Clinical trial NCT06516679: Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia
Official title: Efficacy of Risk-Stratified Treatment in Newly Diagnosed Infant Leukemia: A Multicenter, Prospective Study
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Leukemia, Lymphoid
Lead sponsor: Yonsei University
Sponsor class: OTHER
First posted: 2024-07-24
Enrollment: 40
Sex: ALL
Maximum age (years): 2
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: * 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
Primary purpose: TREATMENT

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

Eligibility criteria: 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

Primary outcomes:
measure: 3-years Overall survival(OS) rate
timeFrame: 3-years
Secondary outcomes:
measure: Overall survival (OS)
timeFrame: Up to 5years
measure: Event Free Survial(EFS)
timeFrame: 3-years and 5-years
measure: The rate of hematopoietic stem cell transplant patients by risk group
timeFrame: Up to 5years
measure: recurred rate
timeFrame: Up to 5years
measure: Death rate related to infusion
timeFrame: Up to 5years
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
Study officials:
References: