Childhood LeukemiaNCT04478006
Advanced Translational Research on Childhood Leukemia
An observational study of Childhood Leukemia, run by Chinese University of Hong Kong at 1 site in China.
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. Read more about this study
- Who is it for?
- Ages Up to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Childhood Leukemia
- What is being tested?
- RNA-seq, whole exon sequencing and Cytogenetics test
- How many people
- 300 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2020to 1 Jun 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Children suspected or diagnosed with leukemia at presentation will be recruited for the study.
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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Hong Kong Children Hospital
Hong Kong, Hksar
Kam Tong LEUNG, Ph. D., 852-35133176, ktleung@cuhk.edu.hk
About this study
Official title: Driving Therapeutic Progress of Childhood Leukemia Through Advanced Translational Research With Immediate and Long-term Impact. Precision Medicine for 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.
Read the detailed 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.
- Conditions
- Childhood Leukemia
- Being tested
- RNA-seq, whole exon sequencing, Cytogenetics test
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RNA-seqGenetic
Gene expression and fusion transcripts analysis
whole exon sequencingGenetic
Genetic alternation analysis
Cytogenetics testOther
Remission and relapse are monitored by cytogenetic analyses.
| Study group | Role | What they receive |
|---|---|---|
| Childhood Leukemia | Peripheral blood and bone marrow samples are collected for genetic analysis, invitro drug sensitivity test and animal experiment. Receives: Genetic: RNA-seq; Genetic: whole exon sequencing; Other: Cytogenetics test |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline
Gene expression profiles of childhood leukemia
Global transcriptome and fusion transcripts of leukemic blasts are identified by RNA-sequencing.
Measured: Baseline
Drug sensitivity profiles
Drug sensitivity results of individual patient blasts-derived ex vivo culture are presented as IC50 and AUC values.
Measured: 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.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2020
- Study start1 Jul 2020
- Primary completion1 Jun 2028 (estimated)
- Study completion1 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood LeukemiaStudies of the same condition, open and under way.
- More trials run by Chinese University of Hong KongThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese University of Hong Kong
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kathy Chan, Chinese University of Hong Kong
- Investigators
- Kam Tong Leung, Ph. D., Principal investigator, Chinese University of Hong Kong
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04478006
- Sponsor's protocol ID
- HKCH-REC-2020-012
- Other IDs
- PAED-2024-082
- Status last verified
- Feb 2026
- First submitted
- 14 Jul 2020
- Last updated
- 3 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Driving Therapeutic Progress of Childhood Leukemia Through Advanced Translational Research With Immediate and Long-term Impact. Precision Medicine for Childhood Leukemia.
- ClinicalTrials.gov ID
- NCT04478006
- Acronym / sponsor ID
- HKCH-REC-2020-012
- Conditions
- Childhood Leukemia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Chinese University of Hong KongUniversity, hospital or other
- Study start
- 1 Jul 2020 (actual)
- Primary completion
- 1 Jun 2028 (estimated)
- Study completion
- 1 Jun 2028 (estimated)
- First posted
- 20 Jul 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Childhood Leukemia | – | Genetic: RNA-seq, Genetic: whole exon sequencing, Other: Cytogenetics testPeripheral blood and bone marrow samples are collected for genetic analysis, invitro drug sensitivity test and animal experiment. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Children suspected or diagnosed with leukemia at presentation will be recruited for the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RNA-seqGene expression and fusion transcripts analysis | Genetic | Childhood Leukemia |
| whole exon sequencingGenetic alternation analysis | Genetic | Childhood Leukemia |
| Cytogenetics testRemission and relapse are monitored by cytogenetic analyses. | Other | Childhood Leukemia |
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2026.
Related trials for Childhood Leukemia
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Acute Lymphatic Leukemia, Lymphoma
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A Multi-site Study to Evaluate the Persistence of Protective Immunity to Routine Childhood Vaccinations in Participants With B-ALL/Ly Who Have Received Blinatumomab
B-Cell ALL, B-Cell Acute Lymphoblastic Leukaemia
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International Study for Treatment of Childhood Relapsed Precursor B-Cell ALL 2020 (IntReALL BCP 2020)
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Pharmacokinetics of Dexamethasone in Childhood ALL and Reduction in Bone Mineral Density
Acute Lymphoblastic Leukemia
Active, not recruitingPhase 3
Combination Chemotherapy With or Without Bortezomib in Treating Younger Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or Stage II-IV T-Cell Lymphoblastic Lymphoma
Adult T Acute Lymphoblastic Leukemia, Ann Arbor Stage II Adult Lymphoblastic Lymphoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kam Tong Leung, Ph. D. principal investigator
Chinese University of Hong Kong
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Childhood Leukemia, run by Chinese University of Hong Kong at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kathy Chan, Ph. D. (852-35052858, kathyyychan@cuhk.edu.hk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Genetic alterations of childhood leukemia (time frame: Baseline). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2026.