Advanced Translational Research on Childhood Leukemia

NCT ID: NCT04478006

Last Updated: 2025-06-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

150 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-07-01

Study Completion Date

2026-06-30

Brief Summary

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Prognosis of children with leukemia, the most common pediatric cancer, has improved markedly. Yet, relapse still occurs in 15-40% of patients with a probability of survival of \<50%, which is unlikely to be boosted by intensification of standard chemotherapy due to overwhelming toxicity. The advent of effective and safe targeted therapies for high-risk cases is therefore imperative. This study constitutes two research projects aiming at driving therapeutic advances.

Detailed Description

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The first part of the study aimed to investigate genomics and drug sensitivity profiling of childhood leukemia and its potential application for precision medicine.

The second part of the study aimed to develop novel antibody for treatment of childhood leukemia by animal model experiments.

Design:

Project 1: Whole-exome and RNA sequencing will be performed on children with leukemia (ALL, AML, MPAL, JMML, MDS) prospectively recruited in the Hong Kong Children's Hospital. Samples will be screened for their sensitivity to preselected, clinically accessible targeted agents in an ex vivo culture system. Results for the high-risk patients will be subjected to the tumor broad for evaluation.

Project 2: Fully human antibody candidates identified by phage display will be engineered into therapeutic forms, and assessed for efficacy and safety in patient-derived xenografts of relapsed/refractory B-ALL and in transgenic mice. The mechanisms of action will be identified by single-cell RNA sequencing.

Significance:

Implementation of functional genomics could identify leukemia patients who will benefit from targeted therapies and enable tailoring of precision medicine. The invented antibodies could be moved forward into clinical trials for salvaging high-risk pediatric B-ALL. Immediate and long-term impact on therapy of childhood leukemia is foreseen.

Conditions

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Childhood Leukemia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Childhood Leukemia

Peripheral blood and bone marrow samples are collected for genetic analysis, invitro drug sensitivity test and animal experiment.

RNA-seq

Intervention Type GENETIC

Gene expression and fusion transcripts analysis

whole exon sequencing

Intervention Type GENETIC

Genetic alternation analysis

Cytogenetics test

Intervention Type OTHER

Remission and relapse are monitored by cytogenetic analyses.

Interventions

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RNA-seq

Gene expression and fusion transcripts analysis

Intervention Type GENETIC

whole exon sequencing

Genetic alternation analysis

Intervention Type GENETIC

Cytogenetics test

Remission and relapse are monitored by cytogenetic analyses.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. acute lymphoblastic leukemia (ALL) or
2. acute myeloid leukemia (AML) or

3 .mixed phenotype acute leukemia (MPAL) or

4\. juvenile myelomonocytic leukemia (JMML) or

5\. myelodysplastic syndromes (MDS) or

6\. normal bone marrow donor

Exclusion Criteria

1. This study will not recruit subjects who are unable to understand English or Chinese.
2. Patient or parent refusal
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Chinese University of Hong Kong

OTHER

Sponsor Role lead

Responsible Party

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Kathy Chan

Scientific Officer

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Kam Tong Leung, Ph. D.

Role: PRINCIPAL_INVESTIGATOR

Chinese University of Hong Kong

Locations

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Hong Kong Children Hospital

Hong Kong, Hksar, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Kathy Chan, Ph. D.

Role: CONTACT

852-35052858

Kam Tong Leung, Ph. D.

Role: CONTACT

852-35133176

Facility Contacts

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Kam Tong LEUNG, Ph. D.

Role: primary

852-35133176

Other Identifiers

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HKCH-REC-2020-012

Identifier Type: -

Identifier Source: org_study_id

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