Phase 2 Study to Assess Safety, Tolerability, and Effectiveness of ARCT-032 Inhaled Treatment in Adults with Cystic Fibrosis Not Using CFTR Modulators

What this Trial Is About

Researchers are evaluating ARCT-032, an investigational therapy, in adults with cystic fibrosis CF who cannot use or do not take CFTR modulator treatments due to intolerance, poor response, or lack of access. This Phase 2, open-label, multicenter study aims to assess the safety, tolerability, and effects of ARCT-032 delivered by inhalation. Participants have confirmed CF and specific lung function ranges depending on their study group. Eligible participants are assigned to one of four groups receiving different daily inhaled doses of ARCT-032. Cohorts 1 to 3 receive nebulized ARCT-032 daily for 4 weeks followed by an 8-week safety follow-up, totaling 12 weeks of participation. Cohort 4 receives nebulized ARCT-032 daily for 12 weeks, then continues safety monitoring for another 12 weeks. The study evaluates different dose levels to understand how the treatment behaves and its impact on lung function and quality of life. During the study, participants undergo regular assessments including monitoring adverse events, measuring drug levels in the blood, lung function tests, and completing quality of life questionnaires. Imaging scans and health questionnaires specific to CF are collected, especially in the longer treatment group. Safety and tolerability are closely observed throughout the treatment and follow-up periods, which can last up to 24 weeks depending on the cohort.

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis