Northfield

Researchers are evaluating the safety of aerosolized RSP-1502 in people with cystic fibrosis who have chronic lung infections caused by Pseudomonas aeruginosa. This phase 1b/2a study compares different doses of RSP-1502 to an active control, aiming to find the maximum tolerated dose (MTD) and assess safety outcomes. Participants must meet specific lung function and infection criteria to join the study. The study involves administering RSP-1502 or an active control solution by inhalation using a nebulizer for 14 days. RSP-1502 contains tobramycin and CaEDTA in a sterile solution, while the active control is a tobramycin inhalation solution. After dose escalation to identify the MTD, a dose expansion phase compares the MTD of RSP-1502 to the active control for another 14 days. Participants will then be followed for 14 days after treatment ends. Participants will have their lung function tested with spirometry and undergo electrocardiograms on specific days during treatment. Researchers will monitor for any treatment-related adverse events and serious adverse events throughout the 28-day treatment and follow-up period. They will also track pulmonary exacerbations and other safety measures. The total participation includes dosing and a 14-day follow-up after treatment completion.

Researchers are evaluating the safety and tolerability of increasing doses of KB407, a nebulized biological treatment, in adults with cystic fibrosis. This Phase 1 study aims to assess how well patients with confirmed cystic fibrosis respond to inhaled KB407, which is designed to express the human cystic fibrosis transmembrane conductance regulator (CFTR) protein. The study focuses on adults who are clinically stable and meet specific lung function and oxygen saturation criteria. Participants will receive nebulized KB407, a solution containing a replication-defective HSV-1 vector that carries the full-length human CFTR gene. The study involves ascending doses to monitor safety and tolerability. The treatment is inhaled through nebulization, and the study may include multiple dosing times to evaluate the response over a six-month period. During the study, participants will be closely monitored for adverse events, changes in physical exams, vital signs, electrocardiograms, and laboratory tests. Safety assessments will track the frequency, severity, and relationship of any side effects to the treatment. The total duration of participation includes safety and monitoring visits spread over six months to thoroughly evaluate the effects and tolerability of KB407.

Researchers are evaluating ARCT-032 in adults with cystic fibrosis (CF) who cannot use CFTR modulator therapies due to intolerance, poor response, or lack of access. This Phase 2, open-label study aims to assess the safety, tolerability, and efficacy of ARCT-032, a CFTR mRNA treatment formulated in lipid nanoparticles, in this specific group of adults with confirmed CF. Participants in cohorts 1 to 3 will receive daily nebulized ARCT-032 for 4 weeks, followed by a 12-week safety monitoring period. Participants in cohort 4 will receive daily nebulized ARCT-032 for 12 weeks, with an additional 12-week safety follow-up. The study uses a multiple-ascending dose design to evaluate different dosing levels of the investigational treatment. During the study, researchers will monitor adverse events related to the treatment over 16 weeks for cohorts 1 to 3 and 24 weeks for cohort 4. Participants will undergo lung function testing and safety assessments throughout the treatment and follow-up periods. The study lasts up to 24 weeks, including dosing and safety monitoring, to fully evaluate how well participants tolerate ARCT-032 and its potential effects.