Actively Recruiting
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
Led by Lantu Biopharma · Updated on 2025-11-25
6
Participants Needed
2
Research Sites
168 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.
CONDITIONS
Official Title
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients or parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedules.
- Diagnosis of beta-thalassemia and a history of red blood cell transfusions.
- Documented baseline or pretransfusion hemoglobin less than 7 g/dL.
- Availability of an adequate and well-documented transfusion history.
You will not qualify if you...
- Active bacterial, viral, fungal, or parasitic infection.
- White blood cell count less than 3 x 10^9/L and/or platelet count less than 100 x 10^9/L not related to hypersplenism.
- Uncorrected bleeding disorder.
- Presence of severe diseases judged incompatible with study procedures, such as severe liver, kidney, lung, or cardiovascular disease.
- Uncontrolled seizure disorder.
- Severe iron overload as judged by the investigator.
- Prior autologous hematopoietic stem cell transplantation.
- Prior receipt of gene therapy.
AI-Screening
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Trial Site Locations
Total: 2 locations
1
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, China, 650100
Actively Recruiting
2
Kunming Hope of Health Hospital
Kunming, Yunnan, China, 650200
Actively Recruiting
Research Team
A
Austin Gao, PhD
CONTACT
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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