*Phase 3 open label study of ELX/TEZ/IVA in Cystic Fibrosis Subjects 2 years and older
Research type
Research Study
Full title
A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
IRAS ID
1004766
Contact name
Alison Buckland
Contact email
Sponsor organisation
Vertex Pharmaceuticals Europe Limited
Eudract number
2020-002239-31
Clinicaltrials.gov Identifier
Research summary
Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Changes in this gene can affect the amount of the protein made from this gene or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky which causes problems in the lungs and other organs. This study is being done to learn about the long term safety and effectiveness of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy- which improves CFTR function in patients with certain mutations in their CF genes- in childre who are 2 years and older. This is a Phase 3, 2-part, multicenter, open-label study for subjects who complete parent study VX20-445-111 Part B and meet eligibility criteria. Subjects who complete Part A will have the opportunity to enroll in Part B for an additional 96 weeks.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
22/NW/0256
Date of REC Opinion
16 Feb 2022
REC opinion
Further Information Favourable Opinion