Nctid:
NCT00001964
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-12-06"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D000740", "term"=>"Anemia"}, {"id"=>"D000741", "term"=>"Anemia, Aplastic"}], "ancestors"=>[{"id"=>"D006402", "term"=>"Hematologic Diseases"}, {"id"=>"D000080983", "term"=>"Bone Marrow Failure Disorders"}, {"id"=>"D001855", "term"=>"Bone Marrow Diseases"}], "browseLeaves"=>[{"id"=>"M4070", "name"=>"Anemia", "asFound"=>"Anemia", "relevance"=>"HIGH"}, {"id"=>"M4071", "name"=>"Anemia, Aplastic", "asFound"=>"Aplastic Anemia", "relevance"=>"HIGH"}, {"id"=>"M13118", "name"=>"Pancytopenia", "relevance"=>"LOW"}, {"id"=>"M9490", "name"=>"Hematologic Diseases", "relevance"=>"LOW"}, {"id"=>"M2241", "name"=>"Bone Marrow Failure Disorders", "relevance"=>"LOW"}, {"id"=>"M5134", "name"=>"Bone Marrow Diseases", "relevance"=>"LOW"}, {"id"=>"T460", "name"=>"Aplastic Anemia", "asFound"=>"Aplastic Anemia", "relevance"=>"HIGH"}], "browseBranches"=>[{"name"=>"Blood and Lymph Conditions", "abbrev"=>"BC15"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}, "interventionBrowseModule"=>{"meshes"=>[{"id"=>"D016572", "term"=>"Cyclosporine"}, {"id"=>"D003524", "term"=>"Cyclosporins"}], "ancestors"=>[{"id"=>"D004791", "term"=>"Enzyme Inhibitors"}, {"id"=>"D045504", "term"=>"Molecular Mechanisms of Pharmacological Action"}, {"id"=>"D007166", "term"=>"Immunosuppressive Agents"}, {"id"=>"D007155", "term"=>"Immunologic Factors"}, {"id"=>"D045505", "term"=>"Physiological Effects of Drugs"}, {"id"=>"D000935", "term"=>"Antifungal Agents"}, {"id"=>"D000890", "term"=>"Anti-Infective Agents"}, {"id"=>"D003879", "term"=>"Dermatologic Agents"}, {"id"=>"D018501", "term"=>"Antirheumatic Agents"}, {"id"=>"D065095", "term"=>"Calcineurin Inhibitors"}], "browseLeaves"=>[{"id"=>"M12128", "name"=>"Mycophenolic Acid", "relevance"=>"LOW"}, {"id"=>"M18961", "name"=>"Cyclosporine", "asFound"=>"Upper", "relevance"=>"HIGH"}, {"id"=>"M6730", "name"=>"Cyclosporins", "asFound"=>"Upper", "relevance"=>"HIGH"}, {"id"=>"M4279", "name"=>"Antilymphocyte Serum", "relevance"=>"LOW"}, {"id"=>"M7951", "name"=>"Enzyme Inhibitors", "relevance"=>"LOW"}, {"id"=>"M10212", "name"=>"Immunosuppressive Agents", "relevance"=>"LOW"}, {"id"=>"M10201", "name"=>"Immunologic Factors", "relevance"=>"LOW"}, {"id"=>"M4254", "name"=>"Antifungal Agents", "relevance"=>"LOW"}, {"id"=>"M6252", "name"=>"Clotrimazole", "relevance"=>"LOW"}, {"id"=>"M11796", "name"=>"Miconazole", "relevance"=>"LOW"}, {"id"=>"M4214", "name"=>"Anti-Infective Agents", "relevance"=>"LOW"}, {"id"=>"M7074", "name"=>"Dermatologic Agents", "relevance"=>"LOW"}, {"id"=>"M20604", "name"=>"Antirheumatic Agents", "relevance"=>"LOW"}, {"id"=>"M30452", "name"=>"Calcineurin Inhibitors", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Anti-Infective Agents", "abbrev"=>"Infe"}, {"name"=>"Antineoplastic Agents", "abbrev"=>"ANeo"}, {"name"=>"All Drugs and Chemicals", "abbrev"=>"All"}, {"name"=>"Antirheumatic Agents", "abbrev"=>"ARhu"}, {"name"=>"Dermatologic Agents", "abbrev"=>"Derm"}]}}, "protocolSection"=>{"designModule"=>{"phases"=>["PHASE2"], "studyType"=>"INTERVENTIONAL", "designInfo"=>{"allocation"=>"NON_RANDOMIZED", "maskingInfo"=>{"masking"=>"NONE"}, "primaryPurpose"=>"TREATMENT", "interventionModel"=>"SINGLE_GROUP"}, "enrollmentInfo"=>{"type"=>"ACTUAL", "count"=>104}}, "statusModule"=>{"overallStatus"=>"COMPLETED", "startDateStruct"=>{"date"=>"2000-03-17"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2021-03", "completionDateStruct"=>{"date"=>"2015-05-12", "type"=>"ACTUAL"}, "lastUpdateSubmitDate"=>"2021-03-12", "studyFirstSubmitDate"=>"2000-01-18", "studyFirstSubmitQcDate"=>"2000-01-18", "lastUpdatePostDateStruct"=>{"date"=>"2021-03-16", "type"=>"ACTUAL"}, "studyFirstPostDateStruct"=>{"date"=>"2000-01-19", "type"=>"ESTIMATED"}, "primaryCompletionDateStruct"=>{"date"=>"2003-12-09", "type"=>"ACTUAL"}}, "outcomesModule"=>{"primaryOutcomes"=>[{"measure"=>"reduction in 24-month relapse", "timeFrame"=>"24 months", "description"=>"reduction in 24-month relapse"}]}, "oversightModule"=>{"isFdaRegulatedDrug"=>true, "isFdaRegulatedDevice"=>false}, "conditionsModule"=>{"keywords"=>["Hematopoiesis", "Bone Marrow Failure", "Aplastic Anemia"], "conditions"=>["Severe Aplastic Anemia"]}, "referencesModule"=>{"references"=>[{"pmid"=>"10422997", "type"=>"BACKGROUND", "citation"=>"Young NS. Acquired aplastic anemia. JAMA. 1999 Jul 21;282(3):271-8. doi: 10.1001/jama.282.3.271. No abstract available. Erratum In: JAMA 2000 Jan 5;283(1):57."