A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
A Phase 2, Randomized, Placebo-Controlled, Double-blind Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
1 other identifier
interventional
41
1 country
9
Brief Summary
The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of TEZ/IVA through Week 72.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Sep 2016
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 31, 2016
CompletedFirst Posted
Study publicly available on registry
April 6, 2016
CompletedStudy Start
First participant enrolled
September 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2018
CompletedResults Posted
Study results publicly available
August 21, 2019
CompletedOctober 23, 2019
October 1, 2019
1.8 years
March 31, 2016
July 30, 2019
October 22, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change in Total Brody/CF-CT Score
The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
From Baseline at Week 72
Secondary Outcomes (1)
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 76
Study Arms (2)
TEZ/IVA
EXPERIMENTALParticipants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
Placebo
PLACEBO COMPARATORParticipants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.
Interventions
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
Eligibility Criteria
You may qualify if:
- Homozygous for the F508del CFTR mutation
- Confirmed diagnosis of CF
- Percent predicted forced expiratory volume (ppFEV1) ≥70% of predicted normal for age, sex, and height during screening.
- Stable CF disease as judged by the investigator
You may not qualify if:
- 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.
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)
- Pregnant or nursing females.
- Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
- Any contraindication to undergoing chest imaging, as per the site's institutional guidelines
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (9)
Unknown Facility
Chermside, Australia
Unknown Facility
Melbourne, Australia
Unknown Facility
Nedlands, Australia
Unknown Facility
New Lambton Heights, Australia
Unknown Facility
Parkville SIC, Australia
Unknown Facility
Randwick, Australia
Unknown Facility
South Brisbane, Australia
Unknown Facility
Subiaco, Australia
Unknown Facility
Westmead, Australia
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 31, 2016
First Posted
April 6, 2016
Study Start
September 1, 2016
Primary Completion
July 1, 2018
Study Completion
July 1, 2018
Last Updated
October 23, 2019
Results First Posted
August 21, 2019
Record last verified: 2019-10