HepatoblastomaPhase 2NCT07300449
A Prospective Multicenter Clinical Study of SCCG Protocol and ctDNA 5hmc in Predicting the Chemotherapy Sensitivity and Monitoring the Recurrence and Metastasis of Hepatoblastoma in Children and Adolescents
A Phase 2 study testing Very low-risk group, Low-risk group and Intermediate-risk group in people with Hepatoblastoma, run by Sun Yat-sen University at 2 sites in China.
Trial summary
Hepatoblastoma is the most common malignant liver tumor in infants and preschool children, comprising 65% of pediatric liver malignancies in those under 15, with its incidence on the rise in recent years [1]. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Hepatoblastoma
- What is being tested?
- Very low-risk group, Low-risk group and Intermediate-risk group
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 2 sitesChina
- Study dates
- 23 Jun 2021to 31 Dec 2028
- Phase
- Phase 2
- 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
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with primary hepatoblastoma confirmed by pathology.
- The age of the subjects was less than 18 years old. ③ Obtain the informed consent from the guardians and sign the informed consent form
Who cannot join
- Recurrent hepatoblastoma or other malignant tumor.
- Age> 18 years old. ③ Patients with other tumors and received chemotherapy and abdominal radiotherapy.
- Heart, brain and kidney failure patients.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, 510060
- Recruiting
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, 510060
Yizhuo Yizhuo Zhang, PhD, 020-87342460, zhangyzh@sysucc.org.cn
About this study
Official title: A Prospective Multicenter Clinical Study of the South China Collaborative Group Protocol for the Treatment of Hepatoblastoma in Children and Adolescents
Hepatoblastoma is the most common malignant liver tumor in infants and preschool children, comprising 65% of pediatric liver malignancies in those under 15, with its incidence on the rise in recent years [1]. Standard therapy combines surgical resection and chemotherapy: early-stage patients boast a survival rate over 90%, yet high-risk cases only reach around 40%, highlighting unmet treatment needs.
Notably, there is no universal definition for high-risk hepatoblastoma. The U.S. COG (AHEP0731) categorizes it as stage 4 disease, AFP <100ng/ml at diagnosis (any stage), or small cell undifferentiated histology; conversely, SIOP includes factors like major vascular invasion (inferior vena cava/portal vein), intra-abdominal extrahepatic spread, distant metastasis, AFP <100ng/ml, or tumor rupture, regardless of PRETEXT stage. To improve outcomes, international teams have tested intensified chemotherapy: Europe's SIOPEL reported that escalated cisplatin-doxorubicin regimens lifted high-risk patients' 3-year overall survival to over 80% [2], though with heightened toxicity. Similarly, Germany's IPA (ifosfamide-cisplatin-doxorubicin) and Japan's ITEC (ifosfamide-doxorubicin-carboplatin-VP-16) regimens delivered significant survival benefits but also amplified side effects [3,4].
Read more
Against this backdrop, the Guangdong Anti-Cancer Association's Pediatric Oncology Committee, led by Sun Yat-sen University Cancer Center and involving 15 hospitals, is launching a multicenter prospective trial to identify optimal chemotherapy regimens for Chinese hepatoblastoma children.
Parallelly, liquid biopsy has become an oncology research priority, offering four core advantages over tissue biopsy: non-invasiveness (peripheral blood sampling avoids tumor seeding), real-time genetic/progression monitoring (eliminating repeated invasive procedures), comprehensive molecular profiling (overcoming intratumoral heterogeneity), and high accuracy (capturing primary tumor-derived data). Given hepatoblastoma's propensity for early distant metastasis and 30-40% advanced-stage survival (with limited late-stage chemo efficacy), the Nano-5hmC-Seal cfDNA epigenetic profiling method holds promise as a novel biomarker for early diagnosis, treatment prediction, recurrence monitoring, and prognosis assessment in this disease.
