The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia

NCT07232134 | Recruiting Phase 3

• 1 other identifier: NCHPOI- 2025-8
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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. The study protocol therapy for all patients includes four 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) Safety to be monitored based on CTCAE v5.0

Conditions

AML (Acute Myelogenous Leukemia); Down Syndrome (DS)

Keywords

AML; Acute Myelogenous Leukemia; Down Syndrome; DS

Outcome Measures

Primary Outcomes (4)

• event-free survival

  Response rate; event-free survival (EFS)

  up to 2 years

• relapse-free survival (RFS)

  up to 2 years

• overall survival (OS)

  up to 2 years

• treatment-related mortality

  up to 2 years

Secondary Outcomes (1)

• CTCAE

  up to 2 years

Study Arms (1)

4 chemotherapy blocks: cytarabine/idarubicin/etoposide, cytarabine/idarubicin, high-dose cytarabine

OTHER

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

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). 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).

4 chemotherapy blocks: cytarabine/idarubicin/etoposide, cytarabine/idarubicin, high-dose cytarabine

Eligibility Criteria

• Age: 1 Day - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 0-18 years
• Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)
• Signed informed consent

You may not qualify if:

• Children with Down syndrome and acute lymphoblastic leukemia (ALL)
• Severe comorbidities with contraindications to the treatment according to the protocol
• Pre-treatment \>14 days with intensive induction therapy
• Refusal of all therapy or important elements of therapy

Sponsors & Collaborators

• Federal Research Institute of Pediatric Hematology, Oncology and Immunology: lead

Study Sites (1)

National medical research center of pediatric haematology, oncology and immulogy named after Dmytriy Rogachyov, Moscow, 117198

Moscow, Russia

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Down Syndrome

Interventions

Cytarabine; Idarubicin; Etoposide; Daunorubicin

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Anthracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Aminoglycosides; Glycosides; Carbohydrates; Podophyllotoxin; Tetrahydronaphthalenes; Naphthalenes; Glucosides

Central Study Contacts

Zalina A Abashidze, MD

CONTACT

89661101770; zalina.abashidze@dgoi.ru

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2025
• First Posted: November 18, 2025
• Study Start: July 24, 2025
• Primary Completion (Estimated): July 24, 2030
• Study Completion (Estimated): December 30, 2032
• Last Updated: November 20, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

RU (1)