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

NCT ID: NCT06989333

Last Updated: 2025-05-25

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-07-01
• Study Completion Date: 2027-06-30

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Spraying GM-CSF

Group Type: EXPERIMENTAL

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

Intervention Type: 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.

Interventions

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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jiuwu Bai

OTHER

Sponsor Role: lead

Responsible Party

Jiuwu Bai

associate chief physician

Responsibility Role: SPONSOR_INVESTIGATOR

Central Contacts

Jiu-Wu Bai Doctor

Role: CONTACT

yv59@163.com

13651602925

Other Identifiers

2025051795

Identifier Type: -

Identifier Source: org_study_id