}, {"pmid"=>"9134878", "type"=>"BACKGROUND", "citation"=>"Young NS, Maciejewski J. The pathophysiology of acquired aplastic anemia. N Engl J Med. 1997 May 8;336(19):1365-72. doi: 10.1056/NEJM199705083361906. No abstract available."}, {"pmid"=>"8043874", "type"=>"BACKGROUND", "citation"=>"Nimer SD, Ireland P, Meshkinpour A, Frane M. An increased HLA DR2 frequency is seen in aplastic anemia patients. Blood. 1994 Aug 1;84(3):923-7."}, {"pmid"=>"34724566", "type"=>"DERIVED", "citation"=>"Zaimoku Y, Patel BA, Adams SD, Shalhoub R, Groarke EM, Lee AAC, Kajigaya S, Feng X, Rios OJ, Eager H, Alemu L, Quinones Raffo D, Wu CO, Flegel WA, Young NS. HLA associations, somatic loss of HLA expression, and clinical outcomes in immune aplastic anemia. Blood. 2021 Dec 30;138(26):2799-2809. doi: 10.1182/blood.2021012895."}], "seeAlsoLinks"=>[{"url"=>"https://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2000-H-0032.html", "label"=>"NIH Clinical Center Detailed Web Page"}]}, "descriptionModule"=>{"briefSummary"=>"This study will test the safety and effectiveness of a combination of three drugs in treating severe aplastic anemia and preventing its recurrence. Two drugs used in this trial ATG and cyclosporine are standard combination therapy for aplastic anemia. This study will try to improve this therapy in three ways: 1) by altering the drug regimen to allow the drugs to work better; 2) by reducing the risk of kidney damage; and 3) by adding a third drug mycophenolate mofetil to try to prevent disease relapse.\n\nPatients with severe aplastic anemia who do not have a suitable bone marrow donor or who decline bone marrow transplantation may participate in this study. Patients will have a skin test for ATG allergy, chest X-ray, blood test, and bone marrow aspiration before treatment begins. ATG will then be started, infused through a vein continuously for 4 days. Ten days after ATG is stopped, cyclosporine treatment will begin, taken twice a day by mouth in either liquid or capsule form and will continue for 6 months. Also, in the first 2 weeks of treatment, patients will be given a full dose of corticosteroid (prednisone) to prevent serum sickness that could develop as a side effect of ATG therapy. The dosage will be decreased after that. Mycophenolate will be started at the same time as ATG, in two daily doses by mouth, and will continue for 18 months.\n\nPatients will be hospitalized at the beginning of the study. During this time, blood will be drawn at 3-week intervals and a bone marrow examination will be repeated 3 months after treatment has begun. Additional tests, including X-rays may be required. After hospital discharge, patients will be followed on an outpatient basis at 3-month intervals. The patients own physician will perform blood tests weekly and kidney and liver function tests every 2 weeks during cyclosporine therapy. Transfusions may be required initially.", "detailedDescription"=>"Severe acquired aplastic anemia (SAA) has a poor prognosis if untreated. Bone marrow transplantation is available to only a minority of patients due to lack of a matched sibling donor, advanced age of the patient, or cost. Clinical studies at NIH and elsewhere have demonstrated excellent response rates and improved survival with immunosuppressive treatments. Laboratory data implicate underlying cytotoxic T-lymphocyte-mediated suppression