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

Sponsor
Peking Union Medical College Hospital
Study ID
NCT07485543
Phase
EARLY_PHASE1
Status
Not Yet Recruiting

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Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • BMD003 (CFTR mRNA) — DRUG
    Nebulized inhalation of BMD003 (CFTR mRNA) lyophilized preparation. The drug is reconstituted with sterile water for injection to the required concentration before use. Participants receive weekly administration for 12 consecutive weeks. Prior to each administration, airway clearance therapy (e.g., active cycle of breathing technique) is performed as standard care, with short-acting bronchodilators permitted if clinically indicated.

Study Details

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.

Key Dates

First listed
Mar 20, 2026
Start date
Mar 31, 2026
Status verified
Mar 2026
Primary completion
Dec 31, 2026
Completion
Dec 31, 2027

Study Design

Enrollment
24 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Inhaled BMD003 (CFTR mRNA) Multiple Administration Arm
    Subjects receive inhaled BMD003 (CFTR mRNA) via vibrating mesh nebulizer once weekly for 12 consecutive weeks. All safety evaluations, efficacy assessments, pharmacokinetic sampling, and follow-up procedures are conducted in accordance with the study protocol.

Primary Outcome Measure

Incidence of adverse events (AE) and serious adverse events (SAE) assessed by CTCAE 6.0 [ Time Frame: From first dose up to approximately 12 months ]

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