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New inhaled mRNA therapy aims to fix cystic fibrosis at its source

NCT ID NCT07485543

Disease control Sponsor: Peking Union Medical College Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tests an inhaled mRNA drug (BMD003) in 24 people aged 12 and older with cystic fibrosis. The goal is to see if it is safe and can improve lung function by delivering instructions to make a working CFTR protein. Participants will receive multiple doses and be monitored for side effects and changes in breathing.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Mar 2026

An estimate. Start dates often move.

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Aged 12 years and above (inclusive), regardless of gender. 2. Voluntarily sign the informed consent form (legal guardians sign for minors \<18 years old, with minor assent if needed). 3. Confirmed diagnosis of cystic fibrosis (CF) with clinically stable condition. 4. Weight ≥40 kg (≥18 years old) or ≥30 kg (\<18 years old), or deemed eligible by the investigator. 5. Predicted FEV1 ≥40% of the normal value and resting SpO2 ≥92% at screening. 6. SAD trial participants: ≥3 months after last dose, no residual CFTR protein/mRNA in nasal epithelial cells (confirmed by lab tests). 7. Quit smoking for at least 2 years. 8. Willing and able to comply with all study procedures and follow-up plans. Exclusion Criteria: 1. Acute respiratory/pulmonary events, significant hemoptysis, or changed CF respiratory medications within 1 month before the first dose. 2. Infected with highly virulent bacteria (e.g., \*Burkholderia cepacia\*, \*Mycobacterium abscessus\*), except for controllable colonization without clinical symptoms. 3. Clinically significant ECG abnormalities (e.g., prolonged QTcF: male \>450ms, female \>460ms) at screening. 4. Abnormal liver/kidney function at screening (TBIL \>ULN, ALT/AST \>3×ULN, CRE ≥1.5×ULN). 5. History of solid organ/bone marrow transplantation or on transplant waiting list. 6. Positive HIV, syphilis, HBsAg or HCV antibodies at screening. 7. Participated in inhaled drug/device studies or used CFTR modulators within 30 days before screening. 8. Pregnant or lactating females. 9. A history of allergies to inhaled drug components or other allergies deemed contraindicated by the investigator. 10. Any other medical conditions/circumstances that may interfere with the trial (judged by the investigator)

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Conditions

The condition(s) this trial relates to.

cystic fibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cystic fibrosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  2. A doctor treating you

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More trials for these conditions

Other studies related to the condition(s) this trial covers.