• Beta-Thalassemia Gene Therapy Clinical Trial

    Gene Therapy

    Phone: 617-632-5064  

    A Phase I/II Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-Thalassemia (TDT)

    Thalassemia is an inherited blood disorder in which the genes that produce hemoglobin, the protein in red blood cells that carries oxygen from the lungs to all parts of the body, are broken. As a result, the red blood cells do not contain enough hemoglobin, causing anemia that can range from mild to life threatening.

    Thalassemia can come in different forms depending on the genetic mutations causing it. One type of thalassemia is beta-thalassemia, which arises from mutations in or the loss of beta globin genes.

    This thalassemia gene therapy clinical trial aims to assess the effectiveness of ST-400 on transfusion-dependent beta-thalassemia (TDT). ST-400 is a type of investigational therapy that consists of transfusing gene edited cells into a patient. In this study, the patient’s own blood stem cells are genetically modified in the laboratory using zinc finger nuclease (ZFN) genome editing technology created by Sangamo Therapeutics. Using this technology, the expression of the BCL11A gene, which normally shuts off the production of fetal hemoglobin shortly after birth, is turned down. After receiving conditioning chemotherapy to make room for the new cells in the bone marrow, the patient will receive a transfusion of edited blood stem cells.

    Ultimately, this approach aims to boost the production of new enthrocytes, or red blood cells, with increased fetal hemoglobin (HbF), which can be substituted for either reduced or absent adult (defective) hemoglobin.

    Inclusion Criteria:

    • Ages 18 to 40
    • Informed consent
    • Clinical diagnosis of transfusion-dependent beta-thalassemia (TDT) with ≥ 8 documented red blood cell (RBC) transfusion events per year on an annualized basis in the 2-years prior to screening
    • Confirmed beta-thalassemia diagnosis by molecular genetic testing
    • Clinically stable and eligible to receive conditioning chemotherapy
    • Able and willing to use double barrier method of contraception for at least 3 months prior to ST-400 infusion and through 6 months post-transplant

    Exclusion Criteria:

    • Previous history of autologous or allogeneic blood stem cell transplantation or solid organ transplantation
    • Pregnant or breastfeeding female
    • Medical contraindication to apheresis
    • Significant liver, lung, heart, or kidney dysfunction
    • Diagnosis of HIV or evidence of active HBV or HCV
    • History of significant bleeding disorder or uncontrolled seizures
    • History of active malignancy in past 5 years (non-melanoma skin cancer or cervical cancer in situ permitted)
    • Currently participating in another clinical trial using an investigational study medication or recent participation in such a trial
    • Previous treatment with gene therapy

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