Read the detailed description
Sun Yat-sen University Cancer Center, the Guangdong Anti-Cancer Association's Pediatric Oncology Committee (15 participating hospitals) is launching a multicenter prospective trial to optimize chemotherapy for Chinese patients.
Liquid biopsy, with advantages of non-invasiveness, real-time monitoring, comprehensive profiling, and high accuracy, is an oncology priority. Given hepatoblastoma's early metastasis and poor advanced-stage outcomes (30-40% survival), Nano-5hmC-Seal cfDNA epigenetic profiling is a promising biomarker for diagnosis, treatment prediction, recurrence monitoring, and prognosis.
- Conditions
- Hepatoblastoma
- Being tested
- Very low-risk group, Low-risk group, Intermediate-risk group, High-risk group, 5hmc dynamic monitoring
- Keywords
- hepatoblastoma
- Medical subject headings
- Hepatoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Very low-risk groupDrug
Very low-risk group: No chemotherapy after pure fetal type surgery, followed up and observed. Chemotherapy with other types of single-agent DDP regimens for 4 courses.
Low-risk groupDrug
Low-risk group: 2 to 4 courses of DDP monotherapy chemotherapy, elective surgery, 2 courses of DDP monotherapy chemotherapy after surgery, totaling 4 to 6 courses.
Intermediate-risk groupDrug
Intermediate risk group: randomized treatment: group A SIOPEL - 3 HR solutions can be 2 ~ 4 course of chemotherapy, continue to A total of 6 course of chemotherapy after surgery. Group B C5VD solution after 2 ~ 4 course of chemotherapy, elective surgical procedures continue to a total of 6 course of chemotherapy after surgery.
High-risk groupDrug
High-risk group：Chemotherapy with the C-CD+ICE+ sorafenib regimen for 3 to 5 courses was followed by elective surgery, with a total of 6 to 7 courses
5hmc dynamic monitoringOther
Peripheral blood was regularly drawn from patients in the very low-risk group, low-risk group, intermediate-risk group and high-risk group for 5hmc dynamic monitoring to evaluate its value in clinical efficacy and recurrence detection
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: Very low-risk group | Experimental | The study used CHIC staging to stage hepatoblastoma Receives: Drug: Very low-risk group; Other: 5hmc dynamic monitoring |
| Arm 2: low risk group | Experimental | Patients received 2 to 4 courses of DDP monotherapy followed by elective surgery, then 2 additional courses of DDP monotherapy postoperatively, totaling 4 to 6 treatment cycles. Receives: Drug: Low-risk group; Other: 5hmc dynamic monitoring |
| Arm 3：intermediate risk group | Experimental | Treatment protocol randomization: Group A received SIOPEL-3 HR chemotherapy for 2-4 cycles followed by elective surgery, with continued chemotherapy for 6 additional cycles postoperatively.
The patients in group B were given C5VD chemotherapy for 2-4 courses and then underwent elective operation. Receives: Drug: Intermediate-risk group; Other: 5hmc dynamic monitoring |
| Arm 4： High risk group | Experimental | Surgical resection after 3-5 courses of C-CD+ICE+solanifin chemotherapy Receives: Drug: High-risk group; Other: 5hmc dynamic monitoring |
What is being measured
The primary outcome is the main question the study is designed to answer.
PFS
progression of the disease or the death
Measured: PFS is defined as the time interval from the start of treatment until the occurrence of disease progression or death of the patient from any cause, whichever happens first (up to 24 months）.
Secondary outcomes (2)
OS
Overall survival (OS) is defined as the time from the initiation of treatment with pucotenlimab in combination with theLenvatinib and Irinotecan regimen until the patient's death from any cause. Death is determined based on official medical records or other reliable documentation. The follow - up for OS will continue throughout this estimated assessment period.
Measured: The assessment of OS will be carried out over an estimated period of up to 24 months.
Consistency
The consistency of 5hmc dynamic monitoring in the evaluation of therapeutic effect and detection of tumor recurrence in hepatoblastoma in each risk group was analyzed.
Measured: The assessment will be carried out over an estimated period of up to 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2025
- Study start23 Jun 2021
- Primary completion30 Dec 2027 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HepatoblastomaStudies of the same condition, open and under way.
- More trials run by Sun Yat-sen UniversityThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sun Yat-sen University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yizhuo Zhang, Sun Yat-sen University
- Investigators
- Junting Huang, Principal investigator, SunYat Sen University Cancer CenterHuang, Study director, SunYat Sen University Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07300449
- Sponsor's protocol ID
- SCCG-HB
- Status last verified
- Dec 2025
- First submitted
- 10 Dec 2025
- Last updated
- 23 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Multicenter Clinical Study of the South China Collaborative Group Protocol for the Treatment of Hepatoblastoma in Children and Adolescents
- ClinicalTrials.gov ID
- NCT07300449
- Acronym / sponsor ID
- SCCG-HB
- Conditions
- Hepatoblastoma
- Keywords
- hepatoblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Sun Yat-sen UniversityUniversity, hospital or other
- Study start
- 23 Jun 2021 (actual)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 23 Dec 2025 (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.
Hepatoblastoma is the most common malignant liver tumor in infants and preschool children, comprising 65% of pediatric liver malignancies in those under 15, with its incidence on the rise in recent years [1]. Standard therapy combines surgical resection and chemotherapy: early-stage patients boast a survival rate over 90%, yet high-risk cases only reach around 40%, highlighting unmet treatment needs.
Notably, there is no universal definition for high-risk hepatoblastoma. The U.S. COG (AHEP0731) categorizes it as stage 4 disease, AFP <100ng/ml at diagnosis (any stage), or small cell undifferentiated histology; conversely, SIOP includes factors like major vascular invasion (inferior vena cava/portal vein), intra-abdominal extrahepatic spread, distant metastasis, AFP <100ng/ml, or tumor rupture, regardless of PRETEXT stage. To improve outcomes, international teams have tested intensified chemotherapy: Europe's SIOPEL reported that escalated cisplatin-doxorubicin regimens lifted high-risk patients' 3-year overall survival to over 80% [2], though with heightened toxicity. Similarly, Germany's IPA (ifosfamide-cisplatin-doxorubicin) and Japan's ITEC (ifosfamide-doxorubicin-carboplatin-VP-16) regimens delivered significant survival benefits but also amplified side effects [3,4].
Against this backdrop, the Guangdong Anti-Cancer Association's Pediatric Oncology Committee, led by Sun Yat-sen University Cancer Center and involving 15 hospitals, is launching a multicenter prospective trial to identify optimal chemotherapy regimens for Chinese hepatoblastoma children.
Parallelly, liquid biopsy has become an oncology research priority, offering four core advantages over tissue biopsy: non-invasiveness (peripheral blood sampling avoids tumor seeding), real-time genetic/progression monitoring (eliminating repeated invasive procedures), comprehensive molecular profiling (overcoming intratumoral heterogeneity), and high accuracy (capturing primary tumor-derived data). Given hepatoblastoma's propensity for early distant metastasis and 30-40% advanced-stage survival (with limited late-stage chemo efficacy), the Nano-5hmC-Seal cfDNA epigenetic profiling method holds promise as a novel biomarker for early diagnosis, treatment prediction, recurrence monitoring, and prognosis assessment in this disease.
Detailed description
Sun Yat-sen University Cancer Center, the Guangdong Anti-Cancer Association's Pediatric Oncology Committee (15 participating hospitals) is launching a multicenter prospective trial to optimize chemotherapy for Chinese patients.
