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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 kwietnia 2017 zaktualizowane przez: 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.

Przegląd badań

Status

Zakończony

Warunki

Interwencja / Leczenie

Szczegółowy opis

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).

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

1164

Faza

  • Faza 3

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

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

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

12 lat i starsze (Dziecko, Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Wszystko

Opis

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)

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Randomizowane
  • Model interwencyjny: Przydział równoległy
  • Maskowanie: Potroić

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: 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.
Lek: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Lek: Ivacaftor
Film-coated tablet, oral use
Eksperymentalny: 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.
Lek: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Lek: Ivacaftor
Film-coated tablet, oral use
Eksperymentalny: 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.
Lek: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Eksperymentalny: 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.
Lek: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Brak interwencji: 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.
Eksperymentalny: 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.
Lek: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Eksperymentalny: 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.
Lek: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Ramy czasowe: 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
Ramy czasowe: 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)

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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)
Ramy czasowe: 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

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Vertex Pharmaceuticals Incorporated

Publikacje i pomocne linki

Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.

Publikacje ogólne

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów

1 października 2013

Zakończenie podstawowe (Rzeczywisty)

1 kwietnia 2016

Ukończenie studiów (Rzeczywisty)

1 kwietnia 2016

Daty rejestracji na studia

Pierwszy przesłany

26 sierpnia 2013

Pierwszy przesłany, który spełnia kryteria kontroli jakości

26 sierpnia 2013

Pierwszy wysłany (Oszacować)

29 sierpnia 2013

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

12 maja 2017

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

3 kwietnia 2017

Ostatnia weryfikacja

1 kwietnia 2017

Więcej informacji

Terminy związane z tym badaniem

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • VX12-809-105

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Nie

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

produkt wyprodukowany i wyeksportowany z USA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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