Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
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
1 other identifier
interventional
52
2 countries
23
Brief Summary
This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to \<24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Feb 2020
Typical duration for phase_3
23 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 15, 2020
CompletedFirst Posted
Study publicly available on registry
January 21, 2020
CompletedStudy Start
First participant enrolled
February 24, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 22, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
August 22, 2023
CompletedResults Posted
Study results publicly available
September 19, 2024
CompletedSeptember 19, 2024
August 1, 2024
3.5 years
January 15, 2020
August 20, 2024
August 20, 2024
Conditions
Outcome Measures
Primary Outcomes (1)
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 120
Secondary Outcomes (1)
Absolute Change in Sweat Chloride (SwCl)
From Baseline at Week 96
Study Arms (1)
LUM/IVA
EXPERIMENTALParticipants weighing 7 to less than (\<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (\>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks.
Interventions
LUM/IVA granules for oral administration
Eligibility Criteria
You may qualify if:
- Participants From Study VX16-809-122 Part B (Study 122)
- Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B
- Participants Not From Study 122
- Subjects will be 1 to less than 2 years of age
- Homozygous for the F508del mutation (F/F)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (23)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Yale New Haven Hospital
New Haven, Connecticut, 06510, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, 63104, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Medical Center of Dallas
Dallas, Texas, 75235, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792, United States
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Canada
The Hospital for Sick Children
Toronto, Canada
British Columbia's Children's Hospital
Vancouver, Canada
Related Publications (2)
Heneghan M, 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. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PMID: 37983082DERIVEDSouthern 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.
PMID: 33331662DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 15, 2020
First Posted
January 21, 2020
Study Start
February 24, 2020
Primary Completion
August 22, 2023
Study Completion
August 22, 2023
Last Updated
September 19, 2024
Results First Posted
September 19, 2024
Record last verified: 2024-08
Data Sharing
- IPD Sharing
- Will not share
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.