A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
A Phase 2, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Exploratory Study to Evaluate Effects of VX-661 in Combination With Ivacaftor on Lung and Extrapulmonary Systems in Subjects Aged 18 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
1 other identifier
interventional
34
1 country
7
Brief Summary
To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
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 Feb 2016
Shorter than P25 for phase_2
7 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
July 23, 2015
CompletedFirst Posted
Study publicly available on registry
July 24, 2015
CompletedStudy Start
First participant enrolled
February 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2017
CompletedResults Posted
Study results publicly available
July 27, 2018
CompletedJuly 19, 2021
June 1, 2018
1.3 years
July 23, 2015
June 29, 2018
June 28, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28
MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.
Baseline, Day 28
Secondary Outcomes (4)
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Baseline, Day 28
Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29
Baseline, Day 29
Absolute Change From Baseline in Sweat Chloride at Day 29
Baseline, Day 29
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Baseline up to Day 57
Study Arms (2)
Placebo
PLACEBO COMPARATORParticipants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.
TEZ/IVA
EXPERIMENTALParticipants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.
Interventions
Eligibility Criteria
You may qualify if:
- Male and female participants, homozygous for the F508del CFTR mutation
- Confirmed diagnosis of CF by sweat chloride testing
- Forced Expiratory Volume in 1 Second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height at Screening Visit
- 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 for pulmonary disease within 28 days before Day 1
- History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.
- History of solid organ or hematological transplantation
- Pregnant or nursing females
- Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study
- In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (7)
Unknown Facility
Birmingham, Alabama, United States
Unknown Facility
Indianapolis, Indiana, United States
Unknown Facility
Baltimore, Maryland, United States
Unknown Facility
Chapel Hill, North Carolina, United States
Unknown Facility
Cincinnati, Ohio, United States
Unknown Facility
Toledo, Ohio, United States
Unknown Facility
Pittsburgh, Pennsylvania, United States
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
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 23, 2015
First Posted
July 24, 2015
Study Start
February 1, 2016
Primary Completion
June 1, 2017
Study Completion
June 1, 2017
Last Updated
July 19, 2021
Results First Posted
July 27, 2018
Record last verified: 2018-06