Low dose treosulfan treatment for Nijmegen Breakage Syndrome
Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Children Nijmegen Breakage Syndrome
This study is testing a gentler treatment plan using low dose treosulfan for patients with Nijmegen Breakage Syndrome who are getting a stem cell transplant to see if it helps them have better outcomes with fewer side effects.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 3 Months to 21 Years |
| Sex | All |
| Sponsor | Federal Research Institute of Pediatric Hematology, Oncology and Immunology Academic / other |
| Drugs / interventions | cyclophosphamide, rituximab, fludarabine |
| Locations | 1 site (Moscow) |
| Trial ID | NCT06198842 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are patients aged 3 months to 21 years diagnosed with Nijmegen Breakage Syndrome who are eligible for allogeneic HSCT.
Not a fit: Patients outside the specified age range or those not diagnosed with Nijmegen Breakage Syndrome may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a safer and more effective treatment option for patients with Nijmegen Breakage Syndrome undergoing HSCT.
How similar studies have performed: While data on treosulfan usage in NBS are sparse, similar approaches in other DNA repair disorders have shown promise, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Moscow
- HSCT department — Moscow, Russia (Recruiting)
Study contacts
- Study coordinator: Dmitry Balashov, MD, PhD
- Email: bala8@yandex.ru
- Phone: +74956647091
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.