A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 844

Inclusion Criteria

Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form, and, when appropriate, an assent form., Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures., Did not withdraw consent from a parent study., Meets at least 1 of the following criteria: •Completed study drug treatment in a parent study. •Had study drug interruption(s) in a parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of a parent study., Willing to remain on a stable Cystic Fibrosis treatment regimen through completion of study participation.

Exclusion Criteria

New development of a comorbidity during the parent study that might post an additional risk in administering study drug to the subject. This includes, but is not limited to, the following: - Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment, that might pose an additional risk in administering study drug to the subject. - Solid organ or hematological transplantation. - Any other comorbidity that, in the opinion of the investigator, might post an additional risk in administering study drug to the subject., Pregnant or breast-feeding females. All female subjects must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug., History of drug intolerance in a 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.), Current participation in an investigational drug trial (other than a 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., The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site. However, an adult (aged 18 years or older) who is a relative of a study staff member may be enrolled in the study provided that: - the adult lives independently of and does not reside with the study staff member, and - the adult participates in the study at a site other than the site at which the family member is employed.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety and tolerability of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in subjects with cystic fibrosis;Secondary Objective: To evaluate the long-term efficacy of VX-121/TEZ/D-IVA;Primary end point(s): Safety and tolerability of long-term treatment with VX-121/TEZ/D-IVA based on adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1);Secondary end point(s):Absolute change from baseline in sweat chloride (SwCl);Secondary end point(s):Number of pulmonary exacerbations (PEx)