Ignite Creation Date:
2025-12-26 @ 11:07 AM
Ignite Modification Date:
2025-12-29 @ 4:15 AM
Study NCT ID:
NCT00004412
Status:
COMPLETED
Last Update Posted:
2015-03-31
First Post:
1999-10-18
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D012883', 'term': 'Skin Ulcer'}, {'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D035583', 'term': 'Rare Diseases'}, {'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D013789', 'term': 'Thalassemia'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C033018', 'term': 'arginine butyrate'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'sperrine@bu.edu', 'phone': '(617) 638-5639', 'title': 'Douglas V. Faller, Ph.D., M.D., Susan P. Perrine, M.D.', 'organization': 'Boston University School of Medicine'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'Standard Local Care Dressing', 'description': 'Standard local care including cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.', 'otherNumAtRisk': 11, 'otherNumAffected': 1, 'seriousNumAtRisk': 11, 'seriousNumAffected': 5}, {'id': 'EG001', 'title': 'Arginine Butyrate', 'description': 'Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.', 'otherNumAtRisk': 15, 'otherNumAffected': 0, 'seriousNumAtRisk': 15, 'seriousNumAffected': 9}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Decreased Appetite', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Vaso-occlusive crisis (VOC))', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 13, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Septic arthritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Clotted Portacath', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Chronic Limb Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Head & Eye Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Eye disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Bloody Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Ulcer Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Possible Celluitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT'}, {'term': 'Pre-existing Osteomyelitis of Left Foot', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pleural effusion and Pericardial Effusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Chest Pain , SOB', 'stats': [{'groupId': 'EG000', 'numAtRisk': 11, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 15, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '1'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Healing Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '12', 'groupId': 'OG001'}]}, {'units': 'number of ulcers', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Standard Local Care Dressing', 'description': 'Standard local care includng cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.'}, {'id': 'OG001', 'title': 'Arginine Butyrate', 'description': 'Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.'}], 'classes': [{'categories': [{'measurements': [{'value': '24', 'groupId': 'OG000'}, {'value': '78', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.05', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'paramValue': '0.5', 'groupDescription': 'Percentage of ulcers undergoing partial and complete healing compared at 12 weeks. Mean Ulcer areas were calculated utilizing computerized planimetry, ulcer area tracings, and photography for baseline and 12 weeks and compared between the two study Arms.', 'statisticalMethod': 'Fisher Exact', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'NUMBER', 'timeFrame': 'participants were followed for an average of 3 months', 'description': 'Treatment Arm: The AB is given as an IV infusion at 500 mg/kg over 6-9 hrs. 5 days per week for 12 weeks. After 12 weeks of therapy, if he ulcer has decreased by 25% , the AB may be continued for additional 8 weeks (twice) or, until ulcer closes plus 2 weeks, additionally.\n\nUlcers photographed, traced, and ulcer areas calculated by computerized planimetry.', 'unitOfMeasure': 'percentage of healed ulcers', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'number of ulcers', 'denomUnitsSelected': 'Participants', 'populationDescription': 'Per protocol'}, {'type': 'SECONDARY', 'title': '% Ulcers Which Completely Healed in Each Group, After 3 Months', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}, {'units': 'Number of ulcers', 'counts': [{'value': '25', 'groupId': 'OG000'}, {'value': '48', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Standard Local Care Dressing', 'description': 'Standard local care includng cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.'}, {'id': 'OG001', 'title': 'Arginine Butyrate', 'description': 'Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.'