Cystic fibrosis
Conditions
Interventions
Study drug refers to VX-445/TEZ/IVA and IVA. Study drugs will be orally
administered as 2 fixed-dose combination (FDC) film-coated tablets of
VX-445/TEZ/IVA in the morning, and 1 film-coated IVA tab
Sponsors
Vertex Pharmaceuticals
Eligibility
Age
16 Years to 64 Years
Inclusion criteria
Inclusion criteria: Part A Subjects transitioning from Study 104 must meet all of the following criteria to be eligible for Part A: 1. Subject (or his or her legally appointed and authorized representative) will sign and date an ICF (informed consent form), 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. Did not withdraw consent from the parent study. 4. Meets at least 1 of the following criteria: - Completed study drug treatment in the parent study. - Had study drug interruption(s) in the parent study, but without prematurely discontinuing study drug in the parent study and completed study visits up to the last scheduled visit of the Treatment Period of the parent study. 5. Willing to remain on a stable CF treatment regimen through completion of study participation. Inclusion criteria for subjects resuming participation in Part A of this study after enrolling in another qualifying Vertex study include all of the criteria above AND the following criterion: 6. Completed the ETT visit in another qualified Vertex study before or on same day as the Returning Visit in this study. If more than 30 days have elapsed since the ETT visit in the other qualified Vertex study, approval of the medical monitor is required. Part B: Subjects who meet all of the following criteria will be eligible for Part B: 1. Subject (or his or her legally appointed and authorized representative) will sign and date an 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. Did not withdraw consent from the parent study or Part A of this study. 4. Meets at least 1 of the following criteria: • Completed study drug treatment in Part A, or • Had study drug interruption(s) in Part A, but without prematurely discontinuing study drug and completed study visits up to the last scheduled visit of the Treatment Period of Part A. 5. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation. Inclusion criteria for subjects resuming participation in Part B of this study after enrolling in another qualifying Vertex study include all of the criteria above AND the following criterion: 6. Completed the ETT visit in another qualified Vertex study before or on same day as the Returning Visit in this study. If more than 30 days have elapsed since the ETT visit in the other qualified Vertex study, approval of the medical monitor is required.
Exclusion criteria
Exclusion criteria: Part A Subjects who meet any of the following criteria will NOT be eligible for Part A: 1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. 2. Pregnant and breast-feeding females. All female subjects, regardless of childbearing potential status, must have a negative pregnancy test at the Part A Day 1 Visit before receiving the first dose of study drug. 3. History of intolerance to study drug in the parent study that would pose an additional risk to the subject in the opinion of the investigator. (e.g., subjects with a history of allergy or hypersensitivity to the study drug). 4. Current participation in an investigational drug study (other than the parent study). Participation in a non-interventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted. Subjects resuming participation in Part A of this study after enrolling in another qualifying Vertex study must not meet Exclusion Criteria 1 through 3, and also must not meet any of the following criteria: 5. Pregnant and nursing females. All female subjects must have a negative pregnancy test at the Returning Visit before receiving the first dose of study drug. 6. History of intolerance to study drug that would pose an additional risk to the subject in the opinion of the investigator. If protocol-defined interruption criteria is met in another qualified Vertex study, then the subject must remain on study drug interruption until the subject meets criteria in Section 9.8 and receives approval by the medical monitor to resume study drug dosing in this study. 7. Has received the first dose of study drug in the Treatment Period of another qualified Vertex study. 8. Has access to commercially available VX-445/TEZ/IVA or is receiving managed access program supplied VX 445/TEZ/IVA. 9. Has departed this study more than once to participate in another qualified Vertex study. Part B: Subjects who meet any of the following criteria will NOT be eligible for this study: 1. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. 2. Pregnant and breast-feeding females. All female subjects, regardless of childbearing potential status, must have a negative pregnancy test at the Part B Day 1 Visit before receiving the first dose of study drug. 3. History of intolerance to study drug that would pose an additional risk to the subject in the opinion of the investigator (e.g., subjects with a history of allergy or hypersensitivity to the study drug). 4. Current participation in an investigational drug study (other than the parent study or the current study). Participation in a non interventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted. Subjects resuming participation in Part B of this study after enrolling in another qualifying Vertex st
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoint (Parts A and B): Safety and tolerability of long-term treatment with VX-445/TEZ/IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints (Part A only): - Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) - Absolute change in sweat chloride (SwCl) - Absolute change in body mass index (BMI) - Absolute change in BMI z-score - Absolute change in body weight - Absolute change in CFQ-R respiratory domain (RD) score | — |
Countries
Netherlands
Outcome results
None listed