A Prospective, Multicenter Study of Transarterial Epirubicin Chemotherapy in the Treatment of Sinonasal Adenoid Cystic Carcinoma
Phase: PHASE2
Conditions: Adenoid Cystic Carcinoma, Sinonasal Carcinoma, Epirubicin
Sponsor: Eye & ENT Hospital of Fudan University

Protocol Summary 
 
Study Title: A Prospective, Multicenter Study of Transarterial Epirubicin Chemoinfusion with 
Response-Adapted Local Therapy for Locally Advanced Sinonasal Adenoid Cystic Carcinoma (SNACC) 
Unique Protocol ID: [EyeEntFudan-2025307] 
Protocol Version: 01 
Version Date: 24 October 2025 
Principal Investigator: Hongmeng Yu; Quan Liu. 
Affiliation: Department of Otolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China. 
 
Executive Summary 
This is a prospective, multicenter, single-arm, Phase 2 interventional study evaluating a novel response-
adapted treatment strategy for patients with locally advanced (T3/T4) sinonasal adenoid cystic carcinoma 
(SNACC). All participants will first receive three cycles of transarterial Epirubicin chemoinfusion (60 
mg/m² per cycle, q4w). Tumor response will be centrally assessed via MRI (RECIST 1.1) 4-6 weeks 
after the third cycle. Based on the assessment, patients will be stratified: those not achieving a complete 
response (CR) will undergo skull base surgery followed by radiotherapy (Path A), while those achieving 
CR will receive definitive radiotherapy alone (Path B). The primary objectives are to assess the objective 
response rate (ORR), 2-year progression-free survival (PFS), and safety. The study aims to enroll 
approximately 100 patients across multiple centers in China to establish a more effective and 
personalized treatment paradigm for this challenging disease. 
1. Background and Rationale 
Sinonasal adenoid cystic carcinoma (SNACC) is a rare malignancy characterized by perineural invasion 
and skull base infiltration, leading to poor long-term prognosis despite surgery and radiotherapy. 
Effective systemic chemotherapy options are limited. Our preclinical drug sensitivity testing using 
patient-derived tumor organoids identified Epirubicin as the most active agent (sensitivity rate: 87.5%, 
n=24, data unpublished). Transarterial chemoinfusion allows for targeted, high-dose drug delivery to the 
tumor bed while minimizing systemic exposure, offering a promising neoadjuvant strategy to improve 
outcomes. 
2. Study Objectives 

2.1 Primary Objectives: 
1. To evaluate the objective response rate (ORR) after three cycles of transarterial Epirubicin 
chemoinfusion. 
2. To evaluate the 2-year progression-free survival (PFS) of the integrated response-adapted strategy. 
3. To evaluate the safety profile of the interventional chemotherapy (CTCAE v5.0). 
2.2 Secondary Objectives: 
1. To assess the complete response (CR) rate post-chemotherapy. 
2. To evaluate the safety of subsequent surgery and/or radiotherapy. 
3. To explore overall survival (OS). 
4. To explore correlations between tumor molecular features and treatment response. 
3. Study Design and Methodology 
3.1 Design: Prospective, multicenter, single-arm, interventional study with a response-adapted design. 
3.2 Participants: Approximately 100 patients with histologically confirmed, locally advanced (AJCC 
9th ed. T3/T4) SNACC. Key inclusion/exclusion criteria are detailed in the main protocol. 
3.3 Intervention & Stratified Pathways: 
• 
Stage 1 (Unified Intervention): Three cycles of transarterial Epirubicin chemoinfusion. 
• 
Stage 2 (Stratified by Central Imaging Review): 
o 
Path A (Non-CR): Skull base surgery + postoperative radiotherapy. 
o 
Path B (CR): Definitive intensity-modulated radiotherapy (IMRT) alone. 
3.4 Assessments: 
• 
Efficacy: Serial contrast-enhanced MRI assessed per RECIST 1.1. 
• 
Safety: Adverse events monitored per CTCAE v5.0, including laboratory tests and cardiac function 
evaluation. 
3.5 Statistical Analysis: 
Sample size is calculated based on superiority in ORR (60% vs. historical 43%, α=0.025, power=90%, 
10% dropout). PFS and OS will be analyzed using Kaplan-Meier method. Analysis follows intention-to-
treat (ITT) principle. 
4. Ethics and Quality Assurance 
The study protocol has been approved by the Institutional Review Board of the Eye & ENT Hospital of 
Fudan University (Approval No: [请填写伦理批件号]) and will be submitted to the IRBs of all 
participating centers. The study will be conducted in compliance with the Declaration of Helsinki and 

Chinese regulations. A Data and Safety Monitoring Board (DSMB) will be established. All study data 
will be kept confidential and retained for at least 5 years after study completion. 
5. Participating Centers (Planned) 
• 
Eye & ENT Hospital, Fudan University (Leading Center) 
• 
Fujian Medical University Affiliated Cancer Hospital 
• 
Fujian Medical University Union Hospital 
• 
Nanjing University Affiliated Drum Tower Hospital 
• 
Ningbo University Affiliated First Hospital 
• 
Qingdao University Affiliated Qilu Hospital (Qingdao) 
• 
The First Affiliated Hospital of Zhejiang University School of Medicine (Shaoyifu Hospital) 
• 
The Second Affiliated Hospital of Guangzhou Medical University (Zhujiang Hospital) 
• 
Yantai Yuhuangding Hospital