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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)

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ClinicalTrials.gov Identifier: NCT03447249
Recruitment Status : Completed
First Posted : February 27, 2018
Last Update Posted : March 8, 2019
Sponsor:
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 heterozygous for F508del and a minimal function mutation (F/MF subjects)

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 385 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
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 Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Actual Study Start Date : March 7, 2018
Actual Primary Completion Date : February 5, 2019
Actual Study Completion Date : February 5, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Triple Combination
Subjects will receive 240 mg VX-659 / 100 mg TEZ / 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
Drug: VX-659
Fixed-dose combination (FDC) tablet (VX-659/TEZ/IVA)

Drug: TEZ
FDC tablet (VX-659/TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (VX-659/TEZ/IVA)
Other Name: ivacaftor; VX-770

Drug: IVA
IVA tablet
Other Name: ivacaftor; VX-770

Placebo Comparator: Placebo Drug: Matched Placebos
Placebo will be used as a comparator.




Primary Outcome Measures :
  1. Absolute change in ppFEV1 [ Time Frame: from baseline at Week 4 ]

Secondary Outcome Measures :
  1. Absolute change in ppFEV1 [ Time Frame: from baseline through Week 24 ]
  2. Number of pulmonary exacerbations (PEx) [ Time Frame: through Week 24 ]
  3. Absolute change in body mass index (BMI) [ Time Frame: from baseline at Week 24 ]
  4. Absolute change in Sweat Chloride (SwCl) [ Time Frame: from baseline at Week 4 and through Week 24 ]
  5. Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Week 4 and through Week 24 ]
  6. Time-to-first PEx [ Time Frame: through Week 24 ]
  7. Absolute change in BMI z score [ Time Frame: from baseline at Week 24 ]
  8. Absolute change in body weight [ Time Frame: from baseline at Week 24 ]
  9. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through 4-week safety follow-up (up to 28 weeks) ]
  10. Observed pre-dose concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through Week 16 ]


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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
Criteria

Key Inclusion Criteria:

  • 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

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): NCT03447249


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Locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, California
Stanford University
Palo Alto, California, United States, 94304
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Connecticut
Hartford Health
Hartford, Connecticut, United States, 06106
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
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Arnold Palmer Hospital
Orlando, Florida, United States, 32806
Johns Hopkins All Children's Hospital Outpatient Care Center
Saint Petersburg, Florida, United States, 33701
United States, Idaho
St. Luke's CF Center of Idaho
Boise, Idaho, United States, 83712
United States, Illinois
Cystic Fibrosis Center of Chicago
Glenview, Illinois, United States, 60025
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, Kentucky
Kentucky Clinic
Lexington, Kentucky, United States, 40536
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, United States, 40202
United States, Maryland
The Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States, 01655
United States, Michigan
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Helen DeVos Children's Hospital CF Center
Grand Rapids, Michigan, United States, 49503
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
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 Hampshire
Dartmouth Hitchcock Medical Center, Lebanon
Lebanon, New Hampshire, United States, 03756
United States, New Jersey
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States, 80901
United States, New York
Albany Medical College
Albany, New York, United States, 12208
Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
Buffalo, New York, United States, 14222
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, North Carolina
Clinical Research of Charlotte
Charlotte, North Carolina, United States, 28277
Duke University Medical Center
Durham, North Carolina, United States, 27710
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, 73112
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Drexel University College of Medicine/ Drexel Adult Cystic Fibrosis Center
Philadelphia, Pennsylvania, United States, 19017
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, 37920
United States, Texas
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
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
Providence Pediatric Pulmonary & Allergy/Immunology Clinic
Spokane, Washington, United States, 99204
Australia, Victoria
The Alfred Hospital
Melbourne, Victoria, Australia
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
Canada, Alberta
Stollery Children's Hospital
Edmonton, Alberta, Canada
Canada, Nova Scotia
Queen Elizabeth II Health Sciences Center
Halifax, Nova Scotia, Canada
Canada
St. Michael's Hospital
Toronto, Canada
Denmark
Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
Germany
Charite Paediatric Pulmonology Department
Berlin, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Clinic of J.W Goethe University
Frankfurt, Germany
Medizinische Hochschule Hannover
Hannover, Germany
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder - und Jugendmedizin
Jena, Germany
University Hospital Cologne
Koeln, Germany
Universitatsklinikum Schleswig-Holstein, Klinik fur Kinder- und Jugendmedizin
Lubeck, Germany
Pneumologische Praxis Pasing
Muenchen, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Ireland
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
Israel
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 HaShomer, Israel
Poland
Instytut Matki i Dziecka
Warsaw, Poland
Spain
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
Sevilla, Spain
Hospital Universitario y Politecnico La Fe
Valencia, Spain
Switzerland
Lindenhofspital - Quartier Bleu
Bern, Switzerland
Kinderspital Zuerich
Zürich, Switzerland
Universitaetsspital Zuerich
Zürich, Switzerland
United Kingdom
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
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03447249     History of Changes
Other Study ID Numbers: VX17-659-102
2017-004132-11 ( EudraCT Number )
First Posted: February 27, 2018    Key Record Dates
Last Update Posted: March 8, 2019
Last Verified: March 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
Fibrosis
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