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Thalassemia clinical trials at UCSF

3 in progress, 2 open to eligible people

Thalassemia is a blood disorder that affects the production of hemoglobin. At UCSF, we are recruiting for clinical trials to improve blood transfusions for people with thalassemia, and to study a new drug for thalassemia and sickle cell disease patients. These trials will look at the safety and effectiveness of these treatments.

Showing trials for
  • Etavopivat in Patients With Thalassemia or Sickle Cell Disease

    open to eligible people ages 12-65

    This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin.

    Oakland, California and other locations

  • Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

    open to all eligible people

    Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.

    Oakland, California and other locations

  • Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

    Sorry, in progress, not accepting new patients

    Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT). This study includes a core period (up to 24 weeks) followed by an extension period (up to 10 years) for eligible participants. 20 participants with NTDT were enrolled. The initial dose of AG-348 was 50 milligrams (mg) twice daily (BID) with one potential dose-level increase to 100 mg BID at the Week 6 visit based on the participant's safety and hemoglobin (Hb) concentrations.

    Oakland, California and other locations

Our lead scientists for Thalassemia research studies include .

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