Denne siden ble automatisk oversatt og nøyaktigheten av oversettelsen er ikke garantert. Vennligst referer til engelsk versjon for en kildetekst.

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

27. april 2021 oppdatert av: 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.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

246

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

6 år og eldre (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Treatment Period 1: LUM/IVA to LUM/IVA
Legemiddel: 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 navn:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Legemiddel: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andre navn:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Eksperimentell: Treatment Period 1: Placebo (PBO) to LUM/IVA
Legemiddel: 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 navn:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Legemiddel: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andre navn:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Ingen inngripen: Treatment Period 1: Observational Cohort
Eksperimentell: Treatment Period 2: LUM/IVA
Legemiddel: 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 navn:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Legemiddel: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andre navn:
  • lumacaftor/ivacaftor
  • VX-809/VX-770

Hva måler studien?

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
Tiltaksbeskrivelse
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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Vertex Pharmaceuticals Incorporated

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

1. august 2015

Primær fullføring (Faktiske)

1. august 2018

Studiet fullført (Faktiske)

1. april 2020

Datoer for studieregistrering

Først innsendt

1. september 2015

Først innsendt som oppfylte QC-kriteriene

4. september 2015

Først lagt ut (Anslag)

9. september 2015

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

24. mai 2021

Siste oppdatering sendt inn som oppfylte QC-kriteriene

27. april 2021

Sist bekreftet

1. februar 2021

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • VX15-809-110

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Kliniske studier på Cystisk fibrose

Kliniske studier på LUM/IVA

Søk i lignende forsøk

Sponsorer og samarbeidspartnere

Medisinsk tilstand

Legemiddelintervensjoner

CROs by country

CROs in Burkina Faso

Forhold

Sjeldne sykdommer

Legemiddelintervensjoner

Kosttilskudd

Sponsor / samarbeidspartnere

Steder

Abonnere