Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis

NCT06989333 — Phase 2, Not yet recruiting, 10 participants, Jiuwu Bai

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A Single-arm Clinical Study of Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis

Registry	NCT06989333, acronym aPAP and GMCSF
Status	Not yet recruiting
Phase	Phase 2
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	10 (Estimated)
Sponsor	Jiuwu Bai (Other)
Conditions	Pulmonary Alveolar Proteinosis
Interventions	GM-CSF (granulocyte-macrophage colony-stimulating factor) (Drug)
Dates	start 2025-07-01 (estimated), primary completion 2027-06-30 (estimated), completion 2027-06-30 (estimated), first posted 2025-05-25, last updated 2025-05-25

-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.

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+Arms and interventions

Arm	Type	Interventions	Description
Spraying GM-CSF	Experimental	Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor)

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.

+Outcome measures

Primary outcomes

Chest HRCT [12 weeks]

+Eligibility

Sex: All, ages: 18 Years to 80 Years

Inclusion Criteria:

1. Age: 18 to 60 years old;
2. 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.

Exclusion Criteria:

1. Secondary PAP (such as secondary to blood diseases, etc.);
2. Patients with obvious pulmonary fibrosis, emphysema or irreversible lung function impairment;
3. Patients in the acute exacerbation stage;
4. Patients with other lung diseases (such as active pulmonary tuberculosis, bronchiectasis with purulent infection or other chronic infections; Have severe asthma, chronic bronchospasm, etc.
5. Have a history of allergy to GM-CSF antibodies or related drug components;
6. Patients who have participated in other clinical drug trials within the past three months;
7. Patients who have experienced severe complications related to bronchoscopy or are intolerant to bronchoscopy;
8. Concurrent with other serious cardiovascular and cerebrovascular diseases, hematological disorders, malignant tumors, etc.
9. Pregnant or lactating women.