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
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 3 months
Maximum Age: 21
Healthy Volunteers: f
View:
• 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
Locations
Other Locations
Russian Federation
HSCT department
RECRUITING
Moscow
Contact Information
Primary
Dmitry Balashov, MD, PhD
bala8@yandex.ru
+74956647091
Backup
Alexandra Laberko, MD, PhD
alexandra.laberko@gmail.com
+74952876570
Time Frame
Start Date: 2023-11-22
Estimated Completion Date: 2029-01-31
Participants
Target number of participants: 10
Treatments
Experimental: intervention/treatment
Fludarabine 150mg/m2 (days -6, -5, -4, -3, -2) Treosulfan 21g/m2 (days -6, -5, -4) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Cyclophosphamide 50mg/kg (days +3, +4)
Related Therapeutic Areas
Sponsors
Leads: Federal Research Institute of Pediatric Hematology, Oncology and Immunology