A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis

Korábbi kezelés

• 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;
• Infected with highly virulent bacteria associated with accelerated decline in lung function and/or shortened survival time (e.g. Burkholderia cepacia, Mycobacterium abscesses). For subjects with a positive culture history, they can be considered uninfected according to the following guidelines:
  • All culture results obtained in the past 12 months have been negative. Subject should have undergone at least 2 cultures within the past 12 months, with a minimum interval of 3 months between each culture, and at least one culture should have been conducted within 6 months prior to screening in this study.
• Participated in inhalation drug or device studies within 30 days prior to the initial screening visit (a 30 day window period applies to inhalation drugs with elimination half-life<6 days. If the elimination half-life of inhalation drugs is ≥ 6 days, the window period should be extended to at least 5 half lives after the last administration);

Más betegség

• History of physical organ or blood transplantation or registered waiting for transplantation;
• During screening, positive for human immunodeficiency virus antibodies (HIV Ab) and positive for Treponema pallidum specific antibodies (TPPA);
• Having a history of drug allergies or other allergies, which investigators consider to be contraindications for participation in the study;
• History of drug allergies or other allergies, and investigators believe it contraindications for participation in the study;

Laborértékek

• 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.
• Investigators believe that any medical indicators (including other laboratory test indicators with clinical significance) or other conditions that may affect clinical trials.

Egyéb

• Individuals who meet any of the following criteria are not eligible for inclusion in this study:
• 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.
• Pregnant (positive blood pregnancy test result) or lactating women;

• Patients must meet all of the following inclusion criteria to be included in this study:
• 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;
• During screening, the subject's body mass index (BMI) is ≥ 17.5 kg/m ² and their weight is ≥ 40 kg;
• Forced Expiratory Volume in 1 second (FEV1) is expected to be ≥ 40% of the normal value;
• Resting oxygen saturation (SpO2) ≥ 92%;
• Quit smoking for at least 2 years;
• The subjects are willing and able to comply with the scheduled visits, treatments, laboratory tests, contraception requirements, and other study procedures.