Ignite Creation Date:
2025-12-24 @ 5:51 PM
Ignite Modification Date:
2025-12-29 @ 11:07 PM
Study NCT ID:
NCT00520468
Status:
COMPLETED
Last Update Posted:
2012-08-07
First Post:
2007-08-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}], 'ancestors': [{'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D004921', 'term': 'Erythropoietin'}, {'id': 'D000068256', 'term': 'Darbepoetin alfa'}, {'id': 'D016572', 'term': 'Cyclosporine'}, {'id': 'D016179', 'term': 'Granulocyte Colony-Stimulating Factor'}, {'id': 'D000069585', 'term': 'Filgrastim'}, {'id': 'D011241', 'term': 'Prednisone'}], 'ancestors': [{'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}, {'id': 'D003524', 'term': 'Cyclosporins'}, {'id': 'D010456', 'term': 'Peptides, Cyclic'}, {'id': 'D047028', 'term': 'Macrocyclic Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D011244', 'term': 'Pregnadienediols'}, {'id': 'D011245', 'term': 'Pregnadienes'}, {'id': 'D011278', 'term': 'Pregnanes'}, {'id': 'D013256', 'term': 'Steroids'}, {'id': 'D000072473', 'term': 'Fused-Ring Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'eharriso@mdanderson.org', 'phone': '713-792-7305', 'title': 'Gautam Borthakur, MBBS / Assistant Professor', 'organization': 'The University of Texas M. D. Anderson Cancer Center'}, 'certainAgreement': {'piSponsorEmployee': True, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': '2 years 2 months', 'eventGroups': [{'id': 'EG000', 'title': 'Cytokine-Immunotherapy', 'description': 'Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily', 'otherNumAtRisk': 14, 'otherNumAffected': 11, 'seriousNumAtRisk': 14, 'seriousNumAffected': 11}], 'otherEvents': [{'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'elevated creatinine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Hypomagnesemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Puritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Blurred vision', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Hyperbilirubinemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}], 'seriousEvents': [{'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'hemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Elevated creatinine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Chest Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Confusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}, {'term': 'Dyspnea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 14, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (3.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Response', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cytokine-Immunotherapy', 'description': 'Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily'}], 'classes': [{'title': 'Complete Remission', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Partial Response', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Hematologic Improvement', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'No Response', 'categories': [{'measurements': [{'value': '6', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Response evaluation within first 3 months from start of therapy, then every 3 to 6 months', 'description': 'Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All treated patients on study included in analysis.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cytokine-Immunotherapy', 'description': 'Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Recruitment Period 6/15/04 - 6/1/09; all patients were registered at The University of Texas M.D. Anderson Cancer Center.', 'preAssignmentDetails': 'Of fifteen patients enrolled, fourteen patients registered were evaluable and one patient taken off study never having received study drug.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Cytokine-Immunotherapy', 'description': 'Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice per week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age Continuous', 'classes': [{'categories': [{'measurements': [{'value': '65', 'groupId': 'BG000', 'lowerLimit': '47', 'upperLimit': '79'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'Years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2004-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2012-08', 'completionDateStruct': {'date': '2009-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2012-08-01', 'studyFirstSubmitDate': '2007-08-23', 'resultsFirstSubmitDate': '2009-09-24', 'studyFirstSubmitQcDate': '2007-08-23', 'lastUpdatePostDateStruct': {'date': '2012-08-07', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2011-03-11', 'studyFirstPostDateStruct': {'date': '2007-08-24', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2011-04-14', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2009-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Response', 'timeFrame': 'Response evaluation within first 3 months from start of therapy, then every 3 to 6 months', 'description': 'Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Myelodysplastic Syndrome', 'MDS', 'Erythropoietin', 'Darbepoetin alfa', 'Aranesp', 'Erythropoiesis stimulating protein', 'G-CSF', 'Filgrastim', 'Neupogen', 'Prednisone', 'Cyclosporin A', 'Sandimmune', 'CYA', 'Cyclosporine'], 'conditions': ['Myelodysplastic Syndrome']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.mdanderson.org', 'label': "M.D. Anderson Cancer Center's Website"}]}, 'descriptionModule': {'briefSummary': 'Objectives:\n\nPrimary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS).