Gene-modified own stem cell treatment for transfusion-dependent thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You’re age 3 to 18, and your parent/guardian can understand the study and sign consent
- You have confirmed transfusion-dependent beta-thalassemia (based on blood tests) and no alpha-chain genetic problems
- You need regular red-blood-cell transfusions (at least 100 mL/kg per year), and your iron chelation (iron-reducing medicine) is stable
- You have no suitable volunteer donor for a bone marrow transplant that matches tissue types, or you are not eligible for that transplant
- Your organs (heart, liver, kidneys, lungs) are healthy enough based on the study’s tests
- You have complete records of transfusions and follow-up for at least the past 2 years before screening
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 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 (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 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.
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