Phase 1 Study of Inhaled KB407 for Treating Cystic Fibrosis in Adults

What this Trial Is About

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.

A Study Assessing KB407 for the Treatment of Cystic Fibrosis