Status:
RECRUITING
Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome
Lead Sponsor:
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Conditions:
Nijmegen Breakage Syndrome
Eligibility:
All Genders
3-21 years
Phase:
PHASE2
Brief 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
Detailed 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 myeloabla...
Eligibility Criteria
Inclusion
- 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
Exclusion
Key Trial Info
Start Date :
November 22 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 31 2029
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06198842
Start Date
November 22 2023
End Date
January 31 2029
Last Update
January 10 2024
Active Locations (1)
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1
HSCT department
Moscow, Russia