A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.
- EligibilityCriteria: Inclusion Criteria:  Male or female age between 3-35 years; 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; Karnofsky performance status ≥70 for participants≥16 years of age; Lansky performance status of ≥70 for participants<16 years of age; Eligible to undergo auto-HSCT; Willing and able to follow the research procedures and conditions, with good compliance; Willing to receive at least the 2 years follow-up; Participant and/or legal guardians voluntarily participated in this clinical trial and signed the informed consent form.  Exclusion Criteria:  Diagnosis of composite α thalassemia; Prior receipt of gene therapy or allo-HSCT; Meet the criteria for allo-HSCT and with an identified willing donor with full HLA match; Participants with severe iron overload at the time of screening; Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody; Known allergy to clinical trial drug (plerixafor or G-CSF or busulfan) or ingredient(DMSO etc.); Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the clinical investigator; 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), Human T-lymphotropic virus antibody (HTLV-Ab), Treponema pallidum antibody (TP-Ab); Uncorrectable coagulation dysfunction or history of severe bleeding disorder;  History of major organ damage including:  Liver function test suggest AST or ALT levels >3× upper limit of normal（ULN）; Total serum bilirubin value>2.5×ULN;if combined with Gilbert syndrome, total bilirubin>3×ULN and direct bilirubin value>2.5×ULN; Left ventricular ejection fraction <45%; Baseline calculated eGFR<60mL/min/1.73m2; Pulmonary function:FEV1/FVC<60% and/or diffusion capacity of carbon monoxide (DLco) <60% of prediction; - HealthyVolunteers: No - Gender: All - MinimumAge: 3 Years - MaximumAge: 35 Years - StdAgeList: Child, Adult