Advanced Translational Research on Childhood Leukemia

NCT04478006 | Recruiting

• 2 other identifiers: HKCH-REC-2020-012PAED-2024-082
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Childhood Leukemia

Outcome Measures

Primary Outcomes (4)

• Genetic alterations of childhood leukemia

  Association of mutation data with drug sensitivity profiles and disease-free survival /overall survival are analysed using standard statistical methods.

  Baseline

• Gene expression profiles of childhood leukemia

  Global transcriptome and fusion transcripts of leukemic blasts are identified by RNA-sequencing.

  Baseline

• Drug sensitivity profiles

  Drug sensitivity results of individual patient blasts-derived ex vivo culture are presented as IC50 and AUC values.

  Baseline

• Antibody efficacy for treatment of childhood leukemia

  In vitro biochemical and biological assays and invivo leukemic patient-derived xenografts are used to characterize the efficacy and toxicity of the novel human anitbodies.

  Up to 1 year

Study Arms (1)

Childhood Leukemia

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

Genetic: RNA-seq; Genetic: whole exon sequencing; Other: Cytogenetics test

Interventions

whole exon sequencing GENETIC

Genetic alternation analysis

Childhood Leukemia

Cytogenetics test OTHER

Remission and relapse are monitored by cytogenetic analyses.

Childhood Leukemia

RNA-seq GENETIC

Gene expression and fusion transcripts analysis

Childhood Leukemia

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children suspected or diagnosed with leukemia at presentation will be recruited for the study.

You may qualify if:

• acute lymphoblastic leukemia (ALL) or
• acute myeloid leukemia (AML) or
• mixed phenotype acute leukemia (MPAL) or
• \. juvenile myelomonocytic leukemia (JMML) or
• \. myelodysplastic syndromes (MDS) or
• \. normal bone marrow donor

You may not qualify if:

• This study will not recruit subjects who are unable to understand English or Chinese.
• Patient or parent refusal

Sponsors & Collaborators

• Chinese University of Hong Kong: lead

Study Sites (1)

Hong Kong Children Hospital

Hong Kong, Hksar, China

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral blood or bone marraw

MeSH Terms

Interventions

RNA-Seq

Intervention Hierarchy (Ancestors)

Gene Expression Profiling; Genetic Techniques; Investigative Techniques; High-Throughput Nucleotide Sequencing; Sequence Analysis; Sequence Analysis, RNA

Study Officials

• Kam Tong Leung, Ph. D.

  Chinese University of Hong Kong

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kathy Chan, Ph. D.

CONTACT

852-35052858; kathyyychan@cuhk.edu.hk

Kam Tong Leung, Ph. D.

CONTACT

852-35133176; ktleung@cuhk.edu.hk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Scientific Officer

Study Record Dates

• First Submitted: July 14, 2020
• First Posted: July 20, 2020
• Study Start: July 1, 2020
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: February 3, 2026

Record last verified: 2026-02

Locations

CN (1)