Liquid biopsy, with advantages of non-invasiveness, real-time monitoring, comprehensive profiling, and high accuracy, is an oncology priority. Given hepatoblastoma's early metastasis and poor advanced-stage outcomes (30-40% survival), Nano-5hmC-Seal cfDNA epigenetic profiling is a promising biomarker for diagnosis, treatment prediction, recurrence monitoring, and prognosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm 1: Very low-risk group | Experimental | Drug: Very low-risk group, Other: 5hmc dynamic monitoringThe study used CHIC staging to stage hepatoblastoma |
| Arm 2: low risk group | Experimental | Drug: Low-risk group, Other: 5hmc dynamic monitoringPatients received 2 to 4 courses of DDP monotherapy followed by elective surgery, then 2 additional courses of DDP monotherapy postoperatively, totaling 4 to 6 treatment cycles. |
| Arm 3：intermediate risk group | Experimental | Drug: Intermediate-risk group, Other: 5hmc dynamic monitoringTreatment protocol randomization: Group A received SIOPEL-3 HR chemotherapy for 2-4 cycles followed by elective surgery, with continued chemotherapy for 6 additional cycles postoperatively. The patients in group B were given C5VD chemotherapy for 2-4 courses and then underwent elective operation. |
| Arm 4： High risk group | Experimental | Drug: High-risk group, Other: 5hmc dynamic monitoringSurgical resection after 3-5 courses of C-CD+ICE+solanifin chemotherapy |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patients with primary hepatoblastoma confirmed by pathology.
Key exclusion criteria (3 listed)
- Recurrent hepatoblastoma or other malignant tumor.
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 |
|---|---|---|
| Very low-risk groupVery low-risk group: No chemotherapy after pure fetal type surgery, followed up and observed. Chemotherapy with other types of single-agent DDP regimens for 4 courses. | Drug | Arm 1: Very low-risk group |
| Low-risk groupLow-risk group: 2 to 4 courses of DDP monotherapy chemotherapy, elective surgery, 2 courses of DDP monotherapy chemotherapy after surgery, totaling 4 to 6 courses. | Drug | Arm 2: low risk group |
| Intermediate-risk groupIntermediate risk group: randomized treatment: group A SIOPEL - 3 HR solutions can be 2 ~ 4 course of chemotherapy, continue to A total of 6 course of chemotherapy after surgery. Group B C5VD solution after 2 ~ 4 course of chemotherapy, elective surgical procedures continue to a total of 6 course of chemotherapy after surgery. | Drug | Arm 3：intermediate risk group |
| High-risk groupHigh-risk group：Chemotherapy with the C-CD+ICE+ sorafenib regimen for 3 to 5 courses was followed by elective surgery, with a total of 6 to 7 courses | Drug | Arm 4： High risk group |
| 5hmc dynamic monitoringPeripheral blood was regularly drawn from patients in the very low-risk group, low-risk group, intermediate-risk group and high-risk group for 5hmc dynamic monitoring to evaluate its value in clinical efficacy and recurrence detection | Other | Arm 1: Very low-risk group, Arm 2: low risk group, Arm 3：intermediate risk group, Arm 4： High risk group |
Primary outcome
PFS
Time frame: PFS is defined as the time interval from the start of treatment until the occurrence of disease progression or death of the patient from any cause, whichever happens first (up to 24 months）.
progression of the disease or the death
2 secondary outcomes
OS
Time frame: The assessment of OS will be carried out over an estimated period of up to 24 months.
Consistency
Time frame: The assessment will be carried out over an estimated period of up to 24 months.
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 23 Dec 2025.
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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.
Junting Huang principal investigator
SunYat Sen University Cancer Center
Huang study director
SunYat Sen University Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Very low-risk group, Low-risk group and Intermediate-risk group in people with Hepatoblastoma, run by Sun Yat-sen University at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. 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 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Yizhuo Zhang, PhD (02087342460, wangjuan@sysucc.org.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PFS (time frame: PFS is defined as the time interval from the start of treatment until the occurrence of disease progression or death of the patient from any cause, whichever happens first (up to 24 months）.). 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 23 Dec 2025.