Nctid:
NCT00001630
Payload:
{"FullStudy"=>{"Rank"=>474073, "Study"=>{"DerivedSection"=>{"MiscInfoModule"=>{"VersionHolder"=>"December 08, 2023"}, "ConditionBrowseModule"=>{"ConditionMeshList"=>{"ConditionMesh"=>[{"ConditionMeshId"=>"D000013921", "ConditionMeshTerm"=>"Thrombocytopenia"}, {"ConditionMeshId"=>"D000000743", "ConditionMeshTerm"=>"Anemia, Hemolytic"}, {"ConditionMeshId"=>"D000000744", "ConditionMeshTerm"=>"Anemia, Hemolytic, Autoimmune"}, {"ConditionMeshId"=>"D000016553", "ConditionMeshTerm"=>"Purpura, Thrombocytopenic, Idiopathic"}, {"ConditionMeshId"=>"D000001327", "ConditionMeshTerm"=>"Autoimmune Diseases"}, {"ConditionMeshId"=>"D000006461", "ConditionMeshTerm"=>"Hemolysis"}]}, "ConditionAncestorList"=>{"ConditionAncestor"=>[{"ConditionAncestorId"=>"D000000740", "ConditionAncestorTerm"=>"Anemia"}, {"ConditionAncestorId"=>"D000006402", "ConditionAncestorTerm"=>"Hematologic Diseases"}, {"ConditionAncestorId"=>"D000001791", "ConditionAncestorTerm"=>"Blood Platelet Disorders"}, {"ConditionAncestorId"=>"D000007154", "ConditionAncestorTerm"=>"Immune System Diseases"}, {"ConditionAncestorId"=>"D000010335", "ConditionAncestorTerm"=>"Pathologic Processes"}, {"ConditionAncestorId"=>"D000011696", "ConditionAncestorTerm"=>"Purpura, Thrombocytopenic"}, {"ConditionAncestorId"=>"D000011693", "ConditionAncestorTerm"=>"Purpura"}, {"ConditionAncestorId"=>"D000001778", "ConditionAncestorTerm"=>"Blood Coagulation Disorders"}, {"ConditionAncestorId"=>"D000057049", "ConditionAncestorTerm"=>"Thrombotic Microangiopathies"}, {"ConditionAncestorId"=>"D000006474", "ConditionAncestorTerm"=>"Hemorrhagic Disorders"}, {"ConditionAncestorId"=>"D000006470", "ConditionAncestorTerm"=>"Hemorrhage"}, {"ConditionAncestorId"=>"D000012877", "ConditionAncestorTerm"=>"Skin Manifestations"}]}, "ConditionBrowseLeafList"=>{"ConditionBrowseLeaf"=>[{"ConditionBrowseLeafId"=>"M16045", "ConditionBrowseLeafName"=>"Syndrome", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M16370", "ConditionBrowseLeafName"=>"Thrombocytopenia", "ConditionBrowseLeafAsFound"=>"Thrombocytopenia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M9246", "ConditionBrowseLeafName"=>"Hemorrhage", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M3760", "ConditionBrowseLeafName"=>"Anemia", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M4319", "ConditionBrowseLeafName"=>"Autoimmune Diseases", "ConditionBrowseLeafAsFound"=>"Autoimmune Diseases", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M18635", "ConditionBrowseLeafName"=>"Purpura, Thrombocytopenic, Idiopathic", "ConditionBrowseLeafAsFound"=>"Autoimmune Thrombocytopenia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M9237", "ConditionBrowseLeafName"=>"Hemolysis", "ConditionBrowseLeafAsFound"=>"Hemolytic", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M3764", "ConditionBrowseLeafName"=>"Anemia, Hemolytic, Autoimmune", "ConditionBrowseLeafAsFound"=>"Autoimmune Hemolytic Anemia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M3763", "ConditionBrowseLeafName"=>"Anemia, Hemolytic", "ConditionBrowseLeafAsFound"=>"Hemolytic Anemia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"M14237", "ConditionBrowseLeafName"=>"Purpura", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M14240", "ConditionBrowseLeafName"=>"Purpura, Thrombocytopenic", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M9180", "ConditionBrowseLeafName"=>"Hematologic Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M4762", "ConditionBrowseLeafName"=>"Blood Platelet Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M9890", "ConditionBrowseLeafName"=>"Immune System Diseases", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M21667", "ConditionBrowseLeafName"=>"Hemostatic Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M4749", "ConditionBrowseLeafName"=>"Blood