Evaluating a new treatment for β-thalassemia using modified stem cells

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

This study is testing a new treatment for kids with β-thalassemia by using their own modified stem cells to see if it can help improve their condition.

Quick facts

• Phase: EARLY_PHASE1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 8 Years to 16 Years
• Sex: All
• Sponsor: Shenzhen Hemogen (industry)
• Locations: 1 site (Shenzhen, Guangdong)
• Trial ID: NCT05745532 on ClinicalTrials.gov

What this trial studies

This open-label study aims to assess the safety and efficacy of β-globin restored autologous hematopoietic stem cells in patients with β-thalassemia major. Participants aged 8-16 will have their own hematopoietic stem cells collected and modified using the LentiHBBT87Q system to restore β-globin expression. After a conditioning regimen, these modified cells will be reinfused into the patients, who will be monitored for two years to evaluate treatment outcomes.

Who should consider this trial

Good fit: Ideal candidates are children aged 8-16 with clinically diagnosed transfusion-dependent β-thalassemia major.

Not a fit: Patients who have received gene therapy or allogeneic hematopoietic stem cell transplantation in the past may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life for patients with β-thalassemia major by reducing their dependence on blood transfusions.

How similar studies have performed: While gene therapy approaches for β-thalassemia are being explored, this specific method using autologous stem cells with β-globin restoration is relatively novel.

Eligibility criteria

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.

Where this trial is running

Shenzhen, Guangdong

• Shenzhen Children's Hospital — Shenzhen, Guangdong, China (RECRUITING)

Study contacts

• Principal investigator: Chao Liu, PHD — Shenzhen Hemogen
• Study coordinator: Haigang Sun

Conditions: β-thalassemia