Hemoglobinopathy clinical trials at UCSF
3 in progress, 2 open to eligible people
Hemoglobinopathy includes diseases where hemoglobin in the blood is abnormal. UCSF is conducting clinical trials for severe sickle cell disease and beta thalassemia that need regular blood transfusions. The trials evaluate how safe and effective new treatments are.
EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
open to eligible people ages 12-50
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent 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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