Ignite Creation Date:
2025-12-24 @ 4:37 PM
Ignite Modification Date:
2026-02-24 @ 8:26 AM
Study NCT ID:
NCT02395666
Status:
COMPLETED
Last Update Posted:
2024-08-06
First Post:
2015-03-05
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009447', 'term': 'Neuroblastoma'}], 'ancestors': [{'id': 'D018241', 'term': 'Neuroectodermal Tumors, Primitive, Peripheral'}, {'id': 'D018242', 'term': 'Neuroectodermal Tumors, Primitive'}, {'id': 'D018302', 'term': 'Neoplasms, Neuroepithelial'}, {'id': 'D017599', 'term': 'Neuroectodermal Tumors'}, {'id': 'D009373', 'term': 'Neoplasms, Germ Cell and Embryonal'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D009375', 'term': 'Neoplasms, Glandular and Epithelial'}, {'id': 'D009380', 'term': 'Neoplasms, Nerve Tissue'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000518', 'term': 'Eflornithine'}], 'ancestors': [{'id': 'D009952', 'term': 'Ornithine'}, {'id': 'D024361', 'term': 'Amino Acids, Basic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D000599', 'term': 'Amino Acids, Diamino'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Giselle.Saulniersholler@atriumhealth.org', 'phone': '704-381-9900', 'title': 'Dr. Giselle Sholler', 'organization': 'Beat Childhood Cancer'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Through study completion plus 30 days, an average of 2 years.', 'description': 'All subjects that received at least one dose of DFMO. Since both arms received the same exact treatment, they were combined into one reporting group.', 'eventGroups': [{'id': 'EG000', 'title': 'All Study Subjects', 'description': 'All subjects that received at least one dose of DFMO. Since both arms received the same exact treatment, they were combined into one reporting group.', 'otherNumAtRisk': 140, 'deathsNumAtRisk': 140, 'otherNumAffected': 41, 'seriousNumAtRisk': 140, 'deathsNumAffected': 0, 'seriousNumAffected': 23}], 'otherEvents': [{'term': 'Neutrophil count decrease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 11, 'numAffected': 11}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Anemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'ALT elevation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 14, 'numAffected': 14}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'AST elevation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 10, 'numAffected': 10}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 9, 'numAffected': 9}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Hearing Loss', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 9, 'numAffected': 9}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Otitis Media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}], 'seriousEvents': [{'term': 'Infection', 'notes': 'All Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Vomiting', 'notes': 'All Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Pain', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Diarrhea', 'notes': 'All Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Fever', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Hyponatremia', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Benign vascular lesion', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Swelling of Eye', 'notes': 'Eye swelling result of brain mass that has been confirmed as metastatic recurrent neuroblastoma.\n\nUnrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Hypoglycemia', 'notes': 'Possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Urticaria', 'notes': 'Possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Constipation', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Respiratory Disorder', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Hypotension', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'hearing loss', 'notes': 'Possibly related', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Fracture', 'notes': 'Femur Fracture- Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Influenza', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Bacteremia', 'notes': 'Unrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}, {'term': 'Obstruction', 'notes': 'gastric obstruction secondary to adhesions from previous surgery. Resolved after surgical correction.\n\nUnrelated', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (4.0)'}], 'frequencyThreshold': '4'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Event Free Survival (EFS) During Study.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '100', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stratum 1', 'description': 'Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}, {'id': 'OG001', 'title': 'Stratum 2', 'description': 'Subjects that are in remission after any previous relapse or refractory therapy.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}], 'classes': [{'categories': [{'measurements': [{'value': '84', 'groupId': 'OG000', 'lowerLimit': '80', 'upperLimit': '88'}, {'value': '51', 'groupId': 'OG001', 'lowerLimit': '43', 'upperLimit': '59'}]}]}], 'paramType': 'MEAN', 'timeFrame': '2 Years', 'description': 'To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)', 'unitOfMeasure': 'percentage of subjects without an event', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'One subject removed from Stratum 1 due to not fitting study criteria upon review'}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Overall Survival (OS)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '100', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stratum 1', 'description': 'Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}, {'id': 'OG001', 'title': 'Stratum 2', 'description': 'Subjects that are in remission after any previous relapse or refractory therapy.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}], 'classes': [{'categories': [{'measurements': [{'value': '97', 'groupId': 'OG000', 'lowerLimit': '95', 'upperLimit': '99'}, {'value': '84', 'groupId': 'OG001', 'lowerLimit': '78', 'upperLimit': '90'}]}]}], 'paramType': 'MEAN', 'timeFrame': '2 Years', 'description': 'To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)', 'unitOfMeasure': 'percentage of subjects without an event', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Adverse Events as a Measure of Safety and Tolerability', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'DFMO Twice Daily', 'description': 'Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.\n\nDFMO: Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}], 'classes': [{'categories': [{'measurements': [{'value': '57', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '2 years', 'description': 'To continue to determine the safety and tolerability of DFMO as a single agent and in pediatric and young adult patients with high risk neuroblastoma that is in remission.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Stratum 1 and 2 were analyzed together as one safety group as per statistical analysis plan.'}, {'type': 'SECONDARY', 'title': 'Test the Association of Survival With ODC1 Genotype', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '140', 'groupId': 'OG001'}, {'value': '140', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'GG, GT, TT', 'description': 'Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.'}, {'id': 'OG001', 'title': 'GG or GT, TT', 'description': 'Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.'}, {'id': 'OG002', 'title': 'GG, GT or TT', 'description': 'Tests of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.96', 'groupId': 'OG000'}, {'value': '0.58', 'groupId': 'OG001'}, {'value': '0.67', 'groupId': 'OG002'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '2 years', 'description': 'Tests (p-value) of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.\n\nBlood: microRNA analysis as predictor of DFMO effect, ornithine decarboxylase (ODC) single nucleotide polymorphism (SNP) analysis in DNA isolated from nucleated cells', 'unitOfMeasure': 'p-value', 'reportingStatus': 'POSTED', 'populationDescription': 'Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.'}, {'type': 'SECONDARY', 'title': 'Peak Plasma Concentration (Cmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Study Subjects Consented to PK Collection', 'description': "Includes study subjects from either stratum that signed an optional consent to have PK's collected"}], 'classes': [{'categories': [{'measurements': [{'value': '11958', 'groupId': 'OG000', 'lowerLimit': '5123', 'upperLimit': '18793'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days', 'description': 'Pharmacokinetic assay Cmax/D\n\nSamples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days.', 'unitOfMeasure': 'ng/mL', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': '12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.'}, {'type': 'SECONDARY', 'title': 'Area Under the Plasma Concentration Versus Time Curve (AUC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Study Subjects Consented to PK Collection', 'description': "Includes study subjects from either stratum that signed an optional consent to have PK's collected"}], 'classes': [{'categories': [{'measurements': [{'value': '47024', 'groupId': 'OG000', 'lowerLimit': '23868', 'upperLimit': '70180'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days', 'description': 'Pharmacokinetic assay AUC(0-6 hr)/D\n\nSamples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days', 'unitOfMeasure': 'hr*ng/mL', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': '12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.'