Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del / F508del-CFTR or F508del / G551D-CFTR

• Published in: American journal of respiratory and critical care medicine Vol. 197; no. 2; pp. 214 - 224
• Main Authors: Donaldson, Scott H., Pilewski, Joseph M., Griese, Matthias, Cooke, Jon, Viswanathan, Lakshmi, Tullis, Elizabeth, Davies, Jane C., Lekstrom-Himes, Julie A., Wang, Linda T.
• Format: Journal Article
• Language: English
• Published: United States American Thoracic Society 15.01.2018
• Subjects: Administration, Oral; Adolescent; Adult; Aminophenols - administration & dosage; Aminophenols - adverse effects; Benzodioxoles - administration & dosage; Benzodioxoles - adverse effects; Chloride; Cystic fibrosis; Cystic Fibrosis - diagnosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cysts; Dose-Response Relationship, Drug; Double-Blind Method; Drug Administration Schedule; Drug dosages; Drug Therapy, Combination; Female; Humans; Indoles - administration & dosage; Indoles - adverse effects; Kinases; Male; Maximum Tolerated Dose; Molecular Targeted Therapy - methods; Mutation; Original; Patients; Pediatrics; Prognosis; Proteins; Quinolones - administration & dosage; Quinolones - adverse effects; Respiratory Function Tests; Risk Assessment; Severity of Illness Index; Treatment Outcome; Young Adult; United States > US; forced expiratory volume; sweat chloride; CFTR modulator; cystic fibrosis transmembrane conductance regulator corrector

Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. To evaluate the safety and efficacy of tezacaftor...