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VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

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

12 Years - undefined (Child, Adult, Older Adult)All Sexes

Inclusion Criteria:

1. Patients who have F/MF genotypes AND who meet at least 1 of the following criteria:

  • The percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 for a minimum of 2 months before the date of the request, OR
  • Documentation of being active on a lung transplant waiting list or documentation of being evaluated for lung transplantation, but deemed unsuitable because of contraindications

Exclusion Criteria:

  1. Patients with severe hepatic impairment (Child-Pugh Class C)
  2. History of any other comorbidity that, in the opinion of the treating physician, might pose undue risk in administering ELX/TEZ/IVA to the patient
  3. Pregnancy

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    August 13, 2019
    Last Updated
    November 19, 2019
    Sponsor
    Vertex Pharmaceuticals Incorporated
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04058210
    Brief Title
    VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)
    Official Title
    VX-445/TEZ/IVA Triple Combination Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function CFTR Mutation
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    November 2019
    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(ELX, VX-445)/tezacaftor(TEZ)/ivacaftor(IVA) combination therapy to CF patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) 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
    150-mg tablet for oral administration.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Years
    Eligibility Criteria
    Inclusion Criteria: 1. Patients who have F/MF genotypes AND who meet at least 1 of the following criteria: The percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 for a minimum of 2 months before the date of the request, OR Documentation of being active on a lung transplant waiting list or documentation of being evaluated for lung transplantation, but deemed unsuitable because of contraindications Exclusion Criteria: Patients with severe hepatic impairment (Child-Pugh Class C) History of any other comorbidity that, in the opinion of the treating physician, might pose undue risk in administering ELX/TEZ/IVA to the patient Pregnancy Other protocol defined Inclusion/Exclusion criteria may apply

    12. IPD Sharing Statement

    Learn more about this trial

    VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

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