Ignite Creation Date:
2025-12-25 @ 2:15 AM
Ignite Modification Date:
2026-02-27 @ 11:18 AM
Study NCT ID:
NCT04088760
Status:
TERMINATED
Last Update Posted:
2023-09-08
First Post:
2019-09-12
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
TCRαβ+/CD19+ Depleted Allogeneic Hematopoietic Stem Cell Transplantation for Malignant and Non-malignant Disorders
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2023-07-10', 'type': 'ACTUAL'}}}}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Rebecca.Marsh@cchmc.org', 'phone': '(513) 803-1139', 'title': 'Rebecca Marsh, MD', 'organization': "Cincinnati Children's Hospital Medical Center"}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events were collected for the first week of the study. Serious Adverse Events and All-Cause Mortality were collected up to 1 year.', 'description': 'Only events that were determined to be attributable to study intervention noted after the first week were recorded and reported.', 'eventGroups': [{'id': 'EG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.', 'otherNumAtRisk': 15, 'deathsNumAtRisk': 15, 'otherNumAffected': 7, 'seriousNumAtRisk': 15, 'deathsNumAffected': 6, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Epistaxis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Anorexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Worsening pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Hypokalemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Bacteremia (strep mitis)', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}], 'seriousEvents': [{'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}, {'term': 'Pericardial effusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 15, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'CTCAE (5.0)'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence of Infusion-related Reactions', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}, {'units': 'infusions', 'counts': [{'value': '16', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '100 days', 'description': 'Number of patients who experienced infusion reactions including rash, fever, difficulty breathing, and blood pressure abnormalities at the time of infusion of stem cells.', 'unitOfMeasure': 'number of infusion reactions', 'reportingStatus': 'POSTED', 'typeUnitsAnalyzed': 'infusions', 'denomUnitsSelected': 'infusions'}, {'type': 'SECONDARY', 'title': 'Engraftment and Sustained Donor Chimerism', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'classes': [{'title': 'Neutrophil Engraftment', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'Sustained donor engraftment at 1 year', 'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '28 days and 1 year', 'description': 'Initial neutrophil engraftment prior to day +28 was determined by monitoring for neutrophil count recovery post-transplant and performing blood tests to confirm presence of donor cells. Sustained donor chimerism at 1 year post transplant was determined again by performing blood tests to confirm presence of donor cells.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Acute GVHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '14', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '100 days', 'description': 'Patients were monitored for symptoms of acute graft versus host disease including rash, diarrhea, and increased bilirubin using the Modified Glucksberg Criteria.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': '14 patients received 15 transplants.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Chronic GVHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'Patients were monitored for symptoms of chronic graft versus host disease using the NIH Consensus Criteria.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Five patients were evaluable for chronic graft versus host disease at 1 year time point.'}, {'type': 'SECONDARY', 'title': 'GVHD-free Survival', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'classes': [{'categories': [{'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': '1 year', 'description': 'GVHD-free survival was determined based on the presence or not of acute or chronic GVHD at 1 year.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}]}], 'dropWithdraws': [{'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '15', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'TCRαβ+/CD19+ Depleted HSCT', 'description': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT): The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '3', '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': '7', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Caucasian', 'measurements': [{'value': '7', 'groupId': 'BG000'}]}, {'title': 'Hispanic', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'African American', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-01-27', 'size': 396954, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2023-06-13T11:16', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 15}}, 'statusModule': {'whyStopped': 'Post-transplant cyclophosphamide and abatacept can be used as standard-of-care approaches to GVHD prevention in the HLA mismatched donor-recipient setting and are simpler to use since study enrollment is not required.