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

27 aprile 2021 aggiornato da: 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.

Panoramica dello studio

Stato

Completato

Condizioni

Intervento / Trattamento

Tipo di studio

Interventistico

Iscrizione (Effettivo)

246

Fase

  • Fase 3

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Luoghi di studio

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

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

6 anni e precedenti (Bambino, Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

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).

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Treatment Period 1: LUM/IVA to LUM/IVA
Droga: 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).

Altri nomi:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Droga: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Altri nomi:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Sperimentale: Treatment Period 1: Placebo (PBO) to LUM/IVA
Droga: 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).

Altri nomi:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Droga: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Altri nomi:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Nessun intervento: Treatment Period 1: Observational Cohort
Sperimentale: Treatment Period 2: LUM/IVA
Droga: 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).

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

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Lasso di tempo
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)
Lasso di tempo: Day 1 up to Week 100
Day 1 up to Week 100

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Absolute Change in Lung Clearance Index (LCI) 2.5
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
Lasso di tempo: 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
Lasso di tempo: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: Day 1 up to Week 168
Day 1 up to Week 168

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Vertex Pharmaceuticals Incorporated

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Pubblicazioni generali

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

1 agosto 2015

Completamento primario (Effettivo)

1 agosto 2018

Completamento dello studio (Effettivo)

1 aprile 2020

Date di iscrizione allo studio

Primo inviato

1 settembre 2015

Primo inviato che soddisfa i criteri di controllo qualità

4 settembre 2015

Primo Inserito (Stima)

9 settembre 2015

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

24 maggio 2021

Ultimo aggiornamento inviato che soddisfa i criteri QC

27 aprile 2021

Ultimo verificato

1 febbraio 2021

Maggiori informazioni

Termini relativi a questo studio

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • VX15-809-110

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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