Doxorubicin Hydrochloride Liposome Combined With Irinotecan (AI Regimen) Versus VIT Regimen in the Treatment of First Relapsed and Refractory Pediatric Rhabdomyosarcoma: a Prospective, Open-label, Randomized Controlled, Multicenter, Phase II Clinical Study

Phase 2

Recruiting

• Conditions: Rhabdomyosarcoma, Child
• Interventions: Drug: Temozolomide+Irinotecan+Vincristine; Drug: Doxorubicin Hydrochloride Liposome+Irinotecan

• Registration Number: NCT05457829
• Lead Sponsor: Sun Yat-sen University

Brief Summary

This multicenter, randomized, controlled, open-label, prospective clinical trial was designed to evaluate the efficacy and safety of doxorubicin hydrochloride liposome injection in combination with irinotican (AI regimen) versus VIT regimen in the treatment of first relapsed and refractory pediatric rhabdomyosarcoma.

Detailed Description

The primary objective of this study was to evaluate the objective response rate (ORR) of first relapsed and refractory rhabdomyosarcoma in children after 2 cycles of chemotherapy with AI or VIT regimens. Secondary objectives were to evaluate the disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety of AI and VIT regimen in the treatment of relapsed and refractory pediatric rhabdomyosarcoma.

Study Timeline

• Study First Submitted: 2022-07-07
• Study First Submitted QC: 2022-07-13
• Study First Posted: 2022-07-14
• Study Start: 2023-12-30
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-13
• Last Update Posted: 2025-04-16
• Primary Completion: 2026-06-30
• Study Completion: 2026-07-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 88

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
VIT Regimen	Temozolomide+Irinotecan+Vincristine	Temozolomide combined with Irinotecan and Vincristine
AI Regimen	Doxorubicin Hydrochloride Liposome+Irinotecan	Doxorubicin hydrochloride liposome injection combined with Irinotecan

Primary Outcome Measures

Name	Time	Method
Objective response rate (ORR)	up to 2 cycles of chemotherapy（each cycle is 21 days）	Objective response rate (ORR) after 2 cycles of chemotherapy with AI or VIT regimen, including complete response (CR) and partial response (PR)

Secondary Outcome Measures

Name	Time	Method
Progression-free survival (PFS)	From date of radomization unit the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 moths	Means from the date of enrollment to the date of first disease progression or death from any cause, whichever comes first. If the subject has no disease progression during the trial period, PFS is defined as the last date until the subject's last confirmed progression-free survival.
Overall survival (OS)	From date of radomization unit the date of first documented date of death from any cause, assessed up to 36 moths	Overall survival (OS) was defined from the date of enrollment to the date of death from any cause.
Adverse events (AE)	Time from the date of enrollment to 30 days after the last dose.	The safety of the drug was evaluated by NCI-CTC AE 5.0 standard.
Disease control rate (DCR)	up to 2 cycles of chemotherapy	Refers to the percentage of patients with confirmed complete response, partial response, and disease stability in patients with evaluable efficacy.

Trial Locations

Locations (1)

Sun Yat-Sen University Cancer Center; 🇨🇳 Guangzhou, China