}], 'classes': [{'categories': [{'measurements': [{'value': '8', 'groupId': 'OG000'}, {'value': '31', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'two additional courses of 8 week cycles', 'description': 'Control Arm: given option to crossover to Treatment Arm if, ulcers have not closed after 12 weeks standard local care alone.', 'unitOfMeasure': 'percentage of completely healed ulcers', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'Number of ulcers', 'denomUnitsSelected': 'Participants', 'populationDescription': 'Per Protocol'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Standard Local Care Dressing', 'description': 'Standard local care includng cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.'}, {'id': 'FG001', 'title': 'Arginine Butyrate', 'description': 'Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.'}, {'id': 'FG002', 'title': 'Crossover', 'description': 'Patients are randomly assigned (following a table of random numbers prepared by a blinded statistician) between two arms of the study. Arm I is Allevyn Hydrophilic PU dressing (standard local care) only, and Arm II is standard local care plus Arginine Butyrate (AB), the Investigational New Drug. Ulcers observed \\& traced weekly. Ulcer area calculated by computerized planimetry. After 12 weeks of therapy, if the ulcer size decreased by at least 25%, the AB may be continued for another 8 weeks (twice), or until the ulcer closes, plus an additional 2 weeks. The patients randomized to the Control Arm ( standard local care) were given the option of crossing over to Arm II If, ulcers did not close after 8 weeks of standard local care.'}], 'periods': [{'title': 'Randomized Period', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '12'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}, {'groupId': 'FG001', 'numSubjects': '12'}, {'groupId': 'FG002', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Moved to Crossover Period', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}, {'title': 'Crossover Period', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'Eight subjects were randomized to Standard of Care dressing Arm', 'groupId': 'FG000', 'numSubjects': '8'}, {'comment': 'Twelve patients were randomized to the Arginine Butyrate plus Standard local care dressing Arm', 'groupId': 'FG001', 'numSubjects': '12'}, {'comment': "Three of Randomized Period's Arm I elected to move to Crossover Period's Crossover Arm.", 'groupId': 'FG002', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Eight subjects in this arm completed study without crossing over.', 'groupId': 'FG000', 'numSubjects': '8'}, {'comment': 'Twelve subjects in this arm completed the study.', 'groupId': 'FG001', 'numSubjects': '12'}, {'comment': 'Three subjects, from Arm I, who crossed- over to Arm II completed the study.', 'groupId': 'FG002', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Study began 1998, contacted physicians at several medical centers with patients under their care for SCD with refractory leg ulcers. Electronic materials describing the study and its sponsor were transmitted to medical centers.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '23', 'groupId': 'BG003'}]}], 'groups': [{'id': 'BG000', 'title': 'Standard Local Care Dressing', 'description': 'Standard local care dressing including cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.'}, {'id': 'BG001', 'title': 'Arginine Butyrate', 'description': 'Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 250 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 8 to 12hours.'}, {'id': 'BG002', 'title': 'Crossover', 'description': 'Patients are randomly assigned (following a table of random numbers prepared by a blinded statistician) between two arms of the study. Arm I is Standard local care dressing only, and Arm II is standard local care plus Arginine Butyrate (AB), the Investigational New Drug. Ulcers observed \\& traced weekly. Ulcer area calculated by computerized planimetry. After 12 weeks of therapy, if the ulcer size decreased by at least 25%, the AB may be continued for another 8 weeks (twice), or until the ulcer closes, plus an additional 2 weeks. The patients randomized to the Control Arm ( standard local care) were given the option of crossing over to Arm II If, ulcers did not close after 8 weeks of standard local care.'