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Clinical Trial Summary

The aim of the current study is to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in HSCT in Nijmegen breakage syndrome


Clinical Trial Description

Nijmegen breakage syndrome (NBS) is a DNA repair disorder. The only curative option for combine immunodeficiency in NBS is allogeneic hematopoietic stem cell transplantation (HSCT). Standard myeloablative conditioning regimens in DNA repair disorders lead to increased morbidity and mortality after HSCT. Low doses of alkylators are used to reduce toxicity rates, which, however, increase the risks of mixed chimerism and graft failure. The data of treosulfan usage in NBS are sparse. To evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in NBS, treosulfan 21g/m2 in combination with fludarabine 150mg/mg, cyclophosphamide 40mg/kg, thymoglobulin (Genzyme) 5mg/kg and rituximab 100mg/m2 will be used from day -6 to -1 day, followed by stem cell infusion. The primary endpoint is event-free survival, where graft failure, death, and malignancies are considered as events. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04400045
Study type Interventional
Source Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Contact Dmitry Balashov, MD, PhD
Phone +74952876570
Email bala8@yandex.ru
Status Recruiting
Phase Phase 2
Start date May 22, 2020
Completion date May 2023

See also
  Status Clinical Trial Phase
Recruiting NCT06198842 - Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome Phase 2