At a glance
ClinicalIndex Comparison Record- ✓Age 12–35 years at consent/assent
- ✓Confirmed diagnosis of β-thalassemia major with transfusion dependence: ≥100 mL/kg/year of pRBCs or ≥8 transfusions/year for the prior 2 years
- ✓Eligible for allogeneic bone marrow transplant
- ✓Treated and followed in a specialized center for ≥2 years with detailed medical records including transfusion history
- ✕HIV 1 or HIV 2 positive
- ✕WBC count <3 × 10⁹/L and/or platelet count <100 × 10⁹/L if not due to hypersplenism
- ✕Uncorrected bleeding disorder
- ✕Prior or current malignancy, myeloproliferative disorder, or immunodeficiency disorder
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)
In Brief
A Phase 2 clinical trial evaluating LentiGlobin BB305 Drug Product for β-thalassemia Major. Completed, enrolled 19 participants across 6 sites in 3 countries.
Detailed Summary
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product \[autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene\].
Study Details
Timeline
Interventions
Transplant of autologous hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector.