Hemoglobinopathy clinical trials at UCSF
3 in progress, 2 open to eligible people
Hemoglobinopathy is a condition that affects the blood and can cause anemia. At UCSF, we are running trials to test treatments for severe sickle cell disease and beta thalassemia. In these trials, we will look at the safety and effectiveness of EDIT-301, a treatment that uses the patient's own stem cells to produce healthy blood cells. Participants must be adults and will be closely monitored throughout the trials.
EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
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 participants with severe sickle cell disease (SCD).
Oakland, California and other locations
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
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 Hemoglobinopathy research studies include Tippi Mackenzie.
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