Advanced Translational Research on Childhood Leukemia

Condition(s) studied

Investigational drug(s) / intervention(s)

RNA-seq: Gene expression and fusion transcripts analysis

whole exon sequencing: Genetic alternation analysis

Cytogenetics test: Remission and relapse are monitored by cytogenetic analyses.

Study 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.

Eligibility

Primary outcome measure(s)

• Genetic alterations of childhood leukemia — 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 — Baseline
  Global transcriptome and fusion transcripts of leukemic blasts are identified by RNA-sequencing.
• Drug sensitivity profiles — 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 — 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.

Trial sites (1)

More Chinese University of Hong Kong trials in China