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

Last updated: October 7, 2024
Sponsor: Lantu Biopharma
Overall Status: Active - Recruiting

Phase

1/2

Condition

Thalassemia

Treatment

Vebeglogene autotemcel

Clinical Study ID

NCT06308159
LT02-101
  • Ages < 35
  • All Genders

Study 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 Criteria

Inclusion

Inclusion Criteria:

  • Patients or parent(s)/legal guardian(s) willing and able to complete the informedconsent 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

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 notrelated 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 cardiovasculardisease.

  • Uncontrolled seizure disorder.

  • Any evidence of severe iron overload that, in the investigator's opinion, warrantsexclusion.

  • Prior autologous hematopoietic stem cell transplantation.

  • Prior receipt of gene therapy.

Study Design

Total Participants: 6
Treatment Group(s): 1
Primary Treatment: Vebeglogene autotemcel
Phase: 1/2
Study Start date:
May 11, 2024
Estimated Completion Date:
August 01, 2027

Study 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.

Connect with a study center

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

    Kunming, Yunnan 650100
    China

    Active - Recruiting

  • Kunming Hope of Health Hospital

    Kunming, Yunnan 650200
    China

    Active - Recruiting

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