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A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF Genotypes

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Completed study drug treatment in parent study (VX19-445-116, NCT04353817), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study

Key Exclusion Criteria:

  • History of study drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • Telethon Kids Institute
  • Queensland Children's Hospital
  • The Children's Hospital at Westmead
  • McGill University Health Centre, Glen Site, Montreal Children's Hospital
  • The Hospital for Sick Children
  • British Columbia Children's Hospital
  • Juliane Marie Center, Rigshospitalet
  • Groupe Hospitaler Pellegrin, CHU De Bordeaux
  • CHU Lyon - Hopital Femme Mere-Enfant
  • Hopital Necker, Enfants Malades
  • Hopital Robert Debre
  • Centre de Perharidy
  • Charite Paediatric Pulmonology Department
  • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
  • Johann Wolfgang Goethe University
  • Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
  • Medizinische Hochschule Hannover
  • Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
  • Universitaetsklinkum Koeln, CF-Studienzentrum
  • Hadassah University Hospital Mount Scopus
  • Schneider Children's Medical Center of Israel
  • Universitair Medisch Centrum Groningen
  • Erasmus Medical Center / Sophia Children's Hospital
  • Hospital Universitari Vall d Hebron
  • Hospital Virgen de la Arrixaca
  • Inselspital - Universitaetsspital Bern
  • Kinderspital Zuerich
  • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
  • Children's Hospital of Wales
  • Royal Hospital for Sick Children
  • Alder Hey Children's NHS Foundation Trust
  • Great Ormond Street Hospital for Sick Children
  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Outcomes

Primary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Absolute Change in Sweat Chloride (SwCl)
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)

Full Information

First Posted
September 3, 2020
Last Updated
April 21, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04545515
Brief Title
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF Genotypes
Official Title
A Phase 3b Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
January 11, 2021 (Actual)
Primary Completion Date
March 24, 2023 (Actual)
Study Completion Date
March 24, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes

5. Study Description

Brief Summary
This study will evaluate the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in subjects with CF who are 6 years of age and older with F/MF genotypes.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
120 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed dose combination (FDC) tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablet for oral administration.
Primary Outcome Measure Information:
Title
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Week 100
Secondary Outcome Measure Information:
Title
Absolute Change in Sweat Chloride (SwCl)
Time Frame
From Baseline up to Week 96
Title
Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)
Time Frame
From Baseline up to Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Completed study drug treatment in parent study (VX19-445-116, NCT04353817), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study Key Exclusion Criteria: History of study drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply
Facility Information:
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
The Children's Hospital at Westmead
City
Westmead
Country
Australia
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montreal
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia Children's Hospital
City
Vancouver
Country
Canada
Facility Name
Juliane Marie Center, Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Groupe Hospitaler Pellegrin, CHU De Bordeaux
City
Bordeaux cedex
Country
France
Facility Name
CHU Lyon - Hopital Femme Mere-Enfant
City
Bron Cedex
Country
France
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Facility Name
Hopital Robert Debre
City
Paris
Country
France
Facility Name
Centre de Perharidy
City
Roscoff cedex
Country
France
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
City
Essen
Country
Germany
Facility Name
Johann Wolfgang Goethe University
City
Frankfurt
Country
Germany
Facility Name
Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
City
Gießen
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
City
Heidelberg
Country
Germany
Facility Name
Universitaetsklinkum Koeln, CF-Studienzentrum
City
Koeln
Country
Germany
Facility Name
Hadassah University Hospital Mount Scopus
City
Jerusalem
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel
City
Petach Tikvah
Country
Israel
Facility Name
Universitair Medisch Centrum Groningen
City
Groningen
Country
Netherlands
Facility Name
Erasmus Medical Center / Sophia Children's Hospital
City
Rotterdam
Country
Netherlands
Facility Name
Hospital Universitari Vall d Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital Virgen de la Arrixaca
City
Murcia
Country
Spain
Facility Name
Inselspital - Universitaetsspital Bern
City
Bern
Country
Switzerland
Facility Name
Kinderspital Zuerich
City
Zurich
Country
Switzerland
Facility Name
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
City
Bristol
Country
United Kingdom
Facility Name
Children's Hospital of Wales
City
Cardiff
Country
United Kingdom
Facility Name
Royal Hospital for Sick Children
City
Edinburgh
Country
United Kingdom
Facility Name
Alder Hey Children's NHS Foundation Trust
City
Liverpool
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Sick Children
City
London
Country
United Kingdom
Facility Name
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
Citations:
PubMed Identifier
33331662
Citation
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived

Learn more about this trial

A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF Genotypes

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