Alpha Thalassemia Major clinical trials at UCSF
2 research studies open to eligible people
Alpha thalassemia major is a serious blood disorder where the body makes less hemoglobin than normal. UCSF is running a study to check the safety of a new stem cell transplant treatment. Another study is collecting data from patients worldwide to understand the disease better.
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
Our lead scientists for Alpha Thalassemia Major research studies include Tippi C MacKenzie, MD.