VX15-770-124 Cystic Fibrosis
Research type
Research Study
Full title
A Phase 3, 2-Part, Open-label Study to Evalute the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation
IRAS ID
199957
Contact name
Paone Antoinette
Contact email
Sponsor organisation
Vertex Pharmaceuticals Limited
Eudract number
2015-001997-16
Duration of Study in the UK
3 years, 0 months, 5 days
Research summary
This is a phase 3, 2 part open-label (no placebo or 'dummy drug)' study to assess the safety and effects of the study drug Ivacaftor in 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 CFTR potentiator (i.e. improves the function) of the aaulty protein, CFTR. It was the first therapy to target the underlying cause of the CF and is licensed for children 2 years and above with a specific group of CFRE gene mutations.
This study of children aged less than 2 years is designed to learn more about the safety and effects of Ivacaftor. Participants will be enrolled to receive Ivacaftor in group's determined by age, starting with the older children and decreasing in age once data have been assessed. Children/babies will be dosed with Ivacaftor based on their weight for 4 days. In Part B, participants will again be enrolled in groups based on age, and will receive Ivacoftor for 24 weeks at a dose determined by their weight. There will be a minimum of 20 participants enrolled in part A and 15 in part B. Participants will also be given the opportunity to enrol in a planned extension study if they meet eligibility criteria.
There will be a minimum of 20 participants enrolled in part A and 15 in part B. Participants will also be given the opportunity to enrol in an extension study if they meet the eligibility criteria.
REC name
London - Brent Research Ethics Committee
REC reference
16/LO/0797
Date of REC Opinion
4 Jul 2016
REC opinion
Further Information Favourable Opinion