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
About this trial
This is an expanded access trial for Cystic Fibrosis
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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT04058210
First Posted
August 13, 2019
Last Updated
November 19, 2019
Sponsor
Vertex Pharmaceuticals Incorporated
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
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Primary Completion Date
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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
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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