Individualized Treatment of Pediatric R/R AML Based on Transcriptomic Profile and in Vitro Drug Sensitivity Test

Not Applicable

Recruiting

• Conditions: Acute Myeloid Leukemia in Children

• Registration Number: NCT06233526
• Lead Sponsor: The Children's Hospital of Zhejiang University School of Medicine

Brief Summary

Acute myeloid leukemia (AML) accounts for about 15% to 20% of childhood leukemia, but the death rate accounts for about 50%. About 20-30% of children with AML did not achieve complete response (CR) after 2 induction treatments, and about 30% of children with CR had relapse within 3 years (including recurrence after hematopoietic stem cell transplantation).Relapsed/refractory (R/R) AML is a major cause of treatment failure and refractory survival. Reinduction chemotherapy for R/R-AML to obtain CR again, followed by hematopoietic stem cell transplantation, is the current treatment. At present, there is no recognized reinduction protocol, and the reinduction remission rate of R/R-AML varies greatly among different treatment regimens, ranging from 23 to 81%. Current guidelines recommend a new combination chemotherapy regimen consisting of new drugs without cross-resistance. This method selects sensitive chemotherapeutic drugs, and then forms a new combination chemotherapy regimen according to the characteristics of drugs, which is the choice of R/R-AML reinduction therapy.This study intends to conduct a clinical study on the individualized treatment of R/R AML patients through in vitro drug sensitivity test combined with patient transcriptomic characteristics.

Detailed Description

Not available

Study Timeline

• Study Start: 2024-01-01
• Study First Submitted: 2024-01-23
• Study First Submitted QC: 2024-01-23
• Study First Posted: 2024-01-31
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-30
• Last Update Posted: 2024-07-03
• Primary Completion: 2025-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• (1) Refractory recurrent acute myeloid leukemia (AML), and after second-line reinduction therapy, such as C+HAG did not reach complete CR.Criteria for complete response, refractory, and recurrence are as follows.

(2) <18 years old; (3) The child had good organ function, could tolerate chemotherapy, and had a physical strength score of 0-3 (WHO standard); (4) Understand the research procedures and voluntarily sign written informed consent.

Exclusion Criteria

• (1) Acute promyelocytic leukemia, chronic myelogenous leukemia, acute mixed cell leukemia or known central nervous system leukemia; (2) AML associated with congenital syndromes such as Down syndrome, Fanconi anemia, Bloom syndrome, Cole's syndrome, or congenital aplastic anemia; (3) secondary to immunodeficiency or positive for human immunodeficiency virus (HIV); (4) Cardiac and renal function were obviously abnormal, and left ventricular ejection fraction was <50%.

(5) There is active systemic infection; (6) any medical history or concomitant condition that the investigator believes would impair the subject's safe completion of the study; (7) The investigator considers that the subject is medically unfit to receive the investigational drug or is unfit for any other reason; (8) a known or suspected allergy to the subject drug or to any drug administered in connection with this test;

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Complete remission rate	4 weeks	Complete remission rate after the course of individualized treatment
Event free survival	one year, five years	The patient's event free survival at one and five years after treatment

Secondary Outcome Measures

Name	Time	Method
Overall Survival	one year, five years	The patient's overall survival at one and five years after treatment

Trial Locations

Locations (1)

Children's Hospital of Zhejiang University School of Medicine; 🇨🇳 Hangzhou, Zhejiang, China