A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
2 other identifiers
interventional
385
11 countries
101
Brief Summary
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Mar 2018
Shorter than P25 for phase_3
101 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
First Submitted
Initial submission to the registry
February 21, 2018
CompletedFirst Posted
Study publicly available on registry
February 27, 2018
CompletedStudy Start
First participant enrolled
March 7, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 5, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
February 5, 2019
CompletedResults Posted
Study results publicly available
March 13, 2020
CompletedMarch 13, 2020
March 1, 2020
11 months
February 21, 2018
February 5, 2020
March 12, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Baseline at Week 4
Secondary Outcomes (12)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline through Week 24
Number of Pulmonary Exacerbations (PEx)
From Baseline through Week 24
Absolute Change in Sweat Chloride (SwCl)
From Baseline through Week 24
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
From Baseline through Week 24
Absolute Change in Body Mass Index (BMI)
From Baseline at Week 24
- +7 more secondary outcomes
Study Arms (2)
Placebo
PLACEBO COMPARATORParticipants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period.
VX-659/TEZ/IVA TC
EXPERIMENTALParticipants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.
Interventions
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Participants received IVA orally once daily in the evening.
Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.
Eligibility Criteria
You may qualify if:
- Heterozygous for F508del and an MF mutation (as defined in the protocol)
- Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height
You may not qualify if:
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (101)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Stanford University
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Hartford Health
Hartford, Connecticut, 06106, United States
Yale New Haven Medical Center
New Haven, Connecticut, 06511, United States
University of Miami/ Miller School of Medicine
Miami, Florida, 33136, United States
Nicklaus Children's Hospital
Miami, Florida, 33155, United States
Arnold Palmer Hospital
Orlando, Florida, 32806, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, 33701, United States
St. Luke's CF Center of Idaho
Boise, Idaho, 83712, United States
Cystic Fibrosis Center of Chicago
Glenview, Illinois, 60025, United States
Advocate Children's Hospital - Park Ridge/ North Suburban Pulmonary and Critical Care Consultants
Niles, Illinois, 60714, United States
Indiana Clinical Research Center, IU Health University Hospital
Indianapolis, Indiana, 46202, United States
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
Kentucky Clinic
Lexington, Kentucky, 40536, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
The Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01655, United States
University of Michigan Health System
Ann Arbor, Michigan, 48109, United States
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan, 49503, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University School of Medicine/ St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Dartmouth Hitchcock Medical Center, Lebanon
Lebanon, New Hampshire, 03756, United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 80901, United States
Albany Medical College
Albany, New York, 12208, United States
Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
Buffalo, New York, 14222, United States
Northwell Health, Long Island Jewish Medical Center
New Hyde Park, New York, 11040, United States
Columbia University Medical Center
New York, New York, 10032, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
Clinical Research of Charlotte
Charlotte, North Carolina, 28277, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Respiratory Diseases of Children and Adolescents
Oklahoma City, Oklahoma, 73112, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Drexel University College of Medicine/ Drexel Adult Cystic Fibrosis Center
Philadelphia, Pennsylvania, 19017, United States
Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
Sanford Research/ USD
Sioux Falls, South Dakota, 57105, United States
University of Tennessee Medical Center - Adult Cystic Fibrosis Clinic
Knoxville, Tennessee, 37920, United States
Children's Foundation Research Center/ Le Bonheur Children's Hospital
Memphis, Tennessee, 38103, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37920, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Providence Pediatric Pulmonary & Allergy/Immunology Clinic
Spokane, Washington, 99204, United States
The Alfred Hospital
Melbourne, Victoria, Australia
Royal Adelaide Hospital
Adelaide, Australia
Prince Charles Hospital
Chermside, Australia
Royal Brisbane & Women's Hospital
Herston, Australia
Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute
New Lambton Heights, Australia
Sydney Children's Hospital, Randwick
Randwick, Australia
Princess Margaret Hospital for Children
Subiaco, Australia
Stollery Children's Hospital
Edmonton, Alberta, Canada
Queen Elizabeth II Health Sciences Center
Halifax, Nova Scotia, Canada
St. Michael's Hospital
Toronto, Canada
Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
Charite Paediatric Pulmonology Department
Berlin, Germany
University Hospital Cologne
Cologne, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Clinic of J.W Goethe University
Frankfurt, Germany
Medizinische Hochschule Hannover
Hanover, Germany
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder - und Jugendmedizin
Jena, Germany
Universitatsklinikum Schleswig-Holstein, Klinik fur Kinder- und Jugendmedizin
Lübeck, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Pneumologische Praxis Pasing
München, Germany
Beaumont Hospital
Dublin, Ireland
Children's University Hospital Temple Street
Dublin, Ireland
Cork University Hospital
Dublin, Ireland
Our Lady's Children's Hospital
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
National University of Ireland
Galway, Ireland
University Hospital Limerick
Limerick, Ireland
Lady Davis Carmel Medical Center
Haifa, Israel
Pediatric Pulmonary Unit Rambam Medical Center
Haifa, Israel
Hadassah Medical Organization
Jerusalem, Israel
Schneider Children's Medical Center
Petah Tikva, Israel
Sheba Medical Center
Tel Litwinsky, Israel
Instytut Matki i Dziecka
Warsaw, Poland
Hospital Universitari Vall d Hebron
Barcelona, Spain
Hospital Universitari Vall d'Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitario Infantil La Paz
Madrid, Spain
Coporacio Sanitaria Parc Tauli
Sabadell, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Hospital Universitario y Politecnico La Fe
Valencia, Spain
Lindenhofspital - Quartier Bleu
Bern, Switzerland
Kinderspital Zuerich
Zurich, Switzerland
Universitaetsspital Zuerich
Zurich, Switzerland
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge, United Kingdom
Queen Elizabeth University Hospital
Glasgow, United Kingdom
The Leeds Teaching Hospitals NHS Trust, St. James University Hospital
Leeds, United Kingdom
Liverpool Heart and Chest Hospital
Liverpool, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, United Kingdom
Wythenshawe Hospital
Manchester, United Kingdom
The Newcastle Upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Nottingham University Hospitals NHS Trust, Queens Medical Center
Nottingham, United Kingdom
University Hospital Llandough
Penarth, United Kingdom
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
- 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
February 21, 2018
First Posted
February 27, 2018
Study Start
March 7, 2018
Primary Completion
February 5, 2019
Study Completion
February 5, 2019
Last Updated
March 13, 2020
Results First Posted
March 13, 2020
Record last verified: 2020-03