}, {'id': 'BG003', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '23', 'groupId': 'BG003'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '31', 'spread': '12.57478', 'groupId': 'BG000'}, {'value': '33', 'spread': '9.14985', 'groupId': 'BG001'}, {'value': '31', 'spread': '2.3094', 'groupId': 'BG002'}, {'value': '32', 'spread': '1.1547', 'groupId': 'BG003'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '11', 'groupId': 'BG003'}]}, {'title': 'Male', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '12', 'groupId': 'BG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '22', 'groupId': 'BG003'}]}]}, {'title': 'Israel', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 23}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1997-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-03', 'completionDateStruct': {'date': '2005-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-03-18', 'studyFirstSubmitDate': '1999-10-18', 'resultsFirstSubmitDate': '2013-07-22', 'studyFirstSubmitQcDate': '1999-10-18', 'lastUpdatePostDateStruct': {'date': '2015-03-31', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2015-03-18', 'studyFirstPostDateStruct': {'date': '1999-10-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-03-31', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2002-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Healing Defined as a Decrease in Ulcer Area by at Least 25% of the Initial Area', 'timeFrame': 'participants were followed for an average of 3 months', 'description': 'Treatment Arm: The AB is given as an IV infusion at 500 mg/kg over 6-9 hrs. 5 days per week for 12 weeks. After 12 weeks of therapy, if he ulcer has decreased by 25% , the AB may be continued for additional 8 weeks (twice) or, until ulcer closes plus 2 weeks, additionally.\n\nUlcers photographed, traced, and ulcer areas calculated by computerized planimetry.'}], 'secondaryOutcomes': [{'measure': '% Ulcers Which Completely Healed in Each Group, After 3 Months', 'timeFrame': 'two additional courses of 8 week cycles', 'description': 'Control Arm: given option to crossover to Treatment Arm if, ulcers have not closed after 12 weeks standard local care alone.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['dermatologic disorders', 'genetic diseases and dysmorphic syndromes', 'hematologic disorders', 'rare disease', 'sickle cell anemia', 'skin ulcers', 'thalassemia major'], 'conditions': ['Skin Ulcers', 'Sickle Cell Anemia']}, 'referencesModule': {'references': [{'pmid': '20955402', 'type': 'RESULT', 'citation': 'McMahon L, Tamary H, Askin M, Adams-Graves P, Eberhardt RT, Sutton M, Wright EC, Castaneda SA, Faller DV, Perrine SP. A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers. Br J Haematol. 2010 Dec;151(5):516-24. doi: 10.1111/j.1365-2141.2010.08395.x. Epub 2010 Oct 19.'}]}, 'descriptionModule': {'briefSummary': 'OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers.\n\nII. Determine the effect of arginine butyrate therapy on tissue factors related to promotion or inhibition of wound healing in these patients.\n\nIII. Determine whether the regimen used in this study is appropriate for testing in pivotal trials.', 'detailedDescription': "PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to one of two treatment arms. Arm I: Patients receive arginine butyrate IV over 6-9 hours at night 5 days a week for 12 weeks, plus concurrent standard local therapy consisting of cleaning, saline irrigation, and dressing changes as prescribed by each patient's physician. Patients who experience progressive healing receive arginine butyrate 3-4 times a week. Arginine butyrate treatment may be discontinued and reinstated following a single 2 week medical complication.\n\nArm II: Patients receive standard local therapy alone for 12 weeks. Patients randomized to arm II may cross over to receive arginine butyrate if no or less than 25% healing is observed after 12 weeks.\n\nPatients whose ulcers have closed by at least 15% per cycle may receive 2 additional 8-week cycles of arginine butyrate therapy and are followed for 2 months after healing is completed."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '60 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL CRITERIA\n\nINCLUSION:\n\n--Disease Characteristics-- Significant sickle cell syndrome including Hemoglobin SS (HbSS), Hemoglobin S-beta thalassemia, and hemoglobin variants. Lower extremity or ankle ulcer (or ulcers) present for at least 6 months without healing --Patient Characteristics-- Age: 16-60 yrs Performance status: Not specified Hematopoietic: Not specified Hepatic: No hepatic compromise Transaminases no greater than 250 IU Renal: No renal compromise Creatinine no greater than 1.2 mg/mL (adults) Creatinine no greater than 0.9 mg/mL (teenagers) Other: Not pregnant Fertile patients must use effective contraception No poorly controlled seizure disorders No other secondary conditions that