Ignite Creation Date:
2025-12-24 @ 6:48 PM
Ignite Modification Date:
2026-01-22 @ 12:10 PM
Study NCT ID:
NCT01983657
Status:
UNKNOWN
Last Update Posted:
2013-11-18
First Post:
2013-11-07
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D011649', 'term': 'Pulmonary Alveolar Proteinosis'}], 'ancestors': [{'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C082856', 'term': 'regramostim'}, {'id': 'D016178', 'term': 'Granulocyte-Macrophage Colony-Stimulating Factor'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}], 'ancestors': [{'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2012-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-11', 'completionDateStruct': {'date': '2014-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2013-11-14', 'studyFirstSubmitDate': '2013-11-07', 'studyFirstSubmitQcDate': '2013-11-07', 'lastUpdatePostDateStruct': {'date': '2013-11-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2013-11-14', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Improvements in double pulmonary diffuse lesions （Chest CT score ）', 'timeFrame': '6 months'}], 'secondaryOutcomes': [{'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', 'description': '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).'}, {'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'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Pulmonary', 'Pulmonary Alveolar Proteinosis', 'PAP'], 'conditions': ['Pulmonary Alveolar Proteinosis']}, 'referencesModule': {'references': [{'pmid': '31898493', 'type': 'DERIVED', '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.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '17 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosed PAP patients\n* Aged 17-80\n* Signed informed consent\n\nExclusion Criteria:\n\n* Secondary PAP\n* Received whole lung lavage therapy within 4 weeks before enrollment\n* Received previous GM-CSF therapy within 6 months before enrollment\n* WBC≥12,000/ul\n* fever≥38℃\n* Severe edema, severe liver, kidney, lung and cardiovascular disease.\n* Pregnant，planning to get pregnant or nursing\n* Inability to express the subjective discomfort\n* Serious drug allergy history, E.coli preparation or rhGM-CSF serious allergy history'}, 'identificationModule': {'nctId': 'NCT01983657', 'acronym': 'PAP', 'briefTitle': 'Study of Subcutaneous Injection of Low-dose rhGM-CSF +/- WLL in PAP.', 'organization': {'class': 'OTHER', 'fullName': 'Shanghai Pulmonary Hospital, Shanghai, China'}, 'officialTitle': 'Study of Subcutaneous Injection of Low-dose Recombinant Granulocyte Macrophage-Colony Stimulating Factor (rhGM-CSF) +/- Whole Lung Lavage(WLL) in Pulmonary Alveolar Proteinosis.', 'orgStudyIdInfo': {'id': '20120401'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'D1', 'description': '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).\n\nIf 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.', 'interventionNames': ['Drug: rhGM-CSF']}, {'type': 'EXPERIMENTAL', 'label': 'D2', 'description': '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).\n\nWhen 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).', 'interventionNames': ['Drug: rhGM-CSF']}, {'type': 'EXPERIMENTAL', 'label': 'D3', 'description': '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).\n\nWhen 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).', 'interventionNames': ['Drug: rhGM-CSF']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'D4', 'description': '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).\n\nWhen 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).', 'interventionNames': ['Drug: rhGM-CSF', 'Procedure: Whole Lung Lavage(WLL)']}], 'interventions': [{'name': 'rhGM-CSF', 'type': 'DRUG', 'otherNames': ['TOPLEUCON', 'GM-CSF', 'Recombinant Granulocyte Macrophage-Colony Stimulating Factor', 'Granulocyte Macrophage-Colony Stimulating Factor'], 'description': 'GM-CSF will be given subcutaneously according to the rule in different groups.', 'armGroupLabels': ['D1', 'D2', 'D3', 'D4']}, {'name': 'Whole Lung Lavage(WLL)', 'type': 'PROCEDURE', 'otherNames': ['Whole Lung Lavage', 'WLL'], 'description': 'using double lumen endotracheal tube (DLT) to selectively lavage one lung', 'armGroupLabels': ['D4']}]}, 'contactsLocationsModule': {'locations': [{'zip': '200433', 'city': 'Shanghai', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Huiping Li, Doctor', 'role': 'CONTACT', 'email': 'liw2013@126.com', 'phone': '86-13817389991'}, {'name': 'Huiping Li, Doctor', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Shanghai Pulmonary Hospital', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}], 'centralContacts': [{'name': 'Huiping Li, Dr', 'role': 'CONTACT', 'email': 'liw2013@126.com', 'phone': '86-13817389991'}], 'overallOfficials': [{'name': 'Huiping Li, Dr', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Shanghai Pulmonary Hospital, Shanghai, China'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shanghai Pulmonary Hospital, Shanghai, China', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor,Chief of Dept. of Respiratory Medicine', 'investigatorFullName': 'Huiping Li', 'investigatorAffiliation': 'Shanghai Pulmonary Hospital, Shanghai, China'}}}}