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Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT05153317
Collaborator
(none)
70
Enrollment
22
Locations
1
Arm
50.4
Anticipated Duration (Months)
3.2
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
70 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 and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
Actual Study Start Date :
Jan 17, 2022
Anticipated Primary Completion Date :
Apr 1, 2026
Anticipated Study Completion Date :
Apr 1, 2026

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 granules or tablets for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor

    • Drug: IVA
    Granules or tablets 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) [From Day 1 up to Week 196]

    Secondary Outcome Measures

    1. Absolute Change in Sweat Chloride (SwCl) [From Baseline up to Week 192]

    2. Absolute Change in Lung Clearance Index (LCI) 2.5 [From Baseline up to Week 192]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No

    key Inclusion Criteria:

    • Completed study drug treatment in the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study
    Key Exclusion Criteria:

    • History of study drug intolerance in the parent study

    • History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study

    Other protocol defined Inclusion/Exclusion criteria may apply

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Banner University of Arizona Medical Center Tucson Arizona United States 85724
    2 Stanford University Palo Alto California United States 94304
    3 Children's Hospital of Colorado Aurora Colorado United States 80045
    4 Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois United States 60611
    5 Riley Hospital for Children at Indiana University Health Indianapolis Indiana United States 46202
    6 Boston Children's Hospital Boston Massachusetts United States 02115
    7 Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota United States 55404
    8 The Children's Mercy Hospital Kansas City Missouri United States 64108
    9 Washington University School of Medicine / St. Louis Children's Hospital Saint Louis Missouri United States 63110
    10 NC TraCS Institute - CTRC University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27599
    11 Oregon Health & Science University Portland Oregon United States 97239
    12 UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania United States 15224
    13 Seattle Children's Hospital Seattle Washington United States 98105
    14 Telethon Kids Institute Nedlands Australia
    15 The Royal Children's Hospital Parkville Australia
    16 Queensland Children's Hospital South Brisbane Australia
    17 The Hospital for Sick Children Toronto Canada
    18 British Columbia Children's Hospital Vancouver Canada
    19 Charite Paediatric Pulmonology Department Berlin Germany
    20 Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie Essen Germany
    21 Alder Hey Children's NHS Foundation Trust Liverpool United Kingdom
    22 Royal Brompton Hospital London 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:
    NCT05153317
    Other Study ID Numbers:
    • VX20-445-112
    • 2020-002239-31
    First Posted:
    Dec 10, 2021
    Last Update Posted:
    Jul 22, 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
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor
    Elexacaftor

    Study Results

    No Results Posted as of Jul 22, 2022