RecruitingEarly Phase 1NCT05745532

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

Sponsor

Shenzhen Hemogen

Enrollment

10 participants

Start Date

Dec 1, 2020

Study Type

INTERVENTIONAL

Conditions

Thalassemia

Summary

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

Eligibility

Min Age: 8 YearsMax Age: 16 Years

Inclusion Criteria4

-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 Criteria5

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.

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