RecruitingPhase 3NCT07232134

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

Prospective Non-randomized Multicenter Trial: the Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia


Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Enrollment

100 participants

Start Date

Jul 24, 2025

Study Type

INTERVENTIONAL

Conditions

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


Eligibility

Min Age: 1 DayMax Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study in Russia is evaluating treatments for children with Down syndrome who have been diagnosed with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) — blood cancers where bone marrow makes abnormal blood cells. Children with Down syndrome have a higher risk of these cancers and may respond differently to treatment. **You may be eligible if...** - Your child is between 0 and 18 years old - Your child has Down syndrome (trisomy 21) and a GATA1 gene mutation - Your child has been diagnosed with AML or MDS - A parent or guardian has signed the consent form **You may NOT be eligible if...** - Your child has acute lymphoblastic leukemia (ALL) rather than AML or MDS - Your child has severe other medical conditions that prevent treatment - Your child has already received more than 14 days of intensive chemotherapy before enrollment - You or your child refuses key parts of the treatment Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUG4 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: 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).


Locations(1)

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

Moscow, Russia

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NCT07232134


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