Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia

NCT06516679 | Recruiting Phase 2 DMC

• 1 other identifier: 4-2023-1263
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 8

Brief Summary

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.

Conditions

Leukemia, Lymphoid

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Overall survival (OS)

  Up to 5years

• Event Free Survial(EFS)

  3-years and 5-years

• The rate of hematopoietic stem cell transplant patients by risk group

  Up to 5years

• recurred rate

  Up to 5years

• Death rate related to infusion

  Up to 5years

Study Arms (3)

Low risk group

EXPERIMENTAL

KMT2A wild type \& minimal residual disease(MRD) (-) after consolidation 1

Drug: Consolidation #4(without daunorubicin)

Intermediate risk group

EXPERIMENTAL

ntermediate risk (If one of the two cases below applies) * KMT2A: MLL mutation (+) \& minimal residual disease (MRD) (-) after consolidation 1 * KMT2A: wild type \& minimal residual disease (MRD) (+) after consolidation 1

Drug: Consolidation #4(with daunorubicin)

High risk group

EXPERIMENTAL

Somatic KMT2A mutation (+) \& minimal residual disease (MRD) (+) after consolidation 1

Drug: Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin)

Interventions

Consolidation #4(without daunorubicin) DRUG

1. Induction chemotherapy(5wks) : Prednisolone, Dexamethasone, Vincristine, Daunorubicin, L-asparaginase, Cytarabine, intrathecal cytarabine, intrathecal methotrexate 2. Low Risk Cosolidation 1 chemotherapy(3wks) : Cytarabine, Etoposide, Cyclophosphamide, intrathecal methotrexate 3. Low Risk Cosolidation 2 chemotherapy(3wks) : Methotrexate, 6-mercaptopurine, intrathecal methotrexate 4. Low Risk Cosolidation 3 chemotherapy(3wks) : Cytarabine, L-asparaginase, intrathecal methotrexate 5. Low Risk Cosolidation 3 chemotherapy(8wks) : Dexamethasone, Vincristine, Daunorubicin, Cytarabine, Cyclophosphamide, 6-mercaptopurine, intrathecal methotrexate 6. Maintenance chemotherapy(about 2yrs) : Vincristine, Dexamethasone, 6-mercaptopurine, intrathecal methotrexate

Low risk group

Consolidation #4(with daunorubicin) DRUG

1. Induction chemotherapy(5wks) : Prednisolone, Dexamethasone, Vincristine, Daunorubicin, L-asparaginase, Cytarabine, intrathecal cytarabine, intrathecal methotrexate 2. High Risk Cosolidation 1 chemotherapy(3wks) : Cytarabine, Etoposide, Cyclophosphamide, Daunorubicin intrathecal methotrexate 3. High Risk Cosolidation 2 chemotherapy(3wks) : Methotrexate, 6-mercaptopurine, intrathecal methotrexate 4. High Risk Cosolidation 3 chemotherapy(3wks) : Cytarabine, L-asparaginase, intrathecal methotrexate 5. High Risk Cosolidation 3 chemotherapy(8wks) : Dexamethasone, Vincristine, Daunorubicin, Cytarabine, Cyclophosphamide, 6-mercaptopurine, intrathecal methotrexate 6. Maintenance chemotherapy(about 2yrs) : Vincristine, Dexamethasone, 6-mercaptopurine, intrathecal methotrexate

Intermediate risk group

Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin) DRUG

1. Induction chemotherapy(5wks) : Prednisolone, Dexamethasone, Vincristine, Daunorubicin, L-asparaginase, Cytarabine, intrathecal cytarabine, intrathecal methotrexate 2. High Risk Cosolidation 1 chemotherapy(3wks) : Cytarabine, Etoposide, Cyclophosphamide, Daunorubicin intrathecal methotrexate 3. High Risk Cosolidation 2 chemotherapy(3wks) : Methotrexate, 6-mercaptopurine, intrathecal methotrexate 4. High Risk Cosolidation 3 chemotherapy(3wks) : Cytarabine, L-asparaginase, intrathecal methotrexate 5. High Risk Cosolidation 3 chemotherapy(8wks) : Dexamethasone, Vincristine, Daunorubicin, Cytarabine, Cyclophosphamide, 6-mercaptopurine, intrathecal methotrexate 6. Allogeneic hematopoietic stem cell transplantation

High risk group

Eligibility Criteria

• Age: Up to 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Yonsei University: lead

Study Sites (8)

Chonnam National University Hwasun Hospital

Hwasun, South Korea

RECRUITING

Jeju National University Hospital

Jeju City, South Korea

RECRUITING

Pusan National University Yangsan Hospital

Pusan, South Korea

RECRUITING

Asan Medical Center

Seoul, South Korea

RECRUITING

Samsung Medical Cente

Seoul, South Korea

RECRUITING

Seoul National University Hospital

Seoul, South Korea

RECRUITING

Seoul saint Mary's Hospital

Seoul, South Korea

RECRUITING

Severance Hospital

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Leukemia, Lymphoid

Condition Hierarchy (Ancestors)

Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: * Low risk group : KMT2A wild type \& minimal residual disease(MRD) (-) after consolidation 1 * Intermediate risk group : Intermediate risk (If one of the two cases below applies) * KMT2A: MLL mutation (+) \& minimal residual disease (MRD) (-) after consolidation 1 * KMT2A: wild type \& minimal residual disease (MRD) (+) after consolidation 1 * High risk group : Somatic KMT2A mutation (+) \& minimal residual disease (MRD) (+) after consolidation 1

Study Record Dates

• First Submitted: June 27, 2024
• First Posted: July 24, 2024
• Study Start: December 11, 2024
• Primary Completion (Estimated): August 31, 2032
• Study Completion (Estimated): December 31, 2032
• Last Updated: July 3, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

KR (8)