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NCT Number: NCT06308159

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

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

Age range

Up to 35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 criteria

  • 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.

Treatment and study plan

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.

Other names: Beta-globin lentiviral vector transduced autologous HSPCs

Primary outcomes

  1. 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

Secondary outcomes

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

    Time frame: From baseline to Month 24

    Participants are monitored for safety from baseline up to the end of the follow-up period.

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

    Time frame: From infusion to Month 24

    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.

  3. Number of days required to achieve successful neutrophil and platelet engraftment

    Time frame: From infusion to Month 24

    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.

  4. Vector copy number (VCN) in peripheral blood over time

    Time frame: From baseline to Month 24

    Quantification of the lentiviral vector copy number in individual peripheral blood cells will be conducted to measure the transduction of HSPCs.

Sponsors and collaborators

Lead sponsor

Lantu Biopharma

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Mar 13, 2024
Registry last updated
Jun 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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