Skip to main content

Phase 3 Beta Thalassemia LentiGlobin BB305 Gene Therapy Trial

Phase 3 Beta Thalassemia LentiGlobin BB305 Gene Therapy Trial

Contact us
This study is no longer recruiting.

Description

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 and criteria


IRB Number:
15-012170
Eligible age range:
Any years - 50 years
Clinical trial phase:
Phase III
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
Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.

Jump back to top