Status:
UNKNOWN
Low Dose Treosulfan Based Conditioning Regimen 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 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 :
May 22 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2023
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04400045
Start Date
May 22 2020
End Date
May 1 2023
Last Update
June 25 2020
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
HSCT department
Moscow, Russia, 117198