NCT04235140

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Months
• Healthy volunteers: Not accepted

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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	LUM/IVA	LUM/IVA granules for oral administration

Timeline

• Start date: 2020-02-24
• Primary completion: 2023-08-22
• Completion: 2023-08-22
• First posted: 2020-01-21
• Last updated: 2024-09-19
• Results posted: 2024-09-19

Locations

23 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study