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

NCT06989333 | Not Yet Recruiting Phase 2

• 1 other identifier: 2025051795
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Pulmonary Alveolar Proteinosis

Outcome Measures

Primary Outcomes (1)

• Chest HRCT

  12 weeks

Study Arms (1)

Spraying GM-CSF

EXPERIMENTAL

Drug: GM-CSF (granulocyte-macrophage colony-stimulating factor)

Interventions

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.

Spraying GM-CSF

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: 18 to 60 years old;
• A clear diagnosis of aPAP must meet at least one of the following diagnostic criteria:
• \) 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.
• \. No other PAP specific treatments (such as WLL, inhaled GM-CSF, biological agents, etc.) have been received recently (for more than 4 weeks).
• \. Agree to participate in this study and sign the informed consent form.

You may not qualify if:

• 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;
• 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.

Sponsors & Collaborators

• Jiuwu Bai: lead

MeSH Terms

Conditions

Pulmonary Alveolar Proteinosis

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Central Study Contacts

Jiu-Wu Bai Doctor

CONTACT

13651602925; yv59@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: associate chief physician

Study Record Dates

• First Submitted: May 17, 2025
• First Posted: May 25, 2025
• Study Start: July 1, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027
• Last Updated: May 25, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share