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

N/AApproved For MarketingNCT04058210

Vertex Pharmaceuticals Incorporated

Overview

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.

Study Type

EXPANDED_ACCESS

Conditions

Cystic Fibrosis

Interventions

ELX/TEZ/IVADRUG

Fixed-dose combination tablet for oral administration.

IVADRUG

150-mg tablet for oral administration.

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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

Data from ClinicalTrials.gov

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