Phase 12 Study of ALS20 Gene Therapy Using Modified Blood Stem Cells for Adults 18 to Under 40 With Transfusion-Dependent Beta Thalassemia

What this Trial Is About

Researchers are studying transfusion-dependent beta thalassemia, a hereditary blood disorder that requires lifelong blood transfusions and leads to serious health issues and reduced quality of life. The trial aims to evaluate the safety and effectiveness of a new gene therapy using autologous blood stem cells modified with a lentiviral vector called ALS20, which carries a healthy beta globin gene. This gene therapy is experimental and has not been approved by the FDA. The study involves collecting hematopoietic stem and progenitor cells from participants, modifying them with the ALS20 lentiviral vector, and then infusing these cells back after the participant undergoes myeloablative conditioning with busulfan chemotherapy. This single-arm pilot trial will include up to 12 subjects aged 18 to less than 36 years with transfusion-dependent beta thalassemia. The treatment is designed to reduce the need for blood transfusions by increasing healthy hemoglobin production. Participants will be closely monitored with tests measuring blood cell recovery, survival, transplant complications such as graft versus host disease, and possible genetic risks related to the therapy over a follow-up period of up to 24 months. Researchers will track neutrophil and platelet engraftment within the first 42 days to one year after infusion and assess hemoglobin levels to evaluate treatment impact. Safety assessments include monitoring for infections, liver function, and blood cancer risks. The total study duration spans from treatment through long-term observation.

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia