Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Subjects 2 Years and Older

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: EUCTR2020-002239-31-DE
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-12-20
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

Part A:
1. Subject’s legal representative or guardian will sign and date an
informed consent form (ICF).
2. As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
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, or
• 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.

Part B
1. Subject’s legal representative or guardian will sign and date an ICF.
2. As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
3. Did not withdraw consent in Part A.
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 did not permanently discontinue 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 70
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

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(s) 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. 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.
4. History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study as deemed by the investigator.

Part B
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(s) to the subject.
2. History of drug intolerance in the parent study or Part A 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. Current participation in an investigational drug trial other than the parent study or the current 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.
4. History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study or Part A, as deemed by the investigator.

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 cystic fibrosis (CF) subjects 2 years of age and older;Secondary Objective: To evaluate the long-term efficacy and 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, standard 12-lead ECGs, vital signs, and pulse oximetry;Timepoint(s) of evaluation of this end point: From signing of ICF until the safety FU visit

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Absolute change in sweat chloride (SwCl)<br>• Absolute change in lung clearance index (LCI)2.5;Timepoint(s) of evaluation of this end point: From signing of ICF until the safety FU visit