A Phase 1 Study of VX-522 in Participants With Cystic Fibrosis (CF)

• Status: Recruiting
• Trial end date: 2024-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Criteria

Key Inclusion Criteria:

• Body mass index (BMI) of greater than or equal to (≥)18.0 to less than (<) 30.0 kilograms per meter square (kg/m^2)

• 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 (<) 94% at screening

Other protocol defined Inclusion/Exclusion criteria may apply.