Phase 2 Study of Inhaled RCT2100 for Safety and Tolerability in Healthy Adults and People With Cystic Fibrosis

What this Trial Is About

Researchers are evaluating the safety, tolerability, and biodistribution of a drug called RCT2100 in both healthy adults and people with cystic fibrosis CF. This phase 2 study aims to provide initial safety data to support future clinical research. The study includes participants aged 18 to 60 years and assesses different dosing regimens and co-administration with another drug, ivacaftor, in those with CF. The study is divided into three parts. Part 1 involves a single ascending dose of inhaled RCT2100 given via nebulizer to healthy participants. Part 2 evaluates multiple ascending doses of inhaled RCT2100 in participants with CF over 4 to 12 weeks. Part 3 assesses the safety of RCT2100 combined with oral ivacaftor for 6 weeks in participants with CF after an initial ivacaftor run-in period. Placebo doses are also used in Part 1 for comparison. Participants will undergo various assessments including monitoring for adverse events and serious adverse events from baseline through follow-up periods up to 24 weeks for parts involving CF participants. They will receive study treatment by inhalation and oral dosing ivacaftor in Part 3 and have regular safety evaluations including clinical labs, vital signs, lung function tests, and ECGs. The study involves randomized allocation and double-blinding to evaluate safety and tolerability comprehensively over the treatment and follow-up periods.

A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF