Study design (if review, criteria of inclusion for studies)
Controlled: Yes.1 Randomised: Yes.2 Open: No.3 Single blind: No.4 Double blind: Yes.5 Parallel group: No.6 Cross over: No
Participants
Subjects aged 12 years or older, on the date of informed consent; Confirmed diagnosis of CF as determined by the investigator. Subject has one of the following genotypes: 1) homozygous for F508del; 2) heterozygous for F508del and a gating (F/G) mutation; 3) heterozygous for F508del and a residual function (F/RF) mutation; at least 1 other TCR CFTR mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation. For subjects currently receiving Vertex CFTR modulator therapy, FEV1 value â¥40% and â¤90% of predicted mean for age, sex, and height at the Screening Visit. All subjects not currently receiving Vertex CFTR modulator therapy must have an FEV1 value â¥40% and â¤80% of predicted mean. Stable CF disease as judged by the investigator.
Interventions
VX-121/TEZ/D-IVA
Outcome measures
Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) through Week 24.