Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT 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
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
10 participants
Nov 22, 2023
INTERVENTIONAL
Conditions
Summary
The aim of the current study is to evaluate the safety and efficacy of low dose treosulfan based conditioning regimen in HSCT with post-transplant cyclophosphamide in Nijmegen breakage syndrome
Eligibility
Inclusion Criteria3
- Patients aged ≥ 3 months and < 21 years
- Patients diagnosed with NBS eligible for an allogeneic HSCT
- Signed written informed consent signed by a parent or legal guardian
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Interventions
Treosulfan 21mg/m2 (days -6, -5, -4)
Locations(1)
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NCT06198842