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

• Status: Recruiting
• Trial end date: 2029-01-31
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
• Treatments: Treosulfan

Criteria

Inclusion Criteria:

1. Patients aged ≥ 3 months and < 21 years

2. Patients diagnosed with NBS eligible for an allogeneic HSCT

3. Signed written informed consent signed by a parent or legal guardian