Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

Institute of Hematology & Blood Diseases Hospital, China

Status

Active, not recruiting

Conditions

Transfusion Dependent Beta-Thalassaemia

Treatments

Biological: ET-01

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT04390971

EDI-001

Details and patient eligibility

About

This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

Full description

After the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and ET-01 infusion to complete therapy. Two years follow-up will be carried out post-transplantation and related data will be collected. Subjects who have been treated by ET-01 will be asked to participate in a long-term follow up study. Monitoring the long-term efficacy and safety up to 15 years after ET-01 transplant.

Enrollment

3 patients

Sex

All

Ages

6 to 35 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms;
6~35 years old, all gender;
Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol;
Eligible for autologous stem cell transplant;
Eligible for autologous stem cell transplant;
Organs in good function.
Other protocol defined inclusion criteria may apply.

Exclusion criteria

Subjects with associated α-thalassemia;
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.