Division of Hematology Research Studies

This clinical trial aims to test a new gene therapy in adults (18 to 35 years old) with beta thalassemia who require regular red cell transfusions.

The Dova study is for children below 18 years with primary Immune Thrombocytopenia (ITP) who have had an insufficient response to previous treatment.

 A gene in stem cells will then be edited to help the stem cells make more fetal hemoglobin. Higher levels of fetal hemoglobin can improve red blood cell sickling.

The ENERGY study is for adults (ages 18 and up) with warm Autoimmune Hemolytic Anemia (wAIHA) who are currently receiving treatment or have previously received treatment. The goal of the study is to see if the study drug, Nipocalimab is safe and effective in the treatment of wAIHA.

This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.

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 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.

ICON 3 is a prospective, open label, randomized, multi-center phase 3 clinical trial comparing eltrombopag with the enrolling physician’s choice of front line therapy.

The LUNA 3 study is for teenagers and adults (ages 12 and up) with primary immune thrombocytopenia (ITP) for which standard medications are no longer working.

The Panzyga Study is looking at a potential medicine (experimental drug) to see if it can result in increasing platelet counts and may potentially treat ITP.

This research study is seeking participants who have hemophilia B and need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also control bleeding events.

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.