Ignite Creation Date:
2025-12-24 @ 11:45 PM
Ignite Modification Date:
2026-01-05 @ 1:04 PM
Study NCT ID:
NCT02327351
Status:
COMPLETED
Last Update Posted:
2021-02-26
First Post:
2014-12-18
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}], 'ancestors': [{'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Dmitriy.Balashov@fccho-moscow.ru', 'phone': '84956647078', 'title': 'Balashov Dmitriy, head of HSCT department', 'phoneExt': '6534', 'organization': 'National Research Center for Pediatric Hematology , Moscow, Russian Federation'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '1 year', 'eventGroups': [{'id': 'EG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion', 'otherNumAtRisk': 98, 'deathsNumAtRisk': 98, 'otherNumAffected': 0, 'seriousNumAtRisk': 98, 'deathsNumAffected': 13, 'seriousNumAffected': 17}], 'seriousEvents': [{'term': 'CMV pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 98, 'numAffected': 6}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'ADV hepatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 98, 'numAffected': 2}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Bacterial sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 98, 'numAffected': 7}], 'organSystem': 'Blood and lymphatic system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'TMA', 'stats': [{'groupId': 'EG000', 'numAtRisk': 98, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Overall Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'OG000'}, {'value': '75', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}]}], 'groups': [{'id': 'OG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}, {'id': 'OG001', 'title': 'Matched Unrelated Donor', 'description': 'type of HSCT donor - HLA-matched unrelated donor'}, {'id': 'OG002', 'title': 'HLA-mismatched Related Donor', 'description': 'type of HSCT donor - HLA-mismatched related'}], 'classes': [{'categories': [{'measurements': [{'value': '86', 'groupId': 'OG000', 'lowerLimit': '79', 'upperLimit': '94'}, {'value': '86', 'groupId': 'OG001', 'lowerLimit': '79', 'upperLimit': '94'}, {'value': '87', 'groupId': 'OG002', 'lowerLimit': '73', 'upperLimit': '100'}]}]}], 'analyses': [{'pValue': '0.95', 'groupIds': ['OG001', 'OG002'], 'paramType': 'survival distribution function', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '86', 'ciLowerLimit': '79', 'ciUpperLimit': '94', 'statisticalMethod': 'Gray test', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': '1 year after HSCT', 'description': 'The probability of overall survival estimated by the Kaplan-Meier method at 1 year after HSCT', 'unitOfMeasure': 'percentage of survival probability', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Transplant Related Mortality (TRM)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'classes': [{'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000', 'lowerLimit': '6', 'upperLimit': '21'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '24 months after transplantation', 'description': 'transplant-related mortality estimated with cumulative incidence curve, considering relapse as a competitive risk', 'unitOfMeasure': 'percentage of cumulative incidence', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Acute Graft Versus Host Diseases (аGVHD)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'classes': [{'categories': [{'measurements': [{'value': '17', 'groupId': 'OG000', 'lowerLimit': '1', 'upperLimit': '28'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '12 months after transplantation', 'description': 'incidence of aGVHD II-IV stage estimated with cumulative incidence curve, considering graft rejection and death as competitive risks', 'unitOfMeasure': 'percentage of cumulative incidence', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Chronic Graft Versus Host Diseases (cGVHD)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'classes': [{'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000', 'lowerLimit': '4', 'upperLimit': '19'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 year after HSCT', 'description': 'incidence of cGVHD estimated with cumulative incidence curve, considering graft rejection and death as competitive risks', 'unitOfMeasure': 'percentage of cumulative incidence', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Cellular Immunological Reconstitution', 'denoms': [{'units': 'Participants', 'counts': [{'value': '70', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'classes': [{'categories': [{'measurements': [{'value': '67', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '2 years after HSCT', 'description': 'Number of participants, who reached immune recovery - CD19+ lymphocytes subsets', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'alive at a time-point 2 years after HSCT'}, {'type': 'SECONDARY', 'title': 'Percentage of Patients With Full Donor Chimerism', 'denoms': [{'units': 'Participants', 'counts': [{'value': '72', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'classes': [{'categories': [{'measurements': [{'value': '48', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'last follow-up', 'description': 'Percentage of patients with full (more than 90%) donor chimerism among survivals', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'survivals at the last folow-up'}, {'type': 'SECONDARY', 'title': 'Viral Infections After Transplant', 'denoms': [{'units': 'Participants', 'counts': [{'value': '74', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Matched Unrelated Donor', 'description': 'donor type - HLA-matched unrelated donor'}, {'id': 'OG001', 'title': 'Mismatched Related Donors', 'description': 'donor type - HLA-mismatch relative'}], 'classes': [{'categories': [{'measurements': [{'value': '50', 'groupId': 'OG000', 'lowerLimit': '38', 'upperLimit': '65'}, {'value': '60', 'groupId': 'OG001', 'lowerLimit': '42', 'upperLimit': '86'}]}]}], 'analyses': [{'pValue': '0.35', 'groupIds': ['OG000', 'OG001'], 'statisticalMethod': 'Log Rank', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'NUMBER', 'timeFrame': '12 months after transplantation', 'description': 'number of patients with CMV reactivation (detection of any grade of CMV viremia after HSCT)', 'unitOfMeasure': 'percentage of cumulative incidence', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '98'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '98'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'TCR Alfa Beta Depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.