A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF genotypes

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2020-001404-42-FR
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-11-16
• Last Update Posted: 5 January 2021

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 108

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 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 did not permanently discontinue study drug, 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 (as defined in Section 9.5) through
completion of study participation.

Are the trial subjects under 18? yes
Number of subjects for this age range: 108
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.History of any comorbidity 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.History of drug intolerance 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).
3.Pregnant and breast-feeding females. Female subjects of childbearing potential (Section 11.4.7.1) must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug.
4.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.

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 elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in subjects with CF;Secondary Objective: •To evaluate the efficacy of ELX/TEZ/IVA<br>•To evaluate the pharmacodynamics (PD) of ELX/TEZ/IVA;Primary end point(s): Safety and tolerability of ELX/TEZ/IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry;Timepoint(s) of evaluation of this end point: From Baseline at each post-baseline visit.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): •Absolute change in sweat chloride (SwCl) from baseline<br>•Absolute change in lung clearance index2.5 (LCI2.5) from baseline<br>;Timepoint(s) of evaluation of this end point: From Baseline at each post-baseline visit.