RecruitingPhase 2NCT06199427

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

Safety and Efficacy of Cyclophosphamide and Ruxolitinib for Graft-versus-host-disease Prophylaxis After Hematopoietic Stem Cell Transplantation With Thymoglobulin Serotherapy in Conditioning Regimen in Patients With Inborn Errors of Immunity


Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Enrollment

100 participants

Start Date

Nov 21, 2023

Study Type

INTERVENTIONAL

Conditions

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)


Eligibility

Min Age: 0 MonthsMax Age: 21 Years

Inclusion Criteria3

  • Patients aged ≥ 0 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 Criteria1

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

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Interventions

DRUGCyclophosphamide

Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD

DRUGRuxolitinib

Ruxolitinib 7 mg/m2 from day +5 after HSCT


Locations(1)

HSCT department

Moscow, Russia

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NCT06199427


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