might inhibit immune function\n\n* Prior/Concurrent Therapy Other: Must be treated with antibiotics prior to entry for complicating cellulitis or secondary infections\n\nEXCLUSION :\n\n--Prior/Concurrent Therapy-- Biologic therapy: chronic transfusion therapy Chemotherapy: prior or concurrent cancer chemotherapy concurrent butyrate derivatives Endocrine therapy: concurrent corticosteroid therapy Radiotherapy: Not specified Surgery: Not specified --Patient Characteristics-- Hepatic: hepatic compromise Transaminases greater than 250 IU Renal: renal compromise Creatinine greater than 1.2 mg/mL (adults) Creatinine greater than 0.9 mg/mL (teenagers) Other: pregnant; Fertile patients with no effective contraception Poorly controlled seizure disorders other secondary conditions that might inhibit immune function'}, 'identificationModule': {'nctId': 'NCT00004412', 'briefTitle': 'Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers', 'organization': {'class': 'OTHER', 'fullName': 'Boston University'}, 'officialTitle': 'Phase II Trial of Arginine Butyrate for Treatment of Refractory Leg Ulcers in Sickle Cell Disease or Beta Thalassemia', 'orgStudyIdInfo': {'id': '199/13302'}, 'secondaryIdInfos': [{'id': 'BUSM-FDR001376', 'type': 'OTHER', 'domain': 'Food & Drug Administration'}, {'id': 'IND 36,957', 'type': 'OTHER', 'domain': 'Food & Drug Administration'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Standard local care dressing', 'description': 'Each subject provided his/her own dressing e.g,standard local care includes cleaning, saline irrigation, dressing changes only for 8 weeks twice a week.', 'interventionNames': ['Other: Standard local care dressing']}, {'type': 'EXPERIMENTAL', 'label': 'Arginine Butyrate', 'description': 'Arginine Butyrate IV plus Standard local care dressing for a total of 12 weeks. Low dose 500 mg/kg or, increased dose 750 mg/kg. First week AB given 5 days in a row, over 6 to 12hours.', 'interventionNames': ['Drug: Arginine Butyrate', 'Other: Standard local care dressing']}, {'type': 'OTHER', 'label': 'Crossover', 'description': 'Patients are randomly assigned (following a table of random numbers prepared by a blinded statistician) between two arms of the study. Arm I is Standard local care dressing only, and Arm II is standard local care plus Arginine Butyrate (AB), the Investigational New Drug. Ulcers observed \\& traced weekly. Ulcer area calculated by computerized planimetry. After 12 weeks of therapy, if the ulcer size decreased by at least 25%, the AB may be continued for another 8 weeks (twice), or until the ulcer closes, plus an additional 2 weeks. The patients randomized to the Control Arm (standard local care) were given the option of crossing over to Arm II If, ulcers did not close after 8 weeks of standard local care.', 'interventionNames': ['Drug: Arginine Butyrate', 'Other: Standard local care dressing']}], 'interventions': [{'name': 'Arginine Butyrate', 'type': 'DRUG', 'description': 'To determine if Arginine Butyrate accelerates healing of refractory leg ulcers over Standard Local Care alone.', 'armGroupLabels': ['Arginine Butyrate', 'Crossover']}, {'name': 'Standard local care dressing', 'type': 'OTHER', 'description': 'To heal leg ulcers.', 'armGroupLabels': ['Arginine Butyrate', 'Crossover', 'Standard local care dressing']}]}, 'contactsLocationsModule': {'locations': [{'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'University of Illinois College of Medicine', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02118', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Boston University School of Medicine', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai School of Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '38103', 'city': 'Memphis', 'state': 'Tennessee', 'country': 'United States', 'facility': 'University of Tennessee, Memphis Cancer Center', 'geoPoint': {'lat': 35.14953, 'lon': -90.04898}}], 'overallOfficials': [{'name': 'Douglas V. Faller', 'role': 'STUDY_CHAIR', 'affiliation': 'Boston University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Susan P. Perrine', 'class': 'OTHER'}, 'collaborators': [{'name': 'Boston Medical Center', 'class': 'OTHER'}, {'name': 'University of Illinois at Chicago', 'class': 'OTHER'}, {'name': 'Icahn School of Medicine at Mount Sinai', 'class': 'OTHER'}, {'name': 'University of Tennessee', 'class': 'OTHER'}, {'name': "Schneider Children's Medical Center, Israel", 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Susan P. Perrine MD, Professor, Boston University School of Medicine', 'investigatorFullName': 'Susan P. Perrine', 'investigatorAffiliation': 'Boston University'}}}}