Ignite Creation Date:
2025-12-25 @ 5:06 AM
Ignite Modification Date:
2025-12-26 @ 4:10 AM
Study NCT ID:
NCT06199427
Status:
RECRUITING
Last Update Posted:
2024-01-10
First Post:
2023-12-28
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'C540383', 'term': 'ruxolitinib'}], 'ancestors': [{'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-11-21', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-12', 'completionDateStruct': {'date': '2027-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-12-28', 'studyFirstSubmitDate': '2023-12-28', 'studyFirstSubmitQcDate': '2023-12-28', 'lastUpdatePostDateStruct': {'date': '2024-01-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-01-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-11-21', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Event-free survival', 'timeFrame': '1 year after HSCT', 'description': 'Events: graft failure, death'}], 'secondaryOutcomes': [{'measure': 'Overall survival', 'timeFrame': '1 year after HSCT'}, {'measure': 'Cumulative incidence of acute graft versus host disease', 'timeFrame': '1 year after HSCT'}, {'measure': 'Cumulative incidence of chronic graft versus host disease', 'timeFrame': '1 year after HSCT'}, {'measure': 'Cumulative incidence of engraftment', 'timeFrame': '100 days'}, {'measure': 'Cumulative incidence of graft failure', 'timeFrame': '1 year after HSCT'}, {'measure': 'Incidence of early organ toxicity', 'timeFrame': '100 days'}, {'measure': 'Cumulative incidence of transplant related mortality', 'timeFrame': '1 year after HSCT'}, {'measure': 'Cumulative incidence of viral infections', 'timeFrame': '1 year after HSCT'}, {'measure': 'Investigation of the concentration of ruxolitinib in the blood To investigate the pharmacokinetics of ruxolitinib', 'timeFrame': '1 month after HSCT', 'description': 'The features of pharmacokinetics in children of different ages'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Inborn Errors of Immunity']}, 'descriptionModule': {'briefSummary': '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)', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '0 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients aged ≥ 0 months and \\< 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'}, 'identificationModule': {'nctId': 'NCT06199427', 'briefTitle': 'PTCy and and Ruxolitinib for GVHD Prophylaxis After HSCT With Thymoglobulin in Conditioning Regimen in Patients With Inborn Errors of Immunity', 'organization': {'class': 'OTHER', 'fullName': 'Federal Research Institute of Pediatric Hematology, Oncology and Immunology'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'IEI-PTCy 2023'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'intervention/treatment', 'description': 'Conditioning regimen containing treosulfan 30-42 g/m2, fludarabine 150 mg/mg, thymoglobulin 5 mg/kg and thiotepa 10 mg/kg or melphalan 140 mg/m2\n\nGVHD prophylaxis regimen for matched unrelated (MUD) and matched related donors (MRD):\n\nCyclophosphamide (PTCY) 25 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5\n\nGVHD prophylaxis regimen for mismatched related donor (MMRD):\n\nCyclophosphamide (PTCY) 50 mg/kg/day (days +3, +4) Ruxolitinib 7 mg/m2 from day +5', 'interventionNames': ['Drug: Cyclophosphamide', 'Drug: Ruxolitinib']}], 'interventions': [{'name': 'Cyclophosphamide', 'type': 'DRUG', 'description': 'Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD', 'armGroupLabels': ['intervention/treatment']}, {'name': 'Ruxolitinib', 'type': 'DRUG', 'description': 'Ruxolitinib 7 mg/m2 from day +5 after HSCT', 'armGroupLabels': ['intervention/treatment']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Moscow', 'status': 'RECRUITING', 'country': 'Russia', 'contacts': [{'name': 'Dmitry Balashov, MD, PhD', 'role': 'CONTACT', 'email': 'bala8@yandex.ru', 'phone': '+74956647091'}], 'facility': 'HSCT department', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}], 'centralContacts': [{'name': 'Dmitry Balashov, MD, PhD', 'role': 'CONTACT', 'email': 'bala8@yandex.ru', 'phone': '+74956647091'}, {'name': 'Alexandra Laberko, MD, PhD', 'role': 'CONTACT', 'email': 'alexandra.laberko@gmail.com', 'phone': '74952876570'}], 'overallOfficials': [{'name': 'Dmitry Balashov, MD, PhD', 'role': 'STUDY_CHAIR', 'affiliation': 'National Research Center for Pediatric Hematology, Moscow, Russia'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Federal Research Institute of Pediatric Hematology, Oncology and Immunology', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}