Nctid:
NCT00001749
Payload:
{"hasResults"=>false, "derivedSection"=>{"miscInfoModule"=>{"versionHolder"=>"2024-12-20"}, "conditionBrowseModule"=>{"meshes"=>[{"id"=>"D005198", "term"=>"Fanconi Syndrome"}, {"id"=>"D000740", "term"=>"Anemia"}, {"id"=>"D006402", "term"=>"Hematologic Diseases"}, {"id"=>"D005199", "term"=>"Fanconi Anemia"}, {"id"=>"D029503", "term"=>"Anemia, Diamond-Blackfan"}], "ancestors"=>[{"id"=>"D029502", "term"=>"Anemia, Hypoplastic, Congenital"}, {"id"=>"D000741", "term"=>"Anemia, Aplastic"}, {"id"=>"D000080984", "term"=>"Congenital Bone Marrow Failure Syndromes"}, {"id"=>"D000080983", "term"=>"Bone Marrow Failure Disorders"}, {"id"=>"D001855", "term"=>"Bone Marrow Diseases"}, {"id"=>"D030342", "term"=>"Genetic Diseases, Inborn"}, {"id"=>"D049914", "term"=>"DNA Repair-Deficiency Disorders"}, {"id"=>"D008659", "term"=>"Metabolic Diseases"}, {"id"=>"D015499", "term"=>"Renal Tubular Transport, Inborn Errors"}, {"id"=>"D007674", "term"=>"Kidney Diseases"}, {"id"=>"D014570", "term"=>"Urologic Diseases"}, {"id"=>"D052776", "term"=>"Female Urogenital Diseases"}, {"id"=>"D005261", "term"=>"Female Urogenital Diseases and Pregnancy Complications"}, {"id"=>"D000091642", "term"=>"Urogenital Diseases"}, {"id"=>"D052801", "term"=>"Male Urogenital Diseases"}, {"id"=>"D012010", "term"=>"Red-Cell Aplasia, Pure"}], "browseLeaves"=>[{"id"=>"M8341", "name"=>"Fanconi Syndrome", "asFound"=>"Fanconi Anemia", "relevance"=>"HIGH"}, {"id"=>"M4070", "name"=>"Anemia", "asFound"=>"Anemia", "relevance"=>"HIGH"}, {"id"=>"M8342", "name"=>"Fanconi Anemia", "asFound"=>"Fanconi Anemia", "relevance"=>"HIGH"}, {"id"=>"M9490", "name"=>"Hematologic Diseases", "asFound"=>"Hematologic Diseases", "relevance"=>"HIGH"}, {"id"=>"M23456", "name"=>"Anemia, Diamond-Blackfan", "asFound"=>"Diamond Blackfan Anemia", "relevance"=>"HIGH"}, {"id"=>"M16355", "name"=>"Syndrome", "relevance"=>"LOW"}, {"id"=>"M23455", "name"=>"Anemia, Hypoplastic, Congenital", "relevance"=>"LOW"}, {"id"=>"M4071", "name"=>"Anemia, Aplastic", "relevance"=>"LOW"}, {"id"=>"M2241", "name"=>"Bone Marrow Failure Disorders", "relevance"=>"LOW"}, {"id"=>"M13118", "name"=>"Pancytopenia", "relevance"=>"LOW"}, {"id"=>"M2242", "name"=>"Congenital Bone Marrow Failure Syndromes", "relevance"=>"LOW"}, {"id"=>"M5134", "name"=>"Bone Marrow Diseases", "relevance"=>"LOW"}, {"id"=>"M23686", "name"=>"Genetic Diseases, Inborn", "relevance"=>"LOW"}, {"id"=>"M26131", "name"=>"DNA Repair-Deficiency Disorders", "relevance"=>"LOW"}, {"id"=>"M11639", "name"=>"Metabolic Diseases", "relevance"=>"LOW"}, {"id"=>"M18151", "name"=>"Renal Tubular Transport, Inborn Errors", "relevance"=>"LOW"}, {"id"=>"M10698", "name"=>"Kidney Diseases", "relevance"=>"LOW"}, {"id"=>"M17319", "name"=>"Urologic Diseases", "relevance"=>"LOW"}, {"id"=>"M2875", "name"=>"Urogenital Diseases", "relevance"=>"LOW"}, {"id"=>"M27093", "name"=>"Female Urogenital Diseases", "relevance"=>"LOW"}, {"id"=>"M14127", "name"=>"Pregnancy Complications", "relevance"=>"LOW"}, {"id"=>"M8399", "name"=>"Female Urogenital Diseases and Pregnancy Complications", "relevance"=>"LOW"}, {"id"=>"M27095", "name"=>"Male Urogenital Diseases", "relevance"=>"LOW"}, {"id"=>"M14852", "name"=>"Red-Cell Aplasia, Pure", "relevance"=>"LOW"}, {"id"=>"T2270", "name"=>"Fanconi Anemia", "asFound"=>"Fanconi Anemia", "relevance"=>"HIGH"}, {"id"=>"T1464", "name"=>"Congenital Aplastic Anemia", "asFound"=>"Fanconi Anemia", "relevance"=>"HIGH"}, {"id"=>"T2272", "name"=>"Fanconi Syndrome", "asFound"=>"Fanconi