The BEACON-CF clinical research study

A Phase 1/2 Dose-Escalation Study Evaluating the Safety, Tolerability, and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy

This is an open-label, dose-escalation, first-in-human study to evaluate the safety, tolerability, and efficacy of VX-522 treatment.

Patients that meet the following criteria may be eligible to participate in this study. Please note this is not a full list of eligibility criteria.

18–65 years of age, inclusive
Body mass index of <30.0 kilogram per square meter (kg/m²)
A total body weight greater than 50 kg
Stable CF disease
Forced expiratory volume in 1 second (FEV1) value of more than or equal to 40% for Part 1 of the study, or more than or equal to 40% and less than or equal to 90% for Part 2 of the study

History of uncontrolled asthma within a year prior to screening
History of solid organ or hematological transplantation
Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7–9), or severe hepatic impairment (Child Pugh Score 10–15)
Oxygen saturation on room air less than 94%
History of severe adverse reaction to any inhaled lipid-based product