Gene therapy for transfusion-dependent beta thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This gene therapy trial uses a modified virus to deliver a healthy gene into your own stem cells. The goal is to help your body make normal red blood cells so you may need fewer or no transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and just under 40 years old
- You have a severe form of beta thalassemia that requires regular blood transfusions
- You have not had a bone marrow or stem cell transplant before
- You have no active cancer or other serious health conditions that would make the treatment risky
- You are willing to discuss other treatment options with your doctor before joining
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a new gene therapy that uses a modified virus to add a working copy of the gene for beta globin, which may help your body make healthy red blood cells. It is designed for people with beta thalassemia who have not had a bone marrow transplant and do not have a matching sibling donor.
This trial tests a new gene therapy that edits your own blood stem cells to help them make more fetal hemoglobin, which can prevent sickling and reduce the need for transfusions. It is for people with severe sickle cell disease or beta thalassemia who do not have a matched sibling donor.
This trial tests a one-time gene therapy using your own stem cells to help you produce healthy red blood cells and reduce or eliminate the need for regular blood transfusions. It is for people with transfusion-dependent beta-thalassemia who are between 3 and 35 years old.
This trial tests a new gene therapy for people with beta-thalassemia who need regular blood transfusions. It uses a virus to deliver a working gene into your own stem cells, then those cells are put back into your body to help your body make healthy red blood cells.
This trial tests a one-time gene therapy using your own blood stem cells to treat severe beta-thalassemia. It may help you stop needing regular blood transfusions.
This trial tests whether giving you your own (autologous) gene-modified blood stem cells can improve transfusion-dependent beta-thalassemia and its safety. It may help people who do not have a fully matched donor and need an alternative approach to care.
Hear when a new Beta-Thalassemia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.