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

27 april 2021 bijgewerkt door: 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.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

246

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

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

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

6 jaar en ouder (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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).

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Treatment Period 1: LUM/IVA to LUM/IVA
Geneesmiddel: 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).

Andere namen:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Geneesmiddel: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andere namen:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Experimenteel: Treatment Period 1: Placebo (PBO) to LUM/IVA
Geneesmiddel: 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).

Andere namen:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Geneesmiddel: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Andere namen:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Geen tussenkomst: Treatment Period 1: Observational Cohort
Experimenteel: Treatment Period 2: LUM/IVA
Geneesmiddel: 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).

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

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Tijdsspanne
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)
Tijdsspanne: Day 1 up to Week 100
Day 1 up to Week 100

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Absolute Change in Lung Clearance Index (LCI) 2.5
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
Tijdsspanne: 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)
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
Tijdsspanne: 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
Tijdsspanne: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
Tijdsspanne: Day 1 up to Week 168
Day 1 up to Week 168

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Vertex Pharmaceuticals Incorporated

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

1 augustus 2015

Primaire voltooiing (Werkelijk)

1 augustus 2018

Studie voltooiing (Werkelijk)

1 april 2020

Studieregistratiedata

Eerst ingediend

1 september 2015

Eerst ingediend dat voldeed aan de QC-criteria

4 september 2015

Eerst geplaatst (Schatting)

9 september 2015

Updates van studierecords

Laatste update geplaatst (Werkelijk)

24 mei 2021

Laatste update ingediend die voldeed aan QC-criteria

27 april 2021

Laatst geverifieerd

1 februari 2021

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • VX15-809-110

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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