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: Yes.6 Cross over: No
Principal inclusion criteria: 1. Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), and, when appropriate, an assent form.2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.3. Age 12 years or older, on the date of informed consent.4. Confirmed diagnosis of CF as determined by the investigator.5. Subject has the F/F genotype. Note: If the screening CFTR genotype result is not received before Day -28, a previous CFTR genotype laboratory report may be used to establish eligibility. Subjects who have been enrolled and whose screening genotype is not confirmed to be F/F must be discontinued from the study (Section 9.9).6. Forced expiratory volume in 1 second (FEV1) value â¥40% and â¤90% of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI])9 at the Screening Visit. Spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria10 for acceptability and repeatability.7. Stable CF disease as judged by the investigator.8. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation.
VX-445 in TC with TEZ and IVA
Primary end point(s): Absolute change in ppFEV1 from baseline at Week 4