A Phase 1 Study of VX-522 in Participants With Cystic Fibrosis (CF)
The purpose of this study is to evaluate the safety and tolerability of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
18 Years - 65 Years
Key inclusion criteria
- Body mass index is less than (
- A total body weight greater than (>) 50 kg
- Stable CF disease
- CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy
- o Example mutations include but are not limited to, mutations that do not produce CFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice mutations (e.g., 621+1G->T)
- Forced expiratory volume in 1 second (FEV1) value, percent of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI])18 ≥40%
Key exclusion criteria
- 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 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
- Arterial oxygen saturation on room air less than (
Other protocol defined Inclusion/Exclusion criteria may apply.
February 2023 - January 2024
Drug : VX-522 mRNA therapy
Interested in this trial?
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