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Clinical Trial 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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04478006
Study type Observational
Source Chinese University of Hong Kong
Contact Kathy Chan, Ph. D.
Phone 852-35052858
Email kathyyychan@cuhk.edu.hk
Status Recruiting
Phase
Start date July 1, 2020
Completion date June 2025

See also
  Status Clinical Trial Phase
Active, not recruiting NCT03336931 - PRecISion Medicine for Children With Cancer
Completed NCT01708421 - Symptom Clusters in Children With Leukemia
Recruiting NCT05504772 - Precision Medicine for Every Child With Cancer