Hemoglobinopathy clinical trials at UCSF

4 in progress, 2 open to eligible people

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

  • EDIT-301 for Autologous HSCT in Subjects With Severe Sickle Cell Disease

    open to eligible people ages 18-50

    The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).

    Oakland, California and other locations

  • In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

    Sorry, accepting new patients by invitation only

    The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.

    San Francisco, California

  • Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

    Sorry, in progress, not accepting new patients

    Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene phosphoramide (TEPA). The goal of this study is to determine what causes some children to have different drug concentrations of thiotepa and TEPA in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and genetic factors cause changes in thiotepa and TEPA drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

    San Francisco, California

Our lead scientists for Hemoglobinopathy research studies include .

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