|E.1 Medical condition or disease under investigation
|Medical condition(s) being investigated
|Medical condition in easily understood language
|Diseases [C] - Respiratory Tract Diseases [C08]
|E.1.2 Medical condition or disease under investigation
|System Organ Class
|10010331 - Congenital, familial and genetic disorders
|Condition being studied is a rare disease
|E.2 Objective of the trial
|Main objective of the trial
|To evaluate the long-term safety and tolerability of VX121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in subjects with cystic fibrosis (CF)
|Secondary objectives of the trial
|To evaluate the long-term efficacy of VX-121/TEZ/D-IVA
|Trial contains a sub-study
|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.Did not withdraw consent from a parent study.
4.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.
5.Willing to remain on a stable CF treatment regimen through completion of study participation.
|Principal exclusion criteria
|1.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 and 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.
2.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.
3.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.)
4.Current participation in an investigational drug trial (other than a 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.
5.The subject or a close relative of the subject is the investigator or a subinvestigator, 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 in 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.
|E.5 End points
|Primary end point(s)
|Safety and tolerability of long-term treatment with VX-121/TEZ/D-IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry
|Timepoint(s) of evaluation of this end point
|Secondary end point(s)
|Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
Absolute change from baseline in sweat chloride(SwCl)
Number of pulmonary exacerbations (PEx)
|Timepoint(s) of evaluation of this end point
|E.6 and E.7 Scope of the trial
|Scope of the trial
|Trial type and phase
|Human pharmacology (Phase I)
|First administration to humans
|Other trial type description
|Therapeutic exploratory (Phase II)
|Therapeutic confirmatory (Phase III)
|Therapeutic use (Phase IV)
|E.8 Design of the trial
| Comparator of controlled trial
|Other medicinal product(s)
|Number of treatment arms in the trial
The trial involves single site in the Member State concerned
| The trial involves multiple sites in the Member State concerned
|Number of sites anticipated in Member State concerned
|The trial involves multiple Member States
|Number of sites anticipated in the EEA
|E.8.6 Trial involving sites outside the EEA
|Trial being conducted both within and outside the EEA
|Trial being conducted completely outside of the EEA
|If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
|Trial has a data monitoring committee
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|E.8.9 Initial estimate of the duration of the trial
|In the Member State concerned years
|In the Member State concerned months
|In the Member State concerned days
|In all countries concerned by the trial years
|In all countries concerned by the trial days