Ignite Creation Date:
2025-12-26 @ 11:10 AM
Ignite Modification Date:
2026-02-25 @ 9:47 PM
Study NCT ID:
NCT01590628
Status:
COMPLETED
Last Update Posted:
2022-05-20
First Post:
2012-04-15
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}, {'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'kelly@gamida-cell.com', 'phone': '026595631', 'title': 'Kelly Myers, CRA', 'organization': 'Gamida Cell'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).', 'eventGroups': [{'id': 'EG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.\n\nThe primary safety objective was to assess the acute toxicity associated with the infusion of omidubicel within 24 hours post-infusion.\n\nPart 1: The primary efficacy objective of Part 1 was to assess the cumulative incidence of donor-derived neutrophil engraftment by Day 42 following co-transplantation of omidubicel and unmanipulated cord blood grafts.', 'otherNumAtRisk': 13, 'deathsNumAtRisk': 13, 'otherNumAffected': 0, 'seriousNumAtRisk': 13, 'deathsNumAffected': 2, 'seriousNumAffected': 13}, {'id': 'EG001', 'title': 'NiCord', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.\n\nThe primary safety objective was to assess the acute toxicity associated with the infusion of omidubicel within 24 hours post-infusion.\n\nPart 2: The primary efficacy objective of Part 2 was to assess the cumulative incidence of donor-derived neutrophil engraftment by Day 42 following transplantation of single unit omidubicel.', 'otherNumAtRisk': 3, 'deathsNumAtRisk': 3, 'otherNumAffected': 0, 'seriousNumAtRisk': 3, 'deathsNumAffected': 0, 'seriousNumAffected': 3}], 'seriousEvents': [{'term': 'Anal fissure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Acute graft versus host disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Anaphylactic reaction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Graft versus host disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Transplant rejection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Gastroenteritis adenovirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Pseudomonal bacteraemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Subdural haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hyperglycaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Osteonecrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Posterior reversible encephalopathy syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Seizure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hypovolaemic shock', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Venous thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Renal Failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 13, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 3, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 23.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'Participants who received NiCord together with an unmanipulated CBU.'}, {'id': 'OG001', 'title': 'NiCord', 'description': 'Participants who received NiCord as a standalone graft.'}], 'classes': [{'title': 'Grade 4-5 acute toxicity event', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Grade 3 acute toxicity event', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 hours post-infusion', 'description': 'Assessment of acute toxicity associated with the infusion of NiCord within 24 hours post-infusion.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Assessment of Cumulative Incidence of Donor-derived Neutrophil Engraftment.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 1 subjects, together with an unmanipulated CBU.'}, {'id': 'OG001', 'title': 'NiCord as a Standalone Graft', 'description': 'NiCord was transplanted as a stand-alone graft in subjects that participated in Part 2.'}], 'classes': [{'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'By Day 42', 'description': 'Neutrophil engraftment is defined as achieving an absolute neutrophil count (ANC) of ≥500/ μL for 3 consecutive measurements on different days by Day 42 inclusive (the day of engraftment was defined as the first of these 3 days).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Proportion of Transplant-related Mortality.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.\n\nThe NiCord was transplanted together with an unmanipulated CBU graft.'}, {'id': 'OG001', 'title': 'NiCord', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.\n\nThe NiCord was transplanted as a standalone product.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'at 100 days', 'description': 'Transplant-related mortality is defined as death not preceded by autologous recovery.', 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Event-free Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 1 subjects, together with an unmanipulated CBU.'}, {'id': 'OG001', 'title': 'NiCord as a Standalone Graft', 'description': 'NiCord was transplanted as a stand-alone graft in subjects that participated in Part 2.'}], 'classes': [{'categories': [{'measurements': [{'value': '92.3', 'groupId': 'OG000', 'lowerLimit': '56.6', 'upperLimit': '98.9'}, {'value': '66.6', 'comment': 'No statistical analysis was performed for this outcome as this number of patients is too small for analysis.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 days post-transplant', 'description': 'Patients with event-free survival at 100 days post-transplant that did not have one of the following events: death, autologous recovery, primary or secondary graft failure.', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'In the first group, 13 patients received NiCord together with a second unmanipulated CBU. In the 2nd group, 3 patients received NiCord as a standalone graft. No statistical analysis was performed for this outcome as this number of patients is too small for analysis.'}, {'type': 'SECONDARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 1 subjects, together with an unmanipulated CBU.'