Division of Hematology Clinical Studies

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HGB-212: Phase 3 Beta Thalassemia LentiGlobin BB305 Gene Therapy Trial

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.

LUNA 3

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.
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