Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated0 个研究点开始时间: 2017年9月11日最近更新: 2018年11月2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

To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719). To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of age and older, homozygous for F508del.

研究设计

研究类型: Expanded Access

入排标准

年龄范围: 12 Years 至 —（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Males and females, 12 years of age and older with confirmed diagnosis of CF.
•completed Vertex clinical studies NCT02565914 or NCT03150719, or
•are homozygous for F508del and have (1) discontinued Orkambi within 12 weeks from start of treatment because of the onset of respiratory AEs considered to be treatment related or (2) cannot initiate Orkambi because of chronic treatment with an essential sensitive CYP3A substrate or a CYP3A substrate with a narrow therapeutic index AND who meet at least 1 of the following criteria:
•the highest percent predicted forced expiratory volume in 1 second (ppFEV1) is <40 in the 6 months before the date of completion of the request form, 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, or
•rapid and persistent loss of lung function, defined as at least a 20% relative decrease in ppFEV1 in the last 6 months and sustained for at least 1 month despite appropriate treatment.

排除标准

•History of any comorbidity that, in the opinion of the treating physician, might pose undue risk in administering TEZ/IVA combination therapy to the patient (e.g., history of advanced liver disease).
•Ongoing or prior participation in an investigational drug study (with the exception of Vertex clinical studies evaluating TEZ/IVA) within 5 terminal half-lives of the previous investigational study drug or 30 days, whichever is longer, of first administration of TEZ/IVA.
•Subjects who are pregnant.
•Patients eligible for participation in ongoing clinical studies evaluating TEZ/IVA or other CFTR modulator therapies.
•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

相似试验

已完成

3 期

A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic FibrosisCystic Fibrosis

NCT03559062Vertex Pharmaceuticals Incorporated67

已完成

3 期

A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR MutationCystic Fibrosis

NCT03537651Vertex Pharmaceuticals Incorporated130

已完成

2 期

A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR MutationCystic Fibrosis

NCT02730208Vertex Pharmaceuticals Incorporated41

终止

4 期

Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon MutationsCystic Fibrosis

NCT03624101University of Alabama at Birmingham1

已完成

3 期

A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)Cystic Fibrosis

NCT03525444Vertex Pharmaceuticals Incorporated405