A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

February 4, 2020 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

67

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • New Lambton Heights, Australia
        • Hunter Medical Research Institute (HMRI)
      • Perth, Australia
        • Princess Margaret Hospital for Children
      • South Brisbane, Australia
        • Lady Cilento Children's Hospital
      • Westmead, Australia
        • The Children's Hospital at Westmead
  • Belgium
      • Brussels, Belgium
        • Universitair Ziekenhuis Brussel - Campus Jette
      • Leuven, Belgium
        • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • Denmark
      • Copenhagen, Denmark
        • University of Copenhagen Rigshospitalet
  • France
      • Bordeaux Cedex, France
        • Groupe Hospitalier Pellegrin - Hôpital des Enfants
      • Paris, France
        • Hôpital Necker - Enfants Malades
  • Germany
      • Essen, Germany
        • Universitaetsklinikum Essen
      • Frankfurt, Germany
        • Klinikum der Johann Wolfgang Goethe-Universitaet
      • Giessen, Germany
        • Universitaetsklinikum Giessen Und Marburg Gmbh Standort Giessen
      • Hannover, Germany
        • Medizinische Hochschule Hannover
      • Heidelberg, Germany
        • Universitaetsklinikum Heidelberg
      • Jena, Germany
        • Universitaetsklinikum Jena
      • Koeln, Germany
        • Universitaetsklinikum Koeln
      • Tuebingen, Germany
        • Universitaetsklinikum Tuebingen
  • Ireland
      • Dublin, Ireland
        • Our Lady's Children's Hospital
      • Limerick, Ireland
        • University Hospital Limerick
  • Poland
      • Dziekanow Lesny, Poland
        • Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
  • Switzerland
      • Bern, Switzerland
        • Inselspital - Universitaetsspital Bern
      • Zuerich, Switzerland
        • Kinderspital Zuerich
  • United Kingdom
      • Edinburgh, United Kingdom
        • Royal Hospital for Sick Children
      • Leeds, United Kingdom
        • Leeds General Infirmary
      • London, United Kingdom
        • Royal Brompton Hospital
      • Nottingham, United Kingdom
        • Nottingham University Hospital City Campus
      • Southampton, United Kingdom
        • Southampton General Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 11 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
  • Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
  • Participants with a screening LCI2.5 result ≥7.5.
  • Participants who are able to swallow tablets.

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension.
  • Colonization with organisms associated with a more rapid decline in pulmonary status.
  • Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Placebo
Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
Drug: Placebo
Placebo matched to TEZ/IVA FDC
Drug: Placebo
Placebo matched to IVA
Experimental: TEZ/IVA
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
Drug: Placebo
Placebo matched to TEZ/IVA FDC
Drug: Placebo
Placebo matched to IVA
Drug: TEZ/IVA
Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.
Other Names:
  • VX-661/VX-770
  • tezacaftor/ivacaftor fixed dose combination
Drug: IVA
Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
Other Names:
  • VX-770
  • ivacaftor
Experimental: Ivacaftor
Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
Drug: Placebo
Placebo matched to TEZ/IVA FDC
Drug: Placebo
Placebo matched to IVA
Drug: IVA
Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
Other Names:
  • VX-770
  • ivacaftor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
Time Frame: From baseline through Week 8
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
From baseline through Week 8

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change in Sweat Chloride At Week 8
Time Frame: From baseline at Week 8
Sweat samples were collected using an approved collection device.
From baseline at Week 8
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
Time Frame: From baseline through Week 8
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 baseline through Week 8
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
Time Frame: From first dose of study drug up to safety follow-up visit (up to Week 12)
From first dose of study drug up to safety follow-up visit (up to Week 12)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 17, 2018

Primary Completion (Actual)

December 21, 2018

Study Completion (Actual)

December 21, 2018

Study Registration Dates

First Submitted

June 5, 2018

First Submitted That Met QC Criteria

June 5, 2018

First Posted (Actual)

June 15, 2018

Study Record Updates

Last Update Posted (Actual)

February 11, 2020

Last Update Submitted That Met QC Criteria

February 4, 2020

Last Verified

February 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX16-661-115
  • 2016-004479-35 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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