Beta Thalassemia clinical trials at UCSF
6 in progress, 3 open to eligible people
Beta thalassemia is a disease that reduces hemoglobin in the blood. UCSF is testing treatments like betibeglogene autotemcel and EDIT-301 for patients who need frequent transfusions. UCSF is also collecting data on alpha thalassemia patients to learn more about the disease.
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
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
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 Tippi C MacKenzie, MD.
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