}, {'id': 'OG001', 'title': 'NiCord as a Standalone Graft', 'description': 'NiCord was transplanted as a stand-alone graft in subjects that participated in Part 2.'}], 'classes': [{'categories': [{'measurements': [{'value': '92.3', 'groupId': 'OG000', 'lowerLimit': '56.6', 'upperLimit': '98.9'}, {'value': '100', 'comment': 'Due to the insufficient number of patients who received a standalone NiCord transplant (3 in total), no statistical anlysis was performed.', 'groupId': 'OG001', 'lowerLimit': 'NA', 'upperLimit': 'NA'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '180 days', 'description': 'Overall survival at 180 days', 'unitOfMeasure': 'percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'In the first group, 13 patients received NiCord together with a second unmanipulated CBU. In the 2nd group, 3 patients received NiCord as a standalone graft.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 1 subjects, together with an unmanipulated CBU.'}, {'id': 'FG001', 'title': 'NiCord', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 2 subjects as a standalone graft.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Death', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'The study enrolled and treated 16 patients in total. 13 patients were treated with omidubicel and an unmanipulated CBU and these served as the primary analysis population for all study endpoints. Three additional patients who were exposed to omidubicel as a single graft source, were included in the safety summaries in order to encompass the entire population exposed to omidubicel.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '13', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'NiCord + Unmanipulated CBU', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.\n\nThe NiCord was transplanted together with an unmanipulated CBU graft.'}, {'id': 'BG001', 'title': 'NiCord', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.\n\nThe NiCord was transplanted as a standalone product.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', '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': '13', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sickle Cell or Thalassemia Disease Type', 'classes': [{'categories': [{'title': 'Hb SS', 'measurements': [{'value': '13', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}, {'title': 'Hb SC', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Hb SBeta0 Thal', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Hb SD', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Hb SOarab', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Other SCD Type', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Beta Thal Major', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Alpha Thal Major', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'CMV Screen (IgG or Total) (Segment S) positive', 'classes': [{'categories': [{'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Performance Status', 'classes': [{'categories': [{'title': '100', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}, {'title': '90', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': '80', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}, {'title': '70', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'The Karnofsky Performance Scale Index allows patients to be classified as to their functional impairment on a scale of 0 to 100%. This can be used to compare effectiveness of different therapies and to assess the prognosis in individual patients. The lower the Karnofsky score, the worse the survival for most serious illnesses.', 'unitOfMeasure': 'Participants'}, {'title': 'Disease characteristics', 'classes': [{'title': 'Three or More Recurrent Painful Events in the Past 2 Years', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}]}, {'title': 'Acute Chest Syndrome (Two or More Episodes in the Past 2 Years)', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'SCD-related & clinically significant neurologic event', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}, {'title': 'Abnormal MRI/MRA', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'chronic PRBC transfusion therapy', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}, {'title': 'Patient has undergone TCD velocity tests', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Hydroxyurea use prior to enrollment', 'classes': [{'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-12-13', 'size': 1111615, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2021-05-19T08:21', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 16}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-04', 'completionDateStruct': {'date': '2019-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-04-27', 'studyFirstSubmitDate': '2012-04-15', 'resultsFirstSubmitDate': '2021-05-19', 'studyFirstSubmitQcDate': '2012-05-02', 'lastUpdatePostDateStruct': {'date': '2022-05-20', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2022-03-31', 'studyFirstPostDateStruct': {'date': '2012-05-03', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2022-04-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity.', 'timeFrame': '24 hours post-infusion', 'description': 'Assessment of acute toxicity associated with the infusion of NiCord within 24 hours post-infusion.'}, {'measure': 'Assessment of Cumulative Incidence of Donor-derived Neutrophil Engraftment.', 'timeFrame': 'By Day 42', 'description': 'Neutrophil engraftment is defined as achieving an absolute neutrophil count (ANC) of ≥500/ μL for 3 consecutive measurements on different days by Day 42 inclusive (the day of engraftment was defined as the first of these 3 days).'}], 'secondaryOutcomes': [{'measure': 'Proportion of Transplant-related Mortality.', 'timeFrame': 'at 100 days', 'description': 'Transplant-related mortality is defined as death not preceded by autologous recovery.'