A Phase 1/2 Study Evaluating Safety, Tolerability, and Biodistribution of RCT2100 via Nebulizer in Healthy Adults and Participants 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