\n\nBiological: TCR alfa beta T cell depletion'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'BG000'}]}], 'categories': [{'title': '<=18 years', 'measurements': [{'value': '98', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '4.2', 'spread': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '23', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '75', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Russia', 'denoms': [{'units': 'Participants', 'counts': [{'value': '98', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '98', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2012-06-15', 'size': 940368, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2021-02-09T06:20', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 98}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-02', 'completionDateStruct': {'date': '2018-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-02-09', 'studyFirstSubmitDate': '2014-12-18', 'resultsFirstSubmitDate': '2020-11-30', 'studyFirstSubmitQcDate': '2014-12-24', 'lastUpdatePostDateStruct': {'date': '2021-02-26', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2021-02-09', 'studyFirstPostDateStruct': {'date': '2014-12-30', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2021-02-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Overall Survival', 'timeFrame': '1 year after HSCT', 'description': 'The probability of overall survival estimated by the Kaplan-Meier method at 1 year after HSCT'}], 'secondaryOutcomes': [{'measure': 'Transplant Related Mortality (TRM)', 'timeFrame': '24 months after transplantation', 'description': 'transplant-related mortality estimated with cumulative incidence curve, considering relapse as a competitive risk'}, {'measure': 'Acute Graft Versus Host Diseases (аGVHD)', 'timeFrame': '12 months after transplantation', 'description': 'incidence of aGVHD II-IV stage estimated with cumulative incidence curve, considering graft rejection and death as competitive risks'}, {'measure': 'Chronic Graft Versus Host Diseases (cGVHD)', 'timeFrame': '1 year after HSCT', 'description': 'incidence of cGVHD estimated with cumulative incidence curve, considering graft rejection and death as competitive risks'}, {'measure': 'Cellular Immunological Reconstitution', 'timeFrame': '2 years after HSCT', 'description': 'Number of participants, who reached immune recovery - CD19+ lymphocytes subsets'}, {'measure': 'Percentage of Patients With Full Donor Chimerism', 'timeFrame': 'last follow-up', 'description': 'Percentage of patients with full (more than 90%) donor chimerism among survivals'}, {'measure': 'Viral Infections After Transplant', 'timeFrame': '12 months after transplantation', 'description': 'number of patients with CMV reactivation (detection of any grade of CMV viremia after HSCT)'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Primary Immune Deficiency Disorder', 'Hematopoietic Stem Cell Transplantation']}, 'referencesModule': {'references': [{'pmid': '26187864', 'type': 'DERIVED', 'citation': 'Balashov D, Shcherbina A, Maschan M, Trakhtman P, Skvortsova Y, Shelikhova L, Laberko A, Livshits A, Novichkova G, Maschan A. Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRalphabeta and CD19 Depletion in Children with Primary Immunodeficiency Syndromes. Biol Blood Marrow Transplant. 2015 Nov;21(11):1955-62. doi: 10.1016/j.bbmt.2015.07.008. Epub 2015 Jul 15.'}]}, 'descriptionModule': {'briefSummary': 'Treatment Study to assess of safety and efficiency of T cells receptor (TCR) alfa beta depleted graft for hematopoietic stem cell transplantation (HSCT) from haploidentical and unrelated donors in patients with primary immunodeficiency diseases', 'detailedDescription': 'Infections, graft versus host diseases (GVHD) and associated morbidity and mortality remains significant problems after unrelated and haploidentical hematopoietic stem sell transplantation (HSCT) in patients with primary immunodeficiency diseases (PID). In this study the hypothesis is that the transplantation of TCR alfa beta depleted peripheral blood stem cells (PBSC) would offers advantages over the use of positively selected CD34+ stem cells in haploidentical HSCT and non-manipulated graft in unrelated HSCT.\n\nThe purpose of this study is to evaluate the safety and efficiency of the selective infusion of TCR alfa beta T cell depleted graft in pediatric patients with PID receiving HSCT from haploidentical and unrelated donors.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '1 Month', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patients aged ≥ 1 months and \\< 19 years\n* Patients diagnosed with Primary Immunodeficiency Diseases eligible for an allogeneic transplantation and lacking a related HLA-matched donor\n* Lansky/Karnofsky score \\> 40, WHO \\> 4\n* Signed written informed consent\n\nExclusion Criteria:\n\n* Dysfunction of liver (ALT/AST \\> 5 times normal value, or bilirubin \\> 3 times normal value), or of renal function (creatinine clearance \\< 30 ml / min)\n* Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \\<40%)\n* Serious concurrent uncontrolled medical disorder\n* Pregnant or breast feeding female patient\n* Lack of parents' informed consent."}, 'identificationModule': {'nctId': 'NCT02327351', 'briefTitle': 'TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID', 'organization': {'class': 'OTHER', 'fullName': 'Federal Research Institute of Pediatric Hematology, Oncology and Immunology'}, 'officialTitle': 'Phase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency Diseases', 'orgStudyIdInfo': {'id': 'TCRabPID-2012'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'TCR alfa beta depletion', 'description': 'TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.', 'interventionNames': ['Other: Biological: TCR alfa beta T cell depletion']}], 'interventions': [{'name': 'Biological: TCR alfa beta T cell depletion', 'type': 'OTHER', 'armGroupLabels': ['TCR alfa beta depletion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '117997', 'city': 'Moscow', 'country': 'Russia', 'facility': 'Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}], 'overallOfficials': [{'name': 'Alexei Maschan, Professor', 'role': 'STUDY_CHAIR', 'affiliation': 'Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology'}, {'name': 'Dmitry Balashov, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Federal Research Institute of Pediatric Hematology, Oncology and Immunology', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}