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A Study Evaluating the Long-term Safety 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

Inclusion Criteria:

  • Currently participating in study VX17-659-105 (NCT03447262)

Exclusion Criteria:

  • History of drug intolerance in study VX17-659-105 that would pose an additional risk to the subject in the opinion of the investigator
  • Current participation in an investigational drug trial (other than study VX17-659-105)

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • University of Alabama at Birmingham
  • Stanford University
  • Children's Hospital Colorado
  • Hartford Hospital
  • Yale New Haven Hospital
  • University of Miami Miller School of Medicine
  • Nicklaus Children's Hospital
  • Arnold Palmer Hospital Pulmonary and Sleep Medicine
  • Johns Hopkins All Children's Hospital Outpatient Care Center
  • St. Luke's CF Center of Idaho
  • Cystic Fibrosis Institute
  • Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
  • Indiana University
  • The University of Iowa Hospitals and Clinics
  • University of Kentucky
  • Kosair Charities Pediatric Clinical Research Unit
  • Johns Hopkins Hospital
  • Boston Children's Hospital
  • UMass Memorial Medical Center
  • Michigan Medicine
  • University of Minnesota
  • University of Mississippi Medical Center
  • The Children's Mercy Hospital
  • Washington University School of Medicine / St. Louis Children's Hospital
  • Dartmouth Hitchcock Medical Center
  • Rutgers Robert Wood Johnson Medical School
  • Albany Medical College, Pulmonary and Critical Care Medicine
  • Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
  • Northwell Health- Long Island Jewish Medical Center
  • Columbia University Medical Center
  • SUNY Upstate Medical University
  • Clinical Research of Charlotte
  • Duke University Medical Center
  • Cincinnati Children's Hospital Medical Center
  • Santiago Reyes, M.D.
  • Oregon Health & Science University
  • Children's Hospital of Pittsburgh of UPMC
  • Sanford Children's Speciality Clinic
  • University of Tennessee Medical Center
  • Children's Foundation Research Center / Le Bonheur Children's Hospital
  • Vanderbilt University Medical Center
  • Cook Children's Medical Center
  • Texas Children's Hospital
  • University of Utah / Primary Children's Medical Center
  • Seattle Children's Hospital
  • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
  • Royal Adelaide Hospital
  • The Prince Charles Hospital
  • Alfred Hospital
  • Institute for Respiratory Health
  • Perth Children's Hospital
  • John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
  • Queensland Children's Hospital
  • Stollery Children's Hospital
  • Queen Elizabeth II Health Sciences Center
  • St. Michael's Hospital
  • Juliane Marie Center, Rigshospitalet
  • Charite Paediatric Pulmonology Department
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
  • Johann Wolfgang Goethe University
  • Hannover Medical School
  • Universitaetsklinikum Jena, Mukoviszidose-Zentrum
  • Universitaetsklinkum Koeln, CF-Studienzentrum
  • Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
  • Pneumologisches Studienzentrum Muenchen-West
  • Klinikum Innenstadt, University of Munich
  • Cork University Hospital
  • Beaumont Hospital
  • Children's Health Ireland at Crumlin
  • Children's Health Ireland at Temple Street
  • St. Vincent's University Hospital
  • University Hospital Galway
  • University Hospital Limerick (Adults)
  • Lady Davis Carmel Medical Center
  • Pediatric Pulmonary Unit Rambam Medical Center
  • Hadassah Medical Organization
  • Schneider Children's Medical Center
  • Sheba Medical Center
  • Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
  • Hospital Universitari Vall d Hebron
  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
  • Hospital Universitario 12 de Octubre
  • Hospital Universitario Infantil La Paz
  • Parc Tauli Sabadell Hospital Universitari
  • Hospital Universitario Virgen del Rocio
  • Hospital Universitario y Politecnico La Fe
  • Lindenhofspital - Quartier Bleu
  • Kinderspital Zuerich
  • Papworth Hospital NHS Foundation Trust, Papworth Everard
  • NHS Greater Glasgow and Clyde, Gartnavel General Hospital
  • St. James University Hospital
  • Liverpool Heart and Chest Hospital
  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
  • Wythenshawe Hospital
  • The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
  • Nottingham University Hospitals NHS Trust, Queens Medical Center
  • All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.

Outcomes

Primary Outcome Measures

Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Full Information

First Posted
July 31, 2019
Last Updated
June 14, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04043806
Brief Title
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
Official Title
A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
August 9, 2019 (Actual)
Primary Completion Date
December 14, 2022 (Actual)
Study Completion Date
December 14, 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 evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).

