Back to resultsEvaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Primary Purpose
Cystic Fibrosis
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
IVA
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
Key Inclusion Criteria:
- Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), 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
- Children's Hospital of Orange County
- Children's Hospital Colorado
- Ann & Robert Lurie Children's Hospital of Chicago
- Boston Children's Hospital
- Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
- The Children's Mercy Hospital
- Northwell Health- Long Island Jewish Medical Center
- Clinical Research of Charlotte
- Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
- Nationwide Children's Hospital
- Oregon Health & Science University
- Texas Children's Hospital
- Seattle Children's Hospital
- Queensland Children's Hospital
- The Children's Hospital at Westmead
- The Hospital for Sick Children
- British Columbia's Children's Hospital
- Children's Health Ireland at Crumlin
- Children's Health Ireland at Temple Street
- Birmingham Children's Hospital - NHS Foundation Trust
- Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton 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 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 Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Absolute change in body mass index (BMI)
Absolute change in BMI-for-age z-score
Number of pulmonary exacerbations (PEx)
Number of CF-related hospitalizations
Absolute change in lung clearance index 2.5 (LCI 2.5)
Absolute change in weight
Absolute change in weight-for-age z-score
Absolute change in height
Absolute change in height-for-age z-score
Full Information
NCT ID
NCT04183790
First Posted
November 28, 2019
Last Updated
February 28, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT04183790
Brief Title
Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Official Title
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
February 17, 2020 (Actual)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
April 2024 (Anticipated)
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, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects 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
64 (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
Mono tablet for oral administration.
Primary Outcome Measure Information:
Title
Safety and tolerability as assessed by adverse events (AEs) and serious adverse events (SAEs)
Time Frame
From Baseline up to Week 196
Secondary Outcome Measure Information:
Title
Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
Time Frame
From Baseline up to Week 192
Title
Absolute change in sweat chloride (SwCl)
Time Frame
From Baseline up to Week 192
Title
Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Time Frame
From Baseline up to Week 192
Title
Absolute change in body mass index (BMI)
Time Frame
From Baseline up to Week 192
Title
Absolute change in BMI-for-age z-score
Time Frame
From Baseline up to Week 192
Title
Number of pulmonary exacerbations (PEx)
Time Frame
From Baseline up to Week 192
Title
Number of CF-related hospitalizations
Time Frame
From Baseline up to Week 192
Title
Absolute change in lung clearance index 2.5 (LCI 2.5)
Time Frame
From Baseline up to Week 192
Title
Absolute change in weight
Time Frame
From Baseline up to Week 192
Title
Absolute change in weight-for-age z-score
Time Frame
From Baseline up to Week 192
Title
Absolute change in height
Time Frame
From Baseline up to Week 192
Title
Absolute change in height-for-age z-score
Time Frame
From Baseline up to Week 192
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 (VX18-445-106 Part B, NCT03691779), 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
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Ann & Robert Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
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
Northwell Health- Long Island Jewish Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Facility Name
Clinical Research of Charlotte
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28277
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
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
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
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia's Children's Hospital
City
Vancouver
Country
Canada
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
Birmingham Children's Hospital - NHS Foundation Trust
City
Birmingham
Country
United Kingdom
Facility Name
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
City
London
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
Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
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