- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04183790
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
March 22, 2024 updated by: Vertex Pharmaceuticals Incorporated
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
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).
Study Overview
Study Type
Interventional
Enrollment (Actual)
64
Phase
- Phase 3
Expanded Access
Approved for sale to the public.
See expanded access record.
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
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South Brisbane, Australia
- Queensland Children's Hospital
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Westmead, Australia
- The Children's Hospital at Westmead
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Toronto, Canada
- The Hospital for Sick Children
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Vancouver, Canada
- British Columbia's Children's Hospital
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Dublin, Ireland
- Children's Health Ireland at Crumlin
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Dublin, Ireland
- Children's Health Ireland at Temple Street
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Birmingham, United Kingdom
- Birmingham Children's Hospital - NHS Foundation Trust
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London, United Kingdom
- Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
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California
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Orange, California, United States, 92868
- Children's Hospital of Orange County
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Illinois
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Chicago, Illinois, United States, 60611
- Ann & Robert Lurie Children's Hospital of Chicago
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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Minnesota
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Minneapolis, Minnesota, United States, 55404
- Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
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Missouri
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Kansas City, Missouri, United States, 64108
- The Children's Mercy Hospital
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New York
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New Hyde Park, New York, United States, 11040
- Northwell Health- Long Island Jewish Medical Center
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North Carolina
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Charlotte, North Carolina, United States, 28277
- Clinical Research of Charlotte
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Ohio
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Cleveland, Ohio, United States, 44106
- Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
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Columbus, Ohio, United States, 43205
- Nationwide Children's Hospital
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health & Science University
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Texas
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Houston, Texas, United States, 77030
- Texas Children's Hospital
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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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 and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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
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: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Triple Combination Arm
Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening.
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Fixed-dose combination (FDC) tablet for oral administration
Other Names:
Mono tablet for oral administration.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Safety and tolerability as assessed by adverse events (AEs) and serious adverse events (SAEs)
Time Frame: From Baseline up to Week 196
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From Baseline up to Week 196
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1)
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in sweat chloride (SwCl)
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in body mass index (BMI)
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in BMI-for-age z-score
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Number of pulmonary exacerbations (PEx)
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Number of CF-related hospitalizations
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in lung clearance index 2.5 (LCI 2.5)
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in weight
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in weight-for-age z-score
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in height
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Absolute change in height-for-age z-score
Time Frame: From Baseline up to Week 192
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From Baseline up to Week 192
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
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.
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)
February 17, 2020
Primary Completion (Actual)
February 24, 2024
Study Completion (Actual)
February 24, 2024
Study Registration Dates
First Submitted
November 28, 2019
First Submitted That Met QC Criteria
November 28, 2019
First Posted (Actual)
December 3, 2019
Study Record Updates
Last Update Posted (Actual)
March 25, 2024
Last Update Submitted That Met QC Criteria
March 22, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Respiratory Tract Diseases
- Lung Diseases
- Infant, Newborn, Diseases
- Genetic Diseases, Inborn
- Pancreatic Diseases
- Fibrosis
- Cystic Fibrosis
- Molecular Mechanisms of Pharmacological Action
- Membrane Transport Modulators
- Chloride Channel Agonists
- Ivacaftor
- Elexacaftor
Other Study ID Numbers
- VX19-445-107
- 2019-001827-11 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
IPD 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
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.
Clinical Trials on Cystic Fibrosis
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University of Colorado, DenverCystic Fibrosis FoundationTerminatedCystic Fibrosis-related Diabetes | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in ChildrenUnited States
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Royal College of Surgeons, IrelandThe Hospital for Sick Children; Imperial College London; Erasmus Medical Center; University College Dublin and other collaboratorsActive, not recruitingCystic Fibrosis | Adherence, Medication | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in Children | Cystic Fibrosis Liver DiseaseUnited Kingdom, Ireland
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Herlev and Gentofte HospitalCopenhagen University Hospital, DenmarkActive, not recruitingMyocardial Infarction | Heart Diseases | Heart Failure | Stroke | Cystic Fibrosis | Heart Failure, Diastolic | Heart Failure, Systolic | Left Ventricular Dysfunction | Cystic Fibrosis-related Diabetes | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of Pancreas | Cystic Fibrosis, Pulmonary | Cystic...Denmark
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The Hospital for Sick ChildrenCanadian Cystic Fibrosis FoundationActive, not recruitingCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis in ChildrenCanada
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Arrowhead PharmaceuticalsTerminatedCystic Fibrosis, PulmonaryAustralia, New Zealand
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AzurRx SASCompletedCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of PancreasTurkey, Hungary
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Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeWithdrawnCystic Fibrosis-related Diabetes | Cystic Fibrosis Liver Disease | CF - Cystic FibrosisUnited States
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University Hospital, BordeauxCompleted
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University of PortsmouthUniversity Hospital Southampton NHS Foundation Trust; Loughborough University; Queen Alexandra HospitalTerminated
Clinical Trials on ELX/TEZ/IVA
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisCanada, France, Germany, United Kingdom, Israel, Australia, Spain, Netherlands, Denmark, Switzerland
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, United Kingdom, New Zealand, Israel, Australia, Ireland, Germany, Sweden, Czechia, Portugal, Hungary
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisSpain, Belgium, Netherlands, France, Canada, Germany, Sweden, Italy, Czechia, Switzerland, Portugal, Austria, Hungary, Norway, Poland
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, United Kingdom, Belgium, Netherlands, France, Denmark, Israel, New Zealand, Australia, Ireland, Sweden, Canada, Germany, Poland, Switzerland, Italy, Austria, Hungary, Greece, Norway
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Canada, Denmark, Germany, Italy, Israel
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisBelgium, United Kingdom, Australia, Germany
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Australia, Belgium, Netherlands
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Vertex Pharmaceuticals IncorporatedRecruitingCystic FibrosisUnited Kingdom, Canada
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisSpain, Belgium, Australia, Canada, Czechia
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Denmark, Germany, Italy, Canada