NCT06760117

UGT1A1 Genotype-drien Phase I Study of Irinotecan in VIT Regimen for the Treatment of Pediatric R/R Solid Tumors

Phase I Study of Irinotecan Dose Adjustment Guided by UGT1A1*6 Genotype in VIT Regimen for the Treatment of Relapsed and Refractory Childhood Solid Tumors

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 39 (estimated)
• Sponsor: Sun Yat-sen University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Irinotecan is a commonly used salvage chemotherapy drug for children with relapsed and refractory solid tumors. Common dose-limiting toxicities of irinotecan include abdominal pain and diarrhea. Studies have shown that patients with UGT1A16 gene mutations have a higher incidence of these side effects, thereby limiting the dosage of irinotecan. The combination of irinotecan with temozolomide and vincristine is a common salvage chemotherapy regimen for children with relapsed and refractory solid tumors. Currently, the recommended dose of irinotecan is 50mg/m², but there is still significant room for improvement in the efficacy of VIT for these children. Whether patients with wild-type UGT1A16 can further increase the dosage of irinotecan, thereby enhancing the efficacy of the VIT regimen, is the focus of our research.

Detailed description

Children with relapsed and refractory solid tumors and wild-type UGT1A1 gene are enrolled in this study. The doses of temozolomide and vincristine are fixed, while irinotecan in the VIT regimen begins at a starting dose of 50mg/m² and escalates across five dosage groups, with the highest dose group being 110mg/m². This study aims to determine the maximum tolerated dose of irinotecan and the safety of incremental irinotecan dosing, as well as the objective response rate (ORR), disease control rate (DCR), progression-free survival (PFS), and overall survival (OS). This will lay the foundation for future Phase II/III clinical studies.

Conditions

• Pediatric Solid Tumor

Interventions

Type	Name	Description
DRUG	Irinotecan (CPT-11)	Irinotecan will start at a dose of 50mg/m² and escalate to explore the maximum tolerated dose.
DRUG	Temozolomide (TMZ)	TMZ：100mg/m2/d，d1-5
DRUG	Vincristine	VCR: 1.5mg/m2/d(≯2mg), d1

Timeline

• Start date: 2022-01-01
• Primary completion: 2025-06-01
• Completion: 2025-12-01
• First posted: 2025-01-06
• Last updated: 2025-01-06

Locations

1 site across 1 country: China