[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100531409":3},{"organization":4,"outcomesModule":7,"designInfo":36,"detailedDescription":42,"studyPopulation":16,"armGroups":43,"interventions":51,"overallOfficials":61,"centralContacts":66,"locations":75,"responsibleParty":90,"collaborators":16,"id":92,"slug":16,"hasResults":93,"nctId":94,"briefTitle":95,"officialTitle":96,"acronym":16,"eligibilityCriteria":97,"healthyVolunteers":93,"sex":98,"minAge":99,"maxAge":100,"enrollmentInfo":101,"targetDuration":16,"studyType":104,"phases":105,"briefSummary":107,"conditions":108,"keywords":16,"overallStatus":78,"whyStopped":16,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":119},{"fullName":5,"class":6},"Federal Research Institute of Pediatric Hematology, Oncology and Immunology","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":16},[9],{"measure":10,"description":11,"timeFrame":12},"Event-free survival","Events: graft failure, death","1 year after HSCT",[14,17,19,21,24,26,28,30,32],{"measure":15,"description":16,"timeFrame":12},"Overall survival",null,{"measure":18,"description":16,"timeFrame":12},"Cumulative incidence of acute graft versus host disease",{"measure":20,"description":16,"timeFrame":12},"Cumulative incidence of chronic graft versus host disease",{"measure":22,"description":16,"timeFrame":23},"Cumulative incidence of engraftment","100 days",{"measure":25,"description":16,"timeFrame":12},"Cumulative incidence of graft failure",{"measure":27,"description":16,"timeFrame":23},"Incidence of early organ toxicity",{"measure":29,"description":16,"timeFrame":12},"Cumulative incidence of transplant related mortality",{"measure":31,"description":16,"timeFrame":12},"Cumulative incidence of viral infections",{"measure":33,"description":34,"timeFrame":35},"Investigation of the concentration of ruxolitinib in the blood To investigate the pharmacokinetics of ruxolitinib","The features of pharmacokinetics in children of different ages","1 month after HSCT",{"allocation":37,"interventionModel":38,"interventionModelDescription":16,"primaryPurpose":39,"observationalModel":16,"timePerspective":16,"maskingInfo":40},"NA","SINGLE_GROUP","TREATMENT",{"masking":41,"maskingDescription":16,"whoMasked":16},"NONE","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\u002Fm2, fludarabine 150 mg\u002Fmg, and thiotepa 10 mg\u002Fkg or melphalan 140 mg\u002Fm2 and GVHD prophylaxis regimen containing cyclophosphamide 50 mg\u002Fkg for MMRD, 25 mg\u002Fkg for matched unrelated and related donors at days +3, 4 post-HSCT and ruxolitinib at dose 7 mg\u002Fm2 from day +5 after HSCT will be used in patients with IEI.",[44],{"label":45,"type":46,"description":47,"interventionNames":48},"intervention\u002Ftreatment","EXPERIMENTAL","Conditioning regimen containing treosulfan 30-42 g\u002Fm2, fludarabine 150 mg\u002Fmg, thymoglobulin 5 mg\u002Fkg and thiotepa 10 mg\u002Fkg or melphalan 140 mg\u002Fm2\n\nGVHD prophylaxis regimen for matched unrelated (MUD) and matched related donors (MRD):\n\nCyclophosphamide (PTCY) 25 mg\u002Fkg\u002Fday (days +3, +4) Ruxolitinib 7 mg\u002Fm2 from day +5\n\nGVHD prophylaxis regimen for mismatched related donor (MMRD):\n\nCyclophosphamide (PTCY) 50 mg\u002Fkg\u002Fday (days +3, +4) Ruxolitinib 7 mg\u002Fm2 from day +5",[49,50],"Drug: Cyclophosphamide","Drug: Ruxolitinib",[52,57],{"type":53,"name":54,"description":55,"armGroupLabels":56,"otherNames":16},"DRUG","Cyclophosphamide","Cyclophosphamide 25mg\u002Fkg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg\u002Fkg (days +3, +4) after HSCT from MMRD",[45],{"type":53,"name":58,"description":59,"armGroupLabels":60,"otherNames":16},"Ruxolitinib","Ruxolitinib 7 mg\u002Fm2 from day +5 after HSCT",[45],[62],{"name":63,"affiliation":64,"role":65},"Dmitry Balashov, MD, PhD","National Research Center for Pediatric Hematology, Moscow, Russia","STUDY_CHAIR",[67,71],{"name":63,"role":68,"phone":69,"phoneExt":16,"email":70},"CONTACT","+74956647091","bala8@yandex.ru",{"name":72,"role":68,"phone":73,"phoneExt":16,"email":74},"Alexandra Laberko, MD, PhD","74952876570","alexandra.laberko@gmail.com",[76],{"facility":77,"status":78,"city":79,"state":16,"zip":16,"country":80,"countryCode":81,"cosmosGeoPoint":82,"geoPoint":87,"contacts":88},"HSCT department","RECRUITING","Moscow","Russia","RU",{"type":83,"coordinates":84},"Point",[85,86],37.61781,55.75204,{"lat":86,"lon":85},[89],{"name":63,"role":68,"phone":69,"phoneExt":16,"email":70},{"type":91,"investigatorFullName":16,"investigatorTitle":16,"investigatorAffiliation":16,"oldNameTitle":16,"oldOrganization":16},"SPONSOR","100531409",false,"NCT06199427","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","Inclusion Criteria:\n\n1. Patients aged ≥ 0 months and \\\u003C 21 years\n2. Patients diagnosed with NBS eligible for an allogeneic HSCT\n3. Signed written informed consent signed by a parent or legal guardian\n\nExclusion Criteria:\n\nConcomitant severe somatic disease associated with an additional risk of severe complications","ALL","0 Months","21 Years",{"count":102,"type":103},100,"ESTIMATED","INTERVENTIONAL",[106],"PHASE2","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)",[109],"Inborn Errors of Immunity","2023-12-28",{"date":112,"type":113},"2024-01-10","ACTUAL",{"date":115,"type":113},"2023-11-21",{"date":117,"type":103},"2027-12-31",{"name":5,"class":6},1]