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A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

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: NCT03447262
Recruitment Status : Active, not recruiting
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 long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 484 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
Actual Study Start Date : July 13, 2018
Estimated Primary Completion Date : April 24, 2021
Estimated Study Completion Date : April 24, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Open-label 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.

Parent studies are Phase 3 Vertex studies investigating VX-659 in combination with TEZ and IVA. This includes Studies VX17-659-102 and VX17-659-103.

Drug: VX-659
Fixed-dose combination (FDC) tablets (VX-659/TEZ/IVA)

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

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

Drug: IVA
IVA tablet
Other Name: ivacaftor




Primary Outcome Measures :
  1. Safety and tolerability of long-term treatment with VX-659 in TC with TEZ and IVA based on adverse events (AEs) [ Time Frame: from baseline through safety follow-up (up to 100 weeks) ]

Secondary Outcome Measures :
  1. Absolute change from baseline in ppFEV1 [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  2. Absolute change in sweat chloride (SwCl) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  3. Number of pulmonary exacerbations (PEx) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  4. Time to first PEx [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  5. Absolute change in body mass index (BMI) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  6. Absolute change in BMI z-score [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  7. Absolute change in body weight [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  8. Absolute change from baseline in CFQ-R respiratory domain score [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]


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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

Inclusion Criteria:

  • Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.

Exclusion Criteria:

  • History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
  • Current participation in an investigational drug trial (other than a parent study)

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): NCT03447262


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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 Hospital
Hartford, Connecticut, United States, 06102
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
Orlando Health, Inc.- Arnold Palmer Hospital for Children (APH)
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
University of Kentucky
Lexington, Kentucky, United States, 40536
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, United States, 40202
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
UMass Memorial Medical Center
Worcester, Massachusetts, United States, 01655
United States, Michigan
Michigan Medicine
Ann Arbor, Michigan, United States, 48109-5212
Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
Grand Rapids, Michigan, United States, 49546
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
The 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, Manchester
Manchester, New Hampshire, United States, 03104
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
CF Therapeutics Development Center of Western New York
Buffalo, New York, United States, 14203
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
Cincinnati, Ohio, United States, 45229
United States, Oklahoma
Santiago Reyes, M.D.
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, 19107
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, South Dakota
Sanford Children's Specialty Clinic
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
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Texas Children's Hospital
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 & Cystic Fibrosis Clinic
Spokane, Washington, United States, 99204
Australia
Royal Adelaide Hospital
Adelaide, Australia
The Prince Charles Hospital
Chermside, Australia
Alfred Hospital
Melbourne, Australia
Institute for Respiratory Health, Sir Charles Gairdner Hospital
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
New Lambton, Australia
Telethon Kids Institute
Perth, Australia
Sydney Children's Hospital
Randwick, Australia
Lady Cilento Children's Hospital
South Brisbane, Australia
Canada
Stollery Children's Hospital
Edmonton, Canada
Queen Elizabeth II Health Sciences Center
Halifax, 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
Universitaetsklinkum Koeln, CF-Studienzentrum
Koeln, Germany
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
Lubeck, Germany
Pneumologische Praxis Pasing
Muenchen, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Ireland
Cork University Hospital
Cork, Ireland
Beaumont Hospital
Dublin, Ireland
Our Lady's Children's Hospital
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
Temple Street Children's University Hospital
Dublin, Ireland
University Hospital Galway
Galway, Ireland
University Hospital Limerick
Limerick, Ireland
Israel
Lady Davis Carmel Medical Center
Haifa, Israel
Rambam Health Care Campus, Liver Unit
Haifa, Israel
Pediatrics Hadassah Medical Center
Jerusalem, Israel
Schneider Children's Medical Center
Petah Tikva, Israel
Sheba Medical Center
Tel HaShomer, Israel
Poland
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Łomianki, 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
Parc Tauli Sabadell Hospital Universitari
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
United Kingdom
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
St. James University Hospital
Leeds, United Kingdom
Liverpool Head and Chest Hospital
Liverpool, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, United Kingdom
Wythenshaw e Hospital
Manchester, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Newcastle Upon Tyne, United Kingdom
Wolfson Cystic Fibrosis Unit, City Campus
Nottingham, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03447262     History of Changes
Other Study ID Numbers: VX17-659-105
2017-004134-29 ( 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