Ignite Creation Date:
2025-12-25 @ 2:12 AM
Ignite Modification Date:
2025-12-26 @ 12:40 AM
Study NCT ID:
NCT00065260
Status:
COMPLETED
Last Update Posted:
2021-07-21
First Post:
2003-07-18
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000741', 'term': 'Anemia, Aplastic'}, {'id': 'D013921', 'term': 'Thrombocytopenia'}, {'id': 'D007970', 'term': 'Leukopenia'}, {'id': 'D009503', 'term': 'Neutropenia'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D012008', 'term': 'Recurrence'}, {'id': 'D000740', 'term': 'Anemia'}], 'ancestors': [{'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D001791', 'term': 'Blood Platelet Disorders'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D000380', 'term': 'Agranulocytosis'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D016572', 'term': 'Cyclosporine'}], 'ancestors': [{'id': 'D061067', 'term': 'Antibodies, Monoclonal, Humanized'}, {'id': 'D000911', 'term': 'Antibodies, Monoclonal'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D003524', 'term': 'Cyclosporins'}, {'id': 'D010456', 'term': 'Peptides, Cyclic'}, {'id': 'D047028', 'term': 'Macrocyclic Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D010455', 'term': 'Peptides'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'bhavisha.patel@nih.gov', 'phone': '301.402.3477', 'title': 'Dr. Bhavisha Patel', 'organization': 'NIH NHLBI'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '3 years', 'eventGroups': [{'id': 'EG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.', 'otherNumAtRisk': 27, 'deathsNumAtRisk': 27, 'otherNumAffected': 7, 'seriousNumAtRisk': 27, 'deathsNumAffected': 9, 'seriousNumAffected': 7}, {'id': 'EG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).', 'otherNumAtRisk': 27, 'deathsNumAtRisk': 27, 'otherNumAffected': 12, 'seriousNumAtRisk': 27, 'deathsNumAffected': 4, 'seriousNumAffected': 12}], 'otherEvents': [{'term': 'infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 27, 'numEvents': 12, 'numAffected': 12}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (2.0)'}], 'seriousEvents': [{'term': 'infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 27, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 27, 'numEvents': 12, 'numAffected': 12}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (2.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Participants no Longer Meeting Criteria for Severe Aplastic Anemia.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'OG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}], 'classes': [{'title': 'No Response', 'categories': [{'measurements': [{'value': '18', 'groupId': 'OG000'}, {'value': '17', 'groupId': 'OG001'}]}]}, {'title': 'Partial Response', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}, {'title': 'Complete Response', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 months', 'description': 'Number of participants no longer meeting the criteria for severe aplastic anemia as measured by response to treatment at 6 months', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': '1 patient was not evaluable at 6 months in the alemtuzumab arm because of a single early death from infectious complications'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Robust Hematologic Recovery With Reticulocyte or Platelet Count', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'OG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}], 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '6 months', 'description': 'Number of participants with robust hematologic recovery with reticulocyte or platelet count ≥ 50,000/uL', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '1 patient was not evaluable at 6 months in the alemtuzumab arm because of a single early death from infectious complications'}, {'type': 'SECONDARY', 'title': 'Percentage of Cumulative Incidence of Relapse in Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'OG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}], 'classes': [{'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '3 year', 'description': 'Percentage of cumulative incidence of relapse of disease in participants', 'unitOfMeasure': 'percentage of cumulative incidence', 'reportingStatus': 'POSTED', 'populationDescription': '1 participants was not evaluable at 6 months in the alemtuzumab arm because of a single early death from infectious complications.'}, {'type': 'SECONDARY', 'title': 'Percentage of Cumulative Incidence of Clonal Evolution in Participants', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'OG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}], 'classes': [{'categories': [{'measurements': [{'value': '16', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '3 years', 'description': 'Percent of cumulative incidence of clonal evolution in participants to either paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia or acute leukemia.', 'unitOfMeasure': 'Percentage of Cumulative Incidence', 'reportingStatus': 'POSTED', 'populationDescription': '1 participant was not evaluable at 6 months in the alemtuzumab arm because of a single early death from infectious complications'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants no Longer Meeting Criteria for Severe Aplastic Anemia.