- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02070744
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
March 14, 2018 updated by: Vertex Pharmaceuticals Incorporated
A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation With an Open-Label Extension
The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
40
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Alabama
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Birmingham, Alabama, United States
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California
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Palo Alto, California, United States
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Stanford, California, United States
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Florida
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Altamonte Springs, Florida, United States
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Miami, Florida, United States
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Orlando, Florida, United States
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Tampa, Florida, United States
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Idaho
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Boise, Idaho, United States
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Illinois
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Chicago, Illinois, United States
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Massachusetts
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Boston, Massachusetts, United States
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New Jersey
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New Brunswick, New Jersey, United States
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New York
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New York, New York, United States
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North Carolina
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Durham, North Carolina, United States
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Ohio
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Cincinnati, Ohio, United States
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Oklahoma
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Oklahoma City, Oklahoma, United States
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Pennsylvania
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Pittsburgh, Pennsylvania, United States
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South Carolina
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Charleston, South Carolina, United States
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Tennessee
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Memphis, Tennessee, United States
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Texas
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Houston, Texas, United States
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Vermont
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Burlington, Vermont, United States
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Colchester, Vermont, United States
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Washington
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Seattle, Washington, United States
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Wisconsin
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Milwaukee, Wisconsin, United States
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Homozygous for the F508del CFTR mutation
- FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
- Stable CF disease as judged by the investigator
Exclusion Criteria:
- 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 participant
- Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE Phase (whichever was applicable)
- Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
- The participant or a close relative of the participant is the investigator or sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h
Participants received VX-661 50 milligram (mg) tablet plus Ivacaftor (IVA) 150 mg tablet every 12 hours (q12h) for 12 weeks.
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Tablet, oral use
Film coated tablet, oral use
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Placebo Comparator: PC Phase: VX 661 placebo q12h + IVA placebo q12h
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
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Tablet, oral use
Film coated tablet, oral use
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Experimental: PC Phase: VX-661 100 mg qd + IVA 150 mg q12h
Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks.
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Tablet, oral use
Film coated tablet, oral use
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Placebo Comparator: PC Phase: VX -661 placebo qd + IVA placebo q12h
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
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Tablet, oral use
Film coated tablet, oral use
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Experimental: OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
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Tablet, oral use
Film coated tablet, oral use
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Baseline (PC Phase) up to 112 days
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AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment.
This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed.
AE includes serious as well as Non-serious AEs.
SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.
AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent.
Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase) up to 112 days
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OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs
Time Frame: Baseline (OLE Phase) up to 364 days
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AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment.
This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed.
AE includes serious as well as Non-serious AEs.
SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event.
AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent.
Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase) up to 364 days
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
Time Frame: Baseline (PC Phase), Through Week 12
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase), Through Week 12
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OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40
Time Frame: Baseline (OLE Phase), Through Week 40
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase), Through Week 40
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PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
Time Frame: Baseline (PC Phase), Through Week 12
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase), Through Week 12
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OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40
Time Frame: Baseline (OLE Phase), Through Week 40
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase), Through Week 40
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PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12
Time Frame: Baseline (PC Phase), Through Week 12
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Sweat samples were collected using an approved collection device.
Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase), Through Week 12
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OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40
Time Frame: Baseline (OLE Phase), Through Week 40
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Sweat samples were collected using an approved collection device.
Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase), Through Week 40
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PC Phase: Absolute Change From Baseline in Body Weight at Week 12
Time Frame: Baseline (PC Phase), Week 12
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Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase), Week 12
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OLE Phase: Absolute Change From Baseline in Body Weight at Week 40
Time Frame: Baseline (OLE Phase), Week 40
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Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase), Week 40
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PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12
Time Frame: Baseline (PC Phase), Week 12
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BMI was calculated using following formula: BMI = Weight in kg/height in square meter (m^2).
Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase), Week 12
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OLE Phase: Absolute Change From Baseline BMI at Week 40
Time Frame: Baseline (OLE Phase), Week 40
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BMI was calculated using following formula: BMI = Weight in kg/height in m^2.
Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase), Week 40
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PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
Time Frame: Baseline (PC Phase), Through Week 12
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis.
Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Baseline was defined as Day 1 of PC Phase.
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Baseline (PC Phase), Through Week 12
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OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40
Time Frame: Baseline (OLE Phase), Through Week 40
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis.
Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Baseline was defined as Day 1 of the OLE Phase.
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Baseline (OLE Phase), Through Week 40
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PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA
Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661
Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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Pharmacokinetic (PK) sampling was performed up to 12 hours post-dose on Day 85.
For Arm VX-661 50 mg q12h + IVA 150 mg q12h (VX-661 q12h regimen), area under the concentration versus time curve from time 0 to 12 hours (AUC0-12h) was multiplied by 2 to obtain AUC0-24h.
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Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA
Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA
Time Frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
March 1, 2014
Primary Completion (Actual)
May 27, 2016
Study Completion (Actual)
May 27, 2016
Study Registration Dates
First Submitted
February 21, 2014
First Submitted That Met QC Criteria
February 21, 2014
First Posted (Estimate)
February 25, 2014
Study Record Updates
Last Update Posted (Actual)
April 13, 2018
Last Update Submitted That Met QC Criteria
March 14, 2018
Last Verified
March 1, 2018
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX13-661-103
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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