VX17-661-116 Open-label safety and efficacy of TEZ/IVA 6 yrs and above
Research type
Research Study
Full title
A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
IRAS ID
241643
Contact name
Timothy Lee
Contact email
Sponsor organisation
Vertex Pharmaceuticals (Europe) Ltd
Eudract number
2017-002968-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 4 months, 1 days
Research summary
Cystic Fibrosis (CF) is a genetic disease caused by mutations in the gene encoding a cell transport protein (ion channel). This ion channel transports chloride ions in and out of cells in multiple organ systems. When ions are not transported correctly the salt and water balance in cells and tissues is not controlled, leading to the production of sticky mucus in the lungs, airways and intestines.
This clinical study is designed to evaluate the long-term safety and efficacy of tezacaftor/ivacaftor in subjects with CF aged 6 years or more, who are homozygous (two copies) or heterozygous (one copy) for F508del along with a second (minimal function) CF mutation. Tezacaftor (TEZ) and ivacaftor (IVA)both address the underlying cause of CF.
The study is designed to evaluate the safety and efficacy of long-term TEZ/IVA treatment in paediatric subjects aged 6 years and older. Subjects who complete treatment in the parent study VX16-661-115 will be offered the opportunity to enrol into VX17-661-116. All participants will receive active study drug for up to 96 weeks.
REC name
East of Scotland Research Ethics Service REC 2
REC reference
18/ES/0054
Date of REC Opinion
28 Jun 2018
REC opinion
Further Information Favourable Opinion