Low-dose treosulfan with PTCy for Nijmegen Breakage Syndrome transplant
Treatments studied
Part of Hormones & metabolism clinical trials.
This trial tests a low-dose chemotherapy regimen (treosulfan) combined with a drug called PTCy to prepare children with Nijmegen Breakage Syndrome for a stem cell transplant. The goal is to reduce side effects while helping the donor cells take hold safely.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 3 months and 21 years old
- You have been diagnosed with Nijmegen Breakage Syndrome (NBS)
- Your doctor has determined you need a stem cell transplant from a donor
- A parent or legal guardian has signed a consent form agreeing to the study
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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This trial tests a chemotherapy drug called treosulfan as part of the preparation for a stem cell transplant (a type of treatment that replaces your bone marrow) in people with Nijmegen breakage syndrome. The goal is to see if this approach is safe and works well.
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This study watches how the chemotherapy drug Treosulfan works in children getting a stem cell transplant. The goal is to make sure the dose is just right for each child.
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This Phase 2 study tests a specific chemotherapy combination (treosulfan, fludarabine, and thiotepa) before an unrelated or matched donor stem cell transplant. It may help people with certain non-cancer blood and immune disorders by trying to reset the immune system using donor stem cells.
This trial tests whether a gentler (reduced-intensity) conditioning treatment before a stem cell transplant works for non-cancer diseases. It uses specific donor stem cell matches and checks heart, lung, liver, and kidney function to help lower transplant stress while still aiming for benefit.
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