Safety and Efficacy of Inhaled BMD003 (CFTR mRNA) in Chinese Cystic Fibrosis Patients Aged ≥12 Years

NCT07485543 | Not Yet Recruiting Early Phase 1

• 1 other identifier: BMD003-002
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a prospective, single-center, open-label clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of inhaled BMD003 (CFTR mRNA) in Chinese patients aged 12 years and above with cystic fibrosis (CF) following multiple administrations. Additionally, the study aims to explore the pharmacokinetic characteristics, immunogenicity, and relevant biomarkers of the drug. The study adopts a multiple-dose escalation design. Eligible patients will be sequentially enrolled into different cohorts, and the next cohort may receive a higher dose only after the safety data review of the previous dose cohort is completed. The entire study consists of four phases: screening period, safety observation period, continuous treatment period, and long-term follow-up period. Participants will receive nebulized inhalation of the study drug at the specified frequency. During the study, blood, sputum samples, and nasal swabs will be collected at designated time points for pharmacokinetic, immunogenicity, cytokine, and other related detections. Moreover, lung function tests, chest imaging, electrocardiograms, sweat chloride concentration tests, and other examinations will be performed at each visit. Meanwhile, the Cystic Fibrosis Questionnaire-Revised (CFQ-R) will be used to assess the patients' health-related quality of life, so as to comprehensively evaluate the safety and efficacy of the study drug. Adverse events will be closely monitored and recorded throughout the study. The safety of the study drug will be comprehensively evaluated by summarizing various safety indicators such as adverse events and laboratory tests. All statistical analyses will be performed using professional statistical software, and descriptive statistical methods will be employed to analyze the safety, efficacy, pharmacokinetic, and other related data of the study drug. The total duration of this study is 1 year, which is intended to provide a basis for the selection of the recommended dose for Phase Ⅱ clinical trials.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events (AE) and serious adverse events (SAE) assessed by CTCAE 6.0

  All adverse events (AEs) and serious adverse events (SAEs) will be monitored and recorded throughout the study. Severity will be graded using the NCI CTCAE version 6.0, and their relationship to the study drug will be evaluated at each study visit.

  From first dose up to approximately 12 months

Secondary Outcomes (2)

• Change in percent predicted forced expiratory volume in 1 second (ppFEV1) measured by spirometry

  Baseline, Week 1, Week 2, Week 4, Week 8, Week 12

• Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) score

  Baseline, Week 2, Week 4, Week 8, Week 12

Other Outcomes (4)

• Change in sweat chloride concentration measured by quantitative pilocarpine iontophoresis

  Baseline, Week 4, Week 12

• Change in daily sputum production volume

  Baseline up to Week 12

• Change in sputum solid content

  Baseline, Week 1, Week 2, Week 4, Week 8, Week 12

Study Arms (1)

Inhaled BMD003 (CFTR mRNA) Multiple Administration Arm

EXPERIMENTAL

Subjects receive inhaled BMD003 (CFTR mRNA) via vibrating mesh nebulizer once weekly for 12 consecutive weeks. All safety evaluations, efficacy assessments, pharmacokinetic sampling, and follow-up procedures are conducted in accordance with the study protocol.

Drug: BMD003 (CFTR mRNA)

Interventions

BMD003 (CFTR mRNA) DRUG

Nebulized inhalation of BMD003 (CFTR mRNA) lyophilized preparation. The drug is reconstituted with sterile water for injection to the required concentration before use. Participants receive weekly administration for 12 consecutive weeks. Prior to each administration, airway clearance therapy (e.g., active cycle of breathing technique) is performed as standard care, with short-acting bronchodilators permitted if clinically indicated.

Also known as: BMD003

Inhaled BMD003 (CFTR mRNA) Multiple Administration Arm

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 12 years and above (inclusive), regardless of gender.
• Voluntarily sign the informed consent form (legal guardians sign for minors \<18 years old, with minor assent if needed).
• Confirmed diagnosis of cystic fibrosis (CF) with clinically stable condition.
• Weight ≥40 kg (≥18 years old) or ≥30 kg (\<18 years old), or deemed eligible by the investigator.
• Predicted FEV1 ≥40% of the normal value and resting SpO2 ≥92% at screening.
• SAD trial participants: ≥3 months after last dose, no residual CFTR protein/mRNA in nasal epithelial cells (confirmed by lab tests).
• Quit smoking for at least 2 years.
• Willing and able to comply with all study procedures and follow-up plans.

You may not qualify if:

• Acute respiratory/pulmonary events, significant hemoptysis, or changed CF respiratory medications within 1 month before the first dose.
• Infected with highly virulent bacteria (e.g., \*Burkholderia cepacia\*, \*Mycobacterium abscessus\*), except for controllable colonization without clinical symptoms.
• Clinically significant ECG abnormalities (e.g., prolonged QTcF: male \>450ms, female \>460ms) at screening.
• Abnormal liver/kidney function at screening (TBIL \>ULN, ALT/AST \>3×ULN, CRE ≥1.5×ULN).
• History of solid organ/bone marrow transplantation or on transplant waiting list.
• Positive HIV, syphilis, HBsAg or HCV antibodies at screening.
• Participated in inhaled drug/device studies or used CFTR modulators within 30 days before screening.
• Pregnant or lactating females.
• A history of allergies to inhaled drug components or other allergies deemed contraindicated by the investigator.
• Any other medical conditions/circumstances that may interfere with the trial (judged by the investigator)

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

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: March 11, 2026
• First Posted: March 20, 2026
• Study Start: March 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: March 20, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share