Tests patient's own gene-corrected stem cells for beta-thalassemia major
Tests patient's own stem cells, corrected for beta-globin, in children with beta-thalassemia major.
Plain-language title generated from the registry record
Registry title: Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
Key facts
- Where
- Shenzhen, ChinaSee the site
- Phase
- Early phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 8 and 16 years old?
Source: NCT05745532, who can take part
Where in China
- Shenzhen Children's HospitalShenzhen, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Shenzhen Hemogen
- Trial code
- NCT05745532
- Record updated
- 29 November 2024
- Original description
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major PatientsRead full original descriptionShow less
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
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
Inclusion: 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: Received gene therapy and allogeneic HSCT in the past; The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation; Other unsuitable conditions identified by doctors; Have an available HLA matched donor; Enrolling in another clinical trial.