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

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).


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 40 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Actual Study Start Date: August 23, 2018
Estimated Primary Completion Date: September 2020
Estimated Study Completion Date: September 2020

Arms:
- Experimental: Part A Cohort 1 [aged 18 to <24 months]
- Experimental: Part A Cohort 2 [12 to <18months]
- Experimental: Part B