VX15-770-126 Cystic Fibrosis
A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation
Vertex Pharmaceuticals Incorporated
Duration of Study in the UK
5 years, 0 months, 27 days
This is a phase 3, 2 part, open-label (no placebo or ‘dummy drug’) study to assess the safety and effects of long-term use of the study drug Ivacaftor in young children with cystic fibrosis (CF). CF is a rare genetic disease, that affects the lungs, the digestive system and other organs.
Ivacaftor (Kalydeco®) is a potentiator (i.e. improves the function) of the faulty protein, CFTR. It was the first therapy to target the underlying cause of CF and is licensed for children aged 2 years and above with a specific group of CFTR gene mutations.
This study of children aged less than 2 years is designed to learn more about the safety and effects of long term Ivacaftor treatment. The study is split into 2 arms; one arm the participants will receive ivacaftor treatment for 104 weeks with a follow up period of 24 weeks and an observational arm with follow-up for 104 weeks (receiving no ivacaftor).
There will be approximately 75 participants enrolled into the study globally
London - Chelsea Research Ethics Committee
Date of REC Opinion
20 Jul 2017