A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.

EdiGene

Status

Terminated

Conditions

Transfusion Dependent Beta-Thalassaemia

Treatments

Biological: ET-01

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT05752123

EDI-001-002

Details and patient eligibility

About

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.

Enrollment

2 patients

Sex

All

Ages

6 to 35 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key 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;
Organs in good function.

Other protocol defined Inclusion criteria may apply.

Key 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.