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 transfusion in subjects with transfusion-dependent β-thalassemia.
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
Documented baseline, or pretransfusion, Hb level≤7 g/dL
Karnofsky performance status ≥70 for subjects≥16 years of age; Lansky performance status of ≥70 for subjects<16 years of age
Presence of clear contraindications for hematopoietic stem cell collection
Diagnosis of composite α thalassemia
A white blood cell (WBC) count <3×10^9/L, and/or platelet count <100×10^9/L not related to hypersplenism
Subjects 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
Single group
KL003 cell injection Drug Product
GeneticNo conditioning regimen for transfusion of auto-HSC transduced with lentiviral vector encoding βA-T87Q-globin gene
The number, frequency and severity of adverse events (AE) after reinfusion of KL003 drug products
The proportion of participants who meet the definition of transfusion independence (TI) for at least 6 months
TI is defined as Hb ≥ 90.0 g/L after reinfusion and without disease-related routine blood transfusion for 6 months
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Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Kanglin Biotech
Collaborator
