Beta Thalassemia clinical trials at UCSF
6 in progress, 2 open to eligible people
Beta thalassemia reduces hemoglobin levels in the blood, causing anemia. UCSF is examining long-term data on people treated with betibeglogene autotemcel for beta-thalassemia. UCSF is also compiling data from an international registry to understand alpha thalassemia.
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 objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
Oakland, California and other locations
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
Sorry, in progress, not accepting new patients
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 Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Sorry, accepting new patients by invitation only
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
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 Tippi C MacKenzie, MD.
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