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
- Registration Number
- NCT04235140
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 52
-
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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description LUM/IVA LUM/IVA Participants 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.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Absolute Change in Sweat Chloride (SwCl) From Baseline at Week 96 Sweat samples were collected using an approved collection device.
Trial Locations
- Locations (23)
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
University of Utah / Primary Children's Medical Center
🇺🇸Salt Lake City, Utah, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Cardinal Glennon Children's Hospital - St. Louis University
🇺🇸Saint Louis, Missouri, United States
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
British Columbia's Children's Hospital
🇨🇦Vancouver, Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital
🇨🇦Montreal, Canada
The Hospital for Sick Children
🇨🇦Toronto, Canada
Wake Forest University Baptist Medical Center
🇺🇸Winston-Salem, North Carolina, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Children's Medical Center of Dallas
🇺🇸Dallas, Texas, United States
Yale New Haven Hospital
🇺🇸New Haven, Connecticut, United States