Northfield

Researchers are evaluating the safety and tolerability of different doses of a nebulized treatment called KB407 in adults with cystic fibrosis, a genetic condition affecting the lungs. This is a Phase 1 study sponsored by Krystal Biotech, Inc., focusing on how well adults with cystic fibrosis tolerate this new inhaled therapy designed to deliver a functional version of the CFTR protein. Participants will receive KB407 through inhalation in one of several dosing schedules a single dose, two doses, four doses, or four consecutive doses followed by weekly doses up to six months. The study includes four open-label groups, each receiving different administration frequencies to assess safety and potential effects over time. During the study, participants will undergo evaluations of adverse events, physical exams, vital signs, ECGs, and laboratory tests over six months to monitor safety and tolerability. Researchers will also measure lung function changes using forced expiratory volume in one second FEV1. Participants will be closely monitored to track any side effects and changes in lung health throughout the treatment period and follow-up.

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.