Anemia", "relevance"=>"HIGH"}, {"id"=>"T1837", "name"=>"Diamond-Blackfan Anemia", "asFound"=>"Diamond Blackfan Anemia", "relevance"=>"HIGH"}, {"id"=>"T4833", "name"=>"Pure Red Cell Aplasia", "relevance"=>"LOW"}], "browseBranches"=>[{"name"=>"Urinary Tract, Sexual Organs, and Pregnancy Conditions", "abbrev"=>"BXS"}, {"name"=>"Diseases and Abnormalities at or Before Birth", "abbrev"=>"BC16"}, {"name"=>"All Conditions", "abbrev"=>"All"}, {"name"=>"Blood and Lymph Conditions", "abbrev"=>"BC15"}, {"name"=>"Nutritional and Metabolic Diseases", "abbrev"=>"BC18"}, {"name"=>"Symptoms and General Pathology", "abbrev"=>"BC23"}, {"name"=>"Rare Diseases", "abbrev"=>"Rare"}]}, "interventionBrowseModule"=>{"meshes"=>[{"id"=>"D016572", "term"=>"Cyclosporine"}, {"id"=>"D003524", "term"=>"Cyclosporins"}, {"id"=>"D000961", "term"=>"Antilymphocyte Serum"}], "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"=>"M18961", "name"=>"Cyclosporine", "asFound"=>"/mL", "relevance"=>"HIGH"}, {"id"=>"M6730", "name"=>"Cyclosporins", "asFound"=>"/mL", "relevance"=>"HIGH"}, {"id"=>"M4279", "name"=>"Antilymphocyte Serum", "asFound"=>"Efavirenz", "relevance"=>"HIGH"}, {"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"=>"Antirheumatic Agents", "abbrev"=>"ARhu"}, {"name"=>"Dermatologic Agents", "abbrev"=>"Derm"}, {"name"=>"All Drugs and Chemicals", "abbrev"=>"All"}]}}, "protocolSection"=>{"designModule"=>{"phases"=>["PHASE2"], "studyType"=>"INTERVENTIONAL", "designInfo"=>{"primaryPurpose"=>"TREATMENT"}, "enrollmentInfo"=>{"count"=>25}}, "statusModule"=>{"overallStatus"=>"COMPLETED", "startDateStruct"=>{"date"=>"1998-07"}, "expandedAccessInfo"=>{"hasExpandedAccess"=>false}, "statusVerifiedDate"=>"2005-07", "completionDateStruct"=>{"date"=>"2005-07"}, "lastUpdateSubmitDate"=>"2008-03-03", "studyFirstSubmitDate"=>"1999-11-03", "studyFirstSubmitQcDate"=>"1999-11-03", "lastUpdatePostDateStruct"=>{"date"=>"2008-03-04", "type"=>"ESTIMATED"}, "studyFirstPostDateStruct"=>{"date"=>"1999-11-04", "type"=>"ESTIMATED"}}, "conditionsModule"=>{"keywords"=>["Immune Suppression", "Erythroid Hypoplasia", "Congenital Anemia", "Reticulocytopenia", "Diamond Blackfan Anemia"], "conditions"=>["Fanconi's Anemia", "Hematologic Disease"]}, "referencesModule"=>{"references"=>[{"pmid"=>"7524624", "type"=>"BACKGROUND", "citation"=>"Casadevall N, Croisille L, Auffray I, Tchernia G, Coulombel L. Age-related alterations in erythroid and granulopoietic progenitors in Diamond-Blackfan anaemia. Br J Haematol. 1994 Jun;87(2):369-75. doi: 10.1111/j.1365-2141.1994.tb04924.x."}, {"pmid"=>"8826887", "type"=>"BACKGROUND", "citation"=>"Ball SE, McGuckin CP, Jenkins G, Gordon-Smith EC. Diamond-Blackfan anaemia in the U.K.: analysis of 80 cases from a 20-year birth cohort. Br J Haematol. 1996 Sep;94(4):645-53. doi: 10.1046/j.1365-2141.1996.d01-1839.x."}, {"pmid"=>"2694854", "type"=>"BACKGROUND", "citation"=>"Halperin DS, Freedman MH. Diamond-blackfan anemia: etiology, pathophysiology, and treatment. Am J Pediatr Hematol Oncol. 1989 Winter;11(4):380-94."}]}, "descriptionModule"=>{"briefSummary"=>"Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood cells and platelets, are normal.\n\nStandard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure, relapse, side-effects, increased morbidity, and even death. Two drugs, antithymocyte globulin (ATG) and cyclosporin have been used to treat DBA, but have only provided occasional responses. No study has ever combined these two drugs for the treatment of DBA.\n\nThis study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA.", "detailedDescription"=>"Diamond Blackfan anemia (DBA) is a constitutional pure red cell aplasia of unknown etiology. There is laboratory evidence for an immune mechanism and most patients respond to corticosteroids. However the relapse and failure rate are high, and corticosteroids are associated with many short and long term side effects. Patients who do not respond or who do not tolerate corticosteriods require lifelong red blood cell transfusion and iron chelation therapy. Allogeneic bone marrow transplantation is an option for those with a related histocompatible donor, but this procedure is associated with high mortality and morbidity. Other therapies have been tried without general success. Occasional responses to either ATG or cyclosporine have been reported, but no study has used both ATG and cyclosporine. In other blood/bone marrow disorders of immune etiology these drugs have synergistic effects. We propose a Phase II study to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of Diamond Blackfan anemia."}, "eligibilityModule"=>{"sex"=>"ALL", "stdAges"=>["CHILD", "ADULT", "OLDER_ADULT"], "healthyVolunteers"=>false, "eligibilityCriteria"=>"INCLUSION CRITERIA:\n\nDiagnosis of DBA as characterized by a hyporegenerative anemia presenting in early childhood with reticulocytopenia, and low or absent erythroid precursors in the bone marrow.\n\nTransfusion-dependence due to steroid failure or intolerance of steroid side effects.\n\nIneligible for or declining an allogeneic transplant.\n\nAges 3 to 75.\n\nEXCLUSION CRITERIA:\n\nSerum creatinine greater than 2 times normal or a creatinine clearance less than 50% normal.\n\nSGPT or SGOT greater than 5 times normal.\n\nHistory of epilepsy (any seizures besides childhood febrile seizures).\n\nCurrent pregnancy or unwillingness to take oral contraceptives if menstruating.\n\nPositive diepoxybutane (DEB) test for Fanconi anemia.\n\nHIV positivity.\n\nInability or unwillingness to sign an informed consent, either by the patient, or in the case of a minor, by the parent or guardian responsible for the patient.\n\nUnderlying organ failure and/or those with a Karnofsky performance status of less than 1.\n\nTreatment with androgens, prednisone greater than 10 mg/day, growth factors, or other immunosuppressive therapies within one month of protocol entry.\n\nOngoing treatment with Beta-adrenergic blocking drugs.\n\nPrevious treatment with ATG and concurrent CSA. Previous treatment with either drug alone is acceptable if greater than one year prior to study entry."}, "identificationModule"=>{"nctId"=>"NCT00001749", "briefTitle"=>"Medical Treatment for Diamond Blackfan Anemia", "organization"=>{"class"=>"NIH", "fullName"=>"National Institutes of Health Clinical Center (CC)"}, "officialTitle"=>"Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A", "orgStudyIdInfo"=>{"id"=>"980144"}, "secondaryIdInfos"=>[{"id"=>"98-H-0144"}]}, "armsInterventionsModule"=>{"interventions"=>[{"name"=>"Antithymocyte globulin", "type"=>"DRUG"}, {"name"=>"Cyclosporine", "type"=>"DRUG"}]}, "contactsLocationsModule"=>{"locations"=>[{"zip"=>"20892", "city"=>"Bethesda", "state"=>"Maryland", "country"=>"United States", "facility"=>"National Heart, Lung and Blood Institute (NHLBI)", "geoPoint"=>{"lat"=>38.98067, "lon"=>-77.10026}}]}, "sponsorCollaboratorsModule"=>{"leadSponsor"=>{"name"=>"National Heart, Lung, and Blood Institute (NHLBI)", "class"=>"NIH"}}}}
