ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Approved for marketing
CT.gov ID
NCT04702360
Collaborator
(none)
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
Study Design
Study Type:
Expanded Access
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
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Ages Eligible for Study:
6 Years
to 11 Years
Sexes Eligible for Study:
All
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
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT04702360
Other Study ID Numbers:
- VX20-445-905
First Posted:
Jan 8, 2021
Last Update Posted:
Jul 15, 2021
Last Verified:
Jul 1, 2021