}, {'measure': 'Event-free Survival', 'timeFrame': '100 days post-transplant', 'description': 'Patients with event-free survival at 100 days post-transplant that did not have one of the following events: death, autologous recovery, primary or secondary graft failure.'}, {'measure': 'Overall Survival', 'timeFrame': '180 days', 'description': 'Overall survival at 180 days'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Sickle Cell Disease & Thalassemia', 'Genetic disorder'], 'conditions': ['Sickle Cell Disease & Thalassemia']}, 'referencesModule': {'references': [{'pmid': '33560399', 'type': 'DERIVED', 'citation': 'Parikh S, Brochstein JA, Galamidi E, Schwarzbach A, Kurtzberg J. Allogeneic stem cell transplantation with omidubicel in sickle cell disease. Blood Adv. 2021 Feb 9;5(3):843-852. doi: 10.1182/bloodadvances.2020003248.'}], 'seeAlsoLinks': [{'url': 'http://www.dukehealth.org/', 'label': 'Duke University Medical Center'}, {'url': 'http://www.gamida-cell.com', 'label': 'Gamida Cell Ltd.'}]}, 'descriptionModule': {'briefSummary': 'Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies', 'detailedDescription': 'Umbilical cord blood (UCB) is an alternative stem cell source for hematopoietic stem cell transplantations (HSCT) and can be used for the treatment of various life-threatening diseases, such as hematological malignancies or genetic blood disorders, in such cases where a matched related stem cell donor is not available. However, the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit (CBU), which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality. To improve outcomes and extend applicability of UCB transplantation, one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells. NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells. NiCord® is based on a novel technology for the ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable the broader application of UCB transplantation, and improve the clinical outcomes of UCB transplantation.\n\nIn Part 1 of this study, NiCord® will be administered to the patient in conjunction with a second, unmanipulated CBU. In Part 2 of this study, NiCord® will be administered to the patient without a second, unmanipulated CBU. The study duration per patient is approximately 270 days from signing of informed consent to last visit on day 180 post-transplant.\n\nThe overall study objectives of part 1 of this study are to evaluate the safety and efficacy of co-transplantation of NiCord® and an unmanipulated CBU in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy. The overall study objectives of part 2 of this study are to evaluate the safety and efficacy of transplantation of NiCord® in patients with Hemoglobinopathies (Sickle Cell Disease (SCD), or thalassemia major) following myeloablative therapy.\n\nThe study hypothesis for part 1 of this study is that the co-transplantation of NiCord® and an unmanipulated unrelated cord blood graft in patients with hemoglobinopathies (SCD, or thalassemia major) following myeloablative preparative therapy will be safe and will enable cord blood engraftment. The study hypothesis for part 2 of this study is that transplantation of NiCord® in patients with hemoglobinopathies (SCD, or thalassemia major) following myeloablative preparative therapy will be safe and will enable cord blood engraftment.\n\nUp to fifteen (15) evaluable patients recruited for part 1 of the study and up to five (5) patients for part 2 of the study should be 2-45 years of age, at least 10 kg in weight, have symptomatic SCD or thalassemia major and should be considered as candidates for allogeneic myeloablative HSCT for the treatment of SCD.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '45 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Must be 2 - 45 years of age and at least 10 kg\n* Must have clinically severe SCD (SS, SC or SBeta0 Thal) or thalassemia major and be eligible for myeloablative SCT\n* Must have two partially HLA-matched CBUs for part 1; and one partially HLA-matched CBU for part 2\n* Back-up autologous stem cells harvested from bone marrow\n* Adequate Karnofsky Performance score or Lansky Play-Performance scale\n* Sufficient physiological reserves\n* Signed written informed consent\n\nExclusion Criteria:\n\n* HLA-matched related donor able to donate\n* Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors\n* Prior allogeneic hematopoietic SCT within the last 12 months or reduced-intensity transplant within the past 6 months\n* Human immunodeficiency virus (HIV) infection\n* Active or uncontrolled infection\n* Pregnancy or lactation'}, 'identificationModule': {'nctId': 'NCT01590628', 'briefTitle': 'Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies', 'organization': {'class': 'INDUSTRY', 'fullName': 'Gamida Cell ltd'}, 'officialTitle': 'Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies', 'orgStudyIdInfo': {'id': 'GC P#02.01.020'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'NiCord', 'description': 'NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.', 'interventionNames': ['Drug: NiCord']}], 'interventions': [{'name': 'NiCord', 'type': 'DRUG', 'description': 'NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells', 'armGroupLabels': ['NiCord']}]}, 'contactsLocationsModule': {'locations': [{'zip': '11040', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': "Steven & Alexandra Cohen Children's Medical Center, New York", 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '77030-4009', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University Of Texas M. D. Anderson Cancer Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'overallOfficials': [{'name': 'Joanne Kurtzberg, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Duke University Medical Center, NC, USA'}, {'name': 'Joel Brochstein, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Steven & Alexandra Cohen Children's Medical Center, New York"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Gamida Cell ltd', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}