Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia

Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia: A Multicenter, Prospective Study

This clinical trial is an open-label, multicenter, prospective phase 2 clinical trial targeting pediatric leukemia patients of infant age. The goal is to improve survival rates by varying the presence or absence of chemotherapy and hematopoietic stem cell transplantation based on genetic characteristics at the time of diagnosis and minimal residual disease (MRD) values measured by various methods after treatment.

In addition, by clearly defining the patient group that requires hematopoietic stem cell transplantation, it is expected that the role of hematopoietic stem cell transplantation in infantile leukemia, for which there have been various guidelines for hematopoietic stem cell transplantation, can be confirmed. Additionally, due to the characteristics of infants, this study aim to identify long-term sequelae or prognosis related to treatment by prospectively collecting side effect data related to treatment during and after treatment.

Study Overview

• Status: Recruiting
• Conditions: Leukemia, Lymphoid
• Intervention / Treatment: Drug: Consolidation #4(without daunorubicin); Drug: Consolidation #4(with daunorubicin); Drug: Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin)

Detailed Description

Infant leukemia patients are classified into low/intermediate/high risk groups and hematopoietic stem cell transplantation is performed after chemotherapy or chemotherapy as shown in the schema below.

• Low risk group : Induction chemotherapy-Low Risk Consolidation chemotherapy 1~4 - Maintenance chemotherapy
• Intermediate risk group : Induction chemotherapy-High Risk Consolidation chemotherapy 1~4 - Maintenance chemotherapy
• High risk group : Induction chemotherapy-High Risk Consolidation chemotherapy 1~4 - hematopoietic stem cell transplantation

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 2

Contacts and Locations

Study Locations

• Korea, Republic of
  • Hwasun, Korea, Republic of
    • Recruiting
    • Chonnam National University Hwasun Hospital
    • Contact: Hee Jo Baek; Phone Number: +82-61-379-8060; Email: swan93@naver.com
  • Jeju, Korea, Republic of
    • Recruiting
    • Jeju National University Hospital
    • Contact: Hyun Sik Kang; Phone Number: +82-64-717-1528; Email: medyapsb@naver.com
  • Pusan, Korea, Republic of
    • Recruiting
    • Pusan National University Yangsan Hospital
    • Contact: Eu Jeen Yang; Phone Number: +82-10-6478-8489; Email: 41sirius@hanmail.net
  • Seoul, Korea, Republic of
    • Recruiting
    • Asan Medical Center
    • Contact: Ho Joon Im, MD; Phone Number: +82-2-3010-3371; Email: hojim@amc.seoul.kr
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul National University Hospital
    • Contact: Hyoung Jin Kang, MD; Phone Number: +82-2-2072-3452; Email: kanghj@snu.ac.kr
  • Seoul, Korea, Republic of
    • Recruiting
    • Severance Hospital
    • Contact: seung min Hahn, MD; Phone Number: +82-2-2228-2050; Email: bluenile88@yuhs.ac
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul Saint Mary's Hospital
    • Contact: Jae Wook Lee, MD; Phone Number: +82-2-2258-6192; Email: dashwood@catholic.ac.kr
  • Seoul, Korea, Republic of
    • Recruiting
    • Samsung Medical Cente
    • Contact: Hee Young Ju, MD; Phone Number: +82-2-3410-0865; Email: heeyoung.ju@samsung.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• The age of diagnosis is less than 1 year old
• The disgnosisi of ALL or ALAL(lymphoid predominant)
• Informed consent of the parents(guardians) before participation in this study

Exclusion Criteria:

• Burkitt leukemia/lymphoma or mature B-cell leukemia
• Down syndrome, Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome or other bone marrow failure syndrome, hematopoietic stem cell transplantation
• Relapsed infant leukemia
• Participants with contraindication to medication
• Administered systemic steroid therapy within 4 weeks prior to this study
• Participants in other interventional studies other than this protocol

