Ignite Creation Date:
2025-12-25 @ 4:07 AM
Ignite Modification Date:
2025-12-26 @ 3:03 AM
Study NCT ID:
NCT04207320
Status:
TERMINATED
Last Update Posted:
2024-12-04
First Post:
2019-12-11
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], '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': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'jcunning@bsd.uchicago.edu', 'phone': '773-702-6808', 'title': 'Dr. John Cunningham', 'organization': 'University of Chicago'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '0 years', 'description': 'For this Phase I study, NCI grade 1-4 AEs were to start being collected for on-study research participants from the initiation of Hydroxyurea/Azathioprine. No subject reached this point of the protocol while on study before their withdrawal. Thus, no AEs occurred for any of the three participants.', 'eventGroups': [{'id': 'EG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety, as Measured by Incidence of Graft Failure, Grade III/IV Irreversible End Organ Toxicity, Grade III/IV aGvHD, or Death Within 100 Days Post-Hap-HSCT', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'OG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'timeFrame': '100 days post-Hap-HSCT', 'description': 'Graft Function: efficacy is defined as stable donor engraftment (\\>5% total nucleated cell DNA) and donor erythropoiesis that corrects the SCD hematologic phenotype (\\<50% HbS in the peripheral blood).\n\nOrgan Toxicity: grade III/IV irreversible end organ toxicity based on NCI grading\n\nGraft Versus Host disease: grade III/IV aGvHD or death within 100 days post- Hap-HSCT', 'reportingStatus': 'POSTED', 'populationDescription': 'No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.'}, {'type': 'SECONDARY', 'title': 'Estimate 1-year Overall and Event-free Survival After Hap-HSCT', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'OG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'timeFrame': '1 year post transplant', 'description': 'Proportion of patients at 1 year who have not died or had graft failure', 'reportingStatus': 'POSTED', 'populationDescription': 'No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.'}, {'type': 'SECONDARY', 'title': 'Observe the Incidence of Grades I Through IV Acute GvHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'OG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'timeFrame': '100 days post transplant', 'description': 'Proportion of subjects with grades I through IV acute GvHD', 'reportingStatus': 'POSTED', 'populationDescription': 'No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.'}, {'type': 'SECONDARY', 'title': 'Observe Incidence of Severe Acute GvHD as Defined by Grades III Through IV', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'OG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'timeFrame': '100 days post transplant', 'description': 'Proportion of subjects with grades III through IV acute GvHD', 'reportingStatus': 'POSTED', 'populationDescription': 'No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.'}, {'type': 'SECONDARY', 'title': 'Observe the Incidence of Grades I Through IV Chronic GvHD', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'OG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'timeFrame': '1 year post transplant', 'description': 'Proportion of subjects with grades I through IV chronic GvHD', 'reportingStatus': 'POSTED', 'populationDescription': 'No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.'}, {'type': 'SECONDARY', 'title': 'Observe Incidence of Severe Chronic GvHD as Defined by Grades III and IV', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'OG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'timeFrame': '1 year post transplant', 'description': 'Proportion of subjects with grades III through IV chronic GvHD', 'reportingStatus': 'POSTED', 'populationDescription': 'No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'FG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'BG001', 'title': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.\n\nαβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', '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': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '20', 'groupId': 'BG000', 'lowerLimit': '16', 'upperLimit': '22'}, {'value': '20', 'groupId': 'BG002', 'lowerLimit': '16', 'upperLimit': '22'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', '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': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', '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': '3', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', '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': '3', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Stage II never opened at our site'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-06-22', 'size': 929123, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2024-11-08T12:23', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'whyStopped': 'Study terminated by PI', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2020-04-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-11', 'completionDateStruct': {'date': '2023-05-22', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-11-08', 'studyFirstSubmitDate': '2019-12-11', 'resultsFirstSubmitDate': '2024-11-08', 'studyFirstSubmitQcDate': '2019-12-19', 'lastUpdatePostDateStruct': {'date': '2024-12-04', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2024-11-08', 'studyFirstPostDateStruct': {'date': '2019-12-20', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2024-12-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-05-22', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety, as Measured by Incidence of Graft Failure, Grade III/IV Irreversible End Organ Toxicity, Grade III/IV aGvHD, or Death Within 100 Days Post-Hap-HSCT', 'timeFrame': '100 days post-Hap-HSCT', 'description': 'Graft Function: efficacy is defined as stable donor engraftment (\\>5% total nucleated cell DNA) and donor erythropoiesis that corrects the SCD hematologic phenotype (\\<50% HbS in the peripheral blood).