Cystic FibrosisEarly Phase 1NCT07485543
Safety and Efficacy of Inhaled BMD003 (CFTR mRNA) in Chinese Cystic Fibrosis Patients Aged ≥12 Years
A Early Phase 1 study testing BMD003 (CFTR mRNA) in people with Cystic Fibrosis, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- BMD003 (CFTR mRNA)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteChina
- Study dates
- 3 Apr 2026to Dec 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Xin-Lun Tian, M.D., +86-10-69155039 ext. 86, xinlun_t@sina.com
About this study
Official title: A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Patients Aged 12 Years and Above With Cystic Fibrosis
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.
Read the detailed description
This is a prospective, single-center, open-label, multiple-dose clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of inhaled BMD003 (CFTR mRNA) administered repeatedly in Chinese patients with cystic fibrosis aged 12 years and older.
BMD003 is an investigational nebulized mRNA therapy that expresses functional human CFTR protein in airway epithelial cells to address the underlying genetic defect of cystic fibrosis. Eligible patients will receive inhaled BMD003, followed by a long-term follow-up period to monitor safety, durability of effect, and clinical outcomes.
The primary objective is to evaluate the safety and tolerability of repeated inhaled BMD003 by assessing adverse events (AEs), serious adverse events (SAEs), clinical laboratory tests, vital signs, physical examinations, 12-lead ECG, pulmonary function, and other safety parameters throughout the study.
The secondary objective is to assess preliminary efficacy by measuring changes from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) and in the score of the Cystic Fibrosis Questionnaire-Revised (CFQ-R).
Exploratory objectives include characterization of pharmacokinetic (PK) profiles in blood and sputum, evaluation of immunogenicity, assessment of sputum properties, sweat chloride concentration, nasal epithelial cell biomarkers, daily sputum volume, and other exploratory endpoints related to disease status and treatment response.
- Conditions
- Cystic Fibrosis
- Being tested
- BMD003 (CFTR mRNA)
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BMD003 (CFTR mRNA)Drug
Also called BMD003
Nebulized inhalation of BMD003 (CFTR mRNA) lyophilized preparation. The drug is reconstituted with sterile water for injection to the required concentration before use.
| Study group | Role | What they receive |
|---|---|---|
| Inhaled BMD003 (CFTR mRNA) Multiple Administration Arm | Experimental | Subjects receive inhaled BMD003 (CFTR mRNA) via vibrating mesh nebulize. All safety evaluations, efficacy assessments, pharmacokinetic sampling, and follow-up procedures are conducted in accordance with the study protocol. Receives: Drug: BMD003 (CFTR mRNA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
ppFEV1 will be measured by standardized spirometry at baseline and scheduled visits. The change from baseline will be calculated and analyzed.
Measured: Baseline, Week 1, Week 2, Week 4, Week 8, Week 12
Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) score
The CFQ-R will be completed by participants at baseline and follow-up visits. The change in total score from baseline will be evaluated.
Measured: Baseline, Week 2, Week 4, Week 8, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2026
- Study start3 Apr 2026
- Primary completionDec 2026 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xinlun Tian, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07485543
- Sponsor's protocol ID
- BMD003-002
- Status last verified
- Sep 2026
- First submitted
- 11 Mar 2026
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Patients Aged 12 Years and Above With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT07485543
- Acronym / sponsor ID
- BMD003-002
- Conditions
- Cystic Fibrosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 3 Apr 2026 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 20 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a prospective, single-center, open-label, multiple-dose clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of inhaled BMD003 (CFTR mRNA) administered repeatedly in Chinese patients with cystic fibrosis aged 12 years and older.
BMD003 is an investigational nebulized mRNA therapy that expresses functional human CFTR protein in airway epithelial cells to address the underlying genetic defect of cystic fibrosis. Eligible patients will receive inhaled BMD003, followed by a long-term follow-up period to monitor safety, durability of effect, and clinical outcomes.
The primary objective is to evaluate the safety and tolerability of repeated inhaled BMD003 by assessing adverse events (AEs), serious adverse events (SAEs), clinical laboratory tests, vital signs, physical examinations, 12-lead ECG, pulmonary function, and other safety parameters throughout the study.
The secondary objective is to assess preliminary efficacy by measuring changes from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) and in the score of the Cystic Fibrosis Questionnaire-Revised (CFQ-R).
Exploratory objectives include characterization of pharmacokinetic (PK) profiles in blood and sputum, evaluation of immunogenicity, assessment of sputum properties, sweat chloride concentration, nasal epithelial cell biomarkers, daily sputum volume, and other exploratory endpoints related to disease status and treatment response.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Inhaled BMD003 (CFTR mRNA) Multiple Administration Arm | Experimental | Drug: BMD003 (CFTR mRNA)Subjects receive inhaled BMD003 (CFTR mRNA) via vibrating mesh nebulize. All safety evaluations, efficacy assessments, pharmacokinetic sampling, and follow-up procedures are conducted in accordance with the study protocol. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BMD003 (CFTR mRNA)Also: BMD003Nebulized inhalation of BMD003 (CFTR mRNA) lyophilized preparation. The drug is reconstituted with sterile water for injection to the required concentration before use. | Drug | Inhaled BMD003 (CFTR mRNA) Multiple Administration Arm |
Primary outcome
Incidence of adverse events (AE) and serious adverse events (SAE) assessed by CTCAE 6.0
Time frame: From first dose up to approximately 12 months
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.
2 secondary outcomes
Change in percent predicted forced expiratory volume in 1 second (ppFEV1) measured by spirometry
Time frame: Baseline, Week 1, Week 2, Week 4, Week 8, Week 12
Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) score
Time frame: Baseline, Week 2, Week 4, Week 8, Week 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing BMD003 (CFTR mRNA) in people with Cystic Fibrosis, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xin-Lun Tian, M.D. (+86-10-69155039 ext. 86, xinlun_t@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (AE) and serious adverse events (SAE) assessed by CTCAE 6.0 (time frame: From first dose up to approximately 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026.