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Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05331183
Collaborator
(none)
270
Enrollment
1
Arm
30
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
270 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 in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
Anticipated Study Start Date :
Sep 1, 2022
Anticipated Primary Completion Date :
Mar 1, 2025
Anticipated Study Completion Date :
Mar 1, 2025

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
    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) [Day 1 up to Week 100]

    Secondary Outcome Measures

    1. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1) [From Baseline up to Week 96]

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

    3. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [From Baseline up to Week 96]

    4. Absolute Change in Body Mass Index (BMI) [From Baseline up to Week 96]

    5. Absolute Change in Weight [From Baseline up to Week 96]

    6. Number of Pulmonary Exacerbations (PEx) [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 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

    No locations specified.

    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:
    NCT05331183
    Other Study ID Numbers:
    • VX21-445-125
    • 2021-005914-33
    First Posted:
    Apr 15, 2022
    Last Update Posted:
    Apr 15, 2022
    Last Verified:
    Apr 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 Apr 15, 2022