A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
1 other identifier
interventional
206
9 countries
53
Brief Summary
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
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 Jul 2015
Shorter than P25 for phase_3
53 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 1, 2015
CompletedFirst Submitted
Initial submission to the registry
July 23, 2015
CompletedFirst Posted
Study publicly available on registry
August 3, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
September 1, 2016
CompletedResults Posted
Study results publicly available
October 23, 2017
CompletedOctober 23, 2017
September 1, 2017
1.2 years
July 23, 2015
September 19, 2017
September 19, 2017
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24
Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Baseline, Through Week 24
Secondary Outcomes (18)
Average Absolute Change From Baseline in Sweat Chloride at Day 15 and Week 4
Baseline, Day 15 and Week 4
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Baseline, Week 24
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24
Baseline, Through Week 24
Absolute Change From Baseline in Lung Clearance Index 5.0 (LCI5.0) Through Week 24
Baseline, Through Week 24
Absolute Change From Baseline in Sweat Chloride at Week 24
Baseline, Week 24
- +13 more secondary outcomes
Study Arms (2)
LUM/IVA
EXPERIMENTALFixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
Placebo
PLACEBO COMPARATORMatching placebo q12h
Interventions
Eligibility Criteria
You may qualify if:
- Subjects who weigh ≥15 kg without shoes a the Screening Visit
- Subjects with confirmed diagnosis of CF at the Screening Visit.
- Subjects who are homozygous for the F508del CFTR mutation
- Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height
- Subjects with a screening LCI2.5 result greater than or equal to 7.5
You may not qualify if:
- History of any comorbidity reviewed at the Screening Visit 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.
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
- Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
- History of solid organ or hematological transplantation at the Screening Visit
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (53)
Unknown Facility
Birmingham, Alabama, United States
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Los Angeles, California, United States
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Palo Alto, California, United States
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Aurora, Colorado, United States
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Wilmington, Delaware, United States
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Orlando, Florida, United States
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Atlanta, Georgia, United States
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Chicago, Illinois, United States
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Iowa City, Iowa, United States
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Boston, Massachusetts, 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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Manchester, New Hampshire, United States
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Chapel Hill, North Carolina, United States
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Cincinnati, Ohio, United States
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Cleveland, Ohio, United States
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Dayton, Ohio, United States
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Philadelphia, Pennsylvania, United States
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Pittsburgh, Pennsylvania, United States
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Charleston, South Carolina, United States
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Salt Lake City, Utah, United States
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Colchester, Vermont, United States
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Charlottesville, Virginia, United States
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Norfolk, Virginia, United States
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Richmond, Virginia, United States
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Seattle, Washington, United States
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Madison, Wisconsin, United States
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Milwaukee, Wisconsin, United States
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Herston, Australia
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New South Wales, Australia
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Parkville, Australia
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Subiaco, Australia
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Westmead, Australia
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Brussels, Belgium
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Leuven, Belgium
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Vancouver, British Columbia, Canada
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Toronto, Ontario, Canada
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Montreal, Quebec, Canada
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Copenhagen, Denmark
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Bordeaux, France
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Bron, France
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Paris, France
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Berlin, Germany
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Cologne, Germany
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Giessen, Germany
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Hanover, Germany
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Munich, Germany
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Stockholm, Sweden
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Edinburgh, Lothian Region, United Kingdom
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Leeds, West Yorkshire, United Kingdom
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Belfast, United Kingdom
Unknown Facility
London, United Kingdom
Related Publications (3)
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PMID: 37983082DERIVEDSouthern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PMID: 33331662DERIVEDRatjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, Milla CE, Robinson PD, Waltz D, Davies JC; VX14-809-109 investigator group. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med. 2017 Jul;5(7):557-567. doi: 10.1016/S2213-2600(17)30215-1. Epub 2017 Jun 9.
PMID: 28606620DERIVED
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
- 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
August 3, 2015
Study Start
July 1, 2015
Primary Completion
September 1, 2016
Study Completion
September 1, 2016
Last Updated
October 23, 2017
Results First Posted
October 23, 2017
Record last verified: 2017-09