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
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Maximum Age: 35
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
China
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
RECRUITING
Kunming
Kunming Hope of Health Hospital
RECRUITING
Kunming
Contact Information
Primary
Austin Gao, PhD
clinicaltrials@lantubiopharma.com
Time Frame
Start Date: 2024-05-11
Estimated Completion Date: 2027-08-01
Participants
Target number of participants: 6
Treatments
Experimental: Vebeglogene autotemcel
One-time infusion of≥5×10\^6/kg beta-globin lentiviral vector transduced HSPCs
Related Therapeutic Areas
Sponsors
Leads: Lantu Biopharma