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Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05274269
Collaborator
(none)
270
Enrollment
41
Locations
2
Arms
11.7
Anticipated Duration (Months)
6.6
Patients Per Site
0.6
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will evaluate the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
270 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation
Actual Study Start Date :
May 9, 2022
Anticipated Primary Completion Date :
Apr 1, 2023
Anticipated Study Completion Date :
May 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) tablets 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

    		•
    Placebo Comparator: Placebo

    Participants will receive placebo matched to ELX/TEZ/IVA in the morning and placebo matched to IVA in the evening.

    Other: Placebo (matched to ELX/TEZ/IVA)
    Placebo matched to ELX/TEZ/IVA for oral administration.

    Other: Placebo (matched to IVA)
    Placebo matched to IVA for oral administration.

    Outcome Measures

    Primary Outcome Measures

    1. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [From Baseline Through Week 24]

    Secondary Outcome Measures

    1. Absolute Change in Sweat Chloride (SwCl) [From Baseline Through Week 24]

    2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [From Baseline Through Week 24]

    3. Absolute Change in Body Mass Index (BMI) [From Baseline at Week 24]

    4. Absolute Change in Weight [From Baseline at Week 24]

    5. Number of Pulmonary Exacerbations (PEx) [From Baseline Through Week 24]

    6. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Day 1 up to Week 28]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Key Inclusion Criteria:

    • Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not have an exclusionary CFTR mutation

    • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height

    Key Exclusion Criteria:

    • History of solid organ or hematological transplantation

    • Clinically significant cirrhosis with or without portal hypertension

    • Lung infection with organisms associated with a more rapid decline in pulmonary status

    Other protocol defined Inclusion/Exclusion criteria may apply.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Universitair Ziekenhuis Gent Gent Belgium
    2 British Columbia Children's Hospital Vancouver Canada
    3 CHU Lyon - Hopital Femme Mere-Enfant Bron Cedex France
    4 Centre Hospitalier Intercommunal Creteil Créteil France
    5 Institut Cœur Poumon, CHU de Lille Lille France
    6 CHU Marseille - Hopital Nord Marseilles France
    7 Hopital Arnaud de Villeneuve Montpellier Cedex 5 France
    8 Centre Hospitalier Universitaire De Nantes - G. R. Laennec Nantes France
    9 Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur Nice France
    10 Hopital Necker, Enfants Malades Paris Cedex 15 France
    11 Hopital Cochin Paris France
    12 Hopital Robert Debre Paris France
    13 Hopital Haut-Leveque - CRCM Adulte Pessac France
    14 Centre Hospitalier Lyon Sud Pierre-Bénite France
    15 Centre de Perharidy Roscoff Cedex France
    16 CHU de Toulouse - Hopital Larre Toulouse France
    17 Hopital Bretonneau Tours France
    18 Charite Paediatric Pulmonology Department Berlin Germany
    19 Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen Essen Germany
    20 Johann Wolfgang Goethe University Frankfurt Germany
    21 Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Gießen Germany
    22 Universitaetsklinikum Jena, Mukoviszidose-Zentrum Jena Germany
    23 Pneumologisches Studienzentrum Muenchen-West Muenchen Germany
    24 Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer Firenze Italy
    25 Azienda Ospedaliera di Verona - Ospedale Civile Maggiore Verona Italy
    26 Academisch Medisch Centrum (Academic Medical Centre) Amsterdam Netherlands
    27 HagaZiekenhuis van den Haag Den Haag Netherlands
    28 UMC St. Radboud Nijmegen Netherlands
    29 Erasmus Medical Center Rotterdam Netherlands
    30 Universitair Medisch Centrum Utrecht Utrecht Netherlands
    31 Hospital Saint Joan de Deu Barcelona Spain
    32 Hospital Universitari Vall d Hebron Barcelona Spain
    33 Hospital Universitari Vall d´Hebron Servicio de Broncoscopia Barcelona Spain
    34 Hospital Infantil Universitario Nino Jesus Madrid Spain
    35 Hospital Universitario 12 de Octubre Madrid Spain
    36 Hospital Universitario Ramon y Cajal Madrid Spain
    37 Hospital Virgen de la Arrixaca Murcia Spain
    38 Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari Sabadell Spain
    39 Hospital Universitario Virgen del Rocio Sevilla Spain
    40 Hospital Universitario y Politecnico La Fe Valencia Spain
    41 Kinderspital Zuerich Zurich Switzerland

    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:
    NCT05274269
    Other Study ID Numbers:
    • VX21-445-124
    • 2021-005320-38
    First Posted:
    Mar 10, 2022
    Last Update Posted:
    Aug 24, 2022
    Last Verified:
    Mar 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 Aug 24, 2022