A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects With Cystic Fibrosis

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: EUCTR2020-004885-21-ES
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-10-19
• Last Update Posted: 30 January 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 143

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. For subjects being considered for resumption of participation in this study after enrolling in another Vertex study of investigational CFTR modulators (referred to as another qualified Vertex study”): Completed the ETT visit in another qualified Vertex study before or on the 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.
6. Willing to remain on a stable CF treatment regimen (as defined in Section 9.5 of the protocol) through completion of study participation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 33
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 107
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

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 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).
3. Pregnant and breast-feeding females. Female subjects 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 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. For subjects being considered for resumption of participation in this study after enrolling in another qualified Vertex study, the following exclusion criteria also apply:
• Subject received the first dose of study drug in the Treatment Period of another qualified Vertex study,
• Subject has access to commercially available ELX/TEZ/IVA or is receiving managed access program supplied ELX/TEZ/IVA, or
• Subject departed this study more than once to participate in another qualified Vertex study.

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 safety and tolerability of Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) in subjects with Cystic Fibrosis (CF).;Secondary Objective: Not applicable;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 signing of ICF until safety Follow up visit.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable