Treosulfan for stem cell transplant in Nijmegen breakage syndrome
Treatments studied
Part of Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of Nijmegen breakage syndrome through genetic testing.
- You or your legal guardian must sign a consent form agreeing to take part in the study.
- You must be healthy enough overall to undergo a stem cell transplant (no major medical problems that would make it unsafe).
- You cannot have any other DNA repair disorder besides Nijmegen breakage syndrome.
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 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.
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.
This trial tests whether the chemotherapy medicine treosulfan (Trecondi® Ideogen) works well and is safe for people with myeloma who are moving on to high-dose chemotherapy plus their own stem cell transplant. It may help strengthen the transplant “conditioning” treatment before the transplant.
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 a gentler chemotherapy regimen before a stem cell transplant from a partially matched family donor for patients with acute myeloid leukemia (AML) who are older or have health conditions that make standard transplants risky. The goal is to help patients achieve long-term remission with fewer serious side effects.
This trial compares two chemotherapy drugs, treosulfan and melphalan, used before a stem cell transplant in people with AML or MDS. The goal is to see which one works better with a special anti-rejection drug called PTCy.
Hear when a new HSCT trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.