Ignite Creation Date:
2025-12-25 @ 2:24 AM
Ignite Modification Date:
2026-02-28 @ 8:47 PM
Study NCT ID:
NCT07232134
Status:
RECRUITING
Last Update Posted:
2025-11-20
First Post:
2025-11-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015470', 'term': 'Leukemia, Myeloid, Acute'}, {'id': 'D004314', 'term': 'Down Syndrome'}], 'ancestors': [{'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003561', 'term': 'Cytarabine'}, {'id': 'D015255', 'term': 'Idarubicin'}, {'id': 'D005047', 'term': 'Etoposide'}, {'id': 'D003630', 'term': 'Daunorubicin'}], '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': 'D018943', 'term': 'Anthracyclines'}, {'id': 'D009279', 'term': 'Naphthacenes'}, {'id': 'D011084', 'term': 'Polycyclic Aromatic Hydrocarbons'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D000617', 'term': 'Aminoglycosides'}, {'id': 'D006027', 'term': 'Glycosides'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D011034', 'term': 'Podophyllotoxin'}, {'id': 'D013764', 'term': 'Tetrahydronaphthalenes'}, {'id': 'D009281', 'term': 'Naphthalenes'}, {'id': 'D005960', 'term': 'Glucosides'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-07-24', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2032-12-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-17', 'studyFirstSubmitDate': '2025-11-14', 'studyFirstSubmitQcDate': '2025-11-14', 'lastUpdatePostDateStruct': {'date': '2025-11-20', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-11-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2030-07-24', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'event-free survival', 'timeFrame': 'up to 2 years', 'description': 'Response rate; event-free survival (EFS)'}, {'measure': 'relapse-free survival (RFS)', 'timeFrame': 'up to 2 years'}, {'measure': 'overall survival (OS)', 'timeFrame': 'up to 2 years'}, {'measure': 'treatment-related mortality', 'timeFrame': 'up to 2 years'}], 'secondaryOutcomes': [{'measure': 'CTCAE', 'timeFrame': 'up to 2 years', 'description': 'Number of adverse events by Common Terminology Criteria for Adverse Events (CTCAE) 5.0'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['AML', 'Acute Myelogenous Leukemia', 'Down Syndrome', 'DS'], 'conditions': ['AML (Acute Myelogenous Leukemia)', 'Down Syndrome (DS)']}, 'descriptionModule': {'briefSummary': 'This prospective non-randomized multicenter trial created based on protocol ML DS 2006 and aimed at standardization of current therapy approaches and creating a national network for diagnostic, treatment and monitoring of children (0-18 years) with AML and Down syndrome in Russia. Based on the results the investigators expect to increase long-term overall and event-free survival in children with AML and DS and reduce the immediate and remote toxicity of chemotherapy by reducing the dose load of chemotherapeutic drugs.\n\nThe study protocol therapy for all patients includes four chemotherapy blocks:\n\nCourse 1 AIE (cytarabine/idarubicin/etoposide) Course 2 AI (cytarabine/idarubicin) Course 3 HAD (high -dose cytarabine (1g)/daunorubicin) Course 4 HA (high-dose cytarabine) Safety to be monitored based on CTCAE v5.0', 'detailedDescription': 'This prospective non-randomized multicenter trial created based on protocol ML DS 2006 and aimed at standardization of current therapy approaches and creating a national network for diagnostic, treatment and monitoring of children (0-18 years) with AML and Down syndrome in Russia. Based on the results the investigators expect to increase long-term overall and event-free survival in children with AML and DS and reduce the immediate and remote toxicity of chemotherapy by reducing the dose load of chemotherapeutic drugs.\n\nPatients to be included: Age 0-18 years\n\n* Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)\n* Signed informed consent\n\nPatients not be included:\n\n* Children with Down syndrome and acute lymphoblastic leukemia (ALL)\n* Severe comorbidities with contraindications to the treatment according to the protocol\n* Pre-treatment \\>14 days with intensive induction therapy\n* Refusal of all therapy or important elements of therapy\n\nNB: Patients with Transient Abnormal Myelopoiesis (TAM) are eligible for enrollment but will be included in the analysis as a separate group.\n\nPatients with M7 AML, trisomy 21 in the tumor clone (without the Down syndrome phenotype) and mutation in the GATA1 gene should be discussed individually with the protocol group to determine the treatment strategy, as patients with M7 AML and trisomy 21 in tumor clone (without the Down syndrome phenotype) are not included in current version of pediatric AML clinical recommendations.\n\nIf a patient meets the inclusion criteria, but cannot/should not receive treatment according to the protocol, he/she can be enrolled in the study as a patient under observation. The results of therapy of patients under observation will not be taken into account in the assessment of treatment efficacy.\n\nThe study protocol therapy for all patients includes four chemotherapy blocks:\n\nCourse 1 AIE (cytarabine/idarubicin/etoposide) Course 2 AI (cytarabine/idarubicin) Course 3 HAD (high-dose cytarabine (1g)/daunorubicin) Course 4 HA (high-dose cytarabine)/ Efficacy parameters include Response rate; event-free survival (EFS); relapse-free survival (RFS); overall survival (OS); treatment-related mortality, myelogram, MRD Safety to be monitored based on CTCAE v5.0 The AML-DS-2025 protocol does not include a maintenance therapy course in order to reduce toxicity and improve quality of life.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '1 Day', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age 0-18 years\n* Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)\n* Signed informed consent\n\nExclusion Criteria:\n\n* Children with Down syndrome and acute lymphoblastic leukemia (ALL)\n* Severe comorbidities with contraindications to the treatment according to the protocol\n* Pre-treatment \\>14 days with intensive induction therapy\n* Refusal of all therapy or important elements of therapy'}, 'identificationModule': {'nctId': 'NCT07232134', 'acronym': 'AML-DS-2025', 'briefTitle': 'The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia', 'organization': {'class': 'OTHER', 'fullName': 'Federal Research Institute of Pediatric Hematology, Oncology and Immunology'}, 'officialTitle': 'Prospective Non-randomized Multicenter Trial: the Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia', 'orgStudyIdInfo': {'id': 'NCHPOI- 2025-8'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': '4 chemotherapy blocks: cytarabine/idarubicin/etoposide, cytarabine/idarubicin, high-dose cytarabine', 'interventionNames': ['Drug: 4 chemotherapy blocks: Course 1 AIE (cytarabine/idarubicin/etoposide), Course 2 AI (cytarabine/idarubicin), Course 3 HAD (high-dose cytarabine (1g)/daunorubicin), Course 4 HA (high-dose cytarabine).']}], 'interventions': [{'name': '4 chemotherapy blocks: Course 1 AIE (cytarabine/idarubicin/etoposide), Course 2 AI (cytarabine/idarubicin), Course 3 HAD (high-dose cytarabine (1g)/daunorubicin), Course 4 HA (high-dose cytarabine).', 'type': 'DRUG', 'description': '4 chemotherapy blocks: Course 1 AIE (cytarabine/idarubicin/etoposide), Course 2 AI (cytarabine/idarubicin), Course 3 HAD (high-dose cytarabine (1g)/daunorubicin), Course 4 HA (high-dose cytarabine).', 'armGroupLabels': ['4 chemotherapy blocks: cytarabine/idarubicin/etoposide, cytarabine/idarubicin, high-dose cytarabine']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Moscow', 'status': 'RECRUITING', 'country': 'Russia', 'contacts': [{'name': 'Lena f Smirnova', 'role': 'CONTACT', 'email': 'lena.smirnova@dgoi.ru', 'phone': '+7(985)130-61-03'}], 'facility': 'National medical research center of pediatric haematology, oncology and immulogy named after Dmytriy Rogachyov, Moscow, 117198', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}], 'centralContacts': [{'name': 'Zalina A Abashidze, MD', 'role': 'CONTACT', 'email': 'zalina.abashidze@dgoi.ru', 'phone': '89661101770'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Federal Research Institute of Pediatric Hematology, Oncology and Immunology', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}