Coagulation Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M28372", "ConditionBrowseLeafName"=>"Thrombotic Microangiopathies", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M9250", "ConditionBrowseLeafName"=>"Hemorrhagic Disorders", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"M15370", "ConditionBrowseLeafName"=>"Skin Manifestations", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"T1303", "ConditionBrowseLeafName"=>"Chronic Graft Versus Host Disease", "ConditionBrowseLeafRelevance"=>"low"}, {"ConditionBrowseLeafId"=>"T3021", "ConditionBrowseLeafName"=>"Immune Thrombocytopenia", "ConditionBrowseLeafAsFound"=>"Autoimmune Thrombocytopenia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T3007", "ConditionBrowseLeafName"=>"Idiopathic Thrombocytopenic Purpura", "ConditionBrowseLeafAsFound"=>"Autoimmune Thrombocytopenia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T557", "ConditionBrowseLeafName"=>"Autoimmune Hemolytic Anemia", "ConditionBrowseLeafAsFound"=>"Autoimmune Hemolytic Anemia", "ConditionBrowseLeafRelevance"=>"high"}, {"ConditionBrowseLeafId"=>"T2147", "ConditionBrowseLeafName"=>"Evans Syndrome", "ConditionBrowseLeafRelevance"=>"low"}]}, "ConditionBrowseBranchList"=>{"ConditionBrowseBranch"=>[{"ConditionBrowseBranchName"=>"Symptoms and General Pathology", "ConditionBrowseBranchAbbrev"=>"BC23"}, {"ConditionBrowseBranchName"=>"All Conditions", "ConditionBrowseBranchAbbrev"=>"All"}, {"ConditionBrowseBranchName"=>"Blood and Lymph Conditions", "ConditionBrowseBranchAbbrev"=>"BC15"}, {"ConditionBrowseBranchName"=>"Immune System Diseases", "ConditionBrowseBranchAbbrev"=>"BC20"}, {"ConditionBrowseBranchName"=>"Heart and Blood Diseases", "ConditionBrowseBranchAbbrev"=>"BC14"}, {"ConditionBrowseBranchName"=>"Rare Diseases", "ConditionBrowseBranchAbbrev"=>"Rare"}]}}, "InterventionBrowseModule"=>{"InterventionBrowseLeafList"=>{"InterventionBrowseLeaf"=>[{"InterventionBrowseLeafId"=>"M6417", "InterventionBrowseLeafName"=>"Cyclophosphamide", "InterventionBrowseLeafRelevance"=>"low"}]}, "InterventionBrowseBranchList"=>{"InterventionBrowseBranch"=>[{"InterventionBrowseBranchName"=>"Antineoplastic Agents", "InterventionBrowseBranchAbbrev"=>"ANeo"}, {"InterventionBrowseBranchName"=>"Antirheumatic Agents", "InterventionBrowseBranchAbbrev"=>"ARhu"}, {"InterventionBrowseBranchName"=>"All Drugs and Chemicals", "InterventionBrowseBranchAbbrev"=>"All"}]}}}, "ProtocolSection"=>{"DesignModule"=>{"PhaseList"=>{"Phase"=>["Phase 1"]}, "StudyType"=>"Interventional", "DesignInfo"=>{"DesignAllocation"=>"Non-Randomized", "DesignMaskingInfo"=>{"DesignMasking"=>"None (Open Label)"}, "DesignPrimaryPurpose"=>"Treatment"}, "EnrollmentInfo"=>{"EnrollmentType"=>"Actual", "EnrollmentCount"=>"29"}}, "StatusModule"=>{"OverallStatus"=>"Completed", "StartDateStruct"=>{"StartDate"=>"July 21, 1997"}, "ExpandedAccessInfo"=>{"HasExpandedAccess"=>"No"}, "StatusVerifiedDate"=>"March 21, 2011", "CompletionDateStruct"=>{"CompletionDate"=>"June 11, 2009", "CompletionDateType"=>"Actual"}, "LastUpdateSubmitDate"=>"June 30, 2017", "StudyFirstSubmitDate"=>"November 3, 1999", "StudyFirstSubmitQCDate"=>"November 3, 1999", "LastUpdatePostDateStruct"=>{"LastUpdatePostDate"=>"July 2, 2017", "LastUpdatePostDateType"=>"Actual"}, "StudyFirstPostDateStruct"=>{"StudyFirstPostDate"=>"November 4, 1999", "StudyFirstPostDateType"=>"Estimate"}}, "OversightModule"=>{}, "ConditionsModule"=>{"KeywordList"=>{"Keyword"=>["Immunosuppression", "Thrombocytopenia", "Autoimmune Disease", "Evan's Syndrome", "Autoimmune Hemolytic Anemia", "Episodic Bleeding"]}, "ConditionList"=>{"Condition"=>["Autoimmune Disease", "Autoimmune Hemolytic Anemia", "Thrombocytopenia"]}}, "ReferencesModule"=>{"ReferenceList"=>{"Reference"=>[{"ReferencePMID"=>"8541715", "ReferenceType"=>"background", "ReferenceCitation"=>"Semple JW, Freedman J. Abnormal cellular immune mechanisms associated with autoimmune thrombocytopenia. Transfus Med Rev. 1995 Oct;9(4):327-38. doi: 10.1016/s0887-7963(05)80080-x. No abstract available."