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

June 30, 2024 updated by: Xiaojun Xu, The Children's Hospital of Zhejiang University School of Medicine

Individualized Treatment of Pediatric Relasped and Refractory Acute Myeloid Leukemia Based on Transcriptomic Profile and in Vitro Drug Sensitivity Test

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

60

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310000
        • Recruiting
        • Children's Hospital of Zhejiang University School of Medicine
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

Yes

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Individualized treatment group
Treating the R/R AML patients based on the transcriptomic profile and in vitro drug sensitivity Test
Other: Chemotherapy regiments based on the transcriptomic profile and in vitro drug sensitivity test
  1. Leukemia cells from newly diagnosed and relapsed children with AML were tested for high throughput in vitro drug sensitivity and resistance.
  2. Combined with multi-omics sequencing technology such as whole exome and transcriptome, the data of drug sensitivity, drug resistance and genomic characteristics of children AML patients were integrated, and the internal rules between drug sensitivity or drug resistance and molecular characteristics such as gene fusion, gene mutation and abnormal gene expression of children AML patients were deeply analyzed, and the molecular characteristics of drug sensitivity in children AML were mapped

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete remission rate
Time Frame: 4 weeks
Complete remission rate after the course of individualized treatment
4 weeks
Event free survival
Time Frame: one year, five years
The patient's event free survival at one and five years after treatment
one year, five years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Survival
Time Frame: one year, five years
The patient's overall survival at one and five years after treatment
one year, five years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Children's Hospital of Zhejiang University School of Medicine

Collaborators

College of Pharmaceutical Science at Zhejiang University

Investigators

  • Principal Investigator: Xiaojun Xu, MD, Children's Hospital, Zhejiang University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2024

Primary Completion (Estimated)

December 31, 2025

Study Completion (Estimated)

December 31, 2025

Study Registration Dates

First Submitted

January 23, 2024

First Submitted That Met QC Criteria

January 23, 2024

First Posted (Actual)

January 31, 2024

Study Record Updates

Last Update Posted (Actual)

July 3, 2024

Last Update Submitted That Met QC Criteria

June 30, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023-IRB-0315-P-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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