Tests chemotherapy in children with acute myeloid leukemia and Down syndrome
Tests 4 chemotherapy courses in children with leukemia and Down syndrome.
Plain-language title generated from the registry record
Registry title: The Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia
Key facts
- Where
- Moscow, RussiaSee the site
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children and teenagers with Down syndrome who have acute myeloid leukemia (AML) or MDS. They would get four blocks of chemotherapy.
- Age
- 0 to 18 years (from 1 day old to 18 years)
- Sex
- All sexes can join.
- Disease stage
- AML or MDS in a person with Down syndrome (constitutional trisomy 21 and a GATA1 gene mutation).
- Earlier treatment
- Cannot have had more than 14 days of intensive induction therapy before joining.
- Biomarker or test result
- Constitutional trisomy 21 and a mutation in the GATA1 gene.
Main reasons for not taking part
- Down syndrome with acute lymphoblastic leukemia (ALL)
- Severe other illnesses that make the protocol treatment unsafe
- More than 14 days of intensive induction therapy before joining
- Refusal of all therapy or important parts of the therapy
This trial only enrols participants with Down syndrome. The registry sentence this rests on: Diagnosis of AML, MDS and presence of Down syndrome (constitutional trisomy 21 and mutation in the GATA1 gene)
Read by machine on 12 November 2026.
Where in Russia
- National medical research center of pediatric haematology, oncology and immulogy named after Dmytriy Rogachyov, Moscow, 117198Moscow, RussiaContact: Lena f SmirnovaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- Trial code
- NCT07232134
- Record updated
- 20 November 2025
- Original description
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.Read full original descriptionShow less
Prospective Non-randomized Multicenter Trial: the Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia
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
Inclusion: 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.
Exclusion: Pre-treatment >14 days with intensive induction therapy; Refusal of all therapy or important elements of therapy; Children with Down syndrome and acute lymphoblastic leukemia (ALL); Severe comorbidities with contraindications to the treatment according to the protocol.