This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
2
Recruited participants will receive ET-01 IV infusion after conditioning.
PLA 923 Hospital
Nanning, Guangxi, China
Frequency and severity of AEs & SAEs identified according to NCI CTCAE 5.0.
Time frame: From ET-01 infusion to 104 weeks post-transplant
All-cause mortality.
Time frame: From signing of informed consent to 104 weeks post-transplant
Incidence of transplant-related mortality.
Time frame: within 100 days post-transplant
Proportion of subjects with engraftment.
Time frame: up to 42 days post-transplant
Change in total hemoglobin from baseline.
Time frame: within 104 weeks post-transplant
Change of HbF from baseline.
Time frame: within 104 weeks post-transplant
Change of proportion of HbF/Hb.
Time frame: within 104 weeks post-transplant
Change of frequency of packed RBC transfusions.
Time frame: From 6 months before recruitment to 104 weeks post-transplant
Change of volume of packed RBC transfusions.
Time frame: From 6 months before recruitment to 104 weeks post-transplant
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