The study adopts a prospective, single-center, open-label clinical study to evaluate the safety and efficacy of inhaled BMD003(CFTR mRNA) in Chinese adult patients with cystic fibrosis This study is a single dose escalation study of BMD003 nebulized inhalation in CF patients.
The study is divided into four cohorts, they are 2mg, 4mg, 8mg, and 16mg dose groups. The first dose group (2mg) is planned to enroll one patient. During the study, if there are ≥ 2 grade treatment-related adverse events (TRAEs) related to the investigational product that occur during the study period, 2 patients will be added at the current dose level. Each of the remaining groups will include 2-3 subjects, regardless of gender. Based on the safety data review of subjects in the previous dose group 4 weeks after administration, subjects in the next dose group will be allowed to receive the next higher dose, while patients in the previous dose group will complete the study.
The study includes three stages: screening period (2 weeks), administration period (1 day), and follow-up period (approximately 13 months). After passing the screening of the subjects, D1 was admitted to the hospital, D1 received a single nebulized inhalation investigational product (BMD003), and D2 completed safety observation before being discharged.
During the study, PK sample collection and safety assessment will be conducted at the corresponding visit points. After the treatment is completed, each patient will be followed up according to the schedule specified in the protocol.This study will conduct multiple dosing studies after the end of a single dose escalation. Multiple dosing doses, dosing intervals, etc. will be revised based on preclinical data and single dose data, and will be implemented after ethical approval.
Gender not limited, 18 years old and above (including threshold values);
The subjects themselves agree to participate in the study and sign an informed consent form
Diagnosed with cystic fibrosis (CF);
According to the investigators' assessment, the subject's cystic fibrosis disease is clinically stable;
Acute respiratory infection, acute exacerbation of lung disease, clinically significant hemoptysis (>30 mL, or as determined by the investigator), or changes in respiratory medications (including antibiotics, oral steroids, etc.) used to treat CF occurred within 4 weeks prior to the first administration;
During screening, the 12 lead electrocardiogram showed prolonged QTcF interval (>450 ms for males and>460 ms for females) and clinically significant abnormalities in other indicators of the 12 lead electrocardiogram.
During screening, total bilirubin exceeded the upper limit of normal, ALT and AST exceeded three times the upper limit of normal, and blood creatinine (CRE) was ≥ 1.5 times the upper limit of normal.
History of physical organ or blood transplantation or registered waiting for transplantation;
Single group
inhaled BMD003(CFTR mRNA)
DrugBMD003 is the world's first freeze-dried CFTR Mrna product (stored at 2-8 ℃), which is reconstituted with injection water before use, nebulized through a vibrating sieve atomizer, and inhaled into the lungs through the mouth and nose.
Observe adverse events (AE) and serious adverse events (SAE)
Expected percentage of forced expiratory volume in one second (ppFEV1) change from baseline
Changes in the revised cystic fibrosis questionnaire (CFQ-R score) compared to baseline
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