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
About this trial
This is an expanded access trial for Cystic Fibrosis
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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT04702360
First Posted
January 6, 2021
Last Updated
July 8, 2021
Sponsor
Vertex Pharmaceuticals Incorporated
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
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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/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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