Alpha Thalassemia Major clinical trials at UCSF
3 in progress, 2 open to eligible people
Alpha thalassemia major is a blood disorder that causes anemia. UCSF is conducting clinical trials to explore treatments for this condition. One trial involves creating an international registry to learn more about the disease and fetal therapy outcomes. Another trial is testing the safety and effectiveness of a treatment called EDIT-301 in adult participants with 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
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
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
Our lead scientists for Alpha Thalassemia Major research studies include Tippi C MacKenzie, MD.
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