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
458 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
Part A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
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
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
From Baseline through Week 100

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Currently participating in study VX17-659-105 (NCT03447262) Exclusion Criteria: History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator Current participation in an investigational drug trial (other than study VX17-659-105) Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Hartford Hospital
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06102
Country
United States
Facility Name
Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
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
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Arnold Palmer Hospital Pulmonary and Sleep Medicine
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Johns Hopkins All Children's Hospital Outpatient Care Center
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
St. Luke's CF Center of Idaho
City
Boise
State/Province
Idaho
ZIP/Postal Code
83702
Country
United States
Facility Name
Cystic Fibrosis Institute
City
Glenview
State/Province
Illinois
ZIP/Postal Code
60025
Country
United States
Facility Name
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
City
Niles
State/Province
Illinois
ZIP/Postal Code
60714
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
The University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Kosair Charities Pediatric Clinical Research Unit
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
UMass Memorial Medical Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
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
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
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
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Rutgers Robert Wood Johnson Medical School
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
Albany Medical College, Pulmonary and Critical Care Medicine
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Facility Name
Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
City
Buffalo
State/Province
New York
ZIP/Postal Code
14222
Country
United States
Facility Name
Northwell Health- Long Island Jewish Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
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
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Clinical Research of Charlotte
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28277
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Santiago Reyes, M.D.
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
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 Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Sanford Children's Speciality Clinic
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57105
Country
United States
Facility Name
University of Tennessee Medical Center
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37920
Country
United States
Facility Name
Children's Foundation Research Center / Le Bonheur Children's Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
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
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
City
Spokane
State/Province
Washington
ZIP/Postal Code
99204
Country
United States
Facility Name
Royal Adelaide Hospital
City
Adelaide
Country
Australia
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Alfred Hospital
City
Melbourne, VIC
Country
Australia
Facility Name
Institute for Respiratory Health
City
Nedlands
Country
Australia
Facility Name
Perth Children's Hospital
City
Nedlands
Country
Australia
Facility Name
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
City
New Lambton
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
Stollery Children's Hospital
City
Edmonton
Country
Canada
Facility Name
Queen Elizabeth II Health Sciences Center
City
Halifax
Country
Canada
Facility Name
St. Michael's Hospital
City
Toronto
Country
Canada
Facility Name
Juliane Marie Center, Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
City
Essen
Country
Germany
Facility Name
Johann Wolfgang Goethe University
City
Frankfurt
Country
Germany
Facility Name
Hannover Medical School
City
Hannover
Country
Germany
Facility Name
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
City
Jena
Country
Germany
Facility Name
Universitaetsklinkum Koeln, CF-Studienzentrum
City
Koeln
Country
Germany
Facility Name
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
City
Lübeck
Country
Germany
Facility Name
Pneumologisches Studienzentrum Muenchen-West
City
Muenchen
Country
Germany
Facility Name
Klinikum Innenstadt, University of Munich
City
München
Country
Germany
Facility Name
Cork University Hospital
City
Cork
Country
Ireland
Facility Name
Beaumont Hospital
City
Dublin
Country
Ireland
Facility Name
Children's Health Ireland at Crumlin
City
Dublin
Country
Ireland
Facility Name
Children's Health Ireland at Temple Street
City
Dublin
Country
Ireland
Facility Name
St. Vincent's University Hospital
City
Dublin
Country
Ireland
Facility Name
University Hospital Galway
City
Galway
Country
Ireland
Facility Name
University Hospital Limerick (Adults)
City
Limerick
Country
Ireland
Facility Name
Lady Davis Carmel Medical Center
City
Haifa
Country
Israel
Facility Name
Pediatric Pulmonary Unit Rambam Medical Center
City
Haifa
Country
Israel
Facility Name
Hadassah Medical Organization
City
Jerusalem
Country
Israel
Facility Name
Schneider Children's Medical Center
City
Petah Tikva
Country
Israel
Facility Name
Sheba Medical Center
City
Tel Hashomer
Country
Israel
Facility Name
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
City
Łomianki
Country
Poland
Facility Name
Hospital Universitari Vall d Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Infantil La Paz
City
Madrid
Country
Spain
Facility Name
Parc Tauli Sabadell Hospital Universitari
City
Sabadell
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
Lindenhofspital - Quartier Bleu
City
Bern
Country
Switzerland
Facility Name
Kinderspital Zuerich
City
Zürich
Country
Switzerland
Facility Name
Papworth Hospital NHS Foundation Trust, Papworth Everard
City
Cambridge
Country
United Kingdom
Facility Name
NHS Greater Glasgow and Clyde, Gartnavel General 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
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
City
Newcastle Upon Tyne
Country
United Kingdom
Facility Name
Nottingham University Hospitals NHS Trust, Queens Medical Center
City
Nottingham
Country
United Kingdom
Facility Name
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
City
Penarth
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 of VX-445 Combination Therapy

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