{ "FullStudy":{ "Rank":218228, "Study":{ "ProtocolSection":{ "IdentificationModule":{ "NCTId":"NCT01511068", "NCTIdAliasList":{ "NCTIdAlias":[ "NCT01534156" ] }, "OrgStudyIdInfo":{ "OrgStudyId":"2011-0959_CCHMC_IRB" }, "Organization":{ "OrgFullName":"Children's Hospital Medical Center, Cincinnati", "OrgClass":"OTHER" }, "BriefTitle":"Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)", "OfficialTitle":"Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)", "Acronym":"FAMPAP" }, "StatusModule":{ "StatusVerifiedDate":"January 2013", "OverallStatus":"Completed", "ExpandedAccessInfo":{ "HasExpandedAccess":"No" }, "StartDateStruct":{ "StartDate":"August 2012" }, "PrimaryCompletionDateStruct":{ "PrimaryCompletionDate":"July 2013", "PrimaryCompletionDateType":"Actual" }, "CompletionDateStruct":{ "CompletionDate":"February 2014", "CompletionDateType":"Actual" }, "StudyFirstSubmitDate":"December 12, 2011", "StudyFirstSubmitQCDate":"January 12, 2012", "StudyFirstPostDateStruct":{ "StudyFirstPostDate":"January 18, 2012", "StudyFirstPostDateType":"Estimate" }, "LastUpdateSubmitDate":"June 3, 2014", "LastUpdatePostDateStruct":{ "LastUpdatePostDate":"June 4, 2014", "LastUpdatePostDateType":"Estimate" } }, "SponsorCollaboratorsModule":{ "ResponsibleParty":{ "ResponsiblePartyType":"Sponsor" }, "LeadSponsor":{ "LeadSponsorName":"Children's Hospital Medical Center, Cincinnati", "LeadSponsorClass":"OTHER" }, "CollaboratorList":{ "Collaborator":[ { "CollaboratorName":"Virginia Commonwealth University", "CollaboratorClass":"OTHER" },{ "CollaboratorName":"Genzyme, a Sanofi Company", "CollaboratorClass":"INDUSTRY" } ] } }, "OversightModule":{ "OversightHasDMC":"Yes" }, "DescriptionModule":{ "BriefSummary":"The purpose of this study is to evaluate the therapeutic efficacy of inhaled recombinant human GM-CSF in individuals with hereditary Pulmonary Alveolar Proteinosis (PAP) due to partial dysfunction of the GM-CSF receptor." }, "ConditionsModule":{ "ConditionList":{ "Condition":[ "Hereditary Pulmonary Alveolar Proteinosis" ] }, "KeywordList":{ "Keyword":[ "PAP" ] } }, "DesignModule":{ "StudyType":"Interventional", "PhaseList":{ "Phase":[ "Phase 2" ] }, "DesignInfo":{ "DesignInterventionModel":"Single Group Assignment", "DesignPrimaryPurpose":"Treatment", "DesignMaskingInfo":{ "DesignMasking":"None (Open Label)" } }, "EnrollmentInfo":{ "EnrollmentCount":"2", "EnrollmentType":"Actual" } }, "ArmsInterventionsModule":{ "ArmGroupList":{ "ArmGroup":[ { "ArmGroupLabel":"inhaled recombinant", "ArmGroupType":"Experimental", "ArmGroupDescription":"inhaled recombinant human GM-CSF in individuals with hereditary Pulmonary Alveolar Proteinosis (PAP) due to partial dysfunction of the GM-CSF receptor", "ArmGroupInterventionList":{ "ArmGroupInterventionName":[ "Drug: Leukine" ] } } ] }, "InterventionList":{ "Intervention":[ { "InterventionType":"Drug", "InterventionName":"Leukine", "InterventionDescription":"Participants will receive inhaled rhGM-CSF (Sargramostim, Leukine) at the dose of 250 mcg one time per week for 12 weeks. Following an interim safety evaluation, participants may be entered into a second 12 week treatment period where participants will receive