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NCT04702360 Clinical Trial

ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Approved for marketing

Administrative data
NCT number NCT04702360
Other study ID # VX20-445-905
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information
Verified date July 2021
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.

Recruitment information / eligibility
Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 6 Years to 11 Years
Eligibility 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

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration.
• IVA
IVA monotablet for oral administration.

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated
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