Clincosm LogoSign in Get a demo

VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Approved for marketing
CT.gov ID
NCT04058210
Collaborator
(none)

Study Details

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.

Condition or Disease Intervention/Treatment Phase

Study Design

Study Type:
Expanded Access
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

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    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:

    1. Patients with severe hepatic impairment (Child-Pugh Class C)

    2. 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

    3. Pregnancy

    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:
    NCT04058210
    Other Study ID Numbers:
    • VX18-445-901
    First Posted:
    Aug 15, 2019
    Last Update Posted:
    Nov 21, 2019
    Last Verified:
    Nov 1, 2019
    Additional relevant MeSH terms:
    Cystic Fibrosis
    Fibrosis
    Ivacaftor
    Elexacaftor

    Study Results

    No Results Posted as of Nov 21, 2019