either 250 mcg or 500 mcg once weekly. At the end of any treatment period, participants will be followed for 12 additional weeks in the absence of inhaled rhGM-CSF to evaluate safety and efficacy.", "InterventionArmGroupLabelList":{ "InterventionArmGroupLabel":[ "inhaled recombinant" ] }, "InterventionOtherNameList":{ "InterventionOtherName":[ "GM-CSF [Leukine (Sargramostim)]" ] } } ] } }, "OutcomesModule":{ "PrimaryOutcomeList":{ "PrimaryOutcome":[ { "PrimaryOutcomeMeasure":"Time for the oxygen saturation to fall below 88%", "PrimaryOutcomeTimeFrame":"6 months" } ] }, "SecondaryOutcomeList":{ "SecondaryOutcome":[ { "SecondaryOutcomeMeasure":"Improvement in pulmonary function", "SecondaryOutcomeTimeFrame":"6 months" },{ "SecondaryOutcomeMeasure":"Improvement in exercise tolerance", "SecondaryOutcomeTimeFrame":"6 months" },{ "SecondaryOutcomeMeasure":"Improvement in CT scan", "SecondaryOutcomeTimeFrame":"6 months" },{ "SecondaryOutcomeMeasure":"Improvement in Quality of Life", "SecondaryOutcomeTimeFrame":"6 months" },{ "SecondaryOutcomeMeasure":"Improvement in dyspnea and fatigue", "SecondaryOutcomeTimeFrame":"6 months" },{ "SecondaryOutcomeMeasure":"Biomarkers will be assayed to detect changes from baseline values: surfactant protein-D (SP-D), KL-6, CEA), GM-CSF, GM-CSF autoantibodies, macrophage colony stimulating factor (M-CSF), and macrophage inflammatory protein -1 alpha MIP-1alpha.", "SecondaryOutcomeTimeFrame":"6 months" } ] } }, "EligibilityModule":{ "EligibilityCriteria":"Inclusion Criteria:\n\nA diagnosis of PAP caused by bi-allelic mutations in CSF2RA or CSF2RB associated with impaired GM-CSF-R-alpha or GM-CSF-R-beta function, respectively, resulting in reduced but non-zero GM-CSF signaling\nAble and willing to give written informed consent / assent as necessary\nClinically stable\n\nExclusion Criteria:\n\nConfirmed diagnosis of a disorder of surfactant production caused by bi-allelic mutations in ABCA3, SFTPB, or SFTPC\nConfirmed diagnosis of autoimmune PAP caused by a high level of GM-CSF autoantibody\nConfirmed diagnosis of secondary PAP caused by an underlying clinical disorder known to be associated with the development of PAP, e.g., inhalation of silica or titanium; myelodysplasia and others\nTreatment with any investigational agent in the 3 months prior to enrollment\nHistory of severe allergic or anaphylactic reactions to GM-CSF or other yeast-derived products\nHistory of asthma or other reactive airways disease\nKnown active, viral, fungal, mycobacterial, or other infection\nA serious medical condition which, in the opinion of the investigator or data and safety monitoring committee, would make the patient unsuitable for the study", "HealthyVolunteers":"No", "Gender":"All", "MinimumAge":"8 Years", "StdAgeList":{ "StdAge":[ "Child", "Adult", "Older Adult" ] } }, "ContactsLocationsModule":{ "OverallOfficialList":{ "OverallOfficial":[ { "OverallOfficialName":"Bruce Trapnell, MD", "OverallOfficialAffiliation":"Children's Hospital Medical Center, Cincinnati", "OverallOfficialRole":"Principal Investigator" },{ "OverallOfficialName":"Bruce Rubin, MD, FRCPC", "OverallOfficialAffiliation":"Virginia Commonwealth