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ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation

Primary Purpose

Cystic Fibrosis

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
ELX/TEZ/IVA
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Cystic Fibrosis

Eligibility Criteria

6 Years - 11 Years (Child)All Sexes

Key Inclusion Criteria:

  • Patients with confirmed diagnosis of CF who have an F/any genotype
  • Prior treatment for at least 6 months with an approved alternative CFTR modulator (if the patient is eligible for such treatment)
  • Patients with percent predicted forced expiratory volume in 1 second (ppFEV1) <40 for a minimum of 60 consecutive days before the date of completion of the request form

Key Exclusion Criteria:

  • Patients with severe hepatic impairment

Other protocol-defined inclusion/exclusion criteria may apply

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    January 6, 2021
    Last Updated
    July 8, 2021
    Sponsor
    Vertex Pharmaceuticals Incorporated
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04702360
    Brief Title
    ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
    Official Title
    ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    July 2021
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Vertex Pharmaceuticals Incorporated

    4. Oversight

    5. Study Description

    Brief Summary
    The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician requests.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystic Fibrosis

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    ELX/TEZ/IVA
    Other Intervention Name(s)
    elexacaftor/tezacaftor/ivacaftor, VX-445/VX-661/VX-770
    Intervention Description
    Fixed-dose combination tablet for oral administration.
    Intervention Type
    Drug
    Intervention Name(s)
    IVA
    Other Intervention Name(s)
    ivacaftor, VX-770
    Intervention Description
    IVA monotablet for oral administration.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    6 Years
    Maximum Age & Unit of Time
    11 Years
    Eligibility Criteria
    Key Inclusion Criteria: Patients with confirmed diagnosis of CF who have an F/any genotype Prior treatment for at least 6 months with an approved alternative CFTR modulator (if the patient is eligible for such treatment) Patients with percent predicted forced expiratory volume in 1 second (ppFEV1) <40 for a minimum of 60 consecutive days before the date of completion of the request form Key Exclusion Criteria: Patients with severe hepatic impairment Other protocol-defined inclusion/exclusion criteria may apply

    12. IPD Sharing Statement

    Learn more about this trial

    ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation

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