX12-809-104, and will be observed (will not receive study drug) in this study VX12-809-105 for up to 2 years.
Experimental: Arm 6 Part B: LUM 400 mg q12h/ IVA 250 mg q12h
Participants who received LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in Cohort 4 of the previous study VX09-809-102, and will receive the same treatment in this study VX12-809-105 up to Week 96.
Medicamento: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Experimental: Arm 7 Part B: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h
Participants who received placebo matched to LUM and IVA tablet in Cohort 4 of the previous study VX09-809-102, and will receive LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in this study VX12-809-105 up to Week 96.
Medicamento: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Prazo: Day 1 up to Week 105 (Study 105)
AE: as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug was considered treatment-emergent.
Day 1 up to Week 105 (Study 105)
Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs
Prazo: Day 1 up to Week 105 (Study 105)
AE: as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug was considered treatment-emergent.
Day 1 up to Week 105 (Study 105)

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Part A Treatment Cohort: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) At Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 103/104/105); Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
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, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4.
Baseline (Study 103/104/105); Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
Part B Treatment Cohort: Absolute Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
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, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7.
Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
Part A Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
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, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4.
Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
Part B Treatment Cohort: Relative Change From Baseline in Percent Predicted FEV1 at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
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, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7.
Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
Part A Treatment Cohort: Absolute Change From Baseline in Body Mass Index (BMI) at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
BMI = (Weight in kilogram [kg]) divided by (Stature in meters [m]) ^2. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4.
Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
Part B Treatment Cohort: Absolute Change From Baseline in BMI at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7.
Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60 , 72 (Study 105)
Part A Treatment Cohort: Number of Pulmonary Exacerbations Events Per Patient-Year
Prazo: Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period)
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. The number of events per patient year were reported, where patient years = total number of days on study/336. Analysis includes all events in the Cumulative Study Period for Arm 1 and 3, and all events in the Current Study period (Study 105) for Arm 2 and 4. As per planned analysis, endpoint evaluation included subjects from the parent study VX12-809-103 and VX12-809-104 as well for cumulative study period.
Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period)
Part A Treatment Cohort: Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score at Day 15, Week 8, 16, 24, 48 and 72
Prazo: Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 48, 72 (Study 105)
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 (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4.
Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 48, 72 (Study 105)
Part B Treatment Cohort: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 15, Week 8, 16, 24, 48 and 72
Prazo: Baseline (Study 102 Study), Day 15, Week 8, 16, 24, 48, 72 (Study 105)
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 (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7.
Baseline (Study 102 Study), Day 15, Week 8, 16, 24, 48, 72 (Study 105)
Part A Treatment Cohort: Absolute Change From Baseline in BMI Z-score at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
z-score is a statistical measure to evaluate how a single data point compares to a standard. It describes whether a mean was above or below the standard and how unusual the measurement is with range from -infinity to +infinity; 0: same mean, >0: a greater mean, and <0: a lesser mean than the standard. BMI-for-age z-score was calculated by using centers for disease control and prevention (CDC) growth charts for the pediatric population. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4.
Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
Part A Treatment Cohort: Absolute Change From Baseline in Body Weight at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4.
Baseline (Study 103/104/105), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
Part B Treatment Cohort: Absolute Change From Baseline in Body Weight at Day 15, Week 8, 16, 24, 36, 48, 60 and 72
Prazo: Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7.
Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
Part A Treatment Cohort: Time-to-First Pulmonary Exacerbation
Prazo: Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period)
Time-to-first pulmonary exacerbation was analyzed using the Kaplan-Meier estimates. 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. Analysis was performed for the Cumulative Study Period for Arm 1 and 3, and for the current study period (Study 105) for Arm 2 and 4. As per planned analysis, endpoint evaluation included subjects from the parent study VX12-809-103 and VX12-809-104 as well for cumulative study period.
Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period)
Part A Treatment Cohort: Percentage of Participants With at Least 1 Pulmonary Exacerbation
Prazo: Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period)
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. Analysis was performed for the Cumulative Study Period for Arm 1 and 3, and for the current study period (Study 105) for Arm 2 and 4. As per planned analysis, endpoint evaluation included subjects from the parent study VX12-809-103 and VX12-809-104 as well for cumulative study period.
Baseline (Study 103/104) up to Week 100 (Study 105) for Arm 1 and 3 (Cumulative study period); Baseline (Study 105) up to Week 100 (Study 105) for Arm 2 and 4 (current study period)
Part A Treatment Cohort: Percentage of Participants With Response Based on Relative Change in Percent Predicted FEV1 From Baseline
Prazo: Baseline (Study 103/104/105); Day 15, Week 8, 16, 24, 36, 48, 60, 72, 84, 96 (Study 105)
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, race, and height). Percentage of participants with at least 5% and 10% relative change in percent predicted FEV1 from baseline were reported. Analysis was performed using baseline of the previous study VX12-809-103 (NCT01807923) and Study VX12-809-104 (NCT01807949) for Arm 1 and 3. Analysis was performed using baseline of the current study VX12-809-105 (NCT01931839) for Arm 2 and 4. As per planned analysis, endpoint evaluation included subjects from the parent study VX12-809-103 and VX12-809-104 as well for cumulative study period.
Baseline (Study 103/104/105); Day 15, Week 8, 16, 24, 36, 48, 60, 72, 84, 96 (Study 105)
Part B Treatment Cohort: Percentage of Participants With Response Based on Relative Change in Percent Predicted FEV1 From Baseline
Prazo: Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
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, race, and height). Percentage of participants with at least 5% relative change in percent predicted FEV1 from Baseline were reported. Analysis was performed using baseline of Cohort 4 of previous study VX09-809-102 (NCT01225211) for Arm 6 and 7. As per planned analysis, endpoint evaluation included subjects from the parent study VX09-809-102 as well.
Baseline (Study 102), Day 15, Week 8, 16, 24, 36, 48, 60, 72 (Study 105)
Part A Observation Cohort: Number of Participants With Serious Adverse Events (SAEs)
Prazo: up to 2 years
AE: as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.
up to 2 years

Colaboradores e Investigadores

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

Patrocinador

Vertex Pharmaceuticals Incorporated

Publicações e links úteis

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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

1 de outubro de 2013

Conclusão Primária (Real)

1 de abril de 2016

Conclusão do estudo (Real)

1 de abril de 2016

Datas de inscrição no estudo

Enviado pela primeira vez

26 de agosto de 2013

Enviado pela primeira vez que atendeu aos critérios de CQ

26 de agosto de 2013

Primeira postagem (Estimativa)

29 de agosto de 2013

Atualizações de registro de estudo

Última Atualização Postada (Real)

12 de maio de 2017

Última atualização enviada que atendeu aos critérios de controle de qualidade

3 de abril de 2017

Última verificação

1 de abril de 2017

Mais Informações

Termos relacionados a este estudo

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • VX12-809-105

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

produto fabricado e exportado dos EUA

Não

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