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

Recruiting now · Early Phase 1

Conditions studied: β-thalassemia

In brief

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

Key facts

Study ID

NCT05745532

Run by

Shenzhen Hemogen

People needed

10

Starts

2020-12-01

Expected to finish

2025-12-30

Last updated by the study team

2024-11-29

Who can join

Age: 8 and older, up to 16. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 it is running

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

Full record on ClinicalTrials.gov

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