}, {'type': 'SECONDARY', 'title': 'Time to Reach Peak Plasma Concentration (Tmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Study Subjects Consented to PK Collection', 'description': "Includes study subjects from either stratum that signed an optional consent to have PK's collected"}], 'classes': [{'categories': [{'measurements': [{'value': '3.3', 'groupId': 'OG000', 'lowerLimit': '1.9', 'upperLimit': '4.7'}]}]}], 'paramType': 'MEAN', 'timeFrame': '0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days', 'description': 'Pharmacokinetic assay- tmax, hr\n\nSamples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days', 'unitOfMeasure': 'hours', 'dispersionType': '90% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': '12 subjects from both Stratum 1 and 2 were analyzed together as one group as per statistical analysis plan.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Stratum 1', 'description': 'Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}, {'id': 'FG001', 'title': 'Stratum 2', 'description': 'Subjects that are in remission after any previous relapse or refractory therapy.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '101'}, {'groupId': 'FG001', 'numSubjects': '39'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '95'}, {'groupId': 'FG001', 'numSubjects': '38'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}, {'groupId': 'FG001', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '101', 'groupId': 'BG000'}, {'value': '39', 'groupId': 'BG001'}, {'value': '140', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Stratum 1', 'description': 'Subjects that are in remission at the end of upfront therapy defined as chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, anti-GD2 antibody therapy with retinoic acid up to 6 cycles.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}, {'id': 'BG001', 'title': 'Stratum 2', 'description': 'Subjects that are in remission after any previous relapse or refractory therapy.\n\nSubjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '4.4', 'groupId': 'BG000', 'lowerLimit': '1', 'upperLimit': '17'}, {'value': '6.8', 'groupId': 'BG001', 'lowerLimit': '1', 'upperLimit': '14'}, {'value': '5.1', 'groupId': 'BG002', 'lowerLimit': '1', 'upperLimit': '17'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '44', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '55', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '57', 'groupId': 'BG000'}, {'value': '28', 'groupId': 'BG001'}, {'value': '85', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '82', 'groupId': 'BG000'}, {'value': '32', 'groupId': 'BG001'}, {'value': '114', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2015-02-18', 'size': 1289267, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_001.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2023-04-04T17:48', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'PREVENTION', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 140}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-03-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-08', 'completionDateStruct': {'date': '2023-08-24', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-08-02', 'studyFirstSubmitDate': '2015-03-05', 'resultsFirstSubmitDate': '2020-02-14', 'studyFirstSubmitQcDate': '2015-03-20', 'lastUpdatePostDateStruct': {'date': '2024-08-06', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-04-01', 'studyFirstPostDateStruct': {'date': '2015-03-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-04-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-03-27', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Event Free Survival (EFS) During Study.', 'timeFrame': '2 Years', 'description': 'To evaluate the preventative activity of DFMO as a single agent in patients that are in remission based on: Event free survival (EFS)'}], 'secondaryOutcomes': [{'measure': 'Percentage of Participants With Overall Survival (OS)', 'timeFrame': '2 Years', 'description': 'To evaluate the preventative activity of DFMO as a single agent in patients with neuroblastoma who are in remission based on: Overall Survival (OS)'}, {'measure': 'Number of Participants With Adverse Events as a Measure of Safety and Tolerability', 'timeFrame': '2 years', 'description': 'To continue to determine the safety and tolerability of DFMO as a single agent and in pediatric and young adult patients with high risk neuroblastoma that is in remission.'}, {'measure': 'Test the Association of Survival With ODC1 Genotype', 'timeFrame': '2 years', 'description': 'Tests (p-value) of the association of survival with ODC1 single nucleotide polymorphism rs2302616 genotype.\n\nBlood: microRNA analysis as predictor of DFMO effect, ornithine decarboxylase (ODC) single nucleotide polymorphism (SNP) analysis in DNA isolated from nucleated cells'}, {'measure': 'Peak Plasma Concentration (Cmax)', 'timeFrame': 'Samples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days', 'description': 'Pharmacokinetic assay Cmax/D\n\nSamples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days.'