Elexacaftor; Tezacaftor; Ivacaftor for Cystic fibrosis

Inclusion criteria

• {"criterion_text":"- Subject (or the subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, when appropriate, an assent form."}
• {"criterion_text":"- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines (as applicable), and other study procedures. •\tFor subjects <18 years of age: as judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned."}
• {"criterion_text":"- Did not withdraw consent from the parent study."}
• {"criterion_text":"- Part A: Meets at least 1 of the following criteria: •\tCompleted study drug treatment in the parent study. •\tHad study drug interruption(s) in the parent study, but completed study visits up to the last scheduled visit of the Treatment Period of the parent study. Part B: Meets at least 1 of the following criteria: •\tCompleted study drug treatment in Part A. •\tHad study drug interruption(s) in Part A, but completed study visits up to the last scheduled visit of the Treatment Period of Part A."}
• {"criterion_text":"- Willing to remain on a stable CF treatment regimen (other than CFTR modulators, as defined in Section 9.5) through completion of study participation."}

Exclusion criteria

• {"criterion_text":"- History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject."}
• {"criterion_text":"- History of drug intolerance in the parent study that would pose an additional risk to the subject. (e.g., subjects with a history of allergy or hypersensitivity to the study drug)."}
• {"criterion_text":"- Pregnant and nursing females. Females of childbearing potential (Section 11.5.6.1) must have a negative pregnancy test at the Day 1 Visit (in Part A and Part B) before receiving the first dose of study drug."}
• {"criterion_text":"- Current participation in an investigational drug trial (other than the parent study). Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted."}

Primary endpoints

• {"endpoint_text":"- Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry","definition_or_measurement_approach":"Safety and tolerability measured by monitoring adverse events (AEs), clinical laboratory values, electrocardiograms (ECGs), vital signs, and pulse oximetry."}

Secondary endpoints

• {"endpoint_text":"- Part A Only: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)","definition_or_measurement_approach":"Absolute change from baseline in percent predicted FEV1 (ppFEV1) measured by spirometry."}
• {"endpoint_text":"- Absolute change from baseline in sweat chloride (SwCl)","definition_or_measurement_approach":"Absolute change from baseline in sweat chloride concentration using standard sweat chloride testing."}
• {"endpoint_text":"- Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain (RD) score","definition_or_measurement_approach":"Absolute change from baseline in CFQ-R respiratory domain score (patient-reported outcome instrument)."}
• {"endpoint_text":"- Absolute change from baseline in body mass index (BMI)","definition_or_measurement_approach":"Absolute change from baseline in BMI calculated from measured weight and height."}
• {"endpoint_text":"- Absolute change from baseline in weight","definition_or_measurement_approach":"Absolute change from baseline in body weight measured in kilograms."}
• {"endpoint_text":"- Number of pulmonary exacerbations (PEx)","definition_or_measurement_approach":"Count of pulmonary exacerbations (PEx) as defined in the protocol (events meeting prespecified clinical criteria)."}

Primary sponsor

Full Name

Vertex Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States