Autologous stem cell treatment to improve beta thalassemia major
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 8 and 16 years old, and you/your parent can consent
- You have a confirmed diagnosis of beta thalassemia major that requires regular blood transfusions
- In the 3 months before stem cell collection, your hemoglobin is at least 9 g/dL
- Your iron level in blood tests is not too high (ferritin at or below 3000 ng/mL), and liver iron is mild or not present
- You must be able to follow the trial plan and come to regular check-ups for up to 2 years after transplant
- You have no past gene therapy or bone marrow transplant from a different donor
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 (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-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 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 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 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.
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