Beta-Thalassemia
Conditions
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.
Interventions
DRUGVebeglogene autotemcel
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.
Sponsors
Lantu Biopharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 35 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
China
Outcome results
None listed