Ignite Creation Date:
2025-12-24 @ 6:26 PM
Ignite Modification Date:
2025-12-28 @ 5:20 PM
Study NCT ID:
NCT00047268
Status:
UNKNOWN
Last Update Posted:
2013-09-17
First Post:
2002-10-03
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D054437', 'term': 'Myelodysplastic-Myeloproliferative Diseases'}, {'id': 'D054429', 'term': 'Leukemia, Myelomonocytic, Juvenile'}, {'id': 'D054438', 'term': 'Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative'}, {'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D015477', 'term': 'Leukemia, Myelomonocytic, Chronic'}], 'ancestors': [{'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'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': 'D003561', 'term': 'Cytarabine'}, {'id': 'D015122', 'term': 'Mercaptopurine'}, {'id': 'D001706', 'term': 'Biopsy'}, {'id': 'D036102', 'term': 'Peripheral Blood Stem Cell Transplantation'}], 'ancestors': [{'id': 'D003562', 'term': 'Cytidine'}, {'id': 'D011741', 'term': 'Pyrimidine Nucleosides'}, {'id': 'D011743', 'term': 'Pyrimidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D001087', 'term': 'Arabinonucleosides'}, {'id': 'D009705', 'term': 'Nucleosides'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D013438', 'term': 'Sulfhydryl Compounds'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D011687', 'term': 'Purines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D003581', 'term': 'Cytodiagnosis'}, {'id': 'D003584', 'term': 'Cytological Techniques'}, {'id': 'D019411', 'term': 'Clinical Laboratory Techniques'}, {'id': 'D019937', 'term': 'Diagnostic Techniques and Procedures'}, {'id': 'D003933', 'term': 'Diagnosis'}, {'id': 'D013048', 'term': 'Specimen Handling'}, {'id': 'D003949', 'term': 'Diagnostic Techniques, Surgical'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D018380', 'term': 'Hematopoietic Stem Cell Transplantation'}, {'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D014180', 'term': 'Transplantation'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'DIAGNOSTIC'}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '1998-07'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2007-07', 'lastUpdateSubmitDate': '2013-09-16', 'studyFirstSubmitDate': '2002-10-03', 'studyFirstSubmitQcDate': '2003-01-26', 'lastUpdatePostDateStruct': {'date': '2013-09-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2003-01-27', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Patient numbers in the different FAB subtypes'}], 'secondaryOutcomes': [{'measure': 'Survival'}, {'measure': 'Event-free survival'}]}, 'conditionsModule': {'keywords': ['juvenile myelomonocytic leukemia', 'childhood myelodysplastic syndromes', 'atypical chronic myeloid leukemia, BCR-ABL1 negative', 'myelodysplastic/myeloproliferative neoplasm, unclassifiable', 'chronic myelomonocytic leukemia'], 'conditions': ['Leukemia', 'Myelodysplastic/Myeloproliferative Neoplasms']}, 'referencesModule': {'references': [{'pmid': '40554414', 'type': 'DERIVED', 'citation': 'Drexler B, Schwarz-Furlan S, Baumann I, Rudelius M, Nollke P, Lebrecht D, Ramamoorthy S, Rotari N, Karow A, Hirabayashi S, Beier F, Behrens YL, Gohring G, Kalb R, Wlodarski MW, Strahm B, Erlacher M, Niemeyer CM, Yoshimi A. Long-term outcomes of patients with refractory cytopenia of childhood under observation only. Blood Adv. 2025 Aug 26;9(16):4279-4285. doi: 10.1182/bloodadvances.2025016136.'}, {'pmid': '29217778', 'type': 'DERIVED', 'citation': 'Pastor VB, Sahoo SS, Boklan J, Schwabe GC, Saribeyoglu E, Strahm B, Lebrecht D, Voss M, Bryceson YT, Erlacher M, Ehninger G, Niewisch M, Schlegelberger B, Baumann I, Achermann JC, Shimamura A, Hochrein J, Tedgard U, Nilsson L, Hasle H, Boerries M, Busch H, Niemeyer CM, Wlodarski MW. Constitutional SAMD9L mutations cause familial myelodysplastic syndrome and transient monosomy 7. Haematologica. 2018 Mar;103(3):427-437. doi: 10.3324/haematol.2017.180778. Epub 2017 Dec 7.'}, {'pmid': '20802024', 'type': 'DERIVED', 'citation': 'Gohring G, Michalova K, Beverloo HB, Betts D, Harbott J, Haas OA, Kerndrup G, Sainati L, Bergstraesser E, Hasle H, Stary J, Trebo M, van den Heuvel-Eibrink MM, Zecca M, van Wering ER, Fischer A, Noellke P, Strahm B, Locatelli F, Niemeyer CM, Schlegelberger B. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood. 2010 Nov 11;116(19):3766-9. doi: 10.1182/blood-2010-04-280313. Epub 2010 Aug 27.'}]}, 'descriptionModule': {'briefSummary': "RATIONALE: Giving chemotherapy before a donor stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether donor stem cell transplant is more effective with or without chemotherapy in treating primary myelodysplastic syndrome.\n\nPURPOSE: This phase III trial is studying how well donor stem cell transplant given with chemotherapy works and compares it with donor stem cell transplant without chemotherapy in treating children with primary myelodysplastic syndrome.", 'detailedDescription': 'OBJECTIVES:\n\n* Determine, by a standard approach, the frequency of different FAB subtypes in children with primary myelodysplastic syndromes.