A Phase 1/2 interventional study of Vebeglogene autotemcel in Beta-Thalassemia, sponsored by Lantu Biopharma. Active, not recruiting at 2 sites in China. Open to participants aged Up to 35 Years. Per ClinicalTrials.gov, last updated 2026-06-15.
Sponsored by Lantu Biopharma · Phase 1/2, Interventional, and Treatment
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.
The participant's autologous HSPCs will be transduced with the self-inactivating lentiviral vector, carrying the functional HBB gene.
Study duration per participant is approximately 27 months including an approximately 30-day screening/baseline period, an approximately 60-day mobilization and product manufacture, an approximately 10-day myeloablative conditioning, 1 treatment day, and an approximately 24-month study observation period.
The endpoints will be used to assess the safety and efficacy profiles in patients with transfusion-dependent beta-thalassemia.
305 studies on the registry are indexed under beta-Thalassemia; 70 are open to participants now.
This study's planned enrollment of 6 is below the median of 38 across 210 interventional studies indexed under beta-Thalassemia.
Browse beta-Thalassemia studies →Lantu Biopharma is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
One-time infusion of≥5×10\^6/kg beta-globin lentiviral vector transduced HSPCs
Drug: Vebeglogene autotemcel
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.
Also known as: Beta-globin lentiviral vector transduced autologous HSPCs
Time and duration of the subject's hemoglobin (Hb)≥9.0 g/dL without receiving red blood cell infusion
Time frame: From baseline to Month 24
The prevalence and severity of adverse events (AEs) and serious adverse events (SAEs)
Participants are monitored for safety from baseline up to the end of the follow-up period.
Time frame: From baseline to Month 24
The reduction of red blood cells (RBCs) transfusion requirement after product infusion compared to previous transfusion records
The annual number of RBCs transfusions prior to product infusion will be compared to the annual number of RBCs transfusions post-infusion, and the requirement reduction duration should be reported.
Time frame: From infusion to Month 24
Number of days required to achieve successful neutrophil and platelet engraftment
Neutrophil engraftment is defined as the time to the first of 3 consecutive days of absolute neutrophil counts (ANC)≥0.5×10\^9/L post-infusion without transfusion. Platelet engraftment is defined as the time to the first of 3 consecutive days of platelet values≥20×10\^9/L post-infusion without transfusion.
Time frame: From infusion to Month 24
Vector copy number (VCN) in peripheral blood over time
Quantification of the lentiviral vector copy number in individual peripheral blood cells will be conducted to measure the transduction of HSPCs.
Time frame: From baseline to Month 24
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Lantu Biopharma