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GEMCAD-REVEAL STUDY - Circulating Tumor DNA as a Predictor of Relapse in Patients With Locally Advanced Rectal Cancer.

Active, not recruiting
Conditions
Patients With Locally Advanced Rectal Cancer
Registration Number
NCT05674422
Lead Sponsor
Grupo Espanol Multidisciplinario del Cancer Digestivo
Brief Summary

The evaluation of the tumor response to chemoradiotherapy/total neoadjuvant therapy (CRT/TNT) remains a challenge. The integration of a blood-based biomarker such as ctDNA with clinico-radiological tools could offer the potential advantage of improving accuracy of assessment of tumor response to neoadjuvant therapy. Furthermore, data on functional outcomes and quality of life after total mesorectal excision (TME) and especially after " Watch And Wait" (WW) is scarce.

REVEAL is a prospective, multicenter study in which the response to TNT in correlation with liquid biopsy (LB) of patients with rectal cancer in Spain will be evaluated. It is planned to include 120 patients. All patients will be aged 18 years or older, with histologically confirmed rectal adenocarcinoma, located in the mid or distal third (with the inferior margin within 12cm from the anal verge), clinically staged II and III (cT3-T4 and/or any TN+), scheduled to undergo TNT will be eligible. All cases and treatment decisions will be discussed by local Multidisciplinary Boards. Patients will be included consecutively when visiting the corresponding health centers for outpatient visits or hospitalization.

The objective of the present study is to evaluate the role of ctDNA in the prediction of relapse in patients diagnosed with locally advanced rectal cancer (LARC) treated with TNT followed by WW or TME based on a clinical assessment of the local response

Detailed Description

The evaluation of the tumor response to chemoradiotherapy/total neoadjuvant therapy (CRT/TNT) remains a challenge. The integration of a blood-based biomarker such as ctDNA with clinico-radiological tools could offer the potential advantage of improving accuracy of assessment of tumor response to neoadjuvant therapy. Furthermore, data on functional outcomes and quality of life after total mesorectal excision (TME) and especially after " Watch And Wait" (WW) is scarce.

REVEAL is a prospective, multicenter study in which the response to TNT in correlation with liquid biopsy (LB) of patients with rectal cancer in Spain will be evaluated. It is planned to include 120 patients. All patients will be aged 18 years or older, with histologically confirmed rectal adenocarcinoma, located in the mid or distal third (with the inferior margin within 12cm from the anal verge), clinically staged II and III (cT3-T4 and/or any TN+), scheduled to undergo TNT will be eligible. All cases and treatment decisions will be discussed by local Multidisciplinary Boards. Patients will be included consecutively when visiting the corresponding health centers for outpatient visits or hospitalization.

The objective of the present study is to evaluate the role of ctDNA in the prediction of relapse in patients diagnosed with locally advanced rectal cancer (LARC) treated with TNT followed by WW or TME based on a clinical assessment of the local response.

HYPOTHESIS ctDNA is a predictor of complete clinical response and could potentially be used to guide patient selection for WW strategy.

ctDNA is an early marker of local tumor relapse and could potentially guide strategy changes during follow-up.

Patients who are enrolled in a WW strategy have better long-term functional outcomes in comparison to patients who undergo surgery.

OBJECTIVES

PRIMARY:

To estimate the positive predictive value (PPV) and the negative predictive value (NPV) of the post-TNT ctDNA measurement to identify relapse (local or distant) in the 2 years after TNT.

SECONDARY:

Secondary objectives related to ctDNA and tumor response to TNT:

* To estimate the concordance between the post-TNT ctDNA measurement and the clinical evaluation of response (endoscopy-biopsy, DRE and MRI) to TNT.

* To estimate the concordance between the ctDNA measured after TNT and the endoscopy-biopsy evaluation of response.

* To estimate the concordance between the ctDNA measured after TNT and the MRI-defined response pattern groups.

Secondary objectives related to ctDNA measure during follow-up and relapse:

* To estimate the positive predictive value and the negative predictive value of ctDNA measured during the follow-up after TNT to identify relapse (local or distant) in the 2 years after TNT.

* To estimate the positive predictive value and the negative predictive value of ctDNA measured during the follow-up after TNT to identify local relapse in the 2 years after TNT

* To estimate the positive predictive value and the negative predictive value of ctDNA measured during the follow-up after TNT to identify distant relapse in the 2 years after TNT

Secondary objective related to functional impact of TME and WW:

-To describe anorectal, urinary and sexual function in the TME and WW groups.

Secondary objective related to ctDNA and survival:

-To assess the association of ctDNA levels (at baseline, post-treatment and during follow-up) with the survival outcomes (overall and disease-free survival, locoregional and distant recurrence, together with the pattern of recurrence).

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
120
Inclusion Criteria
  • Legally capable patients ≥ 18 years of age.
  • Histologically confirmed rectal adenocarcinoma.
  • Any tumor located in the mid or distal rectum, clinically staged II-III (cT3/4 and/or any TN+).
  • Willingness to be enrolled in the WW strategy if cCR or nCCR is achieved.
  • Absence of metastases on imaging.
  • Scheduled to undergo TNT followed by delayed surgery.
  • Patients who have signed the informed consent for this study.

Note: Decision was taken to treat the patient with a specific treatment prior and independently of patient inclusion in this non interventional study.

