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

NCT06963008 | Active Not Recruiting Early Phase 1

• 1 other identifier: BMD003-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The study uses a prospective, single-center, open-label clinical trial design to evaluate the safety and efficacy of inhaled BMD003 (CFTR mRNA) in adult Chinese patients with cystic fibrosis. This is a single-dose escalation study of nebulized inhaled BMD003 in patients with cystic fibrosis. The trial adopts sequential dose-escalation cohorts with flexible subject enrollment. Continuous safety monitoring will be performed throughout the study. If treatment-related adverse events meeting specified grade criteria occur during the trial, additional subjects will be enrolled at the current dose level for further safety observation. Subsequent dose escalation will proceed following a comprehensive safety evaluation of the prior cohort. The trial consists of three phases: a 2-week screening period, a 1-day drug administration period, and a long-term follow-up period of approximately 13 months. After successful screening and meeting eligibility criteria, subjects will be admitted on Day 1 to receive nebulized inhalation of the investigational product. Comprehensive safety observations will be completed on Day 2 prior to hospital discharge. Pharmacokinetic sampling and systematic safety assessments will be conducted at predefined study visits. All subjects will complete protocol-specified follow-up procedures after single-dose administration. Upon completion of this single-dose escalation phase, a multiple-dose study of BMD003 will be initiated. The detailed multiple-dose regimen will be defined based on preclinical findings and clinical data from this single-dose study, and implemented after ethical review and approval.

Conditions

Cystic Fibrosis (CF); Gene Therapy

Outcome Measures

Primary Outcomes (1)

• Observe adverse events (AE) and serious adverse events (SAE)

  To assess the incidence, type, severity, and relatedness of adverse events (AEs) and serious adverse events (SAEs) after single-dose inhalation of BMD003. Safety will be evaluated via physical examination, vital signs, laboratory tests, and adverse event reporting.

  From enrollment to the end of treatment at 13 months

Secondary Outcomes (2)

• Expected percentage of forced expiratory volume in one second (ppFEV1) change from baseline

  From enrollment to the end of treatment at 13 months

• Changes in the revised cystic fibrosis questionnaire (CFQ-R score) compared to baseline

  From enrollment to the end of treatment at 13 months

Study Arms (1)

inhaled BMD003(CFTR mRNA)

EXPERIMENTAL

Single-dose nebulized inhalation of BMD003 (CFTR mRNA). This is a sequential dose-escalation cohort study. Subjects receive a single administration of the investigational product on Day 1, followed by safety and PK assessments during screening, in-hospital observation, and long-term follow-up.

Drug: inhaled BMD003(CFTR mRNA)

Interventions

inhaled BMD003(CFTR mRNA) DRUG

BMD003 is a freeze-dried CFTR mRNA product stored at 2-8°C. It is reconstituted with sterile water for injection before use, nebulized via a vibrating mesh nebulizer, and inhaled into the lungs through the mouth and nose.

Also known as: BMD003

inhaled BMD003(CFTR mRNA)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief physician, professor

Study Record Dates

• First Submitted: April 30, 2025
• First Posted: May 8, 2025
• Study Start: June 27, 2025
• Primary Completion (Estimated): December 13, 2026
• Study Completion (Estimated): May 10, 2027
• Last Updated: October 1, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)