Ignite Creation Date:
2025-12-24 @ 7:09 PM
Ignite Modification Date:
2025-12-31 @ 1:02 AM
Study NCT ID:
NCT01221857
Status:
COMPLETED
Last Update Posted:
2021-08-03
First Post:
2010-10-14
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D054198', 'term': 'Precursor Cell Lymphoblastic Leukemia-Lymphoma'}, {'id': 'D015470', 'term': 'Leukemia, Myeloid, Acute'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D008228', 'term': 'Lymphoma, Non-Hodgkin'}, {'id': 'D006689', 'term': 'Hodgkin Disease'}, {'id': 'D019337', 'term': 'Hematologic Neoplasms'}], 'ancestors': [{'id': 'D007945', 'term': 'Leukemia, Lymphoid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008232', 'term': 'Lymphoproliferative Disorders'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D007160', 'term': 'Immunoproliferative Disorders'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D008223', 'term': 'Lymphoma'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@gamida-cell.com', 'phone': '+972-2-659-5631', 'title': 'Kelly Myers', 'organization': 'Gamida Cell'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'eventGroups': [{'id': 'EG000', 'title': 'NiCord', 'description': 'NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.', 'otherNumAtRisk': 12, 'deathsNumAtRisk': 12, 'otherNumAffected': 11, 'seriousNumAtRisk': 12, 'deathsNumAffected': 3, 'seriousNumAffected': 8}], 'otherEvents': [{'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 7, 'numAffected': 6}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypomagnesemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'aGvHD', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 10, 'numAffected': 7}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'CMV reactivation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'HHV-6 Reactivation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 6, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Staph Coagulase Infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypocalcemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'BK virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Elevated LFTs', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'E coli infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Sinus bradycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Arrhythmia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'RSV infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Pseudomonas infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Enterococcus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'CMV gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypoalbuminemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypoxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Dyspnea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'seriousEvents': [{'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Gastrointestinal Disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Altered mental status', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Graft rejection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Supraventricular tachycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Hematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Acute renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Disease relapse', 'stats': [{'groupId': 'EG000', 'numAtRisk': 12, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Acute Toxicity Associated With the Infusion of NiCord', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'NiCord', 'description': 'Analysis population is patients transplanted with NiCord plus unmanipulated cord blood unit.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '180 days post-transplant', 'description': 'Acute toxicity associated with the infusion of NiCord will be measured by adverse events within 24 hours post-infusion, defined as the acute toxicity period. Known adverse events associated with myeloablation and cord blood transplant were specifically monitored including fever, chills, allergic reaction/hypersensitivity, anaphylaxis, sinus bradycardia, sinus tachycardia, hypertension, hypotension, nausea, vomiting, diarrhea, dyspnea, hypoxia, hemoglobinuria, infection, flank pain and any other skin, CNS, cardiac, pulmonary or other toxicity manifestations.', 'calculatePct': False, 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients transplanted with NiCord'}, {'type': 'PRIMARY', 'title': 'Proportion of Patients With Neutrophil Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Neutrophil Engraftment', 'description': 'Patients transplanted with NiCord plus unmanipulated cord blood unit.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.91', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '42 days', 'description': 'Neutrophil engraftment was defined as achieving an Absolute Neutrophil Count (ANC) of ≥500 mm3 for 3 consecutive measurements on different days by day 42 inclusive (the day of engraftment was defined as the first of these 3 days). The ANC recovery must be of donor origin documented by peripheral blood chimerism assays indicating less than or equal to 10% host cells in peripheral blood.', 'unitOfMeasure': 'proportion of patients', 'reportingStatus': 'POSTED', 'populationDescription': 'Patients transplanted with NiCord'}, {'type': 'SECONDARY', 'title': 'Proportion of Patients Who Developed Acute GvHD Grade II-IV and III-IV', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Grade II-IV GvHD', 'description': 'All patients transplanted were assessed for acute GvHD grade II-IV.'