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

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04043806
Collaborator
(none)
458
Enrollment
97
Locations
1
Arm
46.7
Anticipated Duration (Months)
4.7
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 elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA).

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
458 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 of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
Actual Study Start Date :
Aug 9, 2019
Anticipated Primary Completion Date :
Jul 1, 2023
Anticipated Study Completion Date :
Jul 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: Open-Label Triple Combination

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 through Week 100]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Currently participating in study VX17-659-105 (NCT03447262)
    Exclusion Criteria:

    • History of drug intolerance in study VX17-659-105 that would pose an additional risk to the subject in the opinion of the investigator

    • Current participation in an investigational drug trial (other than study VX17-659-105)

    Other protocol defined Inclusion/Exclusion criteria may apply

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of Alabama at Birmingham Birmingham Alabama United States 35233
    2 Stanford University Palo Alto California United States 94304
    3 Children's Hospital Colorado Aurora Colorado United States 80045
    4 Hartford Hospital Hartford Connecticut United States 06102
    5 Yale New Haven Hospital New Haven Connecticut United States 06510
    6 University of Miami Miller School of Medicine Miami Florida United States 33136
    7 Nicklaus Children's Hospital Miami Florida United States 33155
    8 Arnold Palmer Hospital Pulmonary and Sleep Medicine Orlando Florida United States 32806
    9 Johns Hopkins All Children's Hospital Outpatient Care Center Saint Petersburg Florida United States 33701
    10 St. Luke's CF Center of Idaho Boise Idaho United States 83702
    11 Cystic Fibrosis Institute Glenview Illinois United States 60025
    12 Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants Niles Illinois United States 60714
    13 Indiana University Indianapolis Indiana United States 46202
    14 The University of Iowa Hospitals and Clinics Iowa City Iowa United States 52242
    15 University of Kentucky Lexington Kentucky United States 40536
    16 Kosair Charities Pediatric Clinical Research Unit Louisville Kentucky United States 40202
    17 Johns Hopkins Hospital Baltimore Maryland United States 21287
    18 Boston Children's Hospital Boston Massachusetts United States 02115
    19 UMass Memorial Medical Center Worcester Massachusetts United States 01655
    20 Michigan Medicine Ann Arbor Michigan United States 48109-5212
    21 University of Minnesota Minneapolis Minnesota United States 55455
    22 University of Mississippi Medical Center Jackson Mississippi United States 39216
    23 The Children's Mercy Hospital Kansas City Missouri United States 64108
    24 Washington University School of Medicine / St. Louis Children's Hospital Saint Louis Missouri United States 63110
    25 Dartmouth Hitchcock Medical Center Lebanon New Hampshire United States 03756
    26 Rutgers Robert Wood Johnson Medical School New Brunswick New Jersey United States 08901
    27 Albany Medical College, Pulmonary and Critical Care Medicine Albany New York United States 12208
    28 Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo Buffalo New York United States 14222
    29 Northwell Health- Long Island Jewish Medical Center New Hyde Park New York United States 11040
    30 Columbia University Medical Center New York New York United States 10032
    31 SUNY Upstate Medical University Syracuse New York United States 13210
    32 Clinical Research of Charlotte Charlotte North Carolina United States 28277
    33 Duke University Medical Center Durham North Carolina United States 27710
    34 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    35 Santiago Reyes, M.D. Oklahoma City Oklahoma United States 73112
    36 Oregon Health & Science University Portland Oregon United States 97239
    37 Children's Hospital of Pittsburgh of UPMC Pittsburgh Pennsylvania United States 15224
