New hope for rare genetic disorder: tailored chemo before stem cell transplant

NCT ID NCT07316595

Not yet recruiting Disease control Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HSCT are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Nijmegen breakage syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HSCT NIJMEGEN BREAKAGE SYNDROME TREOSULFAN BASED CONDITIONING

More trials for these conditions

Other studies related to the condition(s) this trial covers.