}, {"ReferencePMID"=>"9167472", "ReferenceType"=>"background", "ReferenceCitation"=>"Karpatkin S. Autoimmune (idiopathic) thrombocytopenic purpura. Lancet. 1997 May 24;349(9064):1531-6. doi: 10.1016/S0140-6736(96)12118-8. No abstract available."}, {"ReferencePMID"=>"7935660", "ReferenceType"=>"background", "ReferenceCitation"=>"George JN, el-Harake MA, Raskob GE. Chronic idiopathic thrombocytopenic purpura. N Engl J Med. 1994 Nov 3;331(18):1207-11. doi: 10.1056/NEJM199411033311807. No abstract available."}]}}, "DescriptionModule"=>{"BriefSummary"=>"Platelets are particles found along with red and white blood cells in the blood that play a role in the process of blood clotting. Disorders affecting the platelets can lower the amount of platelets in the blood and put patients at risk of bleeding. The condition of low platelets is referred to as thrombocytopenia.\n\nThrombocytopenia can be associated with a variety of diseases including cancer, leukemia, tuberculosis, or as a result of an autoimmune reaction. Autoimmune reactions are disorders in which the normal immune system begins attacking itself. Autoimmune thrombocytopenia (AITP) is a disorder of low blood platelet counts in which platelets are destroyed by antibodies produced by the immune system.\n\nUnfortunately, many patients with AITP do not respond to standard treatments for thrombocytopenia. Cyclophosphamide is a drug that works to suppress the activity of the immune system. Researchers believe that combining this drug with transplanted rescued blood stem cells may provide effective treatment for AITP.\n\nThe purpose of this study is to explore the affordability and safety of this therapy for the treatment of AITP. The effectiveness of the therapy will be measured by the number of patients whose platelet levels rise greater than 100,000/m3.\n\nIf this treatment approach appears affordable, this study will form the basis for a larger study to compare alternate treatment approaches.", "DetailedDescription"=>"Autoimmune Thrombocytopenia (AITP) is a disorder of low blood platelet counts in which platelet destruction is caused by antiplatelet autoantibodies. A large proportion of patients with chronic AITP are refractory to standard therapies including corticosteroids, immune globulin and splenectomy. Cyclophosphamide is a cytotoxic immunosuppressive agent which may induce durable remissions of refractory autoimmune diseases. High-dose cyclophosphamide with peripheral blood stem cell (PBPC) rescue has been proposed as a potential definitive therapy for AITP; however, the infusion of autoreactive lymphocytes could result in relapse. The use of PBPC depleted of T-lymphocytes could circumvent this limitation.\n\nThe purpose of this phase I/II study is to explore the feasibility and safety of this approach, and to seek preliminary evidence of effectiveness, of using high-dose cyclophosphamide (50 mg/kg/day x 4) followed by infusion of autologous PBPC enriched for CD34+ cells (concomitantly depleted of CD3+ cells) for the treatment of patients with refractory AITP. Safety/feasibility parameters to be examined will include the ability to mobilize, harvest and purify sufficient PBPC to yield greater than 2 x 10(6) CD34+ cells/kg; symptomatic acceptability and hematologic toxicities of the mobilization regimen (filgrastim 10 micrograms/kg/day IV); tolerability of the leukapheresis procedure, including the central line placement and maintenance; depth and duration of blood cell nadirs following chemotherapy; peritransplant bleeding episodes and transfusion requirements; episodes of febrile neutropenia, culture-proven infections and antibiotic usage. Effectiveness will be gauged by the rapidity and number of patients to achieve complete remission (platelet count greater than 100,000/mm(3) and partial remission (platelet count greater than 50,000/mm(3) or doubling of the platelet count with resolution of bleeding episodes). Ancillary evidence of therapeutic effect will be sought by examining changes in titers of platelet surface glycoprotein antibodies. In addition, alterations in T-lymphocyte subsets will be examined by flow cytometry. If this treatment approach appears feasible, this study will form the basis for a larger trial to compare alternate treatment approaches."