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
About this trial
This is an expanded access trial for Cystic Fibrosis
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.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT03278314
First Posted
September 6, 2017
Last Updated
October 31, 2018
Sponsor
Vertex Pharmaceuticals Incorporated
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
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Primary Completion Date
undefined (undefined)
Study Completion Date
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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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