NCT07300449

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 Prospective Multicenter Clinical Study of the South China Collaborative Group Protocol for the Treatment of Hepatoblastoma in Children and Adolescents

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: Sun Yat-sen University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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

Interventions

Type	Name	Description
DRUG	Very low-risk group	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.
DRUG	Low-risk group	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.
DRUG	Intermediate-risk group	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.
DRUG	High-risk group	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
OTHER	5hmc dynamic monitoring	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

Timeline

• Start date: 2021-06-23
• Primary completion: 2027-12-30
• Completion: 2028-12-31
• First posted: 2025-12-23
• Last updated: 2025-12-23

Locations

2 sites across 1 country: China