Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: GM-CSF (granulocyte-macrophage colony-stimulating factor).
- Who it may be relevant to
- Registry conditions: Pulmonary Alveolar Proteinosis. Basic parameters: 18 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Single-arm Clinical Study of Local Spraying of GM-CSF Via Bronchoscopy in the Treatment of Autoimmune Pulmonary Alveolar Proteinosis
Overview
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.
Interventions
- Drug 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.
Primary outcome measures
- Chest HRCT [Time frame: 12 weeks]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT06989333 · 2025051795