University", "OverallOfficialRole":"Principal Investigator" } ] }, "LocationList":{ "Location":[ { "LocationFacility":"Cincinnati Children's Hospital Medical Center", "LocationCity":"Cincinnati", "LocationState":"Ohio", "LocationZip":"45229", "LocationCountry":"United States" },{ "LocationFacility":"Virginia Commonwealth University", "LocationCity":"Richmond", "LocationState":"Virginia", "LocationZip":"23298-0646", "LocationCountry":"United States" } ] } } }, "AnnotationSection":{ "AnnotationModule":{ "UnpostedAnnotation":{ "UnpostedResponsibleParty":"Children's Hospital Medical Center, Cincinnati", "UnpostedEventList":{ "UnpostedEvent":[ { "UnpostedEventType":"Release", "UnpostedEventDate":"February 26, 2020" },{ "UnpostedEventType":"Reset", "UnpostedEventDate":"March 10, 2020" } ] } } } }, "DerivedSection":{ "MiscInfoModule":{ "VersionHolder":"April 22, 2020" }, "InterventionBrowseModule":{ "InterventionMeshList":{ "InterventionMesh":[ { "InterventionMeshId":"C000081222", "InterventionMeshTerm":"Sargramostim" } ] }, "InterventionAncestorList":{ "InterventionAncestor":[ { "InterventionAncestorId":"D000007155", "InterventionAncestorTerm":"Immunologic Factors" },{ "InterventionAncestorId":"D000045505", "InterventionAncestorTerm":"Physiological Effects of Drugs" } ] }, "InterventionBrowseLeafList":{ "InterventionBrowseLeaf":[ { "InterventionBrowseLeafId":"M267719", "InterventionBrowseLeafName":"Sargramostim", "InterventionBrowseLeafAsFound":"Leukine", "InterventionBrowseLeafRelevance":"high" },{ "InterventionBrowseLeafId":"M8784", "InterventionBrowseLeafName":"Immunologic Factors", "InterventionBrowseLeafRelevance":"low" } ] }, "InterventionBrowseBranchList":{ "InterventionBrowseBranch":[ { "InterventionBrowseBranchAbbrev":"All", "InterventionBrowseBranchName":"All Drugs and Chemicals" } ] } }, "ConditionBrowseModule":{ "ConditionMeshList":{ "ConditionMesh":[ { "ConditionMeshId":"D000011649", "ConditionMeshTerm":"Pulmonary Alveolar Proteinosis" } ] }, "ConditionAncestorList":{ "ConditionAncestor":[ { "ConditionAncestorId":"D000008171", "ConditionAncestorTerm":"Lung Diseases" },{ "ConditionAncestorId":"D000012140", "ConditionAncestorTerm":"Respiratory Tract Diseases" } ] }, "ConditionBrowseLeafList":{ "ConditionBrowseLeaf":[ { "ConditionBrowseLeafId":"M25724", "ConditionBrowseLeafName":"Respiratory Aspiration", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M13086", "ConditionBrowseLeafName":"Pulmonary Alveolar Proteinosis", "ConditionBrowseLeafAsFound":"Pulmonary Alveolar Proteinosis", "ConditionBrowseLeafRelevance":"high" },{ "ConditionBrowseLeafId":"M9751", "ConditionBrowseLeafName":"Lung Diseases", "ConditionBrowseLeafRelevance":"low" },{ "ConditionBrowseLeafId":"M13560", "ConditionBrowseLeafName":"Respiratory Tract Diseases", "ConditionBrowseLeafRelevance":"low" } ] }, "ConditionBrowseBranchList":{ "ConditionBrowseBranch":[ { "ConditionBrowseBranchAbbrev":"BC08", "ConditionBrowseBranchName":"Respiratory Tract (Lung and Bronchial) Diseases" },{ "ConditionBrowseBranchAbbrev":"BC23", "ConditionBrowseBranchName":"Symptoms and General Pathology" },{ "ConditionBrowseBranchAbbrev":"All", "ConditionBrowseBranchName":"All Conditions" } ] } } } } } }