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Accepted for/Published in: JMIR Research Protocols

Date Submitted: Feb 13, 2025
Date Accepted: Jul 25, 2025

The final, peer-reviewed published version of this preprint can be found here:

Surveillance of the Genetic Signature in Circulating Tumor DNA for Guiding Adjuvant Chemotherapy in Urothelial Carcinoma: Protocol for a Pilot Randomized Controlled Trial

Zhan Y, Ruan X, Wu Y, Chun TTS, Yao C, Shi R, Liu J, Ali S, Ma R, Huang D, Gao Y, Xu Y, Chen L, Du Q, Ng ATL, Li CWB, Xu D, Na R

Surveillance of the Genetic Signature in Circulating Tumor DNA for Guiding Adjuvant Chemotherapy in Urothelial Carcinoma: Protocol for a Pilot Randomized Controlled Trial

JMIR Res Protoc 2025;14:e72597

DOI: 10.2196/72597

PMID: 40857715

PMCID: 12421199

Surveillance of the genetic signature in circulating tumor DNA for guiding adjuvant chemotherapy in urothelial carcinoma: protocol for a pilot randomized controlled trial

  • Yongle Zhan; 
  • Xiaohao Ruan; 
  • Yishuo Wu; 
  • Tsun Tsun Stacia Chun; 
  • Chi Yao; 
  • Ruofan Shi; 
  • Jiacheng Liu; 
  • Salida Ali; 
  • Ruochen Ma; 
  • Da Huang; 
  • Yi Gao; 
  • Ying Xu; 
  • Lu Chen; 
  • Qijun Du; 
  • Ada Tsui-Lin Ng; 
  • Cho Wing Bryan Li; 
  • Danfeng Xu; 
  • Rong Na

ABSTRACT

Background:

Urothelial carcinomas are one of the most commonly diagnosed cancers with a poor 5-year survival rate worldwide. As genomics is the backbone of the precision medicine paradigm, the genetic signature from circulating tumor DNA (ctDNA) is emerging as a pivotal biomarker for detecting early-stage cancer and molecular residual disease (MRD).

Objective:

This trial aims to evaluate the feasibility and preliminary effects of ctDNA-based sequencing approach for detecting MRD and guiding adjuvant chemotherapy in post-operational urothelial carcinomas.

Methods:

A two-arm pilot randomized controlled trial will be performed in two tertiary hospitals in Hong Kong, involving patients with urothelial carcinomas (pT2-pT4a N0-2M0) undergoing radical resection. A total of 20 patients will be recruited and randomized to receive a four-cycle of gemcitabine plus cisplatin (GC) chemotherapy (intervention group, n=10) or a standard management (control group, n=10) under a 1:1 ratio. We will test ctDNA MRD using a personalized next-generation sequencing panel which is designed based on the individual’s whole-exome sequencing results from the operation specimen. The primary outcome is radiographic disease-free survival (rDFS). Secondary outcomes include cancer-specific survival and overall survival. The primary analysis will be performed in the intention-to-treat population. Kaplan–Meier survival analysis and proportional hazard cox regression analysis will be performed to evaluate the time-to-event effects. A sensitivity analysis will additionally be performed in the per-protocol population.

Results:

The project was funded in February 2024. Recruitment and data collection for the trial are ongoing, and the results of this study are expected to be published in late 2025.

Conclusions:

Genetic signature in ctDNA is informative for personalized management for postoperative urothelial carcinomas, including personalized treatment and early detection of disease progress. Clinical Trial: ClinicalTrials.gov: NCT06257017


 Citation

Please cite as:

Zhan Y, Ruan X, Wu Y, Chun TTS, Yao C, Shi R, Liu J, Ali S, Ma R, Huang D, Gao Y, Xu Y, Chen L, Du Q, Ng ATL, Li CWB, Xu D, Na R

Surveillance of the Genetic Signature in Circulating Tumor DNA for Guiding Adjuvant Chemotherapy in Urothelial Carcinoma: Protocol for a Pilot Randomized Controlled Trial

JMIR Res Protoc 2025;14:e72597

DOI: 10.2196/72597

PMID: 40857715

PMCID: 12421199

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