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

NCT07300449 | Recruiting Phase 2 DMC

• 1 other identifier: SCCG-HB
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 2

Brief 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 \[1\]. 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.

Conditions

Hepatoblastoma

Keywords

hepatoblastoma

Outcome Measures

Primary Outcomes (1)

• PFS

  progression of the disease or the death

  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

  The assessment of OS will be carried out over an estimated period of up to 24 months.

• Consistency

  The assessment will be carried out over an estimated period of up to 24 months.

Study Arms (4)

Arm 1: Very low-risk group

EXPERIMENTAL

The study used CHIC staging to stage hepatoblastoma

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.

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.

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

Drug: High-risk group; Other: 5hmc dynamic monitoring

Interventions

Very low-risk group DRUG

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.

Arm 1: Very low-risk group

Low-risk group DRUG

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.

Arm 2: low risk group

Intermediate-risk group DRUG

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.

Arm 3：intermediate risk group

High-risk group DRUG

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

Arm 4： High risk group

5hmc dynamic monitoring OTHER

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

Arm 1: Very low-risk group; Arm 2: low risk group; Arm 3：intermediate risk group; Arm 4： High risk group

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sun Yat-sen University: lead

Study Sites (2)

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, 510060, China

RECRUITING

Sun Yat-sen University Cancer Center

Guangzhou, Guangdong, 510060, China

RECRUITING

MeSH Terms

Conditions

Hepatoblastoma

Condition Hierarchy (Ancestors)

Neoplasms, Complex and Mixed; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Junting Huang

  SunYat Sen University Cancer Center

  PRINCIPAL INVESTIGATOR

• Huang

  SunYat Sen University Cancer Center

  STUDY DIRECTOR

Central Study Contacts

Yizhuo Zhang, PhD

CONTACT

02087342460; wangjuan@sysucc.org.cn

Junting Huang, PhD

CONTACT

02087342460; huangjt@sysucc.org.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof

Study Record Dates

• First Submitted: December 10, 2025
• First Posted: December 23, 2025
• Study Start: June 23, 2021
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 31, 2028
• Last Updated: December 23, 2025

Record last verified: 2025-12

Locations

CN (2)