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}, {'value': '26', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'OG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}], 'classes': [{'title': '3 Months', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000', 'lowerLimit': '3', 'upperLimit': '34'}, {'value': '19', 'groupId': 'OG001', 'lowerLimit': '3', 'upperLimit': '34'}]}]}, {'title': '6 Months', 'categories': [{'measurements': [{'value': '33', 'groupId': 'OG000', 'lowerLimit': '14', 'upperLimit': '52'}, {'value': '37', 'groupId': 'OG001', 'lowerLimit': '18', 'upperLimit': '57'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '3 months and 6 months', 'description': 'Percentage of participants no longer meeting the criteria for severe aplastic anemia as measured by response to treatment', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': '1 patient was not evaluable at 6 months in the alemtuzumab arm because of a single early death from infectious complications'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'FG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '27'}, {'groupId': 'FG001', 'numSubjects': '27'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '27'}, {'groupId': 'FG001', 'numSubjects': '26'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'BG000'}, {'value': '27', 'groupId': 'BG001'}, {'value': '54', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'r-ATG /Cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nr-ATG: Rabbit ATG 3.5mg/kg/day for consecutive 5 days\n\nCsA: CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.'}, {'id': 'BG001', 'title': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).\n\nCampath-1H: Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '22', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '36', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '24', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '14', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '27', 'groupId': 'BG000'}, {'value': '27', 'groupId': 'BG001'}, {'value': '54', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 54}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2003-11-06', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-06', 'completionDateStruct': {'date': '2016-02-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-07-02', 'studyFirstSubmitDate': '2003-07-18', 'resultsFirstSubmitDate': '2016-02-03', 'studyFirstSubmitQcDate': '2003-07-18', 'lastUpdatePostDateStruct': {'date': '2021-07-21', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2016-02-03', 'studyFirstPostDateStruct': {'date': '2003-07-21', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2016-03-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-12-29', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Participants no Longer Meeting Criteria for Severe Aplastic Anemia.', 'timeFrame': '6 months', 'description': 'Number of participants no longer meeting the criteria for severe aplastic anemia as measured by response to treatment at 6 months'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Robust Hematologic Recovery With Reticulocyte or Platelet Count', 'timeFrame': '6 months', 'description': 'Number of participants with robust hematologic recovery with reticulocyte or platelet count ≥ 50,000/uL'}, {'measure': 'Percentage of Cumulative Incidence of Relapse in Participants', 'timeFrame': '3 year', 'description': 'Percentage of cumulative incidence of relapse of disease in participants'}, {'measure': 'Percentage of Cumulative Incidence of Clonal Evolution in Participants', 'timeFrame': '3 years', 'description': 'Percent of cumulative incidence of clonal evolution in participants to either paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia or acute leukemia.'}, {'measure': 'Percentage of Participants no Longer Meeting Criteria for Severe Aplastic Anemia.', 'timeFrame': '3 months and 6 months', 'description': 'Percentage of participants no longer meeting the criteria for severe aplastic anemia as measured by response to treatment'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Alemtuzumab (Campath-1H)', 'Rabbit ATG', 'Severe Aplastic Anemia', 'Cyclosporine', 'Thrombocytopenia', 'Leukopenia', 'Neutropenia', 'Autoimmunity', 'Relapse', 'Anemia'], 'conditions': ['Aplastic Anemia']}, 'referencesModule': {'references': [{'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': '4909449', 'type': 'BACKGROUND', 'citation': 'Mathe G, Amiel JL, Schwarzenberg L, Choay J, Trolard P, Schneider M, Hayat M, Schlumberger JR, Jasmin C. Bone marrow graft in man after conditioning by antilymphocytic serum. Br Med J. 1970 Apr 18;2(5702):131-6. doi: 10.1136/bmj.2.5702.131.'}, {'pmid': '7985721', 'type': 'BACKGROUND', 'citation': 'Stein RS, Means RT Jr, Krantz SB, Flexner JM, Greer JP. Treatment of aplastic anemia with an investigational antilymphocyte serum prepared in rabbits. Am J Med Sci. 1994 Dec;308(6):338-43. doi: 10.1097/00000441-199412000-00005.'}, {'pmid': '22067384', 'type': 'DERIVED', 'citation': 'Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Wu CO, Young NS. Activity of alemtuzumab monotherapy in treatment-naive, relapsed, and refractory severe acquired aplastic anemia. Blood. 2012 Jan 12;119(2):345-54. doi: 10.1182/blood-2011-05-352328. Epub 2011 Nov 8.'}], 'seeAlsoLinks': [{'url': 'http://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2003-H-0249.html', 'label': 'NIH Clinical Center Detailed Web Page'}]}, 'descriptionModule': {'briefSummary': 'Severe aplastic anemia, characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation. However, approximately 1/3 of patients will not show blood count improvement after ATG/CsA. General experience and small pilot studies have suggested that such patients may benefit from further immunosuppression. Furthermore, analysis of our own clinical data suggest that patients with poor blood count responses to a single course of ATG, even when transfusion-independence is achieved, have a markedly worse prognosis than patients with robust hematologic improvement. The management of such cases is uncertain.\n\nThis study will enroll patients who are either refractory to h-ATG (continued severe pancytopenia) or who have only modest improvement in blood counts (weak hematologic responders) to receive a further immunosuppressive therapy, delivered either as rabbit ATG (Thymoglobulin, r-ATG) or a humanized monoclonal antibody to T-cells, alemtuzumab (Campath-1H ). Primary endpoint will be response rate at 3 months defined as no longer meeting criteria for severe aplastic anemia. Relapse, robustness of hematopoietic recovery at 3 months, survival and clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia and acute leukemia will be the secondary endpoints.', 'detailedDescription': 'Severe aplastic anemia, characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation. However, approximately 1/3 of patients will not show blood count improvement after ATG/CsA. General experience and small pilot studies have suggested that such patients may benefit from further immunosuppression. Furthermore, analysis of our own clinical data suggest that patients with poor blood count responses to a single course of ATG, even when transfusion-independence is achieved, have a markedly worse prognosis than patients with robust hematologic improvement. The management of such cases is uncertain.\n\nThis study will enroll patients who are either refractory to h-ATG (continued severe pancytopenia) or who have only modest improvement in blood counts (weak hematologic responders) to receive further immunosuppressive therapy, delivered either as rabbit ATG (Thymoglobulin , r-ATG) or a humanized monoclonal antibody to T-cells, alemtuzumab (Campath-1H ). Primary endpoint will be response rate at 6 months defined as no longer meeting criteria for severe aplastic anemia. Relapse, robustness of hematopoietic recovery at 6 months, survival and clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia and acute leukemia will be the secondary endpoints.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "* INCLUSION CRITERIA:\n\nSevere aplastic anemia confirmed at NIH by:\n\nBone marrow cellularity less than 30% (excluding lymphocytes)\n\nAt least two of the following:\n\nAbsolute neutrophil count less than 500/microL;\n\nPlatelet count less than 20,000/ microL;\n\nReticulocyte count less than 60,000/ microL.\n\nSevere aplastic anemia refractory to prior course(s) of h-ATG/CsA defined after 3 months from treatment with less or equal to 4 years from receiving h-ATG.\n\nOR\n\nSuboptimal response to initial immunosuppression with h-ATG/CsA as defined by platelet and reticulocyte count less than 50,000 /microL at 3 months.\n\nAge greater than or equal to 2 years of age\n\nEXCLUSION CRITERIA:\n\nDiagnosis of Fanconi anemia.\n\nEvidence of a clonal disorder on cytogenetics. Patients with super severe neutropenia (ANC less than 200/microL) will not be excluded initially if results of cytogenetics are not available or pending. If evidence of a clonal disorder is later identified, the subject will go off study.\n\nPrior treatment courses with rabbit ATG or high dose cyclophosphamide (200 mg/kg or equivalent).\n\nInfection not adequately responding to appropriate therapy.\n\nUnderlying immunodeficiency state including seropositivity for HIV.\n\nFailure to discontinue the herbal supplements Echinacea purpurea or Usnea barbata (Old Man's Beard) within two weeks of enrollment.\n\nPrevious hypersensitivity to Campath-1H or its components.\n\nMoribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy or that death within 7-10 days is likely.\n\nPotential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible.\n\nSerum creatinine greater than 2.5 mg/dL.\n\nCurrent pregnancy or lactation or unwillingness to take contraceptives.\n\nInability to understand the investigational nature of the study or give informed consent."}, 'identificationModule': {'nctId': 'NCT00065260', 'briefTitle': 'Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia', 'organization': {'class': 'NIH', 'fullName': 'National Institutes of Health Clinical Center (CC)'}, 'officialTitle': 'A Randomized Trial of Immunosuppression in Aplastic Anemia Patients With Refractory Pancytopenia or Suboptimal Hematologic Response After h-ATG/CsA Treatment', 'orgStudyIdInfo': {'id': '030249'}, 'secondaryIdInfos': [{'id': '03-H-0249'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'r-ATG /cyclosporine', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (r-ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).', 'interventionNames': ['Drug: r-ATG', 'Drug: CsA']}, {'type': 'EXPERIMENTAL', 'label': 'Alemtuzumab (Campath-1H)', 'description': 'A randomized trial of rabbit anti-thymocyte globulin (ATG)/ cyclosporine (CsA) versus Campath-1H in aplastic anemia patients with refractory pancytopenia or suboptimal hematological response after horse ATG treatment. Subjects who receive rabbit ATG/ CsA will be given rabbit ATG 3.5mg/kg/day for 5 days and CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs. Subjects who receive Campath-1H will receive an intravenous infusion for 10 days. Adult subjects will receive 10mg/day (children:0.2mg/kg/day).', 'interventionNames': ['Drug: Campath-1H']}], 'interventions': [{'name': 'Campath-1H', 'type': 'DRUG', 'otherNames': ['Alemtuzumab'], 'description': 'Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).', 'armGroupLabels': ['Alemtuzumab (Campath-1H)']}, {'name': 'r-ATG', 'type': 'DRUG', 'otherNames': ['Rabbit Anti-Thymoglobulin'], 'description': 'Rabbit ATG 3.5mg/kg/day for consecutive 5 days', 'armGroupLabels': ['r-ATG /cyclosporine']}, {'name': 'CsA', 'type': 'DRUG', 'otherNames': ['Cyclosporine'], 'description': 'CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.', 'armGroupLabels': ['r-ATG /cyclosporine']}]}, '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': 'Danielle M Townsley, 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'}}}}