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

RecruitingEARLY Phase 1Ages 12 Years - anyAllPeking Union Medical College Hospital
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
Inclusion Criteria: 1. Aged 12 years and above (inclusive), regardless of gender. 2. Voluntarily sign the informed consent form (legal guardians sign for minors \<18 years old, with minor assent if needed). 3. Confirmed diagnosis of cystic fibrosis (CF) with clinically stable condition. 4. Weight ≥40 kg (≥18 years old) or ≥30 kg (\<18 years old), or deemed eligible by the investigator. 5. Predicted FEV1 ≥40% of the normal value and resting SpO2 ≥92% at screening. 6. SAD trial participants: ≥3 months after last dose, no residual CFTR protein/mRNA in nasal epithelial cells (confirmed by lab tests). 7. Quit smoking for at least 2 years. 8. Willing and able to comply with all study procedures and follow-up plans. Exclusion Criteria: 1. Acute respiratory/pulmonary events, significant hemoptysis, or changed CF respiratory medications within 1 month before the first dose. 2. Infected with highly virulent bacteria (e.g., \*Burkholderia cepacia\*, \*Mycobacterium abscessus\*), except for controllable colonization without clinical symptoms. 3. Clinically significant ECG abnormalities (e.g., prolonged QTcF: male \>450ms, female \>460ms) at screening. 4. Abnormal liver/kidney function at screening (TBIL \>ULN, ALT/AST \>3×ULN, CRE ≥1.5×ULN). 5. History of solid organ/bone marrow transplantation or on transplant waiting list. 6. Positive HIV, syphilis, HBsAg or HCV antibodies at screening. 7. Participated in inhaled drug/device studies or used CFTR modulators within 30 days before screening. 8. Pregnant or lactating females. 9. A history of allergies to inhaled drug components or other allergies deemed contraindicated by the investigator. 10. Any other medical conditions/circumstances that may interfere with the trial (judged by the investigator)
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What it tests

  • BMD003 (CFTR mRNA)
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Where

1 site
Peking Union Medical College Hospital
Beijing, China
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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