A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis

The study uses a prospective, single-center, open-label clinical trial design to evaluate the safety and efficacy of inhaled BMD003 (CFTR mRNA) in adult Chinese patients with cystic fibrosis. This is a single-dose escalation study of nebulized inhaled BMD003 in patients with cystic fibrosis. The trial adopts sequential dose-escalation cohorts with flexible subject enrollment. Continuous safety monitoring will be performed throughout the study. If treatment-related adverse events meeting specified grade criteria occur during the trial, additional subjects will be enrolled at the current dose level for further safety observation. Subsequent dose escalation will proceed following a comprehensive safety evaluation of the prior cohort. The trial consists of three phases: a 2-week screening period, a 1-day drug administration period, and a long-term follow-up period of approximately 13 months. After successful screening and meeting eligibility criteria, subjects will be admitted on Day 1 to receive nebulized inhalation of the investigational product. Comprehensive safety observations will be completed on Day 2 prior to hospital discharge. Pharmacokinetic sampling and systematic safety assessments will be conducted at predefined study visits. All subjects will complete protocol-specified follow-up procedures after single-dose administration. Upon completion of this single-dose escalation phase, a multiple-dose study of BMD003 will be initiated. The detailed multiple-dose regimen will be defined based on preclinical findings and clinical data from this single-dose study, and implemented after ethical review and approval.