This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
Age 3-35 years old (inclusive), male or female;
The subject and/or his/her legally recognized representative/parent/guardian fully understands the study and all information related to the study and has signed the informed consent form;
Clinical diagnosis of transfusion-dependent β-thalassemia (TDT) with a blood transfusion record within 2 years (inclusive) prior to screening showing a history of ≥ 10 units (U)/kg/year (or ≥ 100 mL/kg/year) or ≥ 8 times/year of suspended RBC transfusions in at least 1 consecutive 12-month period;
Karnofsky score (for subjects aged ≥ 16 years) or Lansky score (for subjects aged < 16 years) of ≥ 80;
Diagnosis of associated α-thalassemia: > 1 alpha chain deletion or alpha gene functional defect;
Have available HLA-fully matched donors and acceptable for allogeneic hematopoietic stem cell transplantation;
Irregular antibody or platelet antibody positive;
Prior allogeneic bone marrow transplantation or gene therapy;
Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Shanghai Vitalgen BioPharma Co., Ltd.
Collaborator