Gentler chemo tested for rare genetic disorder to make Life-Saving transplant safer
NCT ID NCT07316595
Summary
This study is testing a gentler chemotherapy drug called treosulfan before a stem cell transplant for patients with Nijmegen breakage syndrome, a rare genetic disorder. The goal is to see if using lower doses can reduce severe side effects and transplant-related risks while still effectively controlling the immune problems and cancer risk associated with the disease. The study will include 24 patients and will give different doses based on whether a patient already has cancer.
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Conditions
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