Low dose treosulfan treatment for Nijmegen Breakage Syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the safety and efficacy of a low dose treosulfan-based conditioning regimen combined with post-transplant cyclophosphamide in patients with Nijmegen Breakage Syndrome (NBS) undergoing hematopoietic stem cell transplantation (HSCT). NBS is a DNA repair disorder, and standard conditioning regimens can lead to high morbidity and mortality. The study aims to use a specific combination of treosulfan, fludarabine, thymoglobulin, and rituximab to minimize toxicity while assessing event-free survival as the primary endpoint. The goal is to improve outcomes for patients with this rare condition.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Patients aged ≥ 3 months and \< 21 years
2. Patients diagnosed with NBS eligible for an allogeneic HSCT
3. Signed written informed consent signed by a parent or legal guardian

Where it's running

• HSCT department
  Moscow, Russia Recruiting

Study contacts

• Study coordinator: Dmitry Balashov, MD, PhD
• Email: bala8@yandex.ru
• Phone: +74956647091

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.