Actively Recruiting
Safety and Efficacy Evaluation of b2-globin Restored Autologous Hematopoietic Stem Cells in b2-thalassemia Major Patients
Led by Shenzhen Hemogen · Updated on 2024-11-29
10
Participants Needed
1
Research Sites
30 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of b2-globin restored autologous hematopoietic stem cells in patients with b2-thalassemia major, a condition requiring regular blood transfusions. This early phase 1, open-label study focuses on restoring b2-globin expression to improve patient outcomes. The study is sponsored by Shenzhen Hemogen and targets children and adolescents aged 8 to 16 years with transfusion-dependent b2-thalassemia major. Participants will have their own hematopoietic stem cells collected and modified using the LentiHBBT87Q system to restore b2-globin production. After a conditioning process, these modified stem cells will be reinfused into the patients. Ten subjects aged 8 to 16 years will receive this treatment and be followed for two years to observe effects and safety. During the study, researchers will monitor adverse events, survival rates, engraftment success, viral replication, and viral integration over 24 months. Secondary measures include monitoring viral copy number in blood cells, adult hemoglobin expression, and changes in blood transfusion needs. Participants will undergo regular medical visits, treatments, and assessments throughout the two-year follow-up period to collect comprehensive data on treatment impact and safety.
CONDITIONS
Brief Title
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 8 to 16 years old, with informed consent signed by the subject or legal guardian
- Clinically diagnosed as transfusion-dependent b2-thalassemia major
- Hemoglobin level e9 g/dL with sufficient red blood cell infusion
- Serum ferritin level c= 3000 ng/mL
- Mild or no liver iron overload for at least 3 months before stem cell mobilization
- Willing to follow treatment and attend regular medical check-ups for two years after transplantation
You will not qualify if you...
- Physical condition not suitable for stem cell mobilization and transplantation myeloablation
- Previous gene therapy or allogeneic hematopoietic stem cell transplantation
- Availability of an HLA matched donor
- Currently enrolled in another clinical trial
- Other conditions deemed unsuitable by doctors
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 100 days
Participants receive conditioning followed by reinfusion of their own β-globin-restored hematopoietic stem cells modified with LentiHBBT87Q.
1 treatment visit and multiple monitoring visits during the first 100 days
Duration - Up to 24 months
Participants are followed up for two years after stem cell reinfusion to monitor safety, engraftment, and efficacy outcomes.
Regular visits for up to 24 months
Trial Site Locations
Total: 1 location
1
Shenzhen Children's Hospital
Shenzhen, Guangdong, China, 518083
Actively Recruiting
Research Team
H
Haigang Sun
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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