NCT05745532 Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
| NCT ID | NCT05745532 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Shenzhen Hemogen |
| Condition | β-thalassemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2020-12-01 |
| Primary Completion | 2025-05-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 10 participants in total. It began in 2020-12-01 with a primary completion date of 2025-05-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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.
Contact & Investigator
Chao Liu, PHD
PRINCIPAL INVESTIGATOR
Shenzhen Hemogen
Frequently Asked Questions
Who can join the NCT05745532 clinical trial?
This trial is open to participants of all sexes, aged 8 Years or older, up to 16 Years, studying β-thalassemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05745532 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05745532 currently recruiting?
Yes, NCT05745532 is actively recruiting participants. Contact the research team at sunhaigang@genomics.cn for enrollment information.
Where is the NCT05745532 trial being conducted?
This trial is being conducted at Shenzhen, China.
Who is sponsoring the NCT05745532 clinical trial?
NCT05745532 is sponsored by Shenzhen Hemogen. The principal investigator is Chao Liu, PHD at Shenzhen Hemogen. The trial plans to enroll 10 participants.