Phase 3b Study of ELX/TEZ/IVA in CF subjects 6 Through 11 Years of Age
Research type
Research Study
Full title
A Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
IRAS ID
1003047
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2019-003554-86
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 and causes problems 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 is
designed To evaluate the effectiveness of elexacaftor (VX-445; ELX)/tezacaftor
(TEZ)/ivacaftor (IVA) in participants 6 through 11 years of age with cystic fibrosis
Approximately 108 subjects will be randomized (1:1) to the ELX/TEZ/IVA group or the
placebo group. Excluding the Screening Period, the total study duration is approximately
28 weeks (24 weeks for the Treatment Period, and 4 weeks for the Safety Follow-up
Period).REC name
North West - Haydock Research Ethics Committee
REC reference
20/NW/0195
Date of REC Opinion
1 May 2020
REC opinion
Favourable Opinion