Thalassemia Center 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.

Gladiolus Study

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. All participants enrolled in the study will receive the investigational medication.

We are currently enrolling individuals who meet the following criteria:*

  • Are 12 to 65 years of age
  • Have one of the following diagnoses:
    • SCD and receiving regular RBC transfusions to prevent stroke or recurrence of stroke (Cohort A)
    • Thalassemia and receiving regular RBC transfusions (Cohort B)
    • Thalassemia and not receiving regular RBC transfusions (Cohort C)

      There are additional eligibility requirements, which the investigator will explain to you.