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2019-09-16', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2023-08', 'completionDateStruct': {'date': '2021-09-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-08-16', 'studyFirstSubmitDate': '2019-09-12', 'resultsFirstSubmitDate': '2023-06-13', 'studyFirstSubmitQcDate': '2019-09-12', 'lastUpdatePostDateStruct': {'date': '2023-09-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2023-08-16', 'studyFirstPostDateStruct': {'date': '2019-09-13', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2023-09-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-05-14', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Infusion-related Reactions', 'timeFrame': '100 days', 'description': 'Number of patients who experienced infusion reactions including rash, fever, difficulty breathing, and blood pressure abnormalities at the time of infusion of stem cells.'}], 'secondaryOutcomes': [{'measure': 'Engraftment and Sustained Donor Chimerism', 'timeFrame': '28 days and 1 year', 'description': 'Initial neutrophil engraftment prior to day +28 was determined by monitoring for neutrophil count recovery post-transplant and performing blood tests to confirm presence of donor cells. Sustained donor chimerism at 1 year post transplant was determined again by performing blood tests to confirm presence of donor cells.'}, {'measure': 'Number of Participants With Acute GVHD', 'timeFrame': '100 days', 'description': 'Patients were monitored for symptoms of acute graft versus host disease including rash, diarrhea, and increased bilirubin using the Modified Glucksberg Criteria.'}, {'measure': 'Number of Participants With Chronic GVHD', 'timeFrame': '1 year', 'description': 'Patients were monitored for symptoms of chronic graft versus host disease using the NIH Consensus Criteria.'}, {'measure': 'GVHD-free Survival', 'timeFrame': '1 year', 'description': 'GVHD-free survival was determined based on the presence or not of acute or chronic GVHD at 1 year.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Allogeneic Hematopoietic Cell Transplantation']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to investigate the safety and efficacy of TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) for malignant and non-malignant disorders in children and adolescent/young adult patients using the CliniMACS® immunomagnetic selection device (Miltenyi Biotec).', 'detailedDescription': 'Acute graft versus host disease (GVHD) remains a significant cause of morbidity and mortality and is the biggest barrier to successful allogeneic hematopoietic cell transplantation (HSCT) outcomes. Improved methods of acute GVHD prevention are needed. TCRαβ+/CD19+ depletion of allogeneic hematopoietic stem cell products offers an opportunity to limit the risk of acute GVHD by removing TCRαβ+ T cells and CD19+ B cells which participate in acute GVHD initiation and perpetuation. The purpose of this study is to investigate the safety and efficacy of TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT) for malignant and non-malignant disorders in children and adolescent/young adult patients using the CliniMACS® immunomagnetic selection device (Miltenyi Biotec).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Any patient being treated at Cincinnati Children's Hospital requiring an allogeneic HSCT who lacks an HLA-genotypically matched related donor. Genotypically matched related donors are allowed when there is a clinical desire to avoid the use of GVHD prophylaxis medications.\n\nExclusion Criteria:\n\n* Prior allogeneic transplant with active acute or chronic GVHD, or life-threatening infection. Patients with a prior history of allogenic transplant without active GVHD or life-threatening infection can be considered."}, 'identificationModule': {'nctId': 'NCT04088760', 'briefTitle': 'TCRαβ+/CD19+ Depleted Allogeneic Hematopoietic Stem Cell Transplantation for Malignant and Non-malignant Disorders', 'organization': {'class': 'OTHER', 'fullName': "Children's Hospital Medical Center, Cincinnati"}, 'officialTitle': 'Safety and Efficacy of TCRαβ+/CD19+ Depleted Allogeneic Hematopoietic Stem Cell Transplantation for Malignant and Non-malignant Disorders in Children and Adolescent/Young Adult Patients', 'orgStudyIdInfo': {'id': '2018-8568'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'TCRαβ+/CD19+ depleted HSCT', 'interventionNames': ['Drug: TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT)']}], 'interventions': [{'name': 'TCRαβ+/CD19+ depleted allogeneic hematopoietic stem cell transplant (HSCT)', 'type': 'DRUG', 'description': 'The majority of TCRαβ+ T cells and CD19+ B cells will be removed from the allogeneic graft utilizing the CliniMACS® immunomagnetic selection device (Miltenyi Biotec). The depletion process involves two phases; cell labeling (phase 1) and the automated immunomagnetic depletion process (phase 2). The CD34+ dose may be adjusted by the need to not exceed the TCRαβ+CD3+ dose threshold.', 'armGroupLabels': ['TCRαβ+/CD19+ depleted HSCT']}]}, 'contactsLocationsModule': {'locations': [{'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Children's Hospital Medical Center, Cincinnati", 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}