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Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
tezacaftor/ivacaftor
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:

  • Males and females, 12 years of age and older with confirmed diagnosis of CF.

    1. - completed Vertex clinical studies NCT02565914 or NCT03150719, or
    2. - are homozygous for F508del and have (1) discontinued Orkambi within 12 weeks from start of treatment because of the onset of respiratory AEs considered to be treatment related or (2) cannot initiate Orkambi because of chronic treatment with an essential sensitive CYP3A substrate or a CYP3A substrate with a narrow therapeutic index AND who meet at least 1 of the following criteria:

      • the highest percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 in the 6 months before the date of completion of the request form, 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, or
      • rapid and persistent loss of lung function, defined as at least a 20% relative decrease in ppFEV1 in the last 6 months and sustained for at least 1 month despite appropriate treatment.

Exclusion Criteria:

  • History of any comorbidity that, in the opinion of the treating physician, might pose undue risk in administering TEZ/IVA combination therapy to the patient (e.g., history of advanced liver disease).
  • Ongoing or prior participation in an investigational drug study (with the exception of Vertex clinical studies evaluating TEZ/IVA) within 5 terminal half-lives of the previous investigational study drug or 30 days, whichever is longer, of first administration of TEZ/IVA.
  • Subjects who are pregnant.
  • Patients eligible for participation in ongoing clinical studies evaluating TEZ/IVA or other CFTR modulator therapies.

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    September 6, 2017
    Last Updated
    October 31, 2018
    Sponsor
    Vertex Pharmaceuticals Incorporated
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03278314
    Brief Title
    Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis
    Official Title
    Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    October 2018
    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
    To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719). To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of age and older, homozygous for F508del.

    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)
    tezacaftor/ivacaftor
    Other Intervention Name(s)
    TEZ/IVA
    Intervention Description
    orally administered TEZ/IVA combination therapy

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Years
    Eligibility Criteria
    Inclusion Criteria: Males and females, 12 years of age and older with confirmed diagnosis of CF. - completed Vertex clinical studies NCT02565914 or NCT03150719, or - are homozygous for F508del and have (1) discontinued Orkambi within 12 weeks from start of treatment because of the onset of respiratory AEs considered to be treatment related or (2) cannot initiate Orkambi because of chronic treatment with an essential sensitive CYP3A substrate or a CYP3A substrate with a narrow therapeutic index AND who meet at least 1 of the following criteria: the highest percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 in the 6 months before the date of completion of the request form, 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, or rapid and persistent loss of lung function, defined as at least a 20% relative decrease in ppFEV1 in the last 6 months and sustained for at least 1 month despite appropriate treatment. Exclusion Criteria: History of any comorbidity that, in the opinion of the treating physician, might pose undue risk in administering TEZ/IVA combination therapy to the patient (e.g., history of advanced liver disease). Ongoing or prior participation in an investigational drug study (with the exception of Vertex clinical studies evaluating TEZ/IVA) within 5 terminal half-lives of the previous investigational study drug or 30 days, whichever is longer, of first administration of TEZ/IVA. Subjects who are pregnant. Patients eligible for participation in ongoing clinical studies evaluating TEZ/IVA or other CFTR modulator therapies. Other protocol defined Inclusion/Exclusion criteria may apply.

    12. IPD Sharing Statement

    Learn more about this trial

    Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

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