Exclusion Criteria
  • Patients who do not meet inclusion criteria.
  • Concomitant colorectal tumors.
  • Intolerance or contraindication to planned TNT.
  • Other concurrent malignant diseases. Pregnancy or breastfeeding.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
ctDNA from liquid biopsy2 years after to start treatment total neoadjuvant therapy/2 years per patient

The results from ctDNA will be dichotomized to positive or negative. The investigator will report the percentage of patients with positive or negative ctDNA. Correlation of these values with clinical endpoints of response / survival to total neoadjuvant therapy will be done to fulfill the proposed objectives of this study. The ctDNA isolation test selected for this study is marketed by Guardant Health (Redwood City, California, USA) under the name Guardant RevealTM. It consists of a single state-of-the-art liquid blood biopsy, which is capable of detecting residual or recurrent disease seven days after blood extraction. This test uses a sequencing method that simultaneously evaluates genetic alterations and methylation without the need for a biopsy of the primary tumor tissue to determine specific mutations.

Secondary Outcome Measures
NameTimeMethod
Disease-specific Survival (DSS)at 1 year after the first dose of TNT

Disease-specific Survival (DSS): defined as the time elapsed from the first dose of TNT to the date of progression(only for pts with nCCR), relapse, or death due to the disease, whichever occurs first. We will assess the median DSS, the DSS rate at 1 year, and at the end of study. The 1-year DSS rate is defined as the rate of patients alive and free of event at 1 year after the first dose of TNT, estimated by Kaplan-Meier. Patients alive and free of events at the date of the analysis will be censored at their last known contact.

TNT complianceThroughout the study period, approximately 2 years per patient

TNT compliance: total number of dose reductions and modifications of the treatment throughout the study period.

Response to TNT (cCR,nCCR)Throughout the study period, approximately 2 years per patient

Number of patients will be classified as responders (cCR, nCCR) or non responders based on the International Consensus Recommendations for Organ Preservation in Rectal Cancer. The number and percentage of each category will be reported.

cCR: clinical complete response nCCR: near-complete clinical response

Response to TNT (WW or TME)Throughout the study period, approximately 2 years per patient

The number and percentage of patients allocated to "watch and wait" (WW) or Total Mesorectal Excision (TMT) will be provided. Response rates will be also provided separately depending on the technique empowered to determine the response:

* Endoscopy-biopsy

* MRI

Locoregional failure rate (LRF)Throughout the study period, approximately 2 years per patient

Locoregional failure rate (LRF) defined as the percentage of patients who present progression(only for patients with nCCR)/relapse of disease in the rectum, regional organs and/or regional lymph nodes. Locoregional failure rate will be estimated using the appropriate logistic regression model at 1-year after the first dose of TNT and end of study. Those patients lost to follow-up with no event of local recurrence will be considered not evaluable for LFR endpoint. Patients who die as a direct or secondary cause of the cancer will be considered as an event at the date of death for LFR analysis. Death by other reasons other than cancer progression/relapse will not be considered as an event for LFR.

Distant relapse rate (DRR)Throughout the study period, approximately 2 years per patient

Distant relapse rate (DRR) defined as the percentage of patients who present progression/relapse of disease in distant locations. DRR will be estimated using the appropriate logistic regression model at 1-year after the first dose of TNT and end of study. Those patients lost to follow-up with no event of distal recurrence will be considered not evaluable for DRR endpoint. Patients who die as a direct or secondary cause of the cancer will be considered as an event at the date of death for DRR analysis. Death by other reasons other than cancer progression/relapse will not be considered as an event for DRR.

Overall Survival (OS)at 1 year after the first dose of TNT

Overall Survival (OS) : defined as the time elapsed from the first dose of TNT until death from any cause. We will assess the median OS and the OS rate at 1 year. The 1-year OS rate is defined as the rate of patients alive at 1 year after the first dose of TNT, estimated by Kaplan-Meier. Patients alive and free of events at the date of the analysis will be censored at their last known contact.

Disease-free Survival (DFS)at 1 year after the first dose of TNT and at the end of study (approximately 2 years)

Disease-free Survival (DFS): defined as the time elapsed from the first dose of TNT to progression (only for pts with nCCR), relapse, or death from any cause, whichever occurs first. The investigators will assess the median DFS, the DFS rate at 1 year, and at the end of study. The 1-year DFS rate is defined as the rate of patients alive and free of relapse or progression at 1 year after the first dose of TNT estimated by Kaplan-Meier. Patients alive and free of events at the date of the analysis will be censored at their last known tumor assessment.

MortalityThroughout the study period, approximately 2 years per patient

Mortality: Rate of patients who died during the study. The number and percentage of patients will be provided.

Trial Locations

Locations (15)

Hospital Germans Trias i Pujol - ICO Badalona

🇪🇸

Badalona, Barcelona, Spain

Hospital Universitari Mutua de Terrassa

🇪🇸

Terrassa, Barcelona, Spain

Hospital Universitario 12 de Octubre

🇪🇸

Madrid, Comunidad Autónoma De Madrid, Spain

H. de la Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

H.U. Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital Clínic, Universitat de Barcelona

🇪🇸

Barcelona, Spain

Hospital Del Mar, Barcelona

🇪🇸

Barcelona, Spain

Hospital Sant Joan Despí - Moisés Broggi.

🇪🇸

Barcelona, Spain

ICO-Hospitalet

🇪🇸

Barcelona, Spain

ICO Girona

🇪🇸

Girona, Spain

H. Univeristario La Paz

🇪🇸

Madrid, Spain

Hospital Quirón Salud Valencia

🇪🇸

Valencia, Spain

Hospital Universitari i Politècnic La Fe

🇪🇸

Valencia, Spain

Hospital Clínico Universitario Lozano Blesa

🇪🇸

Zaragoza, Spain

Hospital Universitario Miguel Servet

🇪🇸

Zaragoza, Spain

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