Division of Hematology Research Studies

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.

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 goal of the study is to see if the study drug, Rilzabrutinib can safely increase platelet counts and improve symptoms for people with ITP. Rilzabrutinib is an investigational medication designed to stop your immune system from destroying your platelets. You may be able to join the study if you are:

• 12 years of age or older.
• Diagnosed with primary ITP for at least 6 months for ages 12 through 17 or for at least 3 months for ages 18 and up.
• Have taken IVIg or steroids, but they are no longer working.
• Have platelet counts of less than 30,000. 

Other study requirements will apply.

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