}, {'id': 'OG001', 'title': 'Grade III-IV GvHD', 'description': 'All patients transplanted were assessed for acute GvHD grade III-IV.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.45', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '180 days', 'description': 'Acute GvHD was assessed from transplantation (day 0) until day 99 post-transplant or more frequently as clinically indicated. GvHD was classified according to the Glucksberg Classification (Glucksberg, Storb et al. 1974).\n\nThe overall grade of GvHD, however, was determined by an assessment of skin disease, liver disease and gastrointestinal manifestations.', 'unitOfMeasure': 'proportion of patients', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Non-relapse Mortality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Non-relapse Mortality', 'description': 'proportion of patients with non-relapse mortality at 100 days'}], 'classes': [{'categories': [{'measurements': [{'value': '0.09', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 days', 'description': 'Proportion of patients who had non-relapse mortality at 100 days.', 'unitOfMeasure': 'proportion of patients', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'NiCord', 'description': 'NiCord: NiCord is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'COMPLETED', 'comment': '1 patient received unmanipulated cord only.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': '12 patients were enrolled/transplanted; efficacy results were summarized for 11 patients treated with NiCord. 1 patient was transplanted with unmanipulated cord only. Safety results were summarized for all patients.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'NiCord', 'description': 'NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '45', 'groupId': 'BG000', 'lowerLimit': '21', 'upperLimit': '61'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-08', 'completionDateStruct': {'date': '2013-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-08-01', 'studyFirstSubmitDate': '2010-10-14', 'resultsFirstSubmitDate': '2017-05-24', 'studyFirstSubmitQcDate': '2010-10-14', 'lastUpdatePostDateStruct': {'date': '2021-08-03', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-02-03', 'studyFirstPostDateStruct': {'date': '2010-10-15', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-02-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2012-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Acute Toxicity Associated With the Infusion of NiCord', 'timeFrame': '180 days post-transplant', 'description': 'Acute toxicity associated with the infusion of NiCord will be measured by adverse events within 24 hours post-infusion, defined as the acute toxicity period. Known adverse events associated with myeloablation and cord blood transplant were specifically monitored including fever, chills, allergic reaction/hypersensitivity, anaphylaxis, sinus bradycardia, sinus tachycardia, hypertension, hypotension, nausea, vomiting, diarrhea, dyspnea, hypoxia, hemoglobinuria, infection, flank pain and any other skin, CNS, cardiac, pulmonary or other toxicity manifestations.'}, {'measure': 'Proportion of Patients With Neutrophil Engraftment', 'timeFrame': '42 days', 'description': 'Neutrophil engraftment was defined as achieving an Absolute Neutrophil Count (ANC) of ≥500 mm3 for 3 consecutive measurements on different days by day 42 inclusive (the day of engraftment was defined as the first of these 3 days). The ANC recovery must be of donor origin documented by peripheral blood chimerism assays indicating less than or equal to 10% host cells in peripheral blood.'}], 'secondaryOutcomes': [{'measure': 'Proportion of Patients Who Developed Acute GvHD Grade II-IV and III-IV', 'timeFrame': '180 days', 'description': 'Acute GvHD was assessed from transplantation (day 0) until day 99 post-transplant or more frequently as clinically indicated. GvHD was classified according to the Glucksberg Classification (Glucksberg, Storb et al. 1974).\n\nThe overall grade of GvHD, however, was determined by an assessment of skin disease, liver disease and gastrointestinal manifestations.'}, {'measure': 'Non-relapse Mortality', 'timeFrame': '100 days', 'description': 'Proportion of patients who had non-relapse mortality at 100 days.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Double Umbilical Cord Blood Stem Cell Transplantation', 'Hematological Malignancies', 'HLA Mismatched Donors', 'Cord Blood Transplantation'], 'conditions': ['Acute Lymphoblastic Leukemia (ALL)', 'Acute Myelogenous Leukemia (AML)', 'Myelodysplastic Syndrome (MDS)', "Non-Hodgkin's Lymphoma", "Hodgkin's Disease"]}, 'referencesModule': {'references': [{'pmid': '24911148', 'type': 'DERIVED', 'citation': 'Horwitz ME, Chao NJ, Rizzieri DA, Long GD, Sullivan KM, Gasparetto C, Chute JP, Morris A, McDonald C, Waters-Pick B, Stiff P, Wease S, Peled A, Snyder D, Cohen EG, Shoham H, Landau E, Friend E, Peleg I, Aschengrau D, Yackoubov D, Kurtzberg J, Peled T. Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment. J Clin Invest. 2014 Jul;124(7):3121-8. doi: 10.1172/JCI74556. Epub 2014 Jun 9.'}, {'pmid': '22198152', 'type': 'DERIVED', 'citation': 'Peled T, Shoham H, Aschengrau D, Yackoubov D, Frei G, Rosenheimer G N, Lerrer B, Cohen HY, Nagler A, Fibach E, Peled A. Nicotinamide, a SIRT1 inhibitor, inhibits differentiation and facilitates expansion of hematopoietic progenitor cells with enhanced bone marrow homing and engraftment. Exp Hematol. 2012 Apr;40(4):342-55.e1. doi: 10.1016/j.exphem.2011.12.005. Epub 2011 Dec 20.'}], 'seeAlsoLinks': [{'url': 'http://www.gamida-cell.com', 'label': 'Gamida Cell Ltd.'}, {'url': 'http://www.dukehealth.org', 'label': 'Duke University Medical Center'}, {'url': 'http://www.loyolamedicine.org', 'label': 'Loyola University'}]}, 'descriptionModule': {'briefSummary': 'Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second, Unmanipulated CBU in Patients with Hematological Malignancies', 'detailedDescription': 'Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative procedure for various hematological malignancies, bone marrow failure syndromes and inherited metabolic disorders. The application of allogeneic HSCT is limited by donor availability such that only approximately one-third of the otherwise appropriate candidates have suitably matched family donors. Alternative donors include mismatched family members or matched unrelated donors, but these approaches are often complicated by an increased risk of graft-versus-host disease (GvHD) and a prolonged and cumbersome search and procurement process. In addition, far fewer subjects of racial minorities find suitable human leukocyte antigen (HLA)-matched donors.\n\nUmbilical cord blood has been increasingly used as an alternative source of stem cells and has extended the availability of allogeneic HSCT to patients who would otherwise not be eligible for this curative approach. In the last decade the number of cord blood transplantations from related and unrelated donors has increased dramatically. It is estimated that more than 20,000 patients have undergone cord blood transplantation from unrelated donors to date for a variety of genetic, hematological, immunological, metabolic and oncologic disorders. The major advantages of cord blood transplantation include easy procurement, no risk to donors, reduced incidence of transmitting infections, immediate availability, and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch. Nevertheless, the low cell dose remains a main limitation of this cell source leading to delayed hematopoietic reconstitution, higher risk of graft failure and relatively high treatment related mortality rates as compared to other hematopoeitic cell sources. To improve outcomes and extend applicability of cord blood transplantation, one potential solution is ex vivo expansion of cord blood-derived stem and progenitor cells.\n\nThe Sponsor has undertaken to develop NiCord®, which is based on a novel technology for 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 broader application of umbilical cord blood transplantation and improve clinical outcomes in subjects with high-risk hematological malignancies.\n\nThe main objective of the current study is to evaluate the safety of co-transplantation of NiCord® and an unmanipulated cord blood unit in patients with hematological malignancies following myeloablative therapy.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '8 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Applicable disease and eligible for myeloablative SCT\n* Patients must have two partially HLA-matched CBUs\n* Back-up stem cell source\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* Prior allogeneic HSCT\n* Lymphoma patients with progressive disease\n* Other active malignancy\n* Human immunodeficiency virus (HIV) infection\n* Active or uncontrolled infection\n* Active/symptoms of central nervous system (CNS) disease\n* Pregnancy or lactation'}, 'identificationModule': {'nctId': 'NCT01221857', 'briefTitle': 'Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies', '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 Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies', 'orgStudyIdInfo': {'id': 'GC P#01.01.020'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'NiCord', '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': '60153', 'city': 'Maywood', 'state': 'Illinois', 'country': 'United States', 'facility': 'Loyola University, Cardinal Bernardin Cancer Center', 'geoPoint': {'lat': 41.8792, 'lon': -87.84312}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}], 'overallOfficials': [{'name': 'David Snyder, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Gamida Cell ltd'}, {'name': 'Joanne Kurtzberg, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Duke University'}, {'name': 'Mitchell Horwitz, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Duke University'}, {'name': 'Patrick Stiff, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Loyola University'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Gamida Cell ltd', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}