NCT06308159

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Lantu Biopharma · Industry
• Sex: All
• Age: 35 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2024-05-11
• Primary completion: 2027-08-01
• Completion: 2027-08-01
• First posted: 2024-03-13
• Last updated: 2025-11-25

Locations

2 sites across 1 country: China