PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Cyclophosphamide, Ruxolitinib.
- Who it may be relevant to
- Registry conditions: Inborn Errors of Immunity. Basic parameters: 0 months — 21 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Russia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
Overview
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)
Detailed description
Hematopoietic stem cell transplantation (HSCT) is widely used in inborn errors of immunity (IEI), and risks of graft-versus-host disease (GVHD) remain high. Use of post-transplant cyclophosphamide (PTCY) for GVHD prophylaxis revolutionized the outcomes of HSCT from mismatched related donor (MMRD). Use of ruxolitinib for GVHD prophylaxis demonstrates promising results in adult patients. Another well-known option for GVHD prevention is antithymocyte globulin. To evaluate the efficacy of combination of thymoglobulin with PTCY and ruxolitinib for GVHD prophylaxis, conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, and thiotepa 10 mg/kg or melphalan 140 mg/m2 and GVHD prophylaxis regimen containing cyclophosphamide 50 mg/kg for MMRD, 25 mg/kg for matched unrelated and related donors at days +3, 4 post-HSCT and ruxolitinib at dose 7 mg/m2 from day +5 after HSCT will be used in patients with IEI.
Interventions
- Drug Cyclophosphamide
Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD - Drug Ruxolitinib
Ruxolitinib 7 mg/m2 from day +5 after HSCT
Primary outcome measures
- Event-free survival [Time frame: 1 year after HSCT]
Secondary outcome measures (9)
- Overall survival [Time frame: 1 year after HSCT]
- Cumulative incidence of acute graft versus host disease [Time frame: 1 year after HSCT]
- Cumulative incidence of chronic graft versus host disease [Time frame: 1 year after HSCT]
- Cumulative incidence of engraftment [Time frame: 100 days]
- Cumulative incidence of graft failure [Time frame: 1 year after HSCT]
- Incidence of early organ toxicity [Time frame: 100 days]
- Cumulative incidence of transplant related mortality [Time frame: 1 year after HSCT]
- Cumulative incidence of viral infections [Time frame: 1 year after HSCT]
- Investigation of the concentration of ruxolitinib in the blood To investigate the pharmacokinetics of ruxolitinib [Time frame: 1 month after HSCT]
Eligibility criteria
Inclusion criteria
- 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 criteria
Concomitant severe somatic disease associated with an additional risk of severe complications
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Russia · 1 center
- HSCT department — Moscow
Identifiers
NCT: NCT06199427 · IEI-PTCy 2023