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

N/AApproved For MarketingNCT04702360

Vertex Pharmaceuticals Incorporated

Overview

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.

Study Type

EXPANDED_ACCESS

Conditions

Cystic Fibrosis

Interventions

ELX/TEZ/IVADRUG

Fixed-dose combination tablet for oral administration.

IVADRUG

IVA monotablet for oral administration.

Eligibility

Sex: ALLMin age: 6 YearsMax age: 11 Years

Medical Language ↔ Plain English

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

Data from ClinicalTrials.gov

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