    38 Sanford Children's Speciality Clinic Sioux Falls South Dakota United States 57105
    39 University of Tennessee Medical Center Knoxville Tennessee United States 37920
    40 Children's Foundation Research Center / Le Bonheur Children's Hospital Memphis Tennessee United States 38103
    41 Vanderbilt University Medical Center Nashville Tennessee United States 37232
    42 Cook Children's Medical Center Fort Worth Texas United States 76104
    43 Texas Children's Hospital Houston Texas United States 77030
    44 University of Utah / Primary Children's Medical Center Salt Lake City Utah United States 84132
    45 Seattle Children's Hospital Seattle Washington United States 98105
    46 Providence Pediatric Pulmonary & Cystic Fibrosis Clinic Spokane Washington United States 99204
    47 Royal Adelaide Hospital Adelaide Australia
    48 The Prince Charles Hospital Chermside Australia
    49 Alfred Hospital Melbourne, VIC Australia
    50 Institute for Respiratory Health Nedlands Australia
    51 Perth Children's Hospital Nedlands Australia
    52 John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital New Lambton Australia
    53 Queensland Children's Hospital South Brisbane Australia
    54 Stollery Children's Hospital Edmonton Canada
    55 Queen Elizabeth II Health Sciences Center Halifax Canada
    56 St. Michael's Hospital Toronto Canada
    57 Juliane Marie Center, Rigshospitalet Copenhagen Denmark
    58 Charite Paediatric Pulmonology Department Berlin Germany
    59 Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen Essen Germany
    60 Johann Wolfgang Goethe University Frankfurt Germany
    61 Hannover Medical School Hannover Germany
    62 Universitaetsklinikum Jena, Mukoviszidose-Zentrum Jena Germany
    63 Universitaetsklinkum Koeln, CF-Studienzentrum Koeln Germany
    64 Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin Lübeck Germany
    65 Pneumologisches Studienzentrum Muenchen-West Muenchen Germany
    66 Klinikum Innenstadt, University of Munich München Germany
    67 Cork University Hospital Cork Ireland
    68 Beaumont Hospital Dublin Ireland
    69 Children's Health Ireland at Crumlin Dublin Ireland
    70 Children's Health Ireland at Temple Street Dublin Ireland
    71 St. Vincent's University Hospital Dublin Ireland
    72 University Hospital Galway Galway Ireland
    73 University Hospital Limerick (Adults) Limerick Ireland
    74 Lady Davis Carmel Medical Center Haifa Israel
    75 Pediatric Pulmonary Unit Rambam Medical Center Haifa Israel
    76 Hadassah Medical Organization Jerusalem Israel
    77 Schneider Children's Medical Center Petah Tikva Israel
    78 Sheba Medical Center Tel Hashomer Israel
    79 Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY Łomianki Poland
    80 Hospital Universitari Vall d Hebron Barcelona Spain
    81 Hospital Universitari Vall d´Hebron Servicio de Broncoscopia Barcelona Spain
    82 Hospital Universitario 12 de Octubre Madrid Spain
    83 Hospital Universitario Infantil La Paz Madrid Spain
    84 Parc Tauli Sabadell Hospital Universitari Sabadell Spain
    85 Hospital Universitario Virgen del Rocio Sevilla Spain
    86 Hospital Universitario y Politecnico La Fe Valencia Spain
    87 Lindenhofspital - Quartier Bleu Bern Switzerland
    88 Kinderspital Zuerich Zürich Switzerland
    89 Papworth Hospital NHS Foundation Trust, Papworth Everard Cambridge United Kingdom
    90 NHS Greater Glasgow and Clyde, Gartnavel General Hospital Glasgow United Kingdom
    91 St. James University Hospital Leeds United Kingdom
    92 Liverpool Heart and Chest Hospital Liverpool United Kingdom
    93 Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital London United Kingdom
    94 Wythenshawe Hospital Manchester United Kingdom
    95 The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary Newcastle Upon Tyne United Kingdom
    96 Nottingham University Hospitals NHS Trust, Queens Medical Center Nottingham United Kingdom
    97 All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough Penarth 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:
    NCT04043806
    Other Study ID Numbers:
    • VX18-445-113
    • 2018-004652-38
    First Posted:
    Aug 2, 2019
    Last Update Posted:
    Jun 30, 2022
    Last Verified:
    Jun 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 Jun 30, 2022