Overview
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent -Thalassaemia.
Description
After proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. 2 years' follow up visits will be carried out post-transplantation and related data will be collected.
Eligibility
Inclusion Criteria:
- Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
- 6~35 years old, all gender, weight 30kg;
- Genetically diagnosed -Thalassaemia;
- Diagnosed as transfusion-dependent;
- Eligible for autologous stem cell transplant;
- Organs in good function;
- Other protocol defined Inclusion criteria may apply.
Exclusion Criteria:
- Thalassemia gene type is 0/0;
- Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection;
- HLA identical sibling or unrelated donors are available;
- Prior allo-HSCT or gene therapy;
- Other protocol defined Exclusion criteria may apply.