A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
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ClinicalTrials.gov Identifier: NCT03460990 |
Recruitment Status :
Completed
First Posted : March 9, 2018
Results First Posted : October 17, 2019
Last Update Posted : October 17, 2019
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Condition or disease | Intervention/treatment | Phase |
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Cystic Fibrosis | Drug: VX-659/TEZ/IVA Drug: TEZ/IVA Drug: IVA Drug: Placebo | Phase 3 |
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 |

Arm | Intervention/treatment |
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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.
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Drug: TEZ/IVA
Participants received TEZ/IVA orally once daily in the morning.
Other Names:
Drug: IVA Participants received IVA orally once daily in the evening.
Other Names:
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.
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Drug: VX-659/TEZ/IVA
Participants received VX-659/TEZ/IVA orally once daily in the morning.
Other Names:
Drug: IVA Participants received IVA orally once daily in the evening.
Other Names:
Drug: Placebo Participants received placebo matched TEZ/IVA orally once daily in the morning. |
- 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.
- Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline at Week 4 ]Sweat samples were collected using an approved collection device.
- 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.
- 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) ]
- Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA [ Time Frame: From Day 1 and Week 4 ]

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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

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

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 | |
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 | |
Germany | |
Charite Paediatric Pulmonology Department | |
Berlin, Germany | |
Pneumologische Praxis Pasing | |
Muenchen, Germany | |
Ireland | |
Cork University Hospital | |
Dublin, Ireland | |
Our Lady's Children's Hospital | |
Dublin, Ireland | |
St. Vincent's University Hospital | |
Dublin, Ireland | |
Spain | |
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 |
Documents provided by Vertex Pharmaceuticals Incorporated:
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Cystic Fibrosis Fibrosis VX-659 Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases |
Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Ivacaftor Chloride Channel Agonists Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |