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

ConditionInborn Errors of Immunity
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age0-21
SponsorFederal Research Institute of Pediatric Hematology, Oncology and Immunology

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

  • Cyclophosphamide

    Drug

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

  • Ruxolitinib

    Drug

    Ruxolitinib 7 mg/m2 from day +5 after HSCT

Treatment groups

100 Participants
are divided into 1 treatment group
Group A: intervention/treatmentExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Event-free survival

Events: graft failure, death

Time frame
1 year after HSCT

Secondary outcomes

1

Overall survival

Time frame
1 year after HSCT
2

Cumulative incidence of acute graft versus host disease

Time frame
1 year after HSCT
3

Cumulative incidence of chronic graft versus host disease

Time frame
1 year after HSCT
4

Cumulative incidence of engraftment

Time frame
100 days

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Lead sponsor

Source ClinicalTrials.gov
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