A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy 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: EUCTR2017-004134-29-ES
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-03-21
• Study Completion: 2020-09-09
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 484

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 completed study visits up to the last scheduled visit of the Treatment Period of a parent study.

5. Willing to remain on a stable CF treatment regimen through completion of study participation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 153
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 307
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 nursing females. Females of childbearing potential 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
Timepoint(s) of evaluation of this end point: Treatment period, ETT visit, Safety Follow-up after last dose.;Main Objective: To evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.;Secondary Objective: 1) To evaluate the long-term efficacy of VX-659 in TC with TEZ and IVA<br>2) To evaluate the pharmacodynamics (PD) of VX-659 in TC with TEZ and IVA;Primary end point(s): Safety and tolerability of long-term treatment with VX-659 in TC with TEZ and IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry.