A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 15

Inclusion Criteria

1. Subject (or the subject*s 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 (as applicable), and other study
procedures.
• For subjects <18 years of age: as judged by the investigator, parent or legal
guardian must
be able to understand protocol requirements, restrictions, and instructions and
the parent
or legal 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 a parent study.
4. Part A 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 completed study visits
up to the last
scheduled visit of the Treatment Period of a parent study.
Part B: Meets at least one of the following criteria:
-Completed study frug treatment in Part A
-Had study drug interruption(s) in Part A, but 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 (other than CFTR
modulators, through completion of study participation.

Exclusion Criteria

1. History of any illness or any clinical condition that 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 a parent study that would pose an additional
risk to the subject.
(e.g., subjects with a history of allergy or hypersensitivity to the study
drug).
3. Pregnant and nursing females. Females of childbearing potential must
have a negative pregnancy test at the Day 1 Visit (in Part A and Part B) 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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Safety and tolerability based on adverse events (AEs), clinical laboratory<br /><br>values, ECGs, vital signs, and pulse oximetry</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>PART A ONLY:<br /><br>• Absolute change from baseline in percent predicted forced expiratory<br /><br>volume in 1 second (ppFEV1)<br /><br>• Absolute change from baseline in sweat chloride (SwCl)<br /><br>• Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised<br /><br>(CFQ-R) respiratory domain (RD) score<br /><br>• Absolute change from baseline in body mass index (BMI)<br /><br>• Absolute change from baseline in weight<br /><br>• Number of pulmonary exacerbations (PEx)<br /><br><br /><br>Other endpoints<br /><br>-Absolute change from baseline in BMI z-score (subjects <=20 years of<br /><br>age)<br /><br>-Absolute change from baseline in weight z-score (subjects <=20 years<br /><br>of age) </p><br>