An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
An Open-label Clinical Trial of Ex Vivo Beta-globin Lentiviral Vector Transduction of Autologous CD34+HSPCs (Vebeglogene Autotemcel) for the Treatment of Transfusion Dependent Beta-thalassemia Patients
Lantu Biopharma
6 participants
May 11, 2024
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria4
- 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 RBCs transfusions.
- Documented baseline, or pretransfusion, Hb≤7 g/dL.
- Availability of an adequate and well-documented transfusion history.
Exclusion Criteria8
- Active bacterial, viral, fungal, or parasitic infection.
- A white blood cell (WBC) counts<3×10\^9/L, and/or platelet counts<100×10\^9/L not related to hypersplenism.
- Uncorrected bleeding disorder.
- Presence of severe diseases that judged not compatible with the study procedures, such as severe hepatic disease, kidney disease, lung disease, and/or cardiovascular disease.
- Uncontrolled seizure disorder.
- Any evidence of severe iron overload that, in the investigator's opinion, warrants exclusion.
- Prior autologous hematopoietic stem cell transplantation.
- Prior receipt of gene therapy.
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Interventions
Autologous HSPCs transduced with self-inactivating lentiviral vector encoding functional HBB gene and resuspended in cryopreservative solution in the final immediate container for the intended medical use.
Locations(2)
View Full Details on ClinicalTrials.gov
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NCT06308159