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Trial record 6 of 7 for:    VX-659 | Cystic Fibrosis

A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03460990
Recruitment Status : Completed
First Posted : March 9, 2018
Results First Posted : October 17, 2019
Last Update Posted : October 17, 2019
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-659/TEZ/IVA Drug: TEZ/IVA Drug: IVA Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 116 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : May 1, 2018
Actual Primary Completion Date : September 26, 2018
Actual Study Completion Date : October 8, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Active Comparator: TEZ/IVA
Following 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.
Participants received TEZ/IVA orally once daily in the morning.
Other Names:
  • VX-661/VX-770
  • tezacaftor/ivacaftor

Drug: IVA
Participants received IVA orally once daily in the evening.
Other Names:
  • VX-770
  • ivacaftor

Drug: Placebo
Participants received placebo matched to VX-659/TEZ/IVA orally once daily in the morning.

Experimental: VX-659/TEZ/IVA TC
Following 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.
Drug: VX-659/TEZ/IVA
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Other Names:
  • VX-659/VX-661/VX-770
  • VX-659/tezacaftor/ivacaftor

Drug: IVA
Participants received IVA orally once daily in the evening.
Other Names:
  • VX-770
  • ivacaftor

Drug: Placebo
Participants received placebo matched TEZ/IVA orally once daily in the morning.

Primary Outcome Measures :
  1. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline at Week 4 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Secondary Outcome Measures :
  1. Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline at Week 4 ]
    Sweat samples were collected using an approved collection device.

  2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score [ Time Frame: From Baseline at Week 4 ]
    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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  3. Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 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) ]
  4. Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA [ Time Frame: From Day 1 and Week 4 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • 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

Other protocol defined Inclusion/Exclusion criteria may apply

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03460990

Hide Hide 46 study locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, Connecticut
Yale New Haven Medical Center
New Haven, Connecticut, United States, 06511
United States, Florida
University of Miami/Miller School of Medicine
Miami, Florida, United States, 33136
United States, Idaho
St. Luke's CF Center of Idaho
Boise, Idaho, United States, 83712
United States, Illinois
Advocate Children's Hospital- Park Ridge/ North Suburban Pulmonary and Critical Care Consultants
Niles, Illinois, United States, 60714
United States, Indiana
Indiana Clinical Research Center, IU Health University Hospital
Indianapolis, Indiana, United States, 46202
United States, Iowa
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Maryland
The Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan, United States, 49503
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Washington University School of Medicine / St. Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States, 08901
United States, New York
Albany Medical College
Albany, New York, United States, 12208
Northwell Health, Long Island Jewish Medical Center
New Hyde Park, New York, United States, 11040
Columbia University Medical Center
New York, New York, United States, 10032
SUNY Upstate Medical University
Syracuse, New York, United States, 13210
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Oklahoma
Respiratory Diseases of Children and Adolescents
Oklahoma City, Oklahoma, United States, 73211
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15224
United States, South Dakota
Sanford Research/ USD
Sioux Falls, South Dakota, United States, 57105
United States, Tennessee
University of Tennessee Medical Center-Adult Cystic Fibrosis Clinic
Knoxville, Tennessee, United States, 37920
Children's Foundation Research Center/ Le Bonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Utah
University of Utah/ Primary Children's Medical Center
Salt Lake City, Utah, United States, 84132
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Australia, Victoria
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
Muenchen, 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
Sevilla, Spain
United Kingdom
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
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] April 27, 2018
Statistical Analysis Plan  [PDF] June 7, 2018

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03460990    
Other Study ID Numbers: VX17-659-103
2017-004133-82 ( EudraCT Number )
First Posted: March 9, 2018    Key Record Dates
Results First Posted: October 17, 2019
Last Update Posted: October 17, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action