Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

Shenzhen Hemogen

Status and phase: Enrolling; Early Phase 1

Conditions: β-thalassemia

Treatments: Biological: β-globin restored autologous hematopoietic stem cells

Study type: Interventional

Funder types: Industry

Identifiers

NCT05745532

SZ-101

Details and patient eligibility

About

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients

Full description

We will recruit ß-thalassaemia major patients and collect their autologous hematopoietic stem cells, which will be modified with the LentiHBBT87Q system to restore β-globin expression. After conditioning, the autologous hematopoietic stem cells with restored β-globin will be reinfused to the patients and followed up for two years to collect data.

Enrollment: 10 estimated patients

Sex: All

Ages: 8 to 16 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 8-16 years old. Subject and/or subject's legal guardian fully understand and voluntarily sign informed consent;
• Clinically diagnosed as transfusion-dependent β-thalassemia major;
• With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g/dL, serum ferritin threshold ≤ 3000 ng/mL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;
• Follow the arrangements for treatment and regular medical checks within two years post-transplantation

Exclusion criteria

• The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;
• Received gene therapy and allogeneic HSCT in the past.
• Have an available HLA matched donor.
• Enrolling in another clinical trial.
• Other unsuitable conditions identified by doctors.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

10 participants in 1 patient group

Experimental

Experimental group

Description:

Ten transfusion-dependent β-thalassaemia subjects aged 8-16 years will be reinfused with β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q.

Treatment:

Biological: β-globin restored autologous hematopoietic stem cells