Beta Thalassemia clinical trials at UCSF

5 in progress, 3 open to eligible people

Beta thalassemia is a condition that causes low hemoglobin levels in the blood. At UCSF, researchers are collecting data on people treated with betibeglogene autotemcel. Another study is testing a new stem cell transplant method, EDIT-301, to determine its safety and effectiveness for those who depend on regular blood transfusions.

Showing trials for

  • EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

    open to eligible people ages 18-35

    The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

    Oakland, California and other locations

  • Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

    open to all eligible people

    The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.

    Oakland, California and other locations

  • International Registry of Patients With Alpha Thalassemia

    open to all eligible people

    This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with Alpha thalassemia.

    San Francisco, California

  • Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

    Sorry, in progress, not accepting new patients

    The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).

    Oakland, California and other locations

  • Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

    Sorry, accepting new patients by invitation only

    Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: - Long-term efficacy of the biological treatment effect of BIVV003 in SCD - Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events - Long-term efficacy of the biological treatment effect of ST-400 in TDT - Long-term efficacy of the clinical treatment effect of ST-400 in TDT

    Oakland, California and other locations

Our lead scientists for Beta Thalassemia research studies include .

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