Gene-edited stem cell therapy for beta-thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new treatment that uses your own blood stem cells, edited with CRISPR technology, to reduce or eliminate the need for blood transfusions in people with beta-thalassemia.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 3 and 35 years old.
- You have a confirmed diagnosis of transfusion-dependent beta-thalassemia and have needed frequent blood transfusions (at least 8 times a year or a certain amount of red blood cells) over a 12-month period.
- You are generally healthy enough for a stem cell transplant, as judged by your doctor.
- You have not had a bone marrow transplant or gene therapy before.
- You do not have a fully matched donor available for a standard bone marrow transplant.
- You are willing to use effective contraception for 12 months after treatment if you could become pregnant or father a child.
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-editing treatment called CS-101 for people with beta-thalassemia major who need regular blood transfusions. It aims to see if the treatment is safe and can reduce or eliminate the need for 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.
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 treatment that uses your own (autologous) stem cells that have been changed by gene methods, then returned to you to help manage transfusion-dependent beta-thalassemia. It aims to check safety and whether it can reduce the need for frequent blood transfusions or improve blood health.
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 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.
Hear when a new Thalassemia Major trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.