of hematopoiesis as the likely proximal cause of disease in most patients. In earlier clinical protocols we treated SAA with cyclosporine A (CSA) (86-H-0007), antithymocyte globulin (ATG) (87-H-0124), and combined ATG and CSA (90-H-0146). While intensive immunosuppression is most effective, relapse is common and some patients also develop second hematologic complications like myelodysplasia. In this protocol, we modify our regimen by delaying the introduction of cyclosporine to promote ATG tolerizing effects and adding mycophenolate mofetil (MMF), a new agent that, like ATG may be relatively specific for activated lymphocytes, in an effort to reduce the high relapse rate."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD", "ADULT", "OLDER_ADULT"], "maximumAge"=>"99 years", "minimumAge"=>"1 year", "healthyVolunteers"=>false, "eligibilityCriteria"=>"* INCLUSION CRITERIA:\n\nOnly patients with SAA will be admitted, defined as:\n\nBone marrow cellularity less than 30%.\n\nAt least two of the following blood count findings: absolute granulocyte count less than 500/mm(3); platelet count less than 20,000/mm(3); reticulocyte count less than 60,000/mm(3).\n\nAge greater than or equal to 1 years.\n\nWeight greater than 12 kg.\n\nEXCLUSION CRITERIA:\n\nSerum creatinine greater than 2 mg/dl or estimated creatinine clearance less than 40 ml/min.\n\nUnderlying carcinoma, recent history of radiation or chemotherapy.\n\nCurrent pregnancy or unwillingness to be treated with oral contraceptives.\n\nInability to comprehend the investigational nature of the study.\n\nMoribund status or concurrent hepatic, renal, cardiac, neurologic, or metabolic disease of such severity that death within 7 to 10 days is likely.\n\nEvidence of other etiology than AA for bone marrow failure, including positive clastogenic stress cytogenetic assay for Fanconi anemia and marrow chromosome abnormalities typical of myelodysplasia."}, "identificationModule"=>{"nctId"=>"NCT00001964", "briefTitle"=>"Combination Therapy of Severe Aplastic Anemia", "organization"=>{"class"=>"NIH", "fullName"=>"National Institutes of Health Clinical Center (CC)"}, "officialTitle"=>"Treatment of Severe Aplastic Anemia With Combined Immunosuppression: Antithymocyte Globulin (ATG) and Cyclosporine A (CSA), and Mycophenolate Mofetil (MMF)", "orgStudyIdInfo"=>{"id"=>"000032"}, "secondaryIdInfos"=>[{"id"=>"00-H-0032"}]}, "armsInterventionsModule"=>{"armGroups"=>[{"type"=>"EXPERIMENTAL", "label"=>"single arm", "description"=>"ATG 40 mg/kg/d for 4 d; CsA 2 weeks after at 12 mg/kg/d for 6 months. MMF starting on first day of ATG at 600 mg/m2 twice daily for 18 months", "interventionNames"=>["Drug: Cyclosporine A", "Drug: ATG", "Drug: MMF"]}, {"type"=>"EXPERIMENTAL", "label"=>"single arm 2", "description"=>"ATG at 40 mg/kg/day for 4 days; MMF at 600 mg/m2 twice daily for 18 months and CsA at 12 mg/kg/day for 6 months starting 2 weeks after ATG and MMF", "interventionNames"=>["Drug: Cyclosporine A", "Drug: ATG", "Drug: MMF"]}], "interventions"=>[{"name"=>"Cyclosporine A", "type"=>"DRUG", "description"=>"Cyclosporine A", "armGroupLabels"=>["single arm", "single arm 2"]}, {"name"=>"ATG", "type"=>"DRUG", "description"=>"ATG", "armGroupLabels"=>["single arm", "single arm 2"]}, {"name"=>"MMF", "type"=>"DRUG", "description"=>"MMF", "armGroupLabels"=>["single arm", "single arm 2"]}]}, "contactsLocationsModule"=>{"locations"=>[{"zip"=>"20892", "city"=>"Bethesda", "state"=>"Maryland", "country"=>"United States", "facility"=>"National Institutes of Health Clinical Center, 9000 Rockville Pike", "geoPoint"=>{"lat"=>38.98067, "lon"=>-77.10026}}], "overallOfficials"=>[{"name"=>"Neal S Young, M.D.", "role"=>"PRINCIPAL_INVESTIGATOR", "affiliation"=>"National Heart, Lung, and Blood Institute (NHLBI)"}]}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"National Heart, Lung, and Blood Institute (NHLBI)", "class"=>"NIH"}, "responsibleParty"=>{"type"=>"SPONSOR"}}}}
