Hong Kong Children Hospital
Hong Kong, Hksar, China
Location status: Recruiting
Recruiting
NCT Number: NCT04478006
Advanced Translational Research on Childhood Leukemia
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.
Recruiting
Interested in participating?
Request InfoKey information
Conditions
Age range
Up to 18 year
Sex eligibility
All sexes
Study type
Observational
Primary location
About this study
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.
Who can participate
Healthy volunteers accepted: Yes
Only the study team can determine whether someone qualifies for participation.
List of inclusion criteria:
- acute lymphoblastic leukemia (ALL) or
- acute myeloid leukemia (AML) or
3 .mixed phenotype acute leukemia (MPAL) or
- juvenile myelomonocytic leukemia (JMML) or
- myelodysplastic syndromes (MDS) or
- normal bone marrow donor
List of exclusion criteria:
- This study will not recruit subjects who are unable to understand English or Chinese.
- Patient or parent refusal
Treatment and study plan
RNA-seq
GeneticGene expression and fusion transcripts analysis
whole exon sequencing
GeneticGenetic alternation analysis
Cytogenetics test
OtherRemission and relapse are monitored by cytogenetic analyses.
Primary outcomes
-
Genetic alterations of childhood leukemia
Time frame: Baseline
Association of mutation data with drug sensitivity profiles and disease-free survival /overall survival are analysed using standard statistical methods.
-
Gene expression profiles of childhood leukemia
Time frame: Baseline
Global transcriptome and fusion transcripts of leukemic blasts are identified by RNA-sequencing.
-
Drug sensitivity profiles
Time frame: Baseline
Drug sensitivity results of individual patient blasts-derived ex vivo culture are presented as IC50 and AUC values.
-
Antibody efficacy for treatment of childhood leukemia
Time frame: Up to 1 year
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.
Study contacts
Contact information is provided by the study sponsor or research team.
Kam Tong Leung, Ph. D.
CONTACT
Kathy Chan, Ph. D.
CONTACT
Sponsors and collaborators
Lead sponsor
Chinese University of Hong Kong
Other
Registry information
Official study title
Driving Therapeutic Progress of Childhood Leukemia Through Advanced Translational Research With Immediate and Long-term Impact. Precision Medicine for Childhood Leukemia.
Important dates
- Study start
- 2020
- Primary completion
- 2028
- Study completion
- 2028
- First posted
- Jul 20, 2020
- Registry last updated
- Feb 3, 2026
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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