Inhaled CFTR mRNA for cystic fibrosis in Chinese patients aged 12 and older

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single-center, open-label, early-phase study in Chinese CF patients aged 12 and above to evaluate the safety, tolerability, and preliminary efficacy of repeatedly inhaled BMD003 (CFTR mRNA). The study uses a multiple-dose escalation design across cohorts, with safety reviews guiding dose progression, and includes a screening, safety observation, continuous treatment, and long-term follow-up period. Key measurements include safety labs, vital signs, ECG, pulmonary function, ppFEV1, and CFQ-R, plus exploratory biomarkers in blood, sputum, and other samples to characterize pharmacokinetics and immunogenicity. The trial aims to determine whether repeated inhaled CFTR mRNA can produce meaningful clinical signals and acceptable safety in this population.

Why it matters

Who can join: the full rules

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)

Where it's running

• Peking Union Medical College Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Xin-Lun Tian, M.D.
• Email: xinlun_t@sina.com
• Phone: +86-10-69155039

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.