Individuals with a certain type of beta thalassemia that is treated with regular transfusions, and who are 50 years old and younger, 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.
Thalassemia Center Clinical Studies
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The FORMA 201 study is for teenagers and adults (ages 12and up) with transfusion and non-transfusion dependent Thalassemia and transfusion dependent Sickle Cell Disease. The goal of the study is to see if the study drug, FT-4202 can safely lower the number of blood transfusions required and therefore decrease your iron overload. FT-4202 is an investigational medication designed to improve the production of red blood cells. You may be able to join the study if you are:
- 12 years of age or older,
- Diagnosed with Sickle Cell Disease and receiving regular blood transfusions or
- Diagnosed with Thalassemia.
Other study requirements will apply.