Prospective Non-randomized Multicenter Trial: the Efficacy of Therapy in Patients With Acute Myeloid Leukemia and Down Syndrome in Russia
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Enrollment
- 100
- Locations
- 1
- Primary Endpoint
- overall survival (OS)
Study Overview
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
Detailed 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.
Patients to be included: 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
Patients not be included:
- 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
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 1 Day to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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 Criteria
- •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
Outcomes
Primary Outcomes
overall survival (OS)
Time Frame: up to 2 years
event-free survival
Time Frame: up to 2 years
Response rate; event-free survival (EFS)
relapse-free survival (RFS)
Time Frame: up to 2 years
treatment-related mortality
Time Frame: up to 2 years
Secondary Outcomes
- CTCAE(up to 2 years)