- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04235140
Long-term Safety of Lumacaftor/Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
September 20, 2023 updated by: Vertex Pharmaceuticals Incorporated
A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
This is a Phase 3, multicenter, open-label and roll-over study in subjects who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.
Study Overview
Study Type
Interventional
Enrollment (Actual)
52
Phase
- Phase 3
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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Montreal, Canada
- McGill University Health Centre, Glen Site, Montreal Children's Hospital
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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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Alabama
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Birmingham, Alabama, United States, 35233
- University of Alabama at Birmingham
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Arkansas
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Little Rock, Arkansas, United States, 72202
- Arkansas Children's Hospital
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Connecticut
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New Haven, Connecticut, United States, 06510
- Yale New Haven Hospital
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Georgia
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Atlanta, Georgia, United States, 30329
- Children's Healthcare of Atlanta
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Illinois
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Chicago, Illinois, United States, 60611
- Ann & Robert H. Lurie Children's Hospital of Chicago
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Indiana
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Indianapolis, Indiana, United States, 46202
- Riley Hospital for Children at Indiana University Health
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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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Saint Louis, Missouri, United States, 63104
- Cardinal Glennon Children's Hospital - St. Louis University
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New York
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Rochester, New York, United States, 14642
- University of Rochester Medical Center
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North Carolina
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Chapel Hill, North Carolina, United States, 27599
- NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
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Winston-Salem, North Carolina, United States, 27157
- Wake Forest University Baptist Medical Center
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Texas
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Dallas, Texas, United States, 75235
- Children's Medical Center of Dallas
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Fort Worth, Texas, United States, 76104
- Cook Children's Medical Center
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Utah
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Salt Lake City, Utah, United States, 84132
- University of Utah / Primary Children's Medical Center
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Wisconsin
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Madison, Wisconsin, United States, 53792
- University of Wisconsin Hospital and Clinics
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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
1 year and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Key Inclusion Criteria:
Subjects From Study VX16-809-122 Part B (Study 122)
- Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B
Subjects Not From Study 122
- Subjects will be 1 to less than 2 years of age
- Homozygous for the F508del mutation (F/F)
Key Exclusion Criteria:
- Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject
- Solid organ or hematological transplantation
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: LUM/IVA
Subjects will receive LUM/IVA for 96 weeks.
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LUM/IVA granules for oral administration
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs)
Time Frame: Up to 120 weeks
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Up to 120 weeks
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Absolute change in sweat chloride
Time Frame: From Baseline at Week 96
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From Baseline at Week 96
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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 24, 2020
Primary Completion (Actual)
August 22, 2023
Study Completion (Actual)
August 22, 2023
Study Registration Dates
First Submitted
January 15, 2020
First Submitted That Met QC Criteria
January 15, 2020
First Posted (Actual)
January 21, 2020
Study Record Updates
Last Update Posted (Actual)
September 21, 2023
Last Update Submitted That Met QC Criteria
September 20, 2023
Last Verified
September 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX19-809-124
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.
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AzurRx SASCompletedCystic Fibrosis | Cystic Fibrosis Gastrointestinal Disease | Cystic Fibrosis of PancreasTurkey, Hungary
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Arrowhead PharmaceuticalsTerminatedCystic Fibrosis, PulmonaryAustralia, New Zealand
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University of PortsmouthUniversity Hospital Southampton NHS Foundation Trust; Loughborough University; Queen Alexandra HospitalTerminated
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University Hospital, BordeauxCompleted
Clinical Trials on LUM/IVA
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada
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Vertex Pharmaceuticals IncorporatedCompletedCystic Fibrosis, Homozygous for the F508del CFTR MutationUnited States, France, Spain, Belgium, Canada, Austria, Australia, Germany, United Kingdom, Denmark
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisAustralia, United Kingdom
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisNetherlands
-
Vertex Pharmaceuticals IncorporatedCompletedCystic Fibrosis, Homozygous for the F508del CFTR MutationUnited States, Germany, Canada, Netherlands, Czech Republic, Italy, Ireland, Sweden, United Kingdom, Australia, France
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada
-
Vertex Pharmaceuticals IncorporatedCompleted
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, Canada
-
Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, France, United Kingdom, Germany, Belgium, Denmark, Canada, Australia, Sweden
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Vertex Pharmaceuticals IncorporatedCompletedCystic FibrosisUnited States, France, United Kingdom, Germany, Australia, New Zealand, Belgium