}, "EligibilityModule"=>{"Gender"=>"All", "MaximumAge"=>"65 years", "MinimumAge"=>"18 years", "StdAgeList"=>{"StdAge"=>["Adult", "Older Adult"]}, "HealthyVolunteers"=>"No", "EligibilityCriteria"=>"INCLUSION CRITERIA:\n\nMale or female, ages 18-65 years old.\n\nRefractory severe chronic autoimmune thrombocytopenia, with or without autoimmune hemolytic anemia (Evan's syndrome), with all the following:\n\nPlatelet count frequently below 20,000/mm(3) despite active\n\ntreatment for a period of greater than 6 months.\n\nNormal or increased megakaryocytes on bone marrow\n\naspirate/bx.\n\nNo plausible alternative etiology such as drug-mediated\n\nthrombocytopenia, marrow failure syndrome or thrombocytopenia\n\nrelated to viral or bacterial infection.\n\nFailure of treatment with:\n\ni. conventional-dose steroids (e.g., prednisone or dosage of 40\n\nmg/day or equivalent, followed by dosage taper) for at least 3\n\nmonths.\n\nii. intravenous immunoglobulin.\n\niii. splenectomy.\n\ne. Episodic bleeding requiring transfusions or ecchymoses interfering\n\nwith ordinary daily activities.\n\nEXCLUSION CRITERIA:\n\nECOG performance status greater than 1.\n\nCardiopulmonary disease including:\n\nHistory of coronary artery disease, angina pectoris or congestive heart failure.\nLV ejection fraction less than 40 percent by 2D echocardiogram.\n\nRenal disease, serum creatinine greater than 2.5 mg/dL or creatinine clearance less than 30 mL/min.\n\nSignificant hepatic dysfunction, bilirubin greater than 2 mg/dL or transaminases greater than 2 times UNL.\n\nUncorrected coagulopathy.\n\nBone marrow aplasia (cellularity less than 10 percent), single or multilineage hematopoietic failure, myelodysplastic syndrome, or extensive marrow fibrosis.\n\nHistory or active diagnosis of malignancy (except treated non-melanoma skin cancer or cevical carcinoma in situ).\n\nHIV positive.\n\nPregnancy or lactation, unwillingness to practice adequate birth control in the peritransplant period.\n\nPsychiatric illness or mental incapacity to understand and give informed consent.\n\nOther medical illness or condition which, in the opinion of the Investigators, may contraindicate participation in this study due to patients' risk or compromise of study integrity."}, "IdentificationModule"=>{"NCTId"=>"NCT00001630", "BriefTitle"=>"Treatment of Autoimmune Thrombocytopenia (AITP)", "Organization"=>{"OrgClass"=>"NIH", "OrgFullName"=>"National Institutes of Health Clinical Center (CC)"}, "OfficialTitle"=>"High-Dose Cyclophosphamide With CD34+ Selected Autologous Hematopoietic Cell Support for Treatment of Refractory Chronic Autoimmune Thrombocytopenia", "OrgStudyIdInfo"=>{"OrgStudyId"=>"970154"}, "SecondaryIdInfoList"=>{"SecondaryIdInfo"=>[{"SecondaryId"=>"97-H-0154"}]}}, "ArmsInterventionsModule"=>{"InterventionList"=>{"Intervention"=>[{"InterventionName"=>"Isolex 300i", "InterventionType"=>"Device"}]}}, "ContactsLocationsModule"=>{"LocationList"=>{"Location"=>[{"LocationZip"=>"20892", "LocationCity"=>"Bethesda", "LocationState"=>"Maryland", "LocationCountry"=>"United States", "LocationFacility"=>"National Institutes of Health Clinical Center, 9000 Rockville Pike"}]}}, "SponsorCollaboratorsModule"=>{"LeadSponsor"=>{"LeadSponsorName"=>"National Heart, Lung, and Blood Institute (NHLBI)", "LeadSponsorClass"=>"NIH"}}}}}}
