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A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04362761
Collaborator
(none)
172
Enrollment
29
Locations
1
Arm
36.9
Anticipated Duration (Months)
5.9
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in subjects with cystic fibrosis (CF) who are homozygous for F508del.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
172 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
Actual Study Start Date :
May 4, 2020
Anticipated Primary Completion Date :
Jun 1, 2023
Anticipated Study Completion Date :
Jun 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: ELX/TEZ/IVA

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
    Mono 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 Day 1 up to Week 100]

    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 (VX18-445-109); 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 The Prince Charles Hospital Chermside Australia
    2 Institute for Respiratory Health Nedlands Australia
    3 Telethon Kids Institute, Perth Children's Hospital Nedlands Australia
    4 The Royal Children's Hospital Parkville, VIC Australia
    5 Queensland Children's Hospital South Brisbane Australia
    6 Universitair Ziekenhuis Gent Gent Belgium
    7 Universitair Ziekenhuizen Leuven - Campus Gasthuisberg Leuven Belgium
    8 Charite Paediatric Pulmonology Department Berlin Germany
    9 Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen Essen Germany
    10 Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie Essen Germany
    11 Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin Jena Germany
    12 Universitaetsklinkum Koeln, CF-Studienzentrum Koeln Germany
    13 Klinikum Innenstadt, University of Munich München Germany
    14 Belfast City Hospital Belfast United Kingdom
    15 University Hospitals Birmingham NHS Foundation Trust Birmingham United Kingdom
    16 University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital Bristol United Kingdom
    17 Royal Papworth Hospital NHS Foundation Trust Cambridge United Kingdom
    18 Western General Hospital Edinburgh United Kingdom
    19 Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital Exeter United Kingdom
    20 Clinical Research Facility, Queen Elizabeth University Hospital Glasgow United Kingdom
    21 Leeds General Infirmary Leeds United Kingdom
    22 St. James University Hospital Leeds United Kingdom
    23 Alder Hey Children's NHS Foundation Trust Liverpool United Kingdom
    24 Great Ormond Street Hospital for Sick Children London United Kingdom
    25 London and St Bartholomew's Hospital London United Kingdom
    26 The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary Newcastle Upon Tyne United Kingdom
    27 Nottingham University Hospitals NHS Trust, Queens Medical Center Nottingham United Kingdom
    28 All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough Penarth United Kingdom
    29 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:
    NCT04362761
    Other Study ID Numbers:
    • VX19-445-115
    • 2019-003455-11
    First Posted:
    Apr 27, 2020
    Last Update Posted:
    Jul 5, 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 Jul 5, 2022