Not Yet RecruitingEarly Phase 1

Safety and Efficacy of Inhaled BMD003 (CFTR mRNA) in Chinese Cystic Fibrosis Patients Aged ≥12 Years

24 participantsStarted 2026-03

Plain-language summary

This is a prospective, single-center, open-label clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of inhaled BMD003 (CFTR mRNA) in Chinese patients aged 12 years and above with cystic fibrosis (CF) following multiple administrations. Additionally, the study aims to explore the pharmacokinetic characteristics, immunogenicity, and relevant biomarkers of the drug. The study adopts a multiple-dose escalation design. Eligible patients will be sequentially enrolled into different cohorts, and the next cohort may receive a higher dose only after the safety data review of the previous dose cohort is completed. The entire study consists of four phases: screening period, safety observation period, continuous treatment period, and long-term follow-up period. Participants will receive nebulized inhalation of the study drug at the specified frequency. During the study, blood, sputum samples, and nasal swabs will be collected at designated time points for pharmacokinetic, immunogenicity, cytokine, and other related detections. Moreover, lung function tests, chest imaging, electrocardiograms, sweat chloride concentration tests, and other examinations will be performed at each visit. Meanwhile, the Cystic Fibrosis Questionnaire-Revised (CFQ-R) will be used to assess the patients' health-related quality of life, so as to comprehensively evaluate the safety and efficacy of the study drug. Adverse events will be closely monitored and recorded throughout the study. The safety of the study drug will be comprehensively evaluated by summarizing various safety indicators such as adverse events and laboratory tests. All statistical analyses will be performed using professional statistical software, and descriptive statistical methods will be employed to analyze the safety, efficacy, pharmacokinetic, and other related data of the study drug. The total duration of this study is 1 year, which is intended to provide a basis for the selection of the recommended dose for Phase Ⅱ clinical trials.

Who can participate

Age range12 Years

SexALL

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Inclusion criteria

✓. Aged 12 years and above (inclusive), regardless of gender.
✓. Voluntarily sign the informed consent form (legal guardians sign for minors \<18 years old, with minor assent if needed).
✓. Confirmed diagnosis of cystic fibrosis (CF) with clinically stable condition.
✓. Weight ≥40 kg (≥18 years old) or ≥30 kg (\<18 years old), or deemed eligible by the investigator.
✓. Predicted FEV1 ≥40% of the normal value and resting SpO2 ≥92% at screening.
✓. SAD trial participants: ≥3 months after last dose, no residual CFTR protein/mRNA in nasal epithelial cells (confirmed by lab tests).
✓. Quit smoking for at least 2 years.
✓. Willing and able to comply with all study procedures and follow-up plans.

Exclusion criteria

✕. Acute respiratory/pulmonary events, significant hemoptysis, or changed CF respiratory medications within 1 month before the first dose.

What they're measuring

Incidence of adverse events (AE) and serious adverse events (SAE) assessed by CTCAE 6.0

Timeframe: From first dose up to approximately 12 months

Trial details

NCT IDNCT07485543

SponsorPeking Union Medical College Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-12

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