Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

• Status: Active, not recruiting
• Trial end date: 2022-02-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).

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

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Key Inclusion Criteria:

• Subjects will be 1 to less than 2 years of age on Day 1 of the relevant part of the study.

• Homozygous for F508del (F/F).

Key Exclusion Criteria:

• Any clinically significant laboratory abnormalities at the Screening Visit 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.