Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age

Last updated: October 3, 2025
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Completed

Phase

3

Condition

Cystic Fibrosis

Scar Tissue

Lung Disease

Treatment

IVA

ELX/TEZ/IVA

Clinical Study ID

NCT05882357
VX22-445-122
2023-503230-49-00
  • Ages 12-24
  • All Genders

Study Summary

This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (<) 24 months of age.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation

Exclusion

Key Exclusion Criteria:

History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 70
Treatment Group(s): 2
Primary Treatment: IVA
Phase: 3
Study Start date:
June 27, 2023
Estimated Completion Date:
September 04, 2025

Connect with a study center

  • Telethon Kids Institute

    Nedlands,
    Australia

    Site Not Available

  • Telethon Kids Institute

    Nedlands 2064874,
    Australia

    Site Not Available

  • The Royal Children's Hospital

    Parkville,
    Australia

    Site Not Available

  • The Royal Children's Hospital

    Parkville 2153770,
    Australia

    Site Not Available

  • Queensland Children's Hospital

    South Brisbane,
    Australia

    Site Not Available

  • Queensland Children's Hospital

    South Brisbane 2207259,
    Australia

    Site Not Available

  • The Children's Hospital at Westmead

    Westmead,
    Australia

    Site Not Available

  • The Children's Hospital at Westmead

    Westmead 2143973,
    Australia

    Site Not Available

  • The Hospital for Sick Children

    Toronto,
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto 6167865,
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver,
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver 6173331,
    Canada

    Site Not Available

  • Juliane Marie Center, Rigshospitalet

    Copenhagen,
    Denmark

    Site Not Available

  • Juliane Marie Center, Rigshospitalet

    Copenhagen 2618425,
    Denmark

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin,
    Germany

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin 2950159,
    Germany

    Site Not Available

  • Kinderklinik III, Abt. fur Pneumologie

    Essen,
    Germany

    Site Not Available

  • Kinderklinik III, Abt. fur Pneumologie

    Essen 2928810,
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover

    Hannover,
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover

    Hanover 2910831,
    Germany

    Site Not Available

  • Erasmus Medical Center / Sophia Children's Hospital

    Rotterdam,
    Netherlands

    Site Not Available

  • Erasmus Medical Center / Sophia Children's Hospital

    Rotterdam 2747891,
    Netherlands

    Site Not Available

  • Inselspital - Universitaetsspital Bern

    Bern,
    Switzerland

    Site Not Available

  • Inselspital - Universitaetsspital Bern

    Bern 2661552,
    Switzerland

    Site Not Available

  • Kinderspital Zuerich

    Zurich,
    Switzerland

    Site Not Available

  • Kinderspital Zuerich

    Zurich 2657896,
    Switzerland

    Site Not Available

  • Children and Young Adults Research Unit

    Cardiff,
    United Kingdom

    Site Not Available

  • Children and Young Adults Research Unit

    Cardiff 2653822,
    United Kingdom

    Site Not Available

  • Leeds General Infirmary

    Leeds,
    United Kingdom

    Site Not Available

  • Leeds General Infirmary

    Leeds 2644688,
    United Kingdom

    Site Not Available

  • Alder Hey Children's NHS Foundation Trust

    Liverpool,
    United Kingdom

    Site Not Available

  • Alder Hey Children's NHS Foundation Trust

    Liverpool 2644210,
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children

    London,
    United Kingdom

    Site Not Available

  • Royal Brompton Hospital

    London,
    United Kingdom

    Site Not Available

  • Great Ormond Street Hospital for Children

    London 2643743,
    United Kingdom

    Site Not Available

  • Royal Brompton Hospital

    London 2643743,
    United Kingdom

    Site Not Available

  • Southampton General Hospital

    Southampton,
    United Kingdom

    Site Not Available

  • Southampton General Hospital

    Southampton 2637487,
    United Kingdom

    Site Not Available

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