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

Phase 1

Conditions: Cystic Fibrosis
MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: EUCTR2019-003455-11-GB

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Not specified

Target Recruitment: 158

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 interruptions(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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 32
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 126
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. 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.
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.

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 (VX-445; ELX)/tezacaftor (TEZ)/ivacaftor (IVA);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;<br> Timepoint(s) of evaluation of this end point: AEs throughout the study<br> Vital signs, pulse oximetry, clinical laboratory values Day 1, Day 15, Week 4,12,24, 48 End of trial visit and Safety follow up visit<br> ECG on Day 1, Day 15, Week 12,24, End of trial visit and Safety follow up visit<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): N/A;Timepoint(s) of evaluation of this end point: N/A

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