search
Back to results

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

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

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

Key Inclusion Criteria:

  • Completed study drug treatment in parent study (VX18-445-104); 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 parent study

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • Banner University of Arizona Medical Center
  • Miller Children's Hospital / Long Beach Memorial
  • Stanford University
  • University of California Davis Medical Center
  • University of California San Francisco, Lung Transplant Program
  • National Jewish Health
  • University of Miami Miller School of Medicine
  • Central Florida Pulmonary Group, PA
  • Indiana University
  • University of Kansas Medical Center
  • University of Kentucky
  • Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
  • Boston Children's Hospital
  • Michigan Medicine
  • University of Minnesota
  • Washington University School of Medicine / St. Louis Children's Hospital
  • Mount Sinai Beth Israel
  • Columbia University Medical Center
  • University of North Carolina Hospitals
  • UC Health Holmes
  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
  • Nationwide Children's Hospital
  • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
  • University of Oklahoma Health Sciences Center
  • Oregon Health & Science University
  • Children's Hospital of Philadelphia
  • Children's Hospital of Pittsburgh of UPMC
  • Dell Children's Medical Group
  • The University of Texas Southwestern Medical Center
  • Texas Children's Hospital
  • University of Utah / Primary Children's Medical Center
  • University of Washington Medical Center
  • University of Wisconsin Hospital and Clinics
  • The Prince Charles Hospital
  • Alfred Hospital
  • Telethon Kids Institute
  • The Royal Children's Hospital
  • Mater Adult Hospital
  • Queensland Children's Hospital
  • Westmead Hospital
  • Cliniques Universitaires de Bruxelles Hopital Erasme
  • Universitair Ziekenhuis Brussel - Campus Jette
  • Universitair Ziekenhuis Gent
  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • McGill University Health Center
  • St. Paul's Hospital
  • Juliane Marie Center, Rigshospitalet
  • Centre Hospitalier Lyon Sud
  • Groupe Hospitaler Pellegrin, CHU De Bordeaux
  • CHRU de Lille - Hopital Albert Calmette
  • CHU Marseille - Hopital Nord
  • CHU de Montpellier - Hopital Arnaud de Villeneuve
  • Hopital Necker, Enfants Malades
  • Hopital Cochin
  • Hopital Pontchaillou CHU de Rennes
  • Charite Paediatric Pulmonology Department
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
  • Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
  • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
  • Pneumologisches Studienzentrum Muenchen-West
  • University Hospital Wuerzburg
  • Beaumont Hospital
  • Children's Health Ireland at Crumlin
  • St. Vincent's University Hospital
  • University Hospital Limerick (Adults)
  • Azienda Ospedaliero Universitaria Ospedale Riuniti
  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Ospedale Pediatrico Bambino Gesu
  • Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
  • Academic Medical Center
  • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
  • Hospital Virgen de la Arrixaca
  • Hospital Universitario Virgen del Rocio
  • Hospital Universitario y Politecnico La Fe
  • Papworth Hospital NHS Foundation Trust, Papworth Everard
  • Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
  • Clinical Research Facility, Queen Elizabeth University Hospital
  • St. James University Hospital
  • Liverpool Heart and Chest Hospital
  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
  • Wythenshawe Hospital
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Triple Combination Arm

Arm Description

Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening.

Outcomes

Primary Outcome Measures

Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs)

Secondary Outcome Measures

Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
Absolute change in sweat chloride (SwCl)
Absolute change in body mass index (BMI)
Absolute change in BMI z-score
Absolute change in body weight
Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score

Full Information

First Posted
August 14, 2019
Last Updated
January 20, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
search

1. Study Identification

Unique Protocol Identification Number
NCT04058366
Brief Title
Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
Official Title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Completed
Study Start Date
December 5, 2019 (Actual)
Primary Completion Date
December 16, 2022 (Actual)
Study Completion Date
December 16, 2022 (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

