Ignite Creation Date:
2025-12-24 @ 9:31 PM
Ignite Modification Date:
2025-12-25 @ 7:16 PM
Study NCT ID:
NCT05745532
Status:
RECRUITING
Last Update Posted:
2024-11-29
First Post:
2023-02-16
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-12-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-11', 'completionDateStruct': {'date': '2025-12-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-11-26', 'studyFirstSubmitDate': '2023-02-16', 'studyFirstSubmitQcDate': '2023-02-24', 'lastUpdatePostDateStruct': {'date': '2024-11-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-02-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-05-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)', 'timeFrame': '0-100 days', 'description': 'The number and the percentage of adverse events related to transplantation in 100 days will be summarized according to NCI CTCAE 5.0'}, {'measure': 'Overall survival', 'timeFrame': '0-24 months', 'description': 'Number of patients alive through the whole trial will be record'}, {'measure': 'Proportion of engraftment', 'timeFrame': '0-24 months', 'description': 'Neutrophil count \\[ANC\\] \\>=500 /mm3 for 3 consecutive days and platelet count \\[PLT\\] \\>20,000/mm3 for7 consecutive days'}, {'measure': 'Replication competent lentivirus (RCL)', 'timeFrame': '0-24 months', 'description': 'The percentage of RCL should be negative in the 24 months after transplant'}, {'measure': 'Dynamics of viral integration sites (VIS)', 'timeFrame': '0-24 months', 'description': 'Evaluation of the percentage of participants without abnormal clonal proliferation and polyclonal engraftment . More than 1000 VIS retrieved from peripheral blood should be checked.'}], 'secondaryOutcomes': [{'measure': 'The average Insertion copy number (VCN) in peripheral blood mononuclear cells', 'timeFrame': '18-24 Months', 'description': 'The average insertion copy number (VCN) should be ≥0.1 in peripheral blood mononuclear cells'}, {'measure': 'The expression level of exogenous adult hemoglobin', 'timeFrame': '18-24 Months', 'description': 'Exogenous adult hemoglobin will be evaluated by globin chains and hemoglobin synthesis on peripheral blood by HPLC and the exogenous adult hemoglobin level is ≥2.0g/dL'}, {'measure': 'Change from baseline in annualized frequency of packed RBC transfusions', 'timeFrame': '18-24 Months', 'description': 'Compare the annualized number of pRBC transfusions before gene therapy with the Month 18 and Month 24 period after transplant, the percentage change will be recorded'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['β-thalassemia']}, 'descriptionModule': {'briefSummary': 'This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients', 'detailedDescription': 'We will recruit ß-thalassaemia major patients and collect their autologous hematopoietic stem cells, which will be modified with the LentiHBBT87Q system to restore β-globin expression. After conditioning, the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '16 Years', 'minimumAge': '8 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* 8-16 years old. Subject and/or subject's legal guardian fully understand and voluntarily sign informed consent;\n* Clinically diagnosed as transfusion-dependent β-thalassemia major;\n* With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;\n* Follow the arrangements for treatment and regular medical checks within two years post-transplantation\n\nExclusion Criteria:\n\n* The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;\n* Received gene therapy and allogeneic HSCT in the past.\n* Have an available HLA matched donor.\n* Enrolling in another clinical trial.\n* Other unsuitable conditions identified by doctors."}, 'identificationModule': {'nctId': 'NCT05745532', 'briefTitle': 'Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients', 'nctIdAliases': ['NCT04592458'], 'organization': {'class': 'INDUSTRY', 'fullName': 'Shenzhen Hemogen'}, 'officialTitle': 'Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients', 'orgStudyIdInfo': {'id': 'SZ-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental', 'description': 'Ten transfusion-dependent β-thalassaemia subjects aged 8-16 years will be reinfused with β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q.', 'interventionNames': ['Biological: β-globin restored autologous hematopoietic stem cells']}], 'interventions': [{'name': 'β-globin restored autologous hematopoietic stem cells', 'type': 'BIOLOGICAL', 'description': 'β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q', 'armGroupLabels': ['Experimental']}]}, 'contactsLocationsModule': {'locations': [{'zip': '518083', 'city': 'Shenzhen', 'state': 'Guangdong', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Haigang Sun', 'role': 'CONTACT', 'email': 'sunhaigang@genomics.cn', 'phone': '13823168465'}], 'facility': "Shenzhen Children's Hospital", 'geoPoint': {'lat': 22.54554, 'lon': 114.0683}}], 'centralContacts': [{'name': 'Haigang Sun', 'role': 'CONTACT', 'email': 'sunhaigang@genomics.cn', 'phone': '13823168465'}], 'overallOfficials': [{'name': 'Chao Liu, PHD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Shenzhen Hemogen'}, {'name': 'Sixi Liu, Professor', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Shenzhen Children's Hospital"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shenzhen Hemogen', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}