NCT02797132

Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 2 Years – 5 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	LUM/IVA

Timeline

• Start date: 2016-05-01
• Primary completion: 2017-09-01
• Completion: 2017-09-01
• First posted: 2016-06-13
• Last updated: 2018-10-30
• Results posted: 2018-10-30

Locations

20 sites across 2 countries: United States, Canada