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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. dubna 2017 aktualizováno: 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.

Přehled studie

Postavení

Dokončeno

Podmínky

Intervence / Léčba

Detailní popis

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 studie

Intervenční

Zápis (Aktuální)

1164

Fáze

  • Fáze 3

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

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

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

12 let a starší (Dítě, Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

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)

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Randomizované
  • Intervenční model: Paralelní přiřazení
  • Maskování: Trojnásobný

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: 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.
Lék: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Lék: Ivacaftor
Film-coated tablet, oral use
Experimentální: 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.
Lék: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Lék: Ivacaftor
Film-coated tablet, oral use
Experimentální: 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.
Lék: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Experimentální: 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.
Lék: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Žádný zásah: 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.
Experimentální: 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.
Lék: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use
Experimentální: 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.
Lék: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet, oral use

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Časové okno: 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
Časové okno: 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)

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Vertex Pharmaceuticals Incorporated

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Obecné publikace

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia

1. října 2013

Primární dokončení (Aktuální)

1. dubna 2016

Dokončení studie (Aktuální)

1. dubna 2016

Termíny zápisu do studia

První předloženo

26. srpna 2013

První předloženo, které splnilo kritéria kontroly kvality

26. srpna 2013

První zveřejněno (Odhad)

29. srpna 2013

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

12. května 2017

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

3. dubna 2017

Naposledy ověřeno

1. dubna 2017

Více informací

Termíny související s touto studií

Další relevantní podmínky MeSH

Další identifikační čísla studie

  • VX12-809-105

Informace o lécích a zařízeních, studijní dokumenty

Studuje lékový produkt regulovaný americkým FDA

Ne

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

produkt vyrobený a vyvážený z USA

Ne

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .

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Podmínky

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Sponzor / Spolupracovníci

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