Clinical trial NCT01983657: Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
Official title: Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Pulmonary Alveolar Proteinosis
Keywords: Pulmonary
Pulmonary Alveolar Proteinosis
PAP
Lead sponsor: Shanghai Pulmonary Hospital, Shanghai, China
Sponsor class: OTHER
First posted: 2013-11-14
Enrollment: 40
Sex: ALL
Minimum age (years): 17
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP.

Detailed description: The purpose of this study is to establish an efficient and economic treatment scheme by evaluation of the safety and efficacy of subcutaneous injection of low-dose rhGM-CSF, or of similar injection after whole lung lavage , in patients with PAP. During the observation, study visits will occur at the end of each month. During the 1-year follow-up period which is lasting 6 months after the treatment, all participants will be required to check the various efficacy indicators.

Eligibility criteria: Inclusion Criteria:

* Diagnosed PAP patients
* Aged 17-80
* Signed informed consent

Exclusion Criteria:

* Secondary PAP
* Received whole lung lavage therapy within 4 weeks before enrollment
* Received previous GM-CSF therapy within 6 months before enrollment
* WBC≥12,000/ul
* fever≥38℃
* Severe edema, severe liver, kidney, lung and cardiovascular disease.
* Pregnant，planning to get pregnant or nursing
* Inability to express the subjective discomfort
* Serious drug allergy history, E.coli preparation or rhGM-CSF serious allergy history

Primary outcomes:
measure: Improvements in double pulmonary diffuse lesions （Chest CT score ）
timeFrame: 6 months
Secondary outcomes:
measure: Clinical symptoms observation: shod of breath, cough (according to each score standard）
timeFrame: 6 months
measure: Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change
timeFrame: 6 months
measure: Improvements in pulmonary function
timeFrame: 6 months
measure: Improvements in arterial blood gas, including alveolar-arterial oxygen difference(A-aDO2), partial pressure of oxygen(PaO2), partial pressure of carbon dioxide in artery(PaCO2), arterial oxygen saturation(SaO2).
timeFrame: 6 months
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai Pulmonary Hospital
Study officials:
affiliation: Shanghai Pulmonary Hospital, Shanghai, China
name: Huiping Li, Dr
role: PRINCIPAL_INVESTIGATOR
References:
citation: Zhang F, Weng D, Su Y, Yin C, Shen L, Zhang Y, Zhou Y, Li Q, Hu Y, Li H. Therapeutic effect of subcutaneous injection of low dose recombinant human granulocyte-macrophage colony-stimulating factor on pulmonary alveolar proteinosis. Respir Res. 2020 Jan 2;21(1):1. doi: 10.1186/s12931-019-1261-1.