β-Thalassemia Treatment With KL003 Cell Injection

Ki vehet részt

• 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

Ki nem vehet részt

• Prior receipt of gene therapy or allo-HSCT
• 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
• Presence of unusual antibody of red blood cell antigens or tested positive for platelet antibody
• 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
• Diagnosis of composite α thalassemia
• 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