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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Shenzhen Children's Hospital
  Shenzhen, Guangdong, China Recruiting

Study contacts

• Principal investigator: Chao Liu, PHD — Shenzhen Hemogen
• Study coordinator: Haigang Sun
• Email: sunhaigang@genomics.cn
• Phone: 13823168465

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.