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
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Enrollment
- 52
- Locations
- 23
- Primary Endpoint
- Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Months to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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)
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.
Arms & Interventions
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.
Intervention: LUM/IVA (Drug)
Outcomes
Primary Outcomes
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 120
Secondary Outcomes
- Absolute Change in Sweat Chloride (SwCl)(From Baseline at Week 96)