Ignite Creation Date:
2025-12-25 @ 1:10 AM
Ignite Modification Date:
2026-02-27 @ 7:07 PM
Study NCT ID:
NCT02707393
Status:
COMPLETED
Last Update Posted:
2021-04-15
First Post:
2016-03-01
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Allogeneic Stem Cell Transplantation for Children With CML
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015464', 'term': 'Leukemia, Myelogenous, Chronic, BCR-ABL Positive'}], 'ancestors': [{'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D002908', 'term': 'Chronic Disease'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C024352', 'term': 'fludarabine'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D013852', 'term': 'Thiotepa'}, {'id': 'D008558', 'term': 'Melphalan'}, {'id': 'C512542', 'term': 'thymoglobulin'}, {'id': 'D016572', 'term': 'Cyclosporine'}, {'id': 'D009173', 'term': 'Mycophenolic Acid'}], 'ancestors': [{'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D013721', 'term': 'Triethylenephosphoramide'}, {'id': 'D001388', 'term': 'Aziridines'}, {'id': 'D001389', 'term': 'Azirines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D010649', 'term': 'Phenylalanine'}, {'id': 'D024322', 'term': 'Amino Acids, Aromatic'}, {'id': 'D000598', 'term': 'Amino Acids, Cyclic'}, {'id': 'D000596', 'term': 'Amino Acids'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D003524', 'term': 'Cyclosporins'}, {'id': 'D010456', 'term': 'Peptides, Cyclic'}, {'id': 'D047028', 'term': 'Macrocyclic Compounds'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D002208', 'term': 'Caproates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D005227', 'term': 'Fatty Acids'}, {'id': 'D008055', 'term': 'Lipids'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 13}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-04-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-04', 'completionDateStruct': {'date': '2020-12-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-14', 'studyFirstSubmitDate': '2016-03-01', 'studyFirstSubmitQcDate': '2016-03-11', 'lastUpdatePostDateStruct': {'date': '2021-04-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-03-14', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2020-12-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'transplant related mortality', 'timeFrame': 'one year'}], 'secondaryOutcomes': [{'measure': 'overall survival', 'timeFrame': 'five years'}, {'measure': 'event free survival', 'timeFrame': 'five years'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['stem cell transplantation', 'pediatrics'], 'conditions': ['Chronic Myeloid Leukemia']}, 'descriptionModule': {'briefSummary': 'In children and adolescents with chronic myeloid leukaemia (CML) stem cell transplantation (SCT) may be a valid alternative to the life-long treatment with tyrosinkinase inhibitors (TKI). This trial aims to evaluate the use of a reduced intensity conditioning regimen (RIC), consisting of fludarabine, melphalan and thiotepa in order to minimize transplant related mortality and toxic late effects. Strict post-transplant monitoring and reintroduction of TKI as well as donor lymphocyte infusions (DLI) in case of relevant residual disease are part of the protocol.', 'detailedDescription': 'Chronic myeloid leukaemia (CML) is a rare disease in children with an incidence of 3-5% of all paediatric leukaemias. Since the introduction of tyrosinkinase inhibitors (TKI) stem cell transplantation (SCT) is no longer the first choice treatment for patients with early phase CML.\n\nHowever life-long treatment with TKI may not be feasable in several cases due to side effects such as growth retardation, non-compliance and resistance. This protocol evaluates the feasibility of SCT following a reduced intensity conditioning regimen (RIC) consisting of fludarabine, melphalan, thiotepa and thymoglobuline (ATG). Matched siblings and matched unrelated donors are permitted for stem cell donation. In case of unrelated donors tissue typing has to be done by high resolution molecular typing. Donors with 10/10 or 9/10 identical allels in the human leukocyte antigen (HLA) system are accepted. Preferred stem cell source is bone marrow but peripheral blood stem cells and umbilical cord blood are also allowed. Graft-versus-Host-Disease (GvHD)-prophylaxis is achieved with cyclosporine A and mycophenolate mofetil.