\n\nOrgan Toxicity: grade III/IV irreversible end organ toxicity based on NCI grading\n\nGraft Versus Host disease: grade III/IV aGvHD or death within 100 days post- Hap-HSCT'}], 'secondaryOutcomes': [{'measure': 'Estimate 1-year Overall and Event-free Survival After Hap-HSCT', 'timeFrame': '1 year post transplant', 'description': 'Proportion of patients at 1 year who have not died or had graft failure'}, {'measure': 'Observe the Incidence of Grades I Through IV Acute GvHD', 'timeFrame': '100 days post transplant', 'description': 'Proportion of subjects with grades I through IV acute GvHD'}, {'measure': 'Observe Incidence of Severe Acute GvHD as Defined by Grades III Through IV', 'timeFrame': '100 days post transplant', 'description': 'Proportion of subjects with grades III through IV acute GvHD'}, {'measure': 'Observe the Incidence of Grades I Through IV Chronic GvHD', 'timeFrame': '1 year post transplant', 'description': 'Proportion of subjects with grades I through IV chronic GvHD'}, {'measure': 'Observe Incidence of Severe Chronic GvHD as Defined by Grades III and IV', 'timeFrame': '1 year post transplant', 'description': 'Proportion of subjects with grades III through IV chronic GvHD'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': True}, 'conditionsModule': {'conditions': ['Sickle Cell Disease']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell depletion for children and adolescents with severe sickle cell disease (SCD).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '25 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Hemoglobin SS, SC, S-β0 Thalassemia, or SO-Arab Sickle Cell Disease\n* Between the ages of 2 and 25 years (Stage 1: 10-25 years; Stage II: 2-25 years)\n* Lack a fully matched family donor or fully matched unrelated donor register in the National Marrow Donor Program\n* Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype\n* SCD with Severe Phenotype, defined by the following criteria: Neurologic manifestations of sickle disease including cerebral vascular accident (CVA), transient ischemic event (TIA) or abnormal MRI findings suggestive of silent infarct; Two or more episodes of acute chest syndrome (ACS) requiring admission for transfusional or respiratory support including supplemental oxygen within \\[two years\\] of enrollment in study despite hydroxyurea therapy. Patients who cannot tolerate hydroxyurea and who experience multiple episodes of ACS will also be eligible; History of severe vaso-occlusive (VOC) disease requiring hospitalization and intravenous narcotics on 3 or more occasions per year over the two years prior to enrollment despite hydroxyurea therapy. Patients who cannot tolerate hydroxyurea and who experience multiple episodes of VOC will also be eligible; Other severe phenotype as evidenced by end organ dysfunction related to sickle cell disease.\n\nExclusion Criteria:\n\n* Karnofsky or Lansky score \\< 60%\n* Acute hepatitis or evidence of moderate or severe portal fibrosis on biopsy. (Biopsy will be obtained if patient has been on chronic transfusion therapy \\> 6 months or has a ferritin \\> 1000 ng/ml) or AST or ALT \\>5 times the upper limit of normal\n* Severe renal impairment (as evidenced by creatinine clearance of \\<50ml/minute glomerular filtration rate (GFR) \\< 50% predicted normal)\n* Cardiac function that demonstrates shortening fraction less than 26% by cardiac echocardiogram or pulmonary hypertension.\n* Pregnant Female.\n* Lactating female.\n* Pulmonary function with baseline O2 saturation \\<85% or Diffusing Capacity for Carbon Monoxide (DLCO) on pulmonary function testing (PFT) with a DLCO \\<40%.'}, 'identificationModule': {'nctId': 'NCT04207320', 'briefTitle': 'Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease', 'organization': {'class': 'OTHER', 'fullName': 'University of Chicago'}, 'officialTitle': 'Hematopoietic Stem Cell Transplantation for Patients With Severe Sickle Cell Disease Using Myeloablative Conditioning and αβ+ T-cell Depleted Hematopoietic Stem Cells From Partially Matched Familial Donors', 'orgStudyIdInfo': {'id': 'IRB19-0640'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Stage I', 'description': 'Stage I will include eligible subjects between the ages of 10-25 years.', 'interventionNames': ['Device: αβ+ T-cell depletion with Miltenyi CliniMACS system']}, {'type': 'EXPERIMENTAL', 'label': 'Stage II', 'description': 'Stage II will include eligible subjects between the ages of 2-25 years.', 'interventionNames': ['Device: αβ+ T-cell depletion with Miltenyi CliniMACS system']}], 'interventions': [{'name': 'αβ+ T-cell depletion with Miltenyi CliniMACS system', 'type': 'DEVICE', 'description': 'Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.', 'armGroupLabels': ['Stage I', 'Stage II']}]}, 'contactsLocationsModule': {'locations': [{'zip': '60637', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'The University of Chicago', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}], 'overallOfficials': [{'name': 'John Cunningham, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Chicago'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Chicago', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}