Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia

Yonsei University

Status and phase: Enrolling; Phase 2

Conditions: Leukemia, Lymphoid

Treatments: Drug: Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin); Drug: Consolidation #4(with daunorubicin); Drug: Consolidation #4(without daunorubicin)

Study type: Interventional

Funder types: Other

Identifiers

NCT06516679

4-2023-1263

Details and patient eligibility

About

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.

Full 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

Enrollment: 40 estimated patients

Sex: All

Ages: Under 2 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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40 participants in 3 patient groups

Low risk group

Experimental group

Description:

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

Treatment:

Drug: Consolidation #4(without daunorubicin)

Intermediate risk group

Experimental group

Description:

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

Treatment:

Drug: Consolidation #4(with daunorubicin)

High risk group

Experimental group

Description:

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

Treatment:

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