\n\nMonitoring of the breakpoint cluster region - Abelson (BCR/ABL) rearrangement is performed monthly in the first year after SCT. In case of BCR/ABL positivity TKI are given in the first year after SCT. Followed by donor lymphocyte infusions (DLI) later on if BCR/ABL positivity persists.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* children and adolescents with BCR/ABL positive CML in chronic phase, who are eligible for allogeneic stem cell transplantation, irrespective of the previous treatment strategy\n* availability of a HLA matched sibling donor (MSD), a matched family donor, a matched unrelated donor or a matched unrelated cord blood (MD)\n* informed consent\n\nExclusion Criteria:\n\n* unavailability of MSD or MD\n* patients in accelerated phase or blast crisis\n* pregnancy\n* previous autologous or allogeneic SCT\n* no informed consent'}, 'identificationModule': {'nctId': 'NCT02707393', 'briefTitle': 'Allogeneic Stem Cell Transplantation for Children With CML', 'organization': {'class': 'OTHER', 'fullName': 'St. Anna Kinderkrebsforschung'}, 'officialTitle': 'Allogeneic Stem Cell Transplantation for Children and Adolescents With CML: Conditioning Regimen, Donor Selection, Supportive Care and Diagnostic Procedures', 'orgStudyIdInfo': {'id': 'EudraCT 2008-000569-50'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'single arm', 'description': 'Fludarabine intravenous - daily dose: 40mg/sqm on day -7, -6, -5, -4; Thiotepa intravenous - daily dose: 2 x 5mg/kg on day -3; Melphalan intravenous - daily dose: 140/mg/sqm on day - 2; ATG intravenous - dose according to local standards on day -3, -2, -1; bone marrow or peripheral blood stem cells of an HLA identical sibling or matched unrelated donor on day 0; GvHD propyhlaxis with Mycophenolate Mofetil and Cyclosporine A', 'interventionNames': ['Drug: Fludarabine', 'Drug: Thiotepa', 'Drug: Melphalan', 'Drug: ATG', 'Drug: Cyclosporine A', 'Drug: Mycophenolate mofetil', 'Biological: bone marrow or peripheral blood stem cells']}], 'interventions': [{'name': 'Fludarabine', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': 'infusion', 'armGroupLabels': ['single arm']}, {'name': 'Thiotepa', 'type': 'DRUG', 'otherNames': ['Thioplex', 'TESPA'], 'description': 'infusion', 'armGroupLabels': ['single arm']}, {'name': 'Melphalan', 'type': 'DRUG', 'otherNames': ['Alkeran'], 'description': 'infusion', 'armGroupLabels': ['single arm']}, {'name': 'ATG', 'type': 'DRUG', 'otherNames': ['Thymoglobulin'], 'description': 'infusion', 'armGroupLabels': ['single arm']}, {'name': 'Cyclosporine A', 'type': 'DRUG', 'otherNames': ['Sandimmune'], 'description': 'infusion, orally if possible', 'armGroupLabels': ['single arm']}, {'name': 'Mycophenolate mofetil', 'type': 'DRUG', 'otherNames': ['CellCept'], 'description': 'infusion', 'armGroupLabels': ['single arm']}, {'name': 'bone marrow or peripheral blood stem cells', 'type': 'BIOLOGICAL', 'description': 'infusion', 'armGroupLabels': ['single arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '8036', 'city': 'Graz', 'country': 'Austria', 'facility': 'Universitätsklinik für Kinder- und Jugendheilkunde', 'geoPoint': {'lat': 47.06733, 'lon': 15.44197}}, {'zip': '1050', 'city': 'Vienna', 'country': 'Austria', 'facility': 'St. Anna Kinderspital', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': '15006', 'city': 'Prague', 'country': 'Czechia', 'facility': 'Hospital Motol, Department of Pediatric Hematology and Oncology, BMT Unit', 'geoPoint': {'lat': 50.08804, 'lon': 14.42076}}, {'zip': '20052', 'city': 'Monza', 'country': 'Italy', 'facility': 'Clinica Pediatrica', 'geoPoint': {'lat': 45.58005, 'lon': 9.27246}}], 'overallOfficials': [{'name': 'Susanne Matthes, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'St. Anna Kinderspital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'St. Anna Kinderkrebsforschung', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}