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Currently Recruiting
This trial is actively seeking participants in the UK. Discuss eligibility with your patient.
Status
Recruiting
Phase
PHASE1
NCT ID
NCT05668741
Trial Summary
The purpose of this study is to evaluate the safety, and tolerability and efficacy 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.
Lead Sponsor: Vertex Pharmaceuticals Incorporated
Participants: ALL
Start Date: 2023-02-27
Primary Completion: 2026-12-23
Min Age: 18 Years
Max Age: 65 Years
Eligibility Criteria
Key Inclusion Criteria: * Body mass index is less 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 for SAD: greater than or equal to (≥)40 percent (%), MAD: ≥ 50% to less than or equal to (≤) 90% 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 (
Data sourced from ClinicalTrials.gov. Trial details may change — always check the primary source before clinical decisions.