Clincosm LogoSign in Get a demo

A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03525574
Collaborator
(none)
507
Enrollment
110
Locations
1
Arm
50.8
Anticipated Duration (Months)
4.6
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will evaluate the long-term safety and tolerability of VX-445 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
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
507 participants
Allocation:
N/A
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-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
Actual Study Start Date :
Oct 9, 2018
Anticipated Primary Completion Date :
Jan 1, 2023
Anticipated Study Completion Date :
Jan 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: Open-label Triple Combination

Subjects will receive 200 mg VX-445/ 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-445 in combination with TEZ and IVA. This includes Studies VX17-445-102 and VX17-445-103.

Drug: VX-445
Fixed-dose combination (FDC) tablets (VX-445/TEZ/IVA)

Drug: TEZ
FDC tablets (VX-445/TEZ/IVA)
Other Names:
  • tezacaftor; VX-661

    • Drug: IVA
    FDC tablets (VX-445/TEZ/IVA)
    Other Names:
  • ivacaftor; VX-770

    • Drug: IVA
    IVA tablet
    Other Names:
  • ivacaftor; VX-770

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [from baseline through safety follow-up (up to 196 weeks)]

    Secondary Outcome Measures

    1. Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) [from baseline through last dose of study drug (up to 192 weeks)]

    2. Absolute change in sweat chloride (SwCl) [from baseline through last dose of study drug (up to 192 weeks)]

    3. Number of pulmonary exacerbations (PEx) [from baseline through last dose of study drug (up to 192 weeks)]

    4. Time to first PEx [from baseline through last dose of study drug (up to 192 weeks)]

    5. Absolute change in body mass index (BMI) [from baseline through last dose of study drug (up to 192 weeks)]

    6. Absolute change in BMI z-score [from baseline through last dose of study drug (up to 192 weeks)]

    7. Absolute change in body weight [from baseline through last dose of study drug (up to 192 weeks)]

    8. Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [from baseline through last dose of study drug (up to 192 weeks)]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Banner University of Arizona Medical Center Tucson Arizona United States 85724
    2 University of Arkansas for Medical Sciences Little Rock Arkansas United States 72205
    3 Miller Children's Hospital / Long Beach Memorial Long Beach California United States 90806
    4 Children's Hospital Los Angeles Los Angeles California United States 90027
    5 Valley Children's Hospital Madera California United States 93636
    6 Kaiser Permanente Oakland California United States 94611
    7 University of California Davis Medical Center Sacramento California United States 95817
    8 UCSF Gateway Medical Center San Francisco California United States 94143
    9 National Jewish Health Denver Colorado United States 80206
    10 University of Florida, Shands Hospital Gainesville Florida United States 32610
    11 Joe DiMaggio Cystic Fibrosis & Pulmonary Center Hollywood Florida United States 33021
    12 Nemours Children's Specialty Care Jacksonville Florida United States 32207
    13 Central Florida Pulmonary Group, PA Orlando Florida United States 32803
    14 Nemours Children's Hospital Orlando Florida United States 32827
    15 Tampa General Hospital Cardiac and Lung Transplant Clinic Tampa Florida United States 33606
    16 Children's Specialty Services at North Druid Hills Atlanta Georgia United States 30324
    17 Augusta University Augusta Georgia United States 30912
    18 Ann & Robert Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    19 Northwestern Memorial Hospital Chicago Illinois United States 60611
    20 Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center Peoria Illinois United States 61637
    21 The University of Kansas Medical Center Kansas City Kansas United States 66160
    22 Tulane Medical Center New Orleans Louisiana United States 70112
    23 Maine Medical Partners Portland Maine United States 04102
    24 Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center Boston Massachusetts United States 02114
    25 Harper University Hospital Detroit Michigan United States 48201
    26 Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota United States 55404
    27 Billings Clinic Hospital Billings Montana United States 59101
    28 Nebraska Medical Center Omaha Nebraska United States 68198
    29 Morristown Medical Center Morristown New Jersey United States 07960
    30 UNM Clinical and Translational Science Center Albuquerque New Mexico United States 87131
    31 The Cystic Fibrosis Center, Mount Sinai Beth Israel New York New York United States 10003
    32 New York Medical College Valhalla New York United States 10595
    33 UNC Marsico Clinical Research Center Chapel Hill North Carolina United States 27517
    34 Akron Children's Hospital Akron Ohio United States 44308
    35 UC Health Holmes Cincinnati Ohio United States 45220
    36 Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center Cleveland Ohio United States 44106
    37 Nationwide Children's Hospital Columbus Ohio United States 43205
    38 Dayton Children's Hospital Dayton Ohio United States 45404
