ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation

Vertex Pharmaceuticals Incorporated0 个研究点开始时间: 2021年1月8日最近更新: 2021年7月15日

适应症

试验速览

阶段: 不适用
状态: Approved For Marketing
发起方: Vertex Pharmaceuticals Incorporated

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

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.

研究设计

研究类型: Expanded Access

入排标准

年龄范围: 6 Years 至 11 Years（Child）
性别: All
接受健康志愿者: 否

入选标准

•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

排除标准

•Patients with severe hepatic impairment
•Other protocol-defined inclusion/exclusion criteria may apply

研究者

发起方

Vertex Pharmaceuticals Incorporated

申办方类型

Industry

责任方

Sponsor

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