NCT06308159Active, not recruitingPhase 1/2
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
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
Source: ClinicalTrials.gov · Last updated 15 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 6 (estimated)
- First submitted
- 6 Mar 2024
- Start date
- 11 May 2024
- Primary completion date
- 1 Aug 2027
- Lead sponsor
- Lantu Biopharma
- Diseases
- Thalassaemia
- NCT number
- NCT06308159
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 6
- From 11 May 2024 to the primary completion date, 1 Aug 2027
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior autologous hematopoietic stem cell transplantation.
- Prior receipt of gene therapy.
Other illness
- Active bacterial, viral, fungal, or parasitic infection.
- 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.
Lab values
- A white blood cell (WBC) counts\<3×10\^9/L, and/or platelet counts\<100×10\^9/L not related to hypersplenism.
Other
- Any evidence of severe iron overload that, in the investigator's opinion, warrants exclusion.
Who the study is looking for
- 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.
Age in the registry: - to 35 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China Coordinator of this site? Confirm its status | Kunming | China | Unknown status |
| Kunming Hope of Health Hospital Coordinator of this site? Confirm its status | Kunming | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.