    39 ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center Toledo Ohio United States 43606
    40 University of Oklahoma Health Sciences Center Oklahoma City Oklahoma United States 73104
    41 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    42 Medical University of South Carolina Charleston South Carolina United States 29425
    43 Dell Children's Medical Center of Central Texas Austin Texas United States 78723
    44 The University of Texas Southwestern Medical Center Dallas Texas United States 75390
    45 Vermont Lung Center Colchester Vermont United States 05446
    46 University of Virginia Primary Care Center Charlottesville Virginia United States 22908
    47 Children's Hospital of the King's Daughters Norfolk Virginia United States 23507
    48 Children's Hospital of Richmond at VCU, Children's Pavilion Richmond Virginia United States 23298
    49 University of Washington Medical Center Seattle Washington United States 98195
    50 West Virginia University Morgantown West Virginia United States 26506
    51 University of Wisconsin Hospitals and Clinics Madison Wisconsin United States 53792
    52 CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital Milwaukee Wisconsin United States 53226
    53 Women & Children's Hospital North Adelaide Australia
    54 The Royal Children's Hospital Parkville Australia
    55 Mater Misericordiae Ltd South Brisbane Australia
    56 The Children's Hospital at Westmead Westmead Australia
    57 Westmead Hospital Westmead Australia
    58 University of Graz Graz Austria
    59 Medizinische Universität Innsbruck Innsbruck Austria
    60 LKH - Universitätsklinikum der PMU Salzburg Salzburg Austria
    61 Medizinische Universitat Wien Vienna Austria
    62 Cliniques Universitaires de Bruxelles Hopital Erasme Brussels Belgium
    63 Universitair Ziekenhuis Brussel - Campus Jette Brussels Belgium
    64 UZ Antwerpen Edegem Belgium
    65 Universitair Ziekenhuis Gent Gent Belgium
    66 Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg Leuven Belgium
    67 University of Calgary Medical Clinic of the Foothills Medical Centre Calgary Canada
    68 McGill University Health Centre, Glen Site, Montreal Children's Hospital Montreal Canada
    69 Centre Hospitalier De L'Universite Laval Quebec City Canada
    70 The Hospital for Sick Children Toronto Canada
    71 British Columbia's Children's Hospital Vancouver Canada
    72 St. Paul's Hospital Vancouver Canada
    73 Fakultni Nemocnice Brno Brno Czechia
    74 Fakultni nemocnice v Motole Praha 5 Czechia
    75 Centre Hospitalier Lyon Sud Benite Cedex France
    76 Groupe Hospitaler Pellegrin, CHU De Bordeaux Bordeaux cedex France
    77 CHU Marseille - Hopital Nord Marseille France
    78 CHU de Nice - Hopital Pasteur Nice France
    79 Hopital Cochin Paris France
    80 CHU de Rouen - Hopital Charles Nicolle Rouen France
    81 Hopital Foch (Suresnes), Hopital Foch, Adultes Suresnes France
    82 Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital Erlangen Germany
    83 Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin Giessen Germany
    84 Hannover Medical School Hannover Germany
    85 Johannes Gutenberg-Universitaet Mainz Germany
    86 Dr. von Haunersches Kinderspital München Germany
    87 Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin Tuebingen Germany
    88 University Hospital Wuerzburg Wurzburg Germany
    89 General Hospital of Attika "Sismanoglio"(Adult CF center, NHS) Maroussi Greece
    90 Azienda Ospedaliero Universitaria Ospendali Riuniti Ancona Italy
    91 Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer Firenze Italy
    92 IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico Genova Italy
    93 Azienda Ospedaliera Universitaria Policlinico G. Martino Messina Italy
    94 Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano Italy
    95 Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo Potenza Italy
    96 Azienda Ospedaliera di Verona-Ospedale Civile Maggiore Verona Italy
    97 Academic Medical Center Amsterdam Netherlands
    98 HagaZiekenhuis van den Haag Den Haag Netherlands
    99 University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis Heidelberglaan Netherlands
    100 UMC St. Radboud Nijmegen Netherlands
    101 Erasmus Medical Center Rotterdam Netherlands
    102 Karolinska Universitetssjukhuset, Huddinge Stockholm Sweden
    103 Belfast City Hospital Belfast United Kingdom
    104 Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital Birmingham United Kingdom
    105 Western General Hospital Edinburgh United Kingdom
    106 Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital Exeter United Kingdom
    107 Leeds General Infirmary Leeds United Kingdom
    108 King's College Hospital London United Kingdom
    109 Royal Manchester Children's Hospital Manchester United Kingdom
    110 Southampton General Hospital Southampton United Kingdom

    Sponsors and Collaborators

    • Vertex Pharmaceuticals Incorporated

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Vertex Pharmaceuticals Incorporated
    ClinicalTrials.gov Identifier:
    NCT03525574
    Other Study ID Numbers:
    • VX17-445-105
    • 2018-000185-11
    First Posted:
    May 15, 2018
    Last Update Posted:
    Jul 27, 2022
    Last Verified:
    Jul 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor

    Study Results

    No Results Posted as of Jul 27, 2022