PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity

• Status: Recruiting
• Trial end date: 2027-12-31
• Target enrollment: 0
• Participant gender: All

Summary

The aim of the current study is to evaluate the efficacy of combined regimen of GVHD prophylaxis with thymoglobulin in conditioning regimen and PTCY with ruxolitinib used after HSCT in patients with inborn errors of immunity (IEI)

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

Details

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

Criteria

Inclusion Criteria:

1. Patients aged ≥ 0 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

Exclusion Criteria:

Concomitant severe somatic disease associated with an additional risk of severe complications