\n* Determine the frequency of cytogenetic and molecular abnormalities in these patients.\n* Determine the survival of patients treated with allogeneic stem cell transplantation with or without induction chemotherapy.\n* Determine the rate of complete remission in patients treated with these regimens.\n* Determine the event-free survival of patients treated with these regimens.\n* Determine the relapse rate, morbidity, and mortality of patients treated with these regimens.\n* Determine different subsets of patients who benefit from these regimens.\n\nOUTLINE: This is a multicenter study. Patients are stratified according to FAB subtype (refractory anemia (RA) or RA with ringed sideroblasts (RARS) vs RA with excess blasts (RAEB) vs RAEB in transformation (RAEB-t) vs juvenile myelomonocytic leukemia (JMML)).\n\nPatients undergo complete medical and physical examination. Patients are screened for the following aberrations: -7, +8, +21, t(8;21), t(15;17), and inv(16). Smears of peripheral blood and bone marrow, as well as bone marrow biopsies and all cytogenetic and molecular studies performed on blood or bone marrow, are evaluated by a panel of international experts.\n\nPatients with progressive RA or RARS undergo allogeneic stem cell transplantation (ASCT) according to EWOG-MDS SCT studies. Patients with stable RA or RARS wait for an optimal donor before undergoing ASCT. Patients with RAEB with fewer than 15% bone marrow blasts undergo ASCT. Patients with RAEB with at least 15% bone marrow blasts and patients with RAEB-t with fewer than 30% bone marrow blasts receive standard acute myeloid leukemia (AML) induction therapy and then undergo ASCT. Patients with RAEB-t with at least 30% bone marrow blasts are considered for standard AML induction therapy.\n\nPatients with advanced JMML undergo evaluation for splenectomy and receive chemotherapy with mercaptopurine and cytarabine every 3-4 weeks (for 1-4 doses). Patients then undergo ASCT.\n\nPatients are followed every 6 months.\n\nPROJECTED ACCRUAL: Not specified'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "DISEASE CHARACTERISTICS:\n\n* Morphologically confirmed primary myelodysplastic syndromes (MDS)\n\n * Diagnosed between July 1, 1998 and June 30, 2002\n* No prior aplastic anemia\n* No prior congenital bone marrow failure syndrome, such as:\n\n * Fanconi's anemia\n * Kostmann syndrome\n * Shwachman syndrome\n * Dyskeratosis congenital\n * Amegakaryocytic thrombocytopenia\n * Diamond-Blackfan anemia\n* No Down syndrome\n* None of the following cytogenetic or molecular abnormalities:\n\n * t(8;21)(q22;q22)\n * t(15;17)(q22;q12)\n * inv(16)(p13;q22)\n* No typical clinical and cytogenetic features of acute myeloid leukemia FAB M7 (i.e., acute megakaryocytic leukemia) with fewer than 30% blasts in bone marrow or peripheral blood\n\nPATIENT CHARACTERISTICS:\n\nAge\n\n* Under 19\n\nPerformance status\n\n* Not specified\n\nLife expectancy\n\n* Not specified\n\nHematopoietic\n\n* See Disease Characteristics\n\nHepatic\n\n* Not specified\n\nRenal\n\n* Not specified\n\nOther\n\n* No other concurrent illness that would preclude study\n\nPRIOR CONCURRENT THERAPY:\n\nBiologic therapy\n\n* Not specified\n\nChemotherapy\n\n* No prior chemotherapy for MDS\n\nEndocrine therapy\n\n* Not specified\n\nRadiotherapy\n\n* No prior radiotherapy for MDS\n\nSurgery\n\n* Not specified"}, 'identificationModule': {'nctId': 'NCT00047268', 'briefTitle': 'Donor Stem Cell Transplant With or Without Chemotherapy in Treating Children With Primary Myelodysplastic Syndrome', 'organization': {'class': 'NIH', 'fullName': 'National Cancer Institute (NCI)'}, 'officialTitle': 'Prospective Study of the Diagnosis and Treatment of Myelodysplastic Syndromes (MDS) in Childhood', 'orgStudyIdInfo': {'id': 'CDR0000257581'}, 'secondaryIdInfos': [{'id': 'EWOG-MDS-98'}, {'id': 'EU-20218'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'cytarabine', 'type': 'DRUG'}, {'name': 'mercaptopurine', 'type': 'DRUG'}, {'name': 'laboratory biomarker analysis', 'type': 'OTHER'}, {'name': 'allogeneic bone marrow transplantation', 'type': 'PROCEDURE'}, {'name': 'biopsy', 'type': 'PROCEDURE'}, {'name': 'peripheral blood stem cell transplantation', 'type': 'PROCEDURE'}]}, 'contactsLocationsModule': {'locations': [{'zip': 'D-79106', 'city': 'Freiburg im Breisgau', 'country': 'Germany', 'facility': 'Universitaetskinderklinik - Universitaetsklinikum Freiburg', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}], 'overallOfficials': [{'name': 'Charlotte Niemeyer, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Universitaetskinderklinik - Universitaetsklinikum Freiburg'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'European Working Group of MDS in Childhood', 'class': 'OTHER'}}}}