Pulmonary Alveolar ProteinosisPhase 2aPAP and GMCSFNCT06989333
Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
A Phase 2 study testing GM-CSF (granulocyte-macrophage colony-stimulating factor) in people with Pulmonary Alveolar Proteinosis, run by Jiuwu Bai.
Trial summary
This study aims to explore a new therapeutic approach: the feasibility, safety and preliminary efficacy of directly spraying GM-CSF into the airway through bronchoscopy for the treatment of aPAP. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Pulmonary Alveolar Proteinosis
- What is being tested?
- GM-CSF (granulocyte-macrophage colony-stimulating factor)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2025to 30 Jun 2027
- Phase
- Phase 2
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age: 18 to 60 years old;
- A clear diagnosis of aPAP must meet at least one of the following diagnostic criteria:
- 1) BALF appears "milky white"; Or cytological examination reveals a large amount of PAS-positive protein deposition; 2) HRCT shows typical "paving stone-like changes"; 3) Positive for serum GM-CSF antibody. 3. There are more than one of the following treatment indications: Symptoms such as progressive breathing difficulties, coughing, and shortness of breath after activity occur; 2) Without oxygen inhalation, PaO2 > 65 mmHg 3) Pulmonary function DLCO accounts for % of the predicted value, ranging from 60% to 80%, including the critical value.
- 4. No other PAP specific treatments (such as WLL, inhaled GM-CSF, biological agents, etc.) have been received recently (for more than 4 weeks).
- 5. Agree to participate in this study and sign the informed consent form.
Who cannot join
- Secondary PAP (such as secondary to blood diseases, etc.);
- Patients with obvious pulmonary fibrosis, emphysema or irreversible lung function impairment;
- Patients in the acute exacerbation stage;
- Patients with other lung diseases (such as active pulmonary tuberculosis, bronchiectasis with purulent infection or other chronic infections; Have severe asthma, chronic bronchospasm, etc.
- Have a history of allergy to GM-CSF antibodies or related drug components;
- Patients who have participated in other clinical drug trials within the past three months;
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- Patients who have experienced severe complications related to bronchoscopy or are intolerant to bronchoscopy;
- Concurrent with other serious cardiovascular and cerebrovascular diseases, hematological disorders, malignant tumors, etc.
- Pregnant or lactating women.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Single-arm Clinical Study of Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
This study aims to explore a new therapeutic approach: the feasibility, safety and preliminary efficacy of directly spraying GM-CSF into the airway through bronchoscopy for the treatment of aPAP.
- Conditions
- Pulmonary Alveolar Proteinosis
- Being tested
- GM-CSF (granulocyte-macrophage colony-stimulating factor)
- Medical subject headings
- Pulmonary Alveolar Proteinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GM-CSF (granulocyte-macrophage colony-stimulating factor)Drug
Bronchoscopy under general anesthesia or sedation; The predetermined dose of recombinant GM-CSF was dissolved in normal saline. The diseased lung segments were located through bronchoscopy and the liquid medicine was sprayed.
| Study group | Role | What they receive |
|---|---|---|
| Spraying GM-CSF | Experimental | Not described Receives: Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Chest HRCT
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2025
- Study start1 Jul 2025 (estimated)
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary Alveolar ProteinosisStudies of the same condition, open and under way.
- 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jiuwu Bai
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Jiuwu Bai, Shanghai Pulmonary Hospital, Shanghai, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06989333
- Sponsor's protocol ID
- 2025051795
- Acronym
- aPAP and GMCSF
- Status last verified
- May 2025
- First submitted
- 17 May 2025
- Last updated
- 25 May 2025
- 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 25 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm Clinical Study of Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
- ClinicalTrials.gov ID
- NCT06989333
- Acronym / sponsor ID
- aPAP and GMCSF / 2025051795
- Conditions
- Pulmonary Alveolar Proteinosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Jiuwu BaiUniversity, hospital or other
- Study start
- 1 Jul 2025 (estimated)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 25 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.
This study aims to explore a new therapeutic approach: the feasibility, safety and preliminary efficacy of directly spraying GM-CSF into the airway through bronchoscopy for the treatment of aPAP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Spraying GM-CSF | Experimental | Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor) |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age: 18 to 60 years old;
- A clear diagnosis of aPAP must meet at least one of the following diagnostic criteria:
- 1) BALF appears "milky white"; Or cytological examination reveals a large amount of PAS-positive protein deposition; 2) HRCT shows typical "paving stone-like changes"; 3) Positive for serum GM-CSF antibody. 3. There are more than one of the following treatment indications: Symptoms such as progressive breathing difficulties, coughing, and shortness of breath after activity occur; 2) Without oxygen inhalation, PaO2 > 65 mmHg 3) Pulmonary function DLCO accounts for % of the predicted value, ranging from 60% to 80%, including the critical value.
- 4. No other PAP specific treatments (such as WLL, inhaled GM-CSF, biological agents, etc.) have been received recently (for more than 4 weeks).
- 5. Agree to participate in this study and sign the informed consent form.
Key exclusion criteria (9 listed)
- Secondary PAP (such as secondary to blood diseases, etc.);
- Patients with obvious pulmonary fibrosis, emphysema or irreversible lung function impairment;
- Patients in the acute exacerbation stage;
- Patients with other lung diseases (such as active pulmonary tuberculosis, bronchiectasis with purulent infection or other chronic infections; Have severe asthma, chronic bronchospasm, etc.
- Have a history of allergy to GM-CSF antibodies or related drug components;
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 |
|---|---|---|
| GM-CSF (granulocyte-macrophage colony-stimulating factor)Bronchoscopy under general anesthesia or sedation; The predetermined dose of recombinant GM-CSF was dissolved in normal saline. The diseased lung segments were located through bronchoscopy and the liquid medicine was sprayed. | Drug | Spraying GM-CSF |
Primary outcome
Chest HRCT
Time frame: 12 weeks
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 25 May 2025.
- 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GM-CSF (granulocyte-macrophage colony-stimulating factor) in people with Pulmonary Alveolar Proteinosis, run by Jiuwu Bai.
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 5 inclusion and 9 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: opening soon, as of the sponsor's last update on 25 May 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Jiu-Wu Bai Doctor (13651602925, yv59@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Chest HRCT (time frame: 12 weeks). 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 25 May 2025.