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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
NCT04058366
Collaborator
(none)
251
Enrollment
84
Locations
1
Arm
31.9
Anticipated Duration (Months)
3
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will evaluate the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
251 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 Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Actual Study Start Date :
Dec 5, 2019
Anticipated Primary Completion Date :
Aug 1, 2022
Anticipated Study Completion Date :
Aug 1, 2022

Arms and Interventions

Arm Intervention/Treatment
Experimental: Triple Combination Arm

Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening.

Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor

    • Drug: IVA
    150-mg tablet for oral administration.
    Other Names:
  • VX-770
  • ivacaftor

    		•

    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 up to Week 100]

    Secondary Outcome Measures

    1. Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [From Baseline up to Week 96]

    2. Absolute change in sweat chloride (SwCl) [From Baseline up to Week 96]

    3. Absolute change in body mass index (BMI) [From Baseline up to Week 96]

    4. Absolute change in BMI z-score [From Baseline up to Week 96]

    5. Absolute change in body weight [From Baseline up to Week 96]

    6. Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [From Baseline up to Week 96]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:
    • Completed study drug treatment in parent study (VX18-445-104); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
    Key Exclusion Criteria:
    • History of study drug intolerance in 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 Miller Children's Hospital / Long Beach Memorial Long Beach California United States 90806
    3 Stanford University Palo Alto California United States 94304
    4 University of California Davis Medical Center Sacramento California United States 95817
    5 University of California San Francisco, Lung Transplant Program San Francisco California United States 94143
    6 National Jewish Health Denver Colorado United States 80206
    7 University of Miami Miller School of Medicine Miami Florida United States 33136
    8 Central Florida Pulmonary Group, PA Orlando Florida United States 32803
    9 Indiana University Indianapolis Indiana United States 46202
    10 University of Kansas Medical Center Kansas City Kansas United States 66160
    11 University of Kentucky Lexington Kentucky United States 40536
    12 Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center Boston Massachusetts United States 02114
    13 Boston Children's Hospital Boston Massachusetts United States 02115
    14 Michigan Medicine Ann Arbor Michigan United States 48109-5212
    15 University of Minnesota Minneapolis Minnesota United States 55455
    16 Washington University School of Medicine / St. Louis Children's Hospital Saint Louis Missouri United States 63110
    17 Mount Sinai Beth Israel New York New York United States 10003
    18 Columbia University Medical Center New York New York United States 10032
    19 University of North Carolina Hospitals Chapel Hill North Carolina United States 27514
    20 UC Health Holmes Cincinnati Ohio United States 45220
    21 Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center Cleveland Ohio United States 44106
    22 Nationwide Children's Hospital Columbus Ohio United States 43205
    23 ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center Toledo Ohio United States 43606
    24 University of Oklahoma Health Sciences Center Oklahoma City Oklahoma United States 73104
    25 Oregon Health & Science University Portland Oregon United States 97239
    26 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
    27 Children's Hospital of Pittsburgh of UPMC Pittsburgh Pennsylvania United States 15224
    28 Dell Children's Medical Group Austin Texas United States 78723
    29 The University of Texas Southwestern Medical Center Dallas Texas United States 75390
    30 Texas Children's Hospital Houston Texas United States 77030
    31 University of Utah / Primary Children's Medical Center Salt Lake City Utah United States 84132
    32 University of Washington Medical Center Seattle Washington United States 98195
    33 University of Wisconsin Hospital and Clinics Madison Wisconsin United States 53792
    34 The Prince Charles Hospital Chermside Australia
    35 Alfred Hospital Melbourne Australia
    36 Telethon Kids Institute Nedlands Australia
    37 The Royal Children's Hospital Parkville Australia
    38 Mater Adult Hospital South Brisbane Australia
    39 Queensland Children's Hospital South Brisbane Australia
    40 Westmead Hospital Westmead Australia
    41 Cliniques Universitaires de Bruxelles Hopital Erasme Brussels Belgium
    42 Universitair Ziekenhuis Brussel - Campus Jette Brussels Belgium
    43 Universitair Ziekenhuis Gent Gent Belgium
    44 Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg Leuven Belgium
    45 McGill University Health Center Montréal Canada
    46 St. Paul's Hospital Vancouver Canada
    47 Juliane Marie Center, Rigshospitalet Copenhagen Denmark
    48 Centre Hospitalier Lyon Sud Benite Cedex France
    49 Groupe Hospitaler Pellegrin, CHU De Bordeaux Bordeaux cedex France
    50 CHRU de Lille - Hopital Albert Calmette Lille France
    51 CHU Marseille - Hopital Nord Marseille France
    52 CHU de Montpellier - Hopital Arnaud de Villeneuve Montpellier Cedex 5 France
    53 Hopital Necker, Enfants Malades Paris Cedex 15 France
    54 Hopital Cochin Paris France
    55 Hopital Pontchaillou CHU de Rennes Rennes Cedex France
    56 Charite Paediatric Pulmonology Department Berlin Germany
    57 Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen Essen Germany
    58 Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin Giessen Germany
    59 Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie Halle Germany
    60 Pneumologisches Studienzentrum Muenchen-West Muenchen Germany
    61 University Hospital Wuerzburg Würzburg Germany
    62 Beaumont Hospital Dublin Ireland
    63 Children's Health Ireland at Crumlin Dublin Ireland
    64 St. Vincent's University Hospital Dublin Ireland
    65 University Hospital Limerick (Adults) Limerick Ireland
    66 Azienda Ospedaliero Universitaria Ospedale Riuniti Ancona Italy
    67 IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico Genova Italy
    68 Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milano Italy
    69 Ospedale Pediatrico Bambino Gesu Rome Italy
    70 Azienda Ospedaliera di Verona-Ospedale Civile Maggiore Verona Italy
    71 Academic Medical Center Amsterdam Netherlands
    72 University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis Heidelberglaan Netherlands
    73 Hospital Universitari Vall d´Hebron Servicio de Broncoscopia Barcelona Spain
    74 Hospital Virgen de la Arrixaca Murcia Spain
    75 Hospital Universitario Virgen del Rocio Sevilla Spain
    76 Hospital Universitario y Politecnico La Fe Valencia Spain
    77 Papworth Hospital NHS Foundation Trust, Papworth Everard Cambridge United Kingdom
    78 Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital Exeter United Kingdom
    79 Clinical Research Facility, Queen Elizabeth University Hospital Glasgow United Kingdom
    80 St. James University Hospital Leeds United Kingdom
    81 Liverpool Heart and Chest Hospital Liverpool United Kingdom
    82 Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital London United Kingdom
    83 Wythenshawe Hospital Manchester United Kingdom
    84 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:
    NCT04058366
    Other Study ID Numbers:
    • VX18-445-110
    • 2019-000833-37
    First Posted:
    Aug 15, 2019
    Last Update Posted:
    Jan 12, 2022
    Last Verified:
    Jan 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    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
    Elexacaftor

    Study Results

    No Results Posted as of Jan 12, 2022