Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta™) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocol.

 0 Người đánh giá. Xếp hạng trung bình 0

Tác giả: Cristina Barillá, Erin Barton, Diana J Beltran, Cynthia Chico, Elizabeth M Cox, Joy Dangerfield, Brian R Davis, Jeong S Hong, William R Hunt, Rebecca Kapolka, Candela Manfredi, Andras Rab, Eric J Sorscher, Arlene A Stecenko, Ashleigh Streby, Shingo Suzuki, Colin E Swenson, Adrianna Westbrook

Ngôn ngữ: eng

Ký hiệu phân loại: 936 Europe north and west of Italian Peninsula to ca. 499

Thông tin xuất bản: United States : PloS one , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 88211

BACKGROUND: Non-cystic fibrosis bronchiectasis (NCFBE) is a disease that exhibits dilatation of airways, airflow obstruction, persistent cough, excessive sputum production, and refractory respiratory infections. NCFBE exhibits clinical and pathological manifestations similar to key features of cystic fibrosis (CF) lung disease. In CF, pathogenesis results from dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR), and diagnosis is made by demonstrating elevated sweat chloride concentrations (typically ≥60 mEq/L), two CFTR mutations known to be causal, multi-organ tissue injury, or combination(s) of these findings. OBJECTIVE: Based on a considerable body of evidence, we believe many patients with NCFBE have disease likely to benefit from drugs such as elexacaftor/tezacaftor/ivacaftor (ETI) that activate CFTR-dependent ion transport. ETI is currently prescribed solely for treatment of CF and has not been adequately tested or proposed for patients with NCFBE, many of whom exhibit decreased CFTR function. Accordingly, we are conducting a clinical trial of ETI in subjects carrying a diagnosis of NCFBE. METHODS: Participants will exhibit one disease-causing CFTR mutation and/or sweat chloride measurements of 30-59 mEq/L. Cutaneous punch biopsy or blood samples will be obtained for iPS cell differentiation into airway epithelial monolayers-which will then be tested for response to ETI. Each patient will be given CFTR modulator treatment for approximately four weeks, with monitoring of clinical endpoints that include FEV1 (forced expiratory volume in one second), sweat chloride, quality of life questionnaire, and weight. The study will evaluate response of patients with NCFBE to ETI, and test usefulness of iPSC-derived airway epithelial monolayers as a novel in vitro technology for predicting clinical benefit. TRIAL REGISTRATION: This trial is registered at clinicaltrials.gov (Identifier: NCT05743946. Date: 02/23/2023).
Tạo bộ sưu tập với mã QR

THƯ VIỆN - TRƯỜNG ĐẠI HỌC CÔNG NGHỆ TP.HCM

ĐT: (028) 36225755 | Email: tt.thuvien@hutech.edu.vn

Copyright @2024 THƯ VIỆN HUTECH