Gene therapy for beta-thalassemia in children and teens
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene therapy (BD211) for children and teens with severe beta-thalassemia who need regular blood transfusions. The treatment uses your own blood stem cells, modified with a working gene, to help your body make healthy red blood cells and reduce the need for transfusions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 3 and 18 years old.
- You need regular blood transfusions (at least 2 bags of red blood cells each year).
- You don't have a matched bone marrow donor, or you choose not to have a standard bone marrow transplant.
- You have been treated for at least 2 years and have records of your transfusions.
- Your heart, liver, kidneys, and iron levels are in good enough shape (doctors will check).
- You have not had gene or cell therapy before, and you are not taking certain medicines that could interfere.
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 early-phase trial tests a new way to restore working blood-making cells using your own stem cells for people with transfusion-dependent beta thalassemia major. The goal is to improve safety and blood results, and possibly reduce problems from the disease.
This early-stage study tests the safety and possible benefits of an injection called HGI-001 for people with severe transfusion-dependent beta thalassemia. It may help by preparing you for stem cell transplant, but you must meet several health and lab criteria and be able to return often for follow-up tests.
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 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 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 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.
Hear when a new Thalassemia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.