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low risk group; KMT2A wild type & minimal residual disease(MRD) (-) after consolidation 1	Drug: Consolidation #4(without daunorubicin); Induction chemotherapy(5wks) : Prednisolone, Dexamethasone, Vincristine, Daunorubicin, L-asparaginase, Cytarabine, intrathecal cytarabine, intrathecal methotrexate; Low Risk Cosolidation 1 chemotherapy(3wks) : Cytarabine, Etoposide, Cyclophosphamide, intrathecal methotrexate; Low Risk Cosolidation 2 chemotherapy(3wks) : Methotrexate, 6-mercaptopurine, intrathecal methotrexate; Low Risk Cosolidation 3 chemotherapy(3wks) : Cytarabine, L-asparaginase, intrathecal methotrexate; Low Risk Cosolidation 3 chemotherapy(8wks) : Dexamethasone, Vincristine, Daunorubicin, Cytarabine, Cyclophosphamide, 6-mercaptopurine, intrathecal methotrexate; Maintenance chemotherapy(about 2yrs) : Vincristine, Dexamethasone, 6-mercaptopurine, intrathecal methotrexate
Experimental: Intermediate risk group; ntermediate risk (If one of the two cases below applies); KMT2A: MLL mutation (+) & minimal residual disease (MRD) (-) after consolidation 1KMT2A: wild type & minimal residual disease (MRD) (+) after consolidation 1	Drug: Consolidation #4(with daunorubicin); Induction chemotherapy(5wks) : Prednisolone, Dexamethasone, Vincristine, Daunorubicin, L-asparaginase, Cytarabine, intrathecal cytarabine, intrathecal methotrexate; High Risk Cosolidation 1 chemotherapy(3wks) : Cytarabine, Etoposide, Cyclophosphamide, Daunorubicin intrathecal methotrexate; High Risk Cosolidation 2 chemotherapy(3wks) : Methotrexate, 6-mercaptopurine, intrathecal methotrexate; High Risk Cosolidation 3 chemotherapy(3wks) : Cytarabine, L-asparaginase, intrathecal methotrexate; High Risk Cosolidation 3 chemotherapy(8wks) : Dexamethasone, Vincristine, Daunorubicin, Cytarabine, Cyclophosphamide, 6-mercaptopurine, intrathecal methotrexate; Maintenance chemotherapy(about 2yrs) : Vincristine, Dexamethasone, 6-mercaptopurine, intrathecal methotrexate
Experimental: High risk group; Somatic KMT2A mutation (+) & minimal residual disease (MRD) (+) after consolidation 1	Drug: Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin); Induction chemotherapy(5wks) : Prednisolone, Dexamethasone, Vincristine, Daunorubicin, L-asparaginase, Cytarabine, intrathecal cytarabine, intrathecal methotrexate; High Risk Cosolidation 1 chemotherapy(3wks) : Cytarabine, Etoposide, Cyclophosphamide, Daunorubicin intrathecal methotrexate; High Risk Cosolidation 2 chemotherapy(3wks) : Methotrexate, 6-mercaptopurine, intrathecal methotrexate; High Risk Cosolidation 3 chemotherapy(3wks) : Cytarabine, L-asparaginase, intrathecal methotrexate; High Risk Cosolidation 3 chemotherapy(8wks) : Dexamethasone, Vincristine, Daunorubicin, Cytarabine, Cyclophosphamide, 6-mercaptopurine, intrathecal methotrexate; Allogeneic hematopoietic stem cell transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
3-years Overall survival(OS) rate	The 3-years overall survival rate defined as the percentage of subject in a treatment group who are alive three years after the start of treatm	3-years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival (OS)	The overall survival rate defined as the percentage of subject in a treatment group who are alive five years after the start of treatment.	Up to 5years
Event Free Survial(EFS)	EFS is defined as the period from study enrollment until disease progression, including hematological recurrence of ALL, development of secondary malignancy, or death from any causes, whichever occurs earlier.	3-years and 5-years
The rate of hematopoietic stem cell transplant patients by risk group	through study completion, an average of 1 year	Up to 5years
recurred rate	As a the period from enrollment to disease progression/recurrence	Up to 5years
Death rate related to infusion	The time until defined by date of drug-related mortality from the date of 1st infusion	Up to 5years

Collaborators and Investigators

• Sponsor: Yonsei University

Study record dates

Study Major Dates

• Study Start (Actual): December 11, 2024
• Primary Completion (Estimated): August 31, 2032
• Study Completion (Estimated): December 31, 2032

Study Registration Dates

• First Submitted: June 27, 2024
• First Submitted That Met QC Criteria: July 22, 2024
• First Posted (Actual): July 24, 2024

Study Record Updates

• Last Update Posted (Actual): July 3, 2025
• Last Update Submitted That Met QC Criteria: July 2, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia; Leukemia, Lymphoid; Antibiotics, Antineoplastic; Antineoplastic Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Topoisomerase Inhibitors; Topoisomerase II Inhibitors; Daunorubicin
• Other Study ID Numbers: 4-2023-1263

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No