Ignite Creation Date:
2025-12-24 @ 1:38 PM
Ignite Modification Date:
2026-01-02 @ 2:18 AM
Study NCT ID:
NCT05860595
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-08-05
First Post:
2023-05-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'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'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 3}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-05-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2025-10-24', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-31', 'studyFirstSubmitDate': '2023-05-08', 'studyFirstSubmitQcDate': '2023-05-08', 'lastUpdatePostDateStruct': {'date': '2025-08-05', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-05-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-08-20', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Percentage of participants with successful lentiviral vector transduced CD34+ stem cell engraftment', 'timeFrame': 'Up to 42 days post transplant', 'description': 'Successful engraftment was defined as neutrophil count \\[ANC\\] ≥0.5×10\\^9/L for 3 consecutive days'}, {'measure': 'Engraftment time of neutrophil', 'timeFrame': 'Up to 42 days post transplant', 'description': 'The first day when neutrophils ≥ 0.5×10\\^9/L for 3 consecutive days'}, {'measure': 'Engraftment time of platelet', 'timeFrame': 'Up to 42 days post transplant', 'description': 'The first day of platelet count ≥ 20.0×10\\^9/L for 7 consecutive days after platelet transfusion independence'}, {'measure': 'Transplant-related mortality within 100 days and within 1 year after reinfusion of KL003 drug product', 'timeFrame': 'Up to 1 year post transplant'}, {'measure': 'The number, frequency and severity of adverse events (AE) within 1 year after reinfusion of KL003 drug products', 'timeFrame': 'Up to 24 months post transplant', 'description': 'Frequency and severity of AEs \\& SAEs identified according to NCI CTCAE 5.0'}], 'secondaryOutcomes': [{'measure': 'The proportion of participants who meet the definition of transfusion independence (TI) for at least 6 months', 'timeFrame': 'Up to 24 months post transplant', 'description': 'TI is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 6 months'}, {'measure': 'The duration of transfusion independence', 'timeFrame': 'Up to 24 months post transplant'}, {'measure': 'Changes in the frequency and volume of blood transfusion', 'timeFrame': 'Up to 24 months post transplant'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Beta-Thalassaemia', 'Hematopoietic Stem-Cell Transplantation'], 'conditions': ['Transfusion-dependent Beta-Thalassemia']}, 'descriptionModule': {'briefSummary': 'This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells in subjects with transfusion-dependent β-thalassemia.', 'detailedDescription': 'Subject participation for this study will be 24 months. Subjects who enroll in this study will be asked to participate in a subsequent 13-year follow-up for gene therapy products.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '35 Years', 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female age between 3-35 years\n* Diagnosis of transfusion-dependent β-thalassemia and a history of at least 100 mL/kg/year of pRBCs or ≥8 transfusions of pRBCs per year for the prior 2 years\n* Documented baseline, or pretransfusion, Hb level≤7 g/dL\n* Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects\\<16 years of age\n* Eligible to undergo auto-HSCT\n* Willing and able to follow the research procedures and conditions, with good compliance\n* Willing to receive at least the 2 years follow-up and maintain detailed medical records, including transfusion history\n* Subject and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form, and can complete all follow-up in accordance with the protocol requirements\n\nExclusion Criteria:\n\n* Subjects positive with the following etiological tests: human immunodeficiency virus(HIV-1-2), human cytomegalovirus (HCMV-DNA), EB virus (EBV-DNA), HBV (HBsAg/HBV-DNA positive), HCV antibody (HCV-Ab), Treponema pallidum antibody (TP-Ab)\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator\n* Contraindication to bone marrow collection\n* Any prior or current malignancy or myeloproliferative or significant immunodeficiency disorder\n* A white blood cell (WBC) count \\<3×10\\^9/L, and/or platelet count \\<100×10\\^9/L not related to hypersplenism\n* Diagnosis of composite α thalassemia\n* Participants with severe iron overload at the time of screening: severe iron overload of the liver showed by MRI, serum ferritin ≥ 5000 ng/mL, or moderate to severe iron overload of the heart\n* Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody\n* Meet the criteria for allo-HSCT and with an identified willing donor with a full HLA match\n* Prior receipt of gene therapy or allo-HSCT\n* Immediate family member (i.e. parent or siblings) with a known Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis)\n* Diagnosis of a significant psychiatric disorder of the subject that could seriously impede the ability to participate in the study\n* History of major organ damage including:\n\n 1. Liver function test suggest AST or ALT levels \\>3× upper limit of normal (ULN);\n 2. Total serum bilirubin value \\>2.5×ULN;if combined with Gilbert syndrome, total bilirubin \\>3×ULN and direct bilirubin value \\>2.5×ULN;\n 3. History of bridging fibrosis, cirrhosis;\n 4. Left ventricular ejection fraction \\<45%;\n 5. New York Heart Association (NYHA) class III or IV congestive heart failure;\n 6. Severe arrhythmia requiring medical treatment;\n 7. Uncontrolled hypertension or unstable angina pectoris;\n 8. Myocardial infarction or bypass or stent surgery within 12 months before drug administration;\n 9. Valvular disease with clinical significance;\n 10. Baseline calculated eGFR\\<60mL/min/1.73m2;\n 11. Pulmonary function: FEV1/FVC\\<60% and/or diffusion capacity of carbon monoxide (DLco) \\<60% of prediction;\n 12. Evidence of clinically significant pulmonary hypertension requiring medical intervention.\n* Uncorrectable coagulation dysfunction or history of severe bleeding disorder\n* Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician\n* Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.)\n* Participation in another clinical study with an investigational drug within 30 days of Screening or participating in another clinical study with an investigational drug\n* Inoculated live vaccine within 6 weeks prior to screening\n* Pregnancy or breastfeeding women; Subjects or their sexual partners were unable to take medically recognized effective contraceptive measures during the 27-month study period\n* The subjects or their parents would not comply with the study procedures outlined in the protocol\n* Receipt of hydroxyurea therapy within 3 months before HSCT harvest\n* Patients considered to be ineligible for the study by the investigator for reasons other than the above'}, 'identificationModule': {'nctId': 'NCT05860595', 'briefTitle': 'Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.', 'organization': {'class': 'OTHER', 'fullName': 'Institute of Hematology & Blood Diseases Hospital, China'}, 'officialTitle': 'Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.', 'orgStudyIdInfo': {'id': 'IIT2023021'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'KL003 cell injection Drug Product', 'description': 'Each recruited subject will accept KL003 Transplantation.', 'interventionNames': ['Genetic: KL003 cell injection Drug Product']}], 'interventions': [{'name': 'KL003 cell injection Drug Product', 'type': 'GENETIC', 'description': 'Transplant of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene.', 'armGroupLabels': ['KL003 cell injection Drug Product']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Tianjin', 'state': 'Tianjin Municipality', 'country': 'China', 'facility': 'Regenerative Medicine Center', 'geoPoint': {'lat': 39.14222, 'lon': 117.17667}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Institute of Hematology & Blood Diseases Hospital, China', 'class': 'OTHER'}, 'collaborators': [{'name': 'R&D Kanglin Biotech', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}