Status:
NOT_YET_RECRUITING
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
Lead Sponsor:
Peking Union Medical College Hospital
Conditions:
Cystic Fibrosis (CF)
Gene Therapy
Eligibility:
All Genders
18+ years
Phase:
EARLY_PHASE1
Brief Summary
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...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- Individuals who meet any of the following criteria are not eligible for inclusion in this study:
- 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.
- 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;
- During screening, positive for human immunodeficiency virus antibodies (HIV Ab) and positive for Treponema pallidum specific antibodies (TPPA);
- 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);
- Pregnant (positive blood pregnancy test result) or lactating women;
- 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;
- Investigators believe that any medical indicators (including other laboratory test indicators with clinical significance) or other conditions that may affect clinical trials.
Key Trial Info
Start Date :
May 10 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 10 2027
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06963008
Start Date
May 10 2025
End Date
May 10 2027
Last Update
May 8 2025
Active Locations (0)
Enter a location and click search to find clinical trials sorted by distance.
No Results Found
We couldn’t find results for the location/zipcode entered or within the selected range. Please check your input or adjust your search.