New hope for rare genetic disorder: tailored chemo before stem cell transplant
NCT ID NCT07316595
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether a lower dose of the chemotherapy drug treosulfan, given before a stem cell transplant, can help people with Nijmegen breakage syndrome. The condition causes immune problems and a high risk of cancer. The trial will enroll 24 patients, giving a lower dose to those without cancer and a higher dose to those with cancer, aiming to improve survival and reduce complications.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Treosulfan (a chemotherapy drug)
What this could lead to
If successful, this could make stem cell transplants safer and more effective for people with Nijmegen breakage syndrome, potentially reducing the risk of graft rejection and cancer relapse.
What could go wrong
This is a small, early-phase trial with only 24 participants, so results may not apply to everyone. The drug is still experimental for this condition, and there are risks of serious side effects like graft-versus-host disease or organ toxicity.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
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Contact
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