}, {'measure': 'Area Under the Plasma Concentration Versus Time Curve (AUC)', 'timeFrame': '0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days', 'description': 'Pharmacokinetic assay AUC(0-6 hr)/D\n\nSamples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days'}, {'measure': 'Time to Reach Peak Plasma Concentration (Tmax)', 'timeFrame': '0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose on two different days', 'description': 'Pharmacokinetic assay- tmax, hr\n\nSamples drawn at 5 timepoints: (0 (pre dose), 30min, 1 hour, 3 hours, and 6 hours post-dose) on two different days'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Neuroblastoma in remission', 'Relapsed Neuroblastoma', 'Refractory Neuroblastoma'], 'conditions': ['Neuroblastoma']}, 'referencesModule': {'references': [{'pmid': '38917371', 'type': 'DERIVED', 'citation': 'Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. J Clin Oncol. 2024 Sep 1;42(25):3047-3057. doi: 10.1200/JCO.24.00546. Epub 2024 Jun 25.'}, {'pmid': '37883734', 'type': 'DERIVED', 'citation': 'Oesterheld J, Ferguson W, Kraveka JM, Bergendahl G, Clinch T, Lorenzi E, Berry D, Wada RK, Isakoff MS, Eslin DE, Brown VI, Roberts W, Zage P, Harrod VL, Mitchell DS, Hanson D, Saulnier Sholler GL. Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. J Clin Oncol. 2024 Jan 1;42(1):90-102. doi: 10.1200/JCO.22.02875. Epub 2023 Oct 26.'}, {'pmid': '34522028', 'type': 'DERIVED', 'citation': 'Urban-Wojciuk Z, Graham A, Barker K, Kwok C, Sbirkov Y, Howell L, Campbell J, Woster PM, Poon E, Petrie K, Chesler L. The biguanide polyamine analog verlindamycin promotes differentiation in neuroblastoma via induction of antizyme. Cancer Gene Ther. 2022 Jul;29(7):940-950. doi: 10.1038/s41417-021-00386-6. Epub 2021 Sep 14.'}, {'pmid': '32506485', 'type': 'DERIVED', 'citation': 'Batth IS, Dao L, Satelli A, Mitra A, Yi S, Noh H, Li H, Brownlee Z, Zhou S, Bond J, Wang J, Gill J, Sholler GS, Li S. Cell surface vimentin-positive circulating tumor cell-based relapse prediction in a long-term longitudinal study of postremission neuroblastoma patients. Int J Cancer. 2020 Dec 15;147(12):3550-3559. doi: 10.1002/ijc.33140. Epub 2020 Jun 23.'}, {'pmid': '32391579', 'type': 'DERIVED', 'citation': 'Lewis EC, Kraveka JM, Ferguson W, Eslin D, Brown VI, Bergendahl G, Roberts W, Wada RK, Oesterheld J, Mitchell D, Foley J, Zage P, Rawwas J, Rich M, Lorenzi E, Broglio K, Berry D, Saulnier Sholler GL. A subset analysis of a phase II trial evaluating the use of DFMO as maintenance therapy for high-risk neuroblastoma. Int J Cancer. 2020 Dec 1;147(11):3152-3159. doi: 10.1002/ijc.33044. Epub 2020 May 24.'}], 'seeAlsoLinks': [{'url': 'http://www.beatcc.org', 'label': 'Beat Childhood Cancer Consortium website'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO.\n\nThe safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit.\n\nThe procedures involved in this study include Medical history, Physical exam, Vital signs (blood pressure, pulse, temperature), Blood tests, Urine tests, MRI or CT scan of the tumor(s), meta-iodobenzylguanidine (MIBG) scans, and Bone marrow aspirations. All of these tests and procedures are considered standard of care for this population. Drug administration is also part of this protocol, including an investigational new drug called DFMO.\n\nThe proposed dosing regimen is an oral dose of DFMO tablets two times a day for each day while on study. There will be 27 cycles. Each cycle will be 28 days in length.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age: 0-21 years at the time of diagnosis.\n* Diagnosis: histologic verification at either the time of original diagnosis or a previous relapse of high risk neuroblastoma.\n* Disease Status: Neuroblastoma that is in remission\n* First dose of study medication must be greater than 30 days from completion of cytotoxic and antibody therapy and less than 120 days from previous therapy\n* A negative serum or urine pregnancy test is required for female subjects of child bearing potential (onset of menses or ≥13 years of age).\n* Both male and female post-pubertal study subjects need to agree to use one of the more effective birth control methods during treatment and for six months after treatment is stopped. These methods include total abstinence (no sex), oral contraceptives ("the pill"), an intrauterine device (IUD), levonorgestrel implants (Norplant), or medroxyprogesterone acetate injections (Depo-provera shots). If one of these cannot be used, contraceptive foam with a condom is recommended.\n* Absolute Neutrophil Count (ANC) \\> 500/μl and platelet count \\>50,000/μl\n* Organ Function Requirements: Subjects must have adequate liver function as defined by:\n\n * Aspartate Aminotransferase (AST) and Alanine transaminase (ALT) \\<10x upper limit of normal\n * Serum bilirubin must be ≤ 2.0 mg/dl\n * Serum creatinine based on age/gender\n* Informed Consent: All subjects and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines\n\nExclusion Criteria:\n\n* Lansky score \\< 60%\n* Body Surface Area (BSA) (m2) of \\<0.25\n* Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.