Advanced Translational Research on Childhood Leukemia

  • STATUS
    Recruiting
  • participants needed
    150
  • sponsor
    Chinese University of Hong Kong
Send
Updated on 5 August 2020
See if I qualify

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.

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.

Details
Condition Childhood Leukemia
Age 18years or below
Treatment RNA-seq, whole exon sequencing, Cytogenetics test
Clinical Study IdentifierNCT04478006
SponsorChinese University of Hong Kong
Last Modified on5 August 2020

Eligibility

 Yes No Not Sure

Inclusion Criteria

b'Clinical diagnosis/ defined as'
b'acute lymphoblastic leukemia (ALL) or'
b'acute myeloid leukemia (AML) or'
b'3 .mixed phenotype acute leukemia (MPAL) or'
b'juvenile myelomonocytic leukemia (JMML) or'
b'myelodysplastic syndromes (MDS) or'
b'normal bone marrow donor'

Exclusion Criteria

b'This study will not recruit subjects who are unable to understand English or Chinese.'
b'Patient or parent refusal'
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
Message sent successfully.
We have submitted the information you provided to the research team at the location you chose. For your records, we have sent a copy of the message to your email address.
If you would like to be informed of other studies that may be of interest to you, you may sign up for Patient Notification Service.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.