NCT07355855

A Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma

A Multicenter, Multi-cohort, Prospective Phase II Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 106 (estimated)
• Sponsor: Fudan University · Academic / Other
• Sex: All
• Age: 14 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This study is a prospective, multi-cohort, multi-center clinical trial targeting patients with intermediate-to-high-risk rhabdomyosarcoma who have not previously received systemic anti-tumor treatment. It aims to evaluate the efficacy and safety of all-trans retinoic acid combined with VAC chemotherapy.

Conditions

• Rhabdomyosarcoma

Interventions

Type	Name	Description
DRUG	ATRA+VAC	Cohort 1: Intermediate-risk RMS: ATRA+VAC treatment The specific medication is: Vincristine (V), administered intravenously, at a dose of 1.5 mg/m2 (with a maximum of 2 mg), on days 1, 8, and 15, every three weeks. Dactinomycin (A), intravenous infusion, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w. Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. All-trans-retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd. Capsules, 10mg/capsule, 20mg bid, orally, administered continuously every day, with 21 days constituting one course of treatment. If safety is confirmed, the dosage can be increased to 30mg bid.
DRUG	ATRA+VAC+Anlotinib	Cohort 2: High risk group RMS+: ATRA+VAC, ATRA+anlotinib maintenance therapy The specific medication is: Vincristine (V), static push, 1.5 mg/m2 (maximum not exceeding 2mg), D1, 8, 15, q3w. Dactinomycin (A), intravenous drip, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w. Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. After chemotherapy, Anlotinib, 12mg, d1-d14, po, q3w+ATRA maintenance therapy. Both are maintained for a period of time until disease progression or toxicity is intolerable, or for at least 2 years. All trans retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd., capsule, 10mg/capsule, 20mg bid orally, administered continuously daily for 21 days as a course of treatment. If safety is ensured, the dosage will be increased to 30mg bid.

Timeline

• Start date: 2026-01-01
• Primary completion: 2029-01-01
• Completion: 2029-01-01
• First posted: 2026-01-21
• Last updated: 2026-01-21

Locations

1 site across 1 country: China