Gene therapy for patients with 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
This study is testing a new gene therapy to see if it can help people with transfusion-dependent beta-thalassemia by modifying their own cells to produce healthy hemoglobin.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 6 (estimated) |
| Ages | N/A to 35 Years |
| Sex | All |
| Sponsor | Lantu Biopharma Industry-sponsored |
| Locations | 2 sites (Kunming, Yunnan and 1 other locations) |
| Trial ID | NCT06308159 on ClinicalTrials.gov |
What this trial studies
This interventional study evaluates the safety and efficacy of a gene therapy product called Vebeglogene autotemcel in patients with transfusion-dependent beta-thalassemia. Participants will undergo a process where their own hematopoietic stem and progenitor cells are modified using a lentiviral vector to express a functional hemoglobin subunit beta gene. The study includes a screening period, mobilization and product manufacture, myeloablative conditioning, treatment, and a 24-month observation period to assess outcomes. The primary endpoints focus on the safety and efficacy profiles of the treatment.
Who should consider this trial
Good fit: Ideal candidates are patients diagnosed with transfusion-dependent beta-thalassemia who have a documented history of red blood cell transfusions.
Not a fit: Patients with active infections, severe comorbidities, or prior gene therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly reduce or eliminate the need for blood transfusions in patients with beta-thalassemia.
How similar studies have performed: Other studies using gene therapy for beta-thalassemia have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Kunming, Yunnan and 1 other locations
- 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China — Kunming, Yunnan, China (Recruiting)
- Kunming Hope of Health Hospital — Kunming, Yunnan, China (Recruiting)
Study contacts
- Study coordinator: Austin Gao, PhD
- Email: clinicaltrials@lantubiopharma.com
- Phone: +8617724360504
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.