\n* Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the effects of prior chemotherapy (hematological and bone marrow suppression effects).\n* Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.\n* Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.'}, 'identificationModule': {'nctId': 'NCT02395666', 'briefTitle': 'Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission', 'nctIdAliases': ['NCT01586260'], 'organization': {'class': 'OTHER', 'fullName': 'Milton S. Hershey Medical Center'}, 'officialTitle': 'A Phase II Preventative Trial of DFMO (Eflornithine HCl) as a Single Agent in Patients With High Risk Neuroblastoma in Remission', 'orgStudyIdInfo': {'id': 'NMTRC003B'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'DFMO twice daily', 'description': 'Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.', 'interventionNames': ['Drug: DFMO']}], 'interventions': [{'name': 'DFMO', 'type': 'DRUG', 'otherNames': ['eflornithine HCl', 'Difluoromethylornithine'], 'description': 'Subjects will receive twenty-seven (27) cycles of oral DFMO at a dose of 500 to 1000 mg/m2 BID on each day of a 28 day cycle.', 'armGroupLabels': ['DFMO twice daily']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85016', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': "Phoenix Children's Hospital", 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '72202', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': "Arkansas Children's Hospital", 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '92123', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': "Rady Children's Hospital", 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '06106', 'city': 'Hartford', 'state': 'Connecticut', 'country': 'United States', 'facility': "Connecticut Children's Hospital", 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '32806', 'city': 'Orlando', 'state': 'Florida', 'country': 'United States', 'facility': 'Arnold Palmer Hospital for Children', 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '33701', 'city': 'St. Petersburg', 'state': 'Florida', 'country': 'United States', 'facility': "All Children's Hospital Johns Hopkins Medicine", 'geoPoint': {'lat': 27.77086, 'lon': -82.67927}}, {'zip': '96813', 'city': 'Honolulu', 'state': 'Hawaii', 'country': 'United States', 'facility': 'Kapiolani Medical Center for Women and Children', 'geoPoint': {'lat': 21.30694, 'lon': -157.85833}}, {'zip': '02111', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Tufts Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '49503', 'city': 'Grand Rapids', 'state': 'Michigan', 'country': 'United States', 'facility': "Helen DeVos Children's Hospital", 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '55404', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': "Children's Hospital and Clinics on Minnesota", 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': "Children's Mercy Hospitals and Clinics", 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '63104', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': "Cardinal Glennon Children's Medical Center", 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'country': 'United States', 'facility': "The Children's Hospital at Montefiore", 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}, {'zip': '28204', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': "Levine Children's Hospital", 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Penn State Milton S. Hershey Medical Center and Children's Hospital", 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': "Monroe Carrell Jr. Children's Hospital at Vanderbilt", 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '78723', 'city': 'Austin', 'state': 'Texas', 'country': 'United States', 'facility': "Dell Children's Blood and Cancer Center", 'geoPoint': {'lat': 30.26715, 'lon': -97.74306}}, {'zip': '75235', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': "Children's Medical Center", 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Cancer and Hematology Centers", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': "Primary Children's Hospital", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}], 'overallOfficials': [{'name': 'Giselle Saulnier-Sholler, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Beat Childhood Cancer'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Giselle Sholler', 'class': 'OTHER'}, 'collaborators': [{'name': 'Beat NB Cancer Foundation', 'class': 'OTHER'}, {'name': 'Because of Ezra', 'class': 'OTHER'}, {'name': 'K C Pharmaceuticals Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Beat Childhood Cancer Chair', 'investigatorFullName': 'Giselle Sholler', 'investigatorAffiliation': 'Milton S. Hershey Medical Center'}}}}