Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

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

27. april 2021 opdateret af: Vertex Pharmaceuticals Incorporated

A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

246

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

6 år og ældre (Barn, Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

Subjects entering the Treatment Cohort must meet both of the following criteria:

  • Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
  • Willing to remain on a stable CF medication through the Safety Follow-up Visit.

Subjects entering the Observational Cohort must meet 1 of the following criteria:

  • Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
  • Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.

Exclusion Criteria (Treatment Cohort Only):

  • History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension).
  • Pregnant and nursing females.
  • Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
  • History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
  • History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
  • Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Treatment Period 1: LUM/IVA to LUM/IVA
Medicin: LUM/IVA

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

Andre navne:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Medicin: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andre navne:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Eksperimentel: Treatment Period 1: Placebo (PBO) to LUM/IVA
Medicin: LUM/IVA

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

Andre navne:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Medicin: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andre navne:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Ingen indgriben: Treatment Period 1: Observational Cohort
Eksperimentel: Treatment Period 2: LUM/IVA
Medicin: LUM/IVA

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).

LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

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

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Tidsramme
Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsramme: Day 1 up to Week 100
Day 1 up to Week 100

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Absolute Change in Lung Clearance Index (LCI) 2.5
Tidsramme: From Parent Study Baseline at Week 96
LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
From Parent Study Baseline at Week 96
Absolute Change in Sweat Chloride
Tidsramme: From Parent Study Baseline at Week 96
Sweat samples were collected using an approved collection device.
From Parent Study Baseline at Week 96
Absolute Change in Body Mass Index (BMI)
Tidsramme: From Parent Study Baseline at Week 96
BMI was defined as weight in kilograms divided by height in square meter (m^2).
From Parent Study Baseline at Week 96
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Tidsramme: From Parent Study Baseline at Week 96
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
From Parent Study Baseline at Week 96
Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
Tidsramme: Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
Tidsramme: From Parent Study Baseline at Week 96
LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.
From Parent Study Baseline at Week 96
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Tidsramme: From Parent Study Baseline at Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Parent Study Baseline at Week 96
Relative Change in ppFEV1
Tidsramme: From Parent Study Baseline at Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Parent Study Baseline at Week 96
Absolute Change in BMI-for-age Z-score
Tidsramme: From Parent Study Baseline at Week 96
BMI was defined as weight in kilograms divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Parent Study Baseline at Week 96
Absolute Change in Weight
Tidsramme: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
Tidsramme: From Parent Study Baseline at Week 96
z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Parent Study Baseline at Week 96
Absolute Change in Height
Tidsramme: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
Tidsramme: From Parent Study Baseline at Week 96
z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
From Parent Study Baseline at Week 96
Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score
Tidsramme: From Parent Study Baseline at Week 96
The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction.
From Parent Study Baseline at Week 96
Time-to-first Pulmonary Exacerbation
Tidsramme: From Parent Study Baseline through Week 96
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Parent Study Baseline through Week 96
Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event
Tidsramme: From Parent Study Baseline through Week 96
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Parent Study Baseline through Week 96
Number of Pulmonary Exacerbation Events Per Patient-year
Tidsramme: From Parent Study Baseline through Week 96
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
From Parent Study Baseline through Week 96
Rate of Change in LCI 2.5
Tidsramme: Day 15 after first dose of LUM/IVA through Week 96
Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in LCI 5.0
Tidsramme: Day 15 after first dose of LUM/IVA through Week 96
Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Day 15 after first dose of LUM/IVA through Week 96
Rate of Change in ppFEV1
Tidsramme: Day 15 after first dose of LUM/IVA through Week 96
Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
Day 15 after first dose of LUM/IVA through Week 96
Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsramme: Day 1 up to Week 168
Day 1 up to Week 168

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Vertex Pharmaceuticals Incorporated

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

1. august 2015

Primær færdiggørelse (Faktiske)

1. august 2018

Studieafslutning (Faktiske)

1. april 2020

Datoer for studieregistrering

Først indsendt

1. september 2015

Først indsendt, der opfyldte QC-kriterier

4. september 2015

Først opslået (Skøn)

9. september 2015

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

24. maj 2021

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

27. april 2021

Sidst verificeret

1. februar 2021

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • VX15-809-110

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med LUM/IVA

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Mozambique

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

3
Abonner