Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP. (PAP)
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ClinicalTrials.gov Identifier: NCT01983657 |
Recruitment Status
: Unknown
Verified November 2013 by Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China.
Recruitment status was: Recruiting
First Posted
: November 14, 2013
Last Update Posted
: November 18, 2013
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Pulmonary Alveolar Proteinosis | Drug: rhGM-CSF Procedure: Whole Lung Lavage(WLL) | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 40 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 Start Date : | January 2012 |
Estimated Primary Completion Date : | June 2014 |
Estimated Study Completion Date : | October 2014 |
Arm | Intervention/treatment |
---|---|
Experimental: D1
Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). If the treatment is effective, participants may be entered into low-dose group(D1)(rhGM-CSF administration 1.25 ug/kg/d, qd, sc, for 2 months,then rhGM-CSF administration 1.25 ug/kg/d, qod, sc, for 3 months), when the chest CT absorption≥25% , and /or the PaO2 elevated by 5 mm Hg. |
Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
|
Experimental: D2
Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). When the treatment is ineffective, and anti-GM-CSF antibody titers level ≥1:1000,the dose will be increased to 2.5 ug/kg/d. After 2 months, if the clinical response was optimal, that dose is continued for 3 months, and defined as group 2(D2). |
Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
|
Experimental: D3
Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). When the treatment is ineffective, and anti-GM-CSF antibody titers level ≥1:1000,the dose will be increased to 2.5 ug/kg/d. After 2 months, if the clinical response was not optimal, the patients will receive whole lung lavage(WLL), who are defined as group 3(D3). |
Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
|
Active Comparator: D4
Patients diagnosed with PAP will be received rhGM-CSF 1.25 ug/kg/d subcutaneously for 1 month, before evaluation on the 30th day (±3 day). When the treatment is ineffective, and anti-GM-CSF antibody titers level <1:1000,the patients will receive whole lung lavage(WLL), then give rhGM-CSF administration (1.25 ug/kg/d) for 3 months, which belong to group4(D4). |
Drug: rhGM-CSF
GM-CSF will be given subcutaneously according to the rule in different groups.
Other Names:
Procedure: Whole Lung Lavage(WLL)
using double lumen endotracheal tube (DLT) to selectively lavage one lung
Other Names:
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- Improvements in double pulmonary diffuse lesions (Chest CT score ) [ Time Frame: 6 months ]
- Clinical symptoms observation: shod of breath, cough (according to each score standard) [ Time Frame: 6 months ]
- Granulocyte Macrophage Colony Stimulating Factor(GM-CSF) Antibody titer change [ Time Frame: 6 months ]
- Improvements in pulmonary function [ Time Frame: 6 months ]Pulmonary function tests include residual volume/total lung capacity(RV/TLC), forced vital capacity(FVC), forced expiratory volume in one second/forced vital capacity(FEV1/FVC), diffusing capacity of carbon monoxide(DLCO).
- 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). [ Time Frame: 6 months ]

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Ages Eligible for Study: | 17 Years to 80 Years (Child, Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01983657
Contact: Huiping Li, Dr | 86-13817389991 | liw2013@126.com |
China | |
Shanghai Pulmonary Hospital | Recruiting |
Shanghai, China, 200433 | |
Contact: Huiping Li, Doctor 86-13817389991 liw2013@126.com | |
Principal Investigator: Huiping Li, Doctor |
Principal Investigator: | Huiping Li, Dr | Shanghai Pulmonary Hospital, Shanghai, China |
Responsible Party: | Huiping Li, Professor,Chief of Dept. of Respiratory Medicine, Shanghai Pulmonary Hospital, Shanghai, China |
ClinicalTrials.gov Identifier: | NCT01983657 History of Changes |
Other Study ID Numbers: |
20120401 |
First Posted: | November 14, 2013 Key Record Dates |
Last Update Posted: | November 18, 2013 |
Last Verified: | November 2013 |
Keywords provided by Huiping Li, Shanghai Pulmonary Hospital, Shanghai, China:
Pulmonary Pulmonary Alveolar Proteinosis PAP |
Additional relevant MeSH terms:
Pulmonary Alveolar Proteinosis Lung Diseases Respiratory Tract Diseases |