Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage Syndrome

  • STATUS
    Recruiting
  • participants needed
    10
  • sponsor
    Federal Research Institute of Pediatric Hematology, Oncology and Immunology
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Updated on 5 August 2020
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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

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.

Details
Condition Nijmegen Breakage Syndrome
Age 1years - 21years
Treatment Treosulfan
Clinical Study IdentifierNCT04400045
SponsorFederal Research Institute of Pediatric Hematology, Oncology and Immunology
Last Modified on5 August 2020

Eligibility

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Inclusion Criteria

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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