\n\nSecondary: To evaluate response duration, survival and side effects of the treatment.', 'detailedDescription': 'MDS is a disease that produces low blood counts and may cause anemia, infections, and/or bleeding. Erythropoietin and Granulocyte colony-stimulating factor (G-CSF or GCSF) are drugs that stimulate the production of red cells and white cells. Prednisone and cyclosporin are drugs that work against MDS by affecting your immune system.\n\nBefore you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a complete medical history and physical exam. Routine blood tests (between 4-6 tablespoons) will be performed. You will have a bone marrow aspiration. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.\n\nIf you are found to be eligible to take part in this study, you will receive erythropoietin as an injection under the skin once a week and G-CSF as an injection under the skin 1-2 times a week for as long as you respond well to treatment. You will take prednisone by mouth every day for a month and cyclosporin tablets by mouth every day for 6 months.\n\nDuring this study, you will need to visit your doctor for a physical exam and measurement of your vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once every 3 months.\n\nBlood tests (about 2 teaspoons) will be done about every 1-2 weeks during the first 12 weeks of treatment, then every 2 to 4 weeks for the remainder of the study. The blood samples will be collected for routine lab tests. Periodic (every 3 to 6 months) bone marrow samples will also be taken to check cells related to the disease before, during, and after completion of this study.\n\nYou will be taken off study if the disease gets worse or intolerable side effects occur.\n\nThis is an investigational study. All drugs are FDA approved and commercially available. Up to 60 patients will take part in this study. All will be enrolled at M. D. Anderson.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients with MDS and \\</= 10% blasts or International Prognostic Scoring System (IPSS) low or intermediate 1. No prior intensive chemotherapy or high-dose ara-C (\\>/= 1g/m2). Prior cytokines, biologic therapies, targeted therapies, or single agent chemotherapy allowed. Procrit, G-CSF are allowed before therapy. Patients with blasts \\< 5% must have an indication for therapy, such as transfusion needs, symptomatic anemia or Hb \\< 11g/dl, platelets \\< 100 x 10 9/L, or granulocytes \\< 10 9/L.\n2. Performance 0-2 (Eastern Cooperative Oncology Group (ECOG)). Adequate liver function (bilirubin of \\< 2mg/dl) and renal function (creatinine \\< 2mg/dl). Adequate cardiac functions (New York Heart Association (NYHA) cardiac III-IV excluded)\n3. Signed informed consent\n\nExclusion Criteria:\n\n1. Nursing and pregnant females are excluded. Women of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.\n2. Patients with active and uncontrolled infections.'}, 'identificationModule': {'nctId': 'NCT00520468', 'briefTitle': 'Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS', 'organization': {'class': 'OTHER', 'fullName': 'M.D. Anderson Cancer Center'}, 'officialTitle': 'Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS', 'orgStudyIdInfo': {'id': '2004-0253'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cytokine-Immunotherapy', 'description': 'Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice a week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily', 'interventionNames': ['Drug: Erythropoietin', 'Drug: Cyclosporin A', 'Drug: G-CSF', 'Drug: Prednisone']}], 'interventions': [{'name': 'Erythropoietin', 'type': 'DRUG', 'otherNames': ['Darbepoetin alfa', 'Aranesp', 'Erythropoiesis stimulating protein'], 'description': '40,000 units injected under the skin (SQ) weekly', 'armGroupLabels': ['Cytokine-Immunotherapy']}, {'name': 'Cyclosporin A', 'type': 'DRUG', 'otherNames': ['CYA', 'Cyclosporine', 'Sandimmune'], 'description': '300 mg (tablets) by mouth daily for 6 months', 'armGroupLabels': ['Cytokine-Immunotherapy']}, {'name': 'G-CSF', 'type': 'DRUG', 'otherNames': ['Filgrastim', 'Neupogen'], 'description': '300 mcg injected under the skin (SQ) two times per week', 'armGroupLabels': ['Cytokine-Immunotherapy']}, {'name': 'Prednisone', 'type': 'DRUG', 'description': '60 mg per day for 7 days, taper over 1 month', 'armGroupLabels': ['Cytokine-Immunotherapy']}]}, 'contactsLocationsModule': {'locations': [{'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'U.T.M.D. Anderson Cancer Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'overallOfficials': [{'name': 'Gautam Borthakur, MBBS', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'M.D. Anderson Cancer Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'M.D. Anderson Cancer Center', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}