An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
- Registration Number
- NCT06308159
- Lead Sponsor
- Lantu Biopharma
- 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.
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 6
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Vebeglogene autotemcel Vebeglogene autotemcel One-time infusion of≥5×10\^6/kg beta-globin lentiviral vector transduced HSPCs
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method The prevalence and severity of adverse events (AEs) and serious adverse events (SAEs) From baseline to Month 24 Participants are monitored for safety from baseline up to the end of the follow-up period.
The reduction of red blood cells (RBCs) transfusion requirement after product infusion compared to previous transfusion records 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.
Number of days required to achieve successful neutrophil and platelet engraftment 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.
Vector copy number (VCN) in peripheral blood over time 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.
Trial Locations
- Locations (2)
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
🇨🇳Kunming, Yunnan, China
Kunming Hope of Health Hospital
🇨🇳Kunming, Yunnan, China