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

• Status: Enrolling by invitation
• Trial end date: 2024-02-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

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.