Phase 3 Beta Thalassemia LentiGlobin BB305 Gene Therapy Trial



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Individuals with certain types of beta thalassemia that is treated with regular transfusions and who are up to 50 years old may be able to participate. A number of screening tests will be done to determine if subjects can participate. These include review of your past medical history, blood and urine tests, bone marrow aspirate/biopsy, bone age/ DEXA scan, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and liver biopsy. If eligible, blood stem cells will be collected by apheresis or by bone marrow harvest. These stem cells will then have a healthy beta globin gene inserted. You will then be hospitalized and chemotherapy will be given to empty out the bone marrow. The stem cells, with the inserted gene, will then be given back to you. You will be followed for side effects and to see if the inserted gene helps you make hemoglobin.

Eligibility & Criteria

IRB #:
Official Title:
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent ß-Thalassemia, who do not have ß0/ß0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral ßA-T87Q-Globin Vector in Subjects =50 Years of Age
Study Phase:
Phase III
Eligible Age Range:
0 - 50 Years
Study Categories: