Gene therapy for severe beta-thalassemia
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 6 and 35 years old.
- You have a confirmed diagnosis of severe transfusion-dependent beta-thalassemia without other alpha-thalassemia.
- You have needed many blood transfusions (more than 8 per year or more than 100 mL per kg per year) in the last 2 years.
- You have been on full transfusions for at least 3 months and your hemoglobin level is kept at 9.0 g/dL or higher.
- Your iron levels in blood and organs (heart and liver by MRI) are not too high.
- You do not have a fully matched donor or have had prior gene therapy or stem cell transplant.
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 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 (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.
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 early-phase trial studies HGI-002 to see if it’s safe and can help people with severe alpha thalassemia who need frequent blood transfusions. It also requires planning for a special treatment approach that includes busulfan pre-treatment and stem cell transplantation.
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
Hear when a new Thalassemia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.