Lower-toxicity bone marrow conditioning for high-risk thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a modified, lower-toxicity preparation (conditioning) before a bone marrow transplant in children and teens with high-risk thalassemia. It aims to reduce treatment side effects while still allowing transplant success for people who need a donor.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Ages 1–18 with transfusion-dependent alpha or beta thalassemia (with a confirmed 3 or 4 gene deletion)
- Thalassemia type must be confirmed by genetic (DNA) testing
- You must have at least one high-risk feature (for example: age over 7, enlarged liver, poor iron control, severe antibody reactions, or trouble tolerating iron chelation)
- You must have recent iron testing (blood ferritin and heart/liver MRI within 6 months; liver stiffness scan within 3 months if done)
- A suitable family donor is needed (an HLA-matched sibling or a close HLA half-match donor, approved to give stem cells)
- You must be able to take and stick with oral study medicines and be well enough for transplant (Karnofsky/Lansky score at least 70%)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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