A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
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 Homozygous for the F508del Mutation (F/F)
2 other identifiers
interventional
116
6 countries
46
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 homozygous for the F508del mutation (F/F).
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 May 2018
Shorter than P25 for phase_3
46 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
March 1, 2018
CompletedFirst Posted
Study publicly available on registry
March 9, 2018
CompletedStudy Start
First participant enrolled
May 1, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 26, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
October 8, 2018
CompletedResults Posted
Study results publicly available
October 17, 2019
CompletedOctober 17, 2019
September 1, 2019
5 months
March 1, 2018
September 26, 2019
September 26, 2019
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 (4)
Absolute Change in Sweat Chloride (SwCl)
From Baseline at Week 4
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
From Baseline at Week 4
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8)
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA
From Day 1 and Week 4
Study Arms (2)
TEZ/IVA
ACTIVE COMPARATORFollowing a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period.
VX-659/TEZ/IVA TC
EXPERIMENTALFollowing a run-in period of 4 weeks with TEZ/IVA, participants received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period.
Interventions
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Participants received TEZ/IVA orally once daily in the morning.
Participants received IVA orally once daily in the evening.
Participants received placebo matched TEZ/IVA orally once daily in the morning.
Eligibility Criteria
You may qualify if:
- Homozygous for the F508del mutation (F/F)
- 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 (46)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, 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
St. Luke's CF Center of Idaho
Boise, Idaho, 83712, 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
The Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan, 49503, 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
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
Albany Medical College
Albany, New York, 12208, 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
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Respiratory Diseases of Children and Adolescents
Oklahoma City, Oklahoma, 73211, United States
Oregon Health & Science University
Portland, Oregon, 97239, 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, 37232, 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
The Alfred Hospital
Melbourne, Victoria, Australia
Royal Adelaide Hospital
Adelaide, Australia
Prince Charles Hospital
Chermside, Australia
Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute
New Lambton Heights, Australia
Charite Paediatric Pulmonology Department
Berlin, Germany
Pneumologische Praxis Pasing
München, Germany
Cork University Hospital
Dublin, Ireland
Our Lady's Children's Hospital
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
Hospital Universitari Vall d Hebron
Barcelona, Spain
Hospital Universitari Vall d'Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge, 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
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
- 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
March 1, 2018
First Posted
March 9, 2018
Study Start
May 1, 2018
Primary Completion
September 26, 2018
Study Completion
October 8, 2018
Last Updated
October 17, 2019
Results First Posted
October 17, 2019
Record last verified: 2019-09