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Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia

Phase 2
Not yet recruiting
Conditions
Leukemia, Lymphoid
Interventions
Drug: Consolidation #4(without daunorubicin)
Drug: Allogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin)
Drug: Consolidation #4(with daunorubicin)
Registration Number
NCT06516679
Lead Sponsor
Yonsei University
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.

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

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
40
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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Low risk groupConsolidation #4(without daunorubicin)KMT2A wild type \& minimal residual disease(MRD) (-) after consolidation 1
High risk groupAllogeneic hematopoietic stem cell transplantation after Consolidation #4(with daunorubicin)Somatic KMT2A mutation (+) \& minimal residual disease (MRD) (+) after consolidation 1
Intermediate risk groupConsolidation #4(with daunorubicin)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
Primary Outcome Measures
NameTimeMethod
3-years Overall survival(OS) rate3-years

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

Secondary Outcome Measures
NameTimeMethod
recurred rateUp to 5years

As a the period from enrollment to disease progression/recurrence

Overall survival (OS)Up to 5years

The overall survival rate defined as the percentage of subject in a treatment group who are alive five years after the start of treatment.

Event Free Survial(EFS)3-years and 5-years

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.

Death rate related to infusionUp to 5years

The time until defined by date of drug-related mortality from the date of 1st infusion

The rate of hematopoietic stem cell transplant patients by risk groupUp to 5years

through study completion, an average of 1 year

Trial Locations

Locations (1)

Severance Hospital

🇰🇷

Seoul, Korea, Republic of

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