Cystic Fibrosis (CF)Early Phase 1NCT06963008
A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis
A Early Phase 1 study testing inhaled BMD003(CFTR mRNA) in people with Cystic Fibrosis (CF) and Gene Therapy, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis (CF), Gene Therapy
- What is being tested?
- inhaled BMD003(CFTR mRNA)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 27 Jun 2025to 10 May 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
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 Adult Patients With Cystic Fibrosis
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.
Read more
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.
Read the detailed description
This is a prospective, single-center, open-label, single-dose-escalation clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of nebulized inhaled BMD003 (CFTR mRNA) in adult patients with cystic fibrosis.
The study adopts a sequential cohort dose-escalation design. Enrollment flexibility is applied to ensure adequate safety observation at each study level. Continuous safety monitoring will be conducted throughout the screening, dosing, and follow-up phases. Dose escalation to the next cohort will only be permitted after completion of a predefined safety observation period and comprehensive safety review of the previous dose level. Additional subjects may be enrolled within the current cohort if significant treatment-related adverse events are observed, to further characterize the safety profile of BMD003.
Each eligible subject will complete a screening period of up to 2 weeks to confirm study eligibility. Qualified participants will be hospitalized for study drug administration on Day 1. Subjects will receive a single nebulized inhalation of BMD003 under medical supervision. In-hospital safety observation will be conducted for 24 hours after dosing, and subjects will be discharged upon satisfactory safety assessment on Day 2.
Pharmacokinetic blood sampling, vital sign monitoring, physical examinations, laboratory tests, and adverse event recording will be performed at scheduled time points throughout the study. After discharge, subjects will continue long-term follow-up for approximately 13 months to monitor delayed safety events and collect continuous clinical outcome data.
Following completion of this single-dose escalation study, a multiple-dose clinical investigation of BMD003 will be conducted. The dosing regimen, dosing frequency, and study design of the multiple-dose phase will be determined based on preclinical study results and the clinical safety and PK data obtained from this single-dose trial, and will be implemented after ethical approval.
- Conditions
- Cystic Fibrosis (CF), Gene Therapy
- Being tested
- inhaled 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.
inhaled BMD003(CFTR mRNA)Drug
Also called BMD003
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: inhaled BMD003(CFTR mRNA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
To assess the change from baseline in expected percentage of forced expiratory volume in one second (ppFEV1) following single-dose inhalation of BMD003. Lung function will be measured at scheduled study visits to evaluate preliminary efficacy.
Measured: From enrollment to the end of treatment at 13 months
Changes in the revised cystic fibrosis questionnaire (CFQ-R score) compared to baseline
To evaluate changes from baseline in the Revised Cystic Fibrosis Questionnaire (CFQ-R) total and domain scores after single-dose inhalation of BMD003. The CFQ-R questionnaire will be completed by subjects at scheduled study visits to assess patient-reported outcomes.
Measured: From enrollment to the end of treatment at 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2025
- Study start27 Jun 2025
- Primary completion13 Dec 2026 (estimated)
- Study completion10 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis (CF)Studies 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
- 12 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
- NCT06963008
- Sponsor's protocol ID
- BMD003-001
- Status last verified
- Sep 2026
- First submitted
- 30 Apr 2025
- Last updated
- 1 Oct 2026
- Results posted
- 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 Adult Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT06963008
- Acronym / sponsor ID
- BMD003-001
- Conditions
- Cystic Fibrosis (CF), Gene Therapy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 27 Jun 2025 (actual)
- Primary completion
- 13 Dec 2026 (estimated)
- Study completion
- 10 May 2027 (estimated)
- First posted
- 8 May 2025 (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.
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.
Detailed description
This is a prospective, single-center, open-label, single-dose-escalation clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of nebulized inhaled BMD003 (CFTR mRNA) in adult patients with cystic fibrosis.
The study adopts a sequential cohort dose-escalation design. Enrollment flexibility is applied to ensure adequate safety observation at each study level. Continuous safety monitoring will be conducted throughout the screening, dosing, and follow-up phases. Dose escalation to the next cohort will only be permitted after completion of a predefined safety observation period and comprehensive safety review of the previous dose level. Additional subjects may be enrolled within the current cohort if significant treatment-related adverse events are observed, to further characterize the safety profile of BMD003.
Each eligible subject will complete a screening period of up to 2 weeks to confirm study eligibility. Qualified participants will be hospitalized for study drug administration on Day 1. Subjects will receive a single nebulized inhalation of BMD003 under medical supervision. In-hospital safety observation will be conducted for 24 hours after dosing, and subjects will be discharged upon satisfactory safety assessment on Day 2.
Pharmacokinetic blood sampling, vital sign monitoring, physical examinations, laboratory tests, and adverse event recording will be performed at scheduled time points throughout the study. After discharge, subjects will continue long-term follow-up for approximately 13 months to monitor delayed safety events and collect continuous clinical outcome data.
Following completion of this single-dose escalation study, a multiple-dose clinical investigation of BMD003 will be conducted. The dosing regimen, dosing frequency, and study design of the multiple-dose phase will be determined based on preclinical study results and the clinical safety and PK data obtained from this single-dose trial, and will be implemented after ethical approval.
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) | Experimental | Drug: inhaled BMD003(CFTR mRNA)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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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;
Key exclusion criteria (13 listed)
- 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.
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 |
|---|---|---|
| inhaled BMD003(CFTR mRNA)Also: BMD003BMD003 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. | Drug | inhaled BMD003(CFTR mRNA) |
Primary outcome
Observe adverse events (AE) and serious adverse events (SAE)
Time frame: From enrollment to the end of treatment at 13 months
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.
2 secondary outcomes
Expected percentage of forced expiratory volume in one second (ppFEV1) change from baseline
Time frame: From enrollment to the end of treatment at 13 months
Changes in the revised cystic fibrosis questionnaire (CFQ-R score) compared to baseline
Time frame: From enrollment to the end of treatment at 13 months
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
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 inhaled BMD003(CFTR mRNA) in people with Cystic Fibrosis (CF) and Gene Therapy, 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 10 inclusion and 13 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: under way, not looking for participants, 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Observe adverse events (AE) and serious adverse events (SAE) (time frame: From enrollment to the end of treatment at 13 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.