Cette page a été traduite automatiquement et l'exactitude de la traduction n'est pas garantie. Veuillez vous référer au version anglaise pour un texte source.

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

27 avril 2021 mis à jour par: 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.

Aperçu de l'étude

Statut

Complété

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Réel)

246

Phase

  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

6 ans et plus (Enfant, Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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).

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Non randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: Treatment Period 1: LUM/IVA to LUM/IVA
Médicament: 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).

Autres noms:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Médicament: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Autres noms:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Expérimental: Treatment Period 1: Placebo (PBO) to LUM/IVA
Médicament: 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).

Autres noms:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Médicament: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Autres noms:
  • lumacaftor/ivacaftor
  • VX-809/VX-770
Aucune intervention: Treatment Period 1: Observational Cohort
Expérimental: Treatment Period 2: LUM/IVA
Médicament: 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).

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

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Délai
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)
Délai: Day 1 up to Week 100
Day 1 up to Week 100

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Absolute Change in Lung Clearance Index (LCI) 2.5
Délai: 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
Délai: 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)
Délai: 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
Délai: 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)
Délai: Day 1 up to Week 100
Day 1 up to Week 100
Absolute Change in LCI 5.0
Délai: 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)
Délai: 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
Délai: 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
Délai: 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
Délai: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Weight-for-age Z-score
Délai: 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
Délai: From Parent Study Baseline at Week 96
From Parent Study Baseline at Week 96
Absolute Change in Height-for-age Z-score
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: Day 1 up to Week 168
Day 1 up to Week 168

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Vertex Pharmaceuticals Incorporated

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

1 août 2015

Achèvement primaire (Réel)

1 août 2018

Achèvement de l'étude (Réel)

1 avril 2020

Dates d'inscription aux études

Première soumission

1 septembre 2015

Première soumission répondant aux critères de contrôle qualité

4 septembre 2015

Première publication (Estimation)

9 septembre 2015

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

24 mai 2021

Dernière mise à jour soumise répondant aux critères de contrôle qualité

27 avril 2021

Dernière vérification

1 février 2021

Plus d'information

Termes liés à cette étude

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • VX15-809-110

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

Essais cliniques sur LUM/IVA

Rechercher des essais similaires

Sponsors et collaborateurs

Les conditions médicales

Interventions en matière de drogue

CROs by country

CROs in Indonesia

Conditions

Maladies rares

Interventions en matière de drogue

Compléments alimentaires

Commanditaire / collaborateurs

Emplacements

S'abonner