Beta Thalassemia

This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused.

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.

This study assesses whether early chelation with deferiprone is effective at delaying the progression of iron overload in infants and young children.

Individuals with beta thalassemia that is treated with regular transfusions, and who are between the ages of 18 and 40 years old, may be able to participate.

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.

The Rivella Laboratory uses lentiviral vectors for modulation of gene expression and gene transfer for the cure of hemoglobinopathies.