A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR)
2 other identifiers
interventional
168
6 countries
36
Brief Summary
Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene and with a second CFTR mutation that is not likely to respond to VX-661 and/or IVA therapy (F508del/not responsive \[NR\]).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Aug 2015
Shorter than P25 for phase_3
36 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 28, 2015
CompletedStudy Start
First participant enrolled
August 1, 2015
CompletedFirst Posted
Study publicly available on registry
August 5, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 7, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
June 7, 2016
CompletedResults Posted
Study results publicly available
June 12, 2018
CompletedJune 12, 2018
May 1, 2018
10 months
July 28, 2015
March 14, 2018
May 8, 2018
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.
Baseline, Through Week 12
Secondary Outcomes (11)
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
Baseline, Through Week 12
Number of Pulmonary Exacerbation Events
Baseline through Week 12
Number of Pulmonary Exacerbation Events Per Year
Baseline through Week 12
Absolute Change From Baseline in Body Mass Index (BMI) at Week 12
Baseline, Week 12
Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
Baseline, Through Week 12
- +6 more secondary outcomes
Study Arms (2)
VX-661/IVA
EXPERIMENTALVX-661 100 milligram (mg) plus IVA 150 mg fixed dose combination (FDC) tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.
Placebo
PLACEBO COMPARATORPlacebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.
Interventions
Eligibility Criteria
You may qualify if:
- Confirmed diagnosis of CF defined as a sweat chloride value greater than or equal to (\>=)60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis.
- Heterozygous for the F508del-CFTR mutation and with a second CFTR mutation that is not likely to respond to VX-661 and/or ivacaftor therapy, genotype to be confirmed via assessment at the Screening Visit.
- Forced Expiratory Volume in 1 Second (FEV1) \>=40 percent (%) and less than or equal to (\<=)90% of predicted normal for age, sex, and height at Screening Visit.
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 participant.
- 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).
- History of solid organ or hematological transplantation.
- Ongoing or prior participation in an investigational drug study or use of commercially available CFTR modulator within 30 days of screening.
- Pregnant or nursing females.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (41)
Unknown Facility
Birmingham, Alabama, United States
Unknown Facility
La Jolla, California, United States
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Los Angeles, California, United States
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Denver, Colorado, United States
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Miami, Florida, United States
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Atlanta, Georgia, United States
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Chicago, Illinois, United States
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Indianapolis, Indiana, United States
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New Orleans, Louisiana, United States
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Ann Arbor, Michigan, United States
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Detroit, Michigan, United States
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Minneapolis, Minnesota, United States
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Kansas City, Missouri, United States
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Omaha, Nebraska, United States
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New York, New York, United States
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Cincinnati, Ohio, United States
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Memphis, Tennessee, United States
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Austin, Texas, United States
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Dallas, Texas, United States
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Richmond, Virginia, United States
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Seattle, Washington, United States
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Spokane, Washington, United States
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Brisban, Queensland, Australia
Unknown Facility
Chermside, Australia
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Herston, Australia
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Innsbruck, Australia
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Westmead, Australia
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Graz, Austria
Unknown Facility
Innsbruck, Austria
Unknown Facility
Salzburg, Austria
Unknown Facility
Vancouver, British Columbia, Canada
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Montreal, Canada
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Bron, France
Unknown Facility
Montpellier, France
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Paris, France
Unknown Facility
Haifa, Israel
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Hashomer, Israel
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Jerusalem, Israel
Unknown Facility
Petah Tikva, Israel
Unknown Facility
Barcelona, Spain
Unknown Facility
Valencia, Spain
Related Publications (1)
Munck A, Kerem E, Ellemunter H, Campbell D, Wang LT, Ahluwalia N, Owen CA, Wainwright C. Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations. J Cyst Fibros. 2020 Nov;19(6):962-968. doi: 10.1016/j.jcf.2020.04.015. Epub 2020 Jun 13.
PMID: 32546431DERIVED
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 3
- 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
July 28, 2015
First Posted
August 5, 2015
Study Start
August 1, 2015
Primary Completion
June 7, 2016
Study Completion
June 7, 2016
Last Updated
June 12, 2018
Results First Posted
June 12, 2018
Record last verified: 2018-05