Clinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage Syndrome
Who is this study for? Child to young adult patients with Nijmegan Breakage Syndrome
What treatments are being studied? Treosulfan
Status: Unknown
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 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
+74952876570
Backup
Alexandra Laberko, MD
alexandra.laberko@gmail.com
74952876570
Time Frame
Start Date: 2020-05-22
Completion Date: 2023-05
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) Cyclophosphamide 40mg/kg (days -3, -2) Thymoglobulin (Genzyme) 5mg/kg (days -5, -4) Rituximab 100mg/m2 (day -1) Stem cell infusion - day 0
Related Therapeutic Areas
Sponsors
Leads: Federal Research Institute of Pediatric Hematology, Oncology and Immunology