Phase 3b OLE Study of ELX/TEZ/IVA in CF subjects 6 years and older
Research type
Research Study
Full title
A Phase 3b Open label Study Evaluating the Long term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
IRAS ID
1003390
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2020-001404-42
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 or it does not work properly, some fluids in the body become
sticky and causes pcan affect the amount of the protein made from this gene or how well
the protein works. When there is not enough protein roblems in the lungs and other
organs. This study will help
researchers learn if the study drugs VX-445/TEZ/IVA is safe and effective in the tested
doses. VX- 445, tezacaftor and ivacaftor are a triple combination of therapies targeted at
improving the function of this protein. There is a strong rationale for preventing the
progress of the disease by treating patients earlier in life. Currently Vertex
VX-445/TEZ/IVA therapy in Phase 3 studies in adult and young CF subjects with 1 or 2
copies of the F508del mutation, namely those
with F/MF and F/F genotypes. Furthermore this triple combination therapy has been
approved by FDA for people with CF aged 12 years and older who have at least one
copy of the F508del mutation, regardless of their second mutation. Given the clinical
benefit seen in previous studies of adults with CF,this study To evaluate the long-term
safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in subjects
with CF.REC name
North West - Haydock Research Ethics Committee
REC reference
20/NW/0371
Date of REC Opinion
12 Oct 2020
REC opinion
Further Information Favourable Opinion