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A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF Genotypes

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04545515
Collaborator
(none)
120
Enrollment
34
Locations
1
Arm
26.6
Anticipated Duration (Months)
3.5
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 efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in subjects with CF who are 6 years of age and older with F/MF genotypes.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
120 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3b Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Actual Study Start Date :
Jan 11, 2021
Anticipated Primary Completion Date :
Apr 1, 2023
Anticipated Study Completion Date :
Apr 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: ELX/TEZ/IVA

Participants will receive ELX/TEZ/IVA in the morning and IVA 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
    Tablet for oral administration.
    Other Names:
  • VX-770
  • ivacaftor

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Day 1 up to Week 100]

    Secondary Outcome Measures

    1. Absolute Change in Sweat Chloride (SwCl) [From Baseline up to Week 96]

    2. Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) [From Baseline up to Week 96]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:
    • Completed study drug treatment in parent study (VX19-445-116, NCT04353817), 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 the parent study

    Other protocol defined Inclusion/Exclusion criteria may apply

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Telethon Kids Institute Nedlands Australia
    2 Queensland Children's Hospital South Brisbane Australia
    3 The Children's Hospital at Westmead Westmead Australia
    4 McGill University Health Centre, Glen Site, Montreal Children's Hospital Montreal Canada
    5 The Hospital for Sick Children Toronto Canada
    6 British Columbia Children's Hospital Vancouver Canada
    7 Juliane Marie Center, Rigshospitalet Copenhagen Denmark
    8 Groupe Hospitaler Pellegrin, CHU De Bordeaux Bordeaux cedex France
    9 CHU Lyon - Hopital Femme Mere-Enfant Bron Cedex France
    10 Hopital Necker, Enfants Malades Paris Cedex 15 France
    11 Hopital Robert Debre Paris France
    12 Centre de Perharidy Roscoff cedex France
    13 Charite Paediatric Pulmonology Department Berlin Germany
    14 Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie Essen Germany
    15 Johann Wolfgang Goethe University Frankfurt Germany
    16 Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin Gießen Germany
    17 Medizinische Hochschule Hannover Hannover Germany
    18 Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin Heidelberg Germany
    19 Universitaetsklinkum Koeln, CF-Studienzentrum Koeln Germany
    20 Hadassah University Hospital Mount Scopus Jerusalem Israel
    21 Schneider Children's Medical Center of Israel Petach Tikvah Israel
    22 Universitair Medisch Centrum Groningen Groningen Netherlands
    23 Erasmus Medical Center / Sophia Children's Hospital Rotterdam Netherlands
    24 Hospital Universitari Vall d Hebron Barcelona Spain
    25 Hospital Virgen de la Arrixaca Murcia Spain
    26 Inselspital - Universitaetsspital Bern Bern Switzerland
    27 Kinderspital Zuerich Zurich Switzerland
    28 University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital Bristol United Kingdom
    29 Children's Hospital of Wales Cardiff United Kingdom
    30 Royal Hospital for Sick Children Edinburgh United Kingdom
    31 Alder Hey Children's NHS Foundation Trust Liverpool United Kingdom
    32 Great Ormond Street Hospital for Sick Children London United Kingdom
    33 Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital London United Kingdom
    34 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:
    NCT04545515
    Other Study ID Numbers:
    • VX20-445-119
    • 2020-001404-42
    First Posted:
    Sep 11, 2020
    Last Update Posted:
    Feb 7, 2022
    Last Verified:
    Feb 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
    Product Manufactured in and Exported from the U.S.:
    Yes
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor
    Elexacaftor

    Study Results

    No Results Posted as of Feb 7, 2022