5. Study Description

Brief Summary
This study will evaluate the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF 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
251 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Triple Combination Arm
Arm Type
Experimental
Arm Description
Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet 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
150-mg tablet for oral administration.
Primary Outcome Measure Information:
Title
Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs)
Time Frame
From Baseline up to Week 100
Secondary Outcome Measure Information:
Title
Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
Time Frame
From Baseline up to Week 96
Title
Absolute change in sweat chloride (SwCl)
Time Frame
From Baseline up to Week 96
Title
Absolute change in body mass index (BMI)
Time Frame
From Baseline up to Week 96
Title
Absolute change in BMI z-score
Time Frame
From Baseline up to Week 96
Title
Absolute change in body weight
Time Frame
From Baseline up to Week 96
Title
Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Time Frame
From Baseline up to Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Completed study drug treatment in parent study (VX18-445-104); 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 parent study Other protocol defined Inclusion/Exclusion criteria may apply
Facility Information:
Facility Name
Banner University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Miller Children's Hospital / Long Beach Memorial
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California San Francisco, Lung Transplant Program
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Central Florida Pulmonary Group, PA
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Michigan Medicine
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-5212
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Washington University School of Medicine / St. Louis Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Mount Sinai Beth Israel
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
University of North Carolina Hospitals
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
UC Health Holmes
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45220
Country
United States
Facility Name
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Dell Children's Medical Group
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
The University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah / Primary Children's Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Alfred Hospital
City
Melbourne
Country
Australia
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville
Country
Australia
Facility Name
Mater Adult Hospital
City
South Brisbane
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
Country
Australia
Facility Name
Cliniques Universitaires de Bruxelles Hopital Erasme
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Brussel - Campus Jette
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
McGill University Health Center
City
Montréal
Country
Canada
Facility Name
St. Paul's Hospital
City
Vancouver
Country
Canada
Facility Name
Juliane Marie Center, Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Centre Hospitalier Lyon Sud
City
Benite Cedex
Country
France
Facility Name
Groupe Hospitaler Pellegrin, CHU De Bordeaux
City
Bordeaux cedex
Country
France
Facility Name
CHRU de Lille - Hopital Albert Calmette
City
Lille
Country
France
Facility Name
CHU Marseille - Hopital Nord
City
Marseille
Country
France
Facility Name
CHU de Montpellier - Hopital Arnaud de Villeneuve
City
Montpellier Cedex 5
Country
France
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Facility Name
Hopital Cochin
City
Paris
Country
France
Facility Name
Hopital Pontchaillou CHU de Rennes
City
Rennes Cedex
Country
France
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
City
Essen
Country
Germany
Facility Name
Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
City
Giessen
Country
Germany
Facility Name
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
City
Halle
Country
Germany
Facility Name
Pneumologisches Studienzentrum Muenchen-West
City
Muenchen
Country
Germany
Facility Name
University Hospital Wuerzburg
City
Würzburg
Country
Germany
Facility Name
Beaumont Hospital
City
Dublin
Country
Ireland
Facility Name
Children's Health Ireland at Crumlin
City
Dublin
Country
Ireland
Facility Name
St. Vincent's University Hospital
City
Dublin
Country
Ireland
Facility Name
University Hospital Limerick (Adults)
City
Limerick
Country
Ireland
Facility Name
Azienda Ospedaliero Universitaria Ospedale Riuniti
City
Ancona
Country
Italy
Facility Name
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
City
Genova
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesu
City
Rome
Country
Italy
Facility Name
Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
City
Verona
Country
Italy
Facility Name
Academic Medical Center
City
Amsterdam
Country
Netherlands
Facility Name
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
City
Heidelberglaan
Country
Netherlands
Facility Name
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
City
Barcelona
Country
Spain
Facility Name
Hospital Virgen de la Arrixaca
City
Murcia
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
Country
Spain
Facility Name
Hospital Universitario y Politecnico La Fe
City
Valencia
Country
Spain
Facility Name
Papworth Hospital NHS Foundation Trust, Papworth Everard
City
Cambridge
Country
United Kingdom
Facility Name
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
City
Exeter
Country
United Kingdom
Facility Name
Clinical Research Facility, Queen Elizabeth University Hospital
City
Glasgow
Country
United Kingdom
Facility Name
St. James University Hospital
City
Leeds
Country
United Kingdom
Facility Name
Liverpool Heart and Chest Hospital
City
Liverpool
Country
United Kingdom
Facility Name
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
Wythenshawe Hospital
City
Manchester
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

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

We'll reach out to this number within 24 hrs