An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

NCT06308159 | Recruiting Phase 1

• 1 other identifier: LT02-101
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Beta-Thalassemia

Outcome Measures

Primary Outcomes (1)

• Time and duration of the subject's hemoglobin (Hb)≥9.0 g/dL without receiving red blood cell infusion

  From baseline to Month 24

Secondary Outcomes (4)

• The prevalence and severity of adverse events (AEs) and serious adverse events (SAEs)

  From baseline to Month 24

• The reduction of red blood cells (RBCs) transfusion requirement after product infusion compared to previous transfusion records

  From infusion to Month 24

• Number of days required to achieve successful neutrophil and platelet engraftment

  From infusion to Month 24

• Vector copy number (VCN) in peripheral blood over time

  From baseline to Month 24

Study Arms (1)

Vebeglogene autotemcel

EXPERIMENTAL

One-time infusion of≥5×10\^6/kg beta-globin lentiviral vector transduced HSPCs

Drug: Vebeglogene autotemcel

Interventions

Vebeglogene autotemcel DRUG

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

Vebeglogene autotemcel

Eligibility Criteria

• Age: Up to 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Prior autologous hematopoietic stem cell transplantation.
• Prior receipt of gene therapy.

Sponsors & Collaborators

• Lantu Biopharma: lead

Study Sites (2)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Kunming, Yunnan, 650100, China

RECRUITING

Kunming Hope of Health Hospital

Kunming, Yunnan, 650200, China

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Austin Gao, PhD

CONTACT

+8617724360504; clinicaltrials@lantubiopharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2024
• First Posted: March 13, 2024
• Study Start: May 11, 2024
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): August 1, 2027
• Last Updated: November 25, 2025

Record last verified: 2025-11

Locations

CN (2)