Active Surveillance and Chemotherapy Before Surgery in Treating Participants With Stage II-III Rectal Cancer

March 7, 2024 updated by: M.D. Anderson Cancer Center

Organ Preservation With Active Surveillance After Chemoradiation in Rectal Cancer (OPTION)

This pilot trial studies how well active surveillance and chemotherapy before surgery work in treating participants with stage II-III rectal cancer. Active surveillance involves monitoring participants for additional tumor growth after receiving cancer treatment. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. It is not yet known whether deferring surgery after active surveillance and chemotherapy will work better in treating participants with stage II-III rectal cancer.

Study Overview

Detailed Description

PRIMARY OBJECTIVES:

I. To quantify the rates of organ preservation and tumor regrowth with non-operative management of locally advanced rectal cancer in patients achieving a clinical complete response (cCR).

SECONDARY OBJECTIVES:

I. To correlate clinical, radiographic, and pathologic findings after neoadjuvant therapy for rectal cancer.

II. To determine the impact of active surveillance with deferral of surgery on oncologic outcomes.

III. To assess decision quality for patients with rectal cancer facing multiple treatment options.

IV. To explore the impact of patient-provider communication on patient decisions for surgical versus nonsurgical treatment decision for rectal cancer.

V. To assess safety of deferral of surgery in distal rectal cancer patients with possibility of cohort expansion to more proximal locally advanced rectal cancer patients.

CORRELATIVE OBJECTIVES:

I. Obtain tissue to monitor treatment response and any future biomarker analyses

OUTLINE: Participants are assigned to 1 of 2 groups.

GROUP I: Participants who have achieved clinical complete response undergo standard surgical resection.

GROUP II: Participants who have achieved clinical complete response receive active surveillance and consolidated chemotherapy for up to 4 months in the absence of disease progression or unacceptable toxicity. Participants with incomplete response or regrowth of tumor, undergo surgical resection as in Group I.

After the completion of study treatment, participants in Group I are followed up at 6 and 12 months, and then once a year for up to 3 years. Participants in Group II are followed up every 3 months for 18 months, every 6 months for 2 years, and then every year for up to 3 years.

Study Type

Interventional

Enrollment (Estimated)

120

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Histologically confirmed diagnosis of rectal adenocarcinoma
  • Eligible for curative resection of rectal adenocarcinoma
  • Rectal tumor location =< 12 cm from the anal verge as determined by endoscopy or magnetic resonance imaging (MRI) (if endoscopy report is not available or deemed inadequate my treating oncologist)
  • Nodal involvement confined to the radiation field
  • Radiologically measurable or clinically evaluable disease as defined in the protocol
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS): 0, 1 or 2
  • Clinical Stage: Stage II and III. N2 disease is to be estimated as four or more lymph nodes that are >= 10 mm. Clinical staging should be estimated based on the combination of the following assessments: physical exam by the primary surgeon including digital rectal exam (DRE), computed tomography (CT) or positron emission tomography (PET)/CT scan of the chest/abdomen/pelvis and a pelvic MRI. If a pelvic MRI is performed, it is acceptable to perform CT of the chest/abdomen, omitting CT imaging of the pelvis. PET/CT is optional.
  • No known contraindication to standard (fluoropyrimidine-based) pelvic chemoradiation (e.g. dihydropyrimidine dehydrogenase [DPD] deficiency)
  • Patient of child-bearing potential is willing to employ adequate contraception during treatment and after treatment, as directed by treating clinical team
  • Willing to provide written informed consent
  • Willing to return to enrolling medical site for all study assessments

Exclusion Criteria:

  • Diagnosis of inflammatory bowel disease (IBD)
  • Diagnosis of MSI-H colorectal cancer at time of consent
  • Recurrent rectal cancer
  • Tumor is causing symptomatic bowel obstruction (patients who have diverting ostomy are eligible)
  • Any prior pelvic radiation
  • Other invasive malignancy undergoing active treatment. Patients receiving prior treatment that precludes standard chemoradiation or ability to receive consolidation/adjuvant chemotherapy will be excluded from survival analyses
  • Patients unwilling or unable to undergo pelvic MRI

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Group I (surgical resection)
Participants who have achieved clinical complete response undergo standard surgical resection.
Ancillary studies
Undergo surgical resection
Other Names:
  • Proctectomy
Experimental: Group II (active surveillance)
Participants who have achieved clinical complete response receive active surveillance and consolidated chemotherapy for up to 4 months in the absence of disease progression or unacceptable toxicity. Participants with incomplete response or regrowth of tumor, undergo surgical resection as in Group I.
Ancillary studies
Receive chemotherapy
Other Names:
  • Chemo
  • Chemotherapy (NOS)
  • Chemotherapy, Cancer, General
Undergo surgical resection
Other Names:
  • Proctectomy
Receive active surveillance
Other Names:
  • observation
  • Active Surveillance
  • deferred therapy
  • expectant management
  • Watchful Waiting

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall organ preservation rate
Time Frame: At 12 months
The study will estimate overall organ preservation rate at 12 months and the corresponding 95% confidence interval (95% CI). The exact confidence interval will be computed when observed number of events is limited. The 12-month organ preservation rate corresponds to the proportion of patients alive and not having surgery within 12 months. The study will use Kaplan-Meier methods to estimate probability of overall organ preservation at 12 months at 12 months for all patients and for deferral patients respectively.
At 12 months
Local tumor regrowth rate
Time Frame: At 12 months
The study will estimate local tumor regrowth rate at 12 months and the corresponding 95% confidence interval (95% CI). The exact confidence interval will be computed when observed number of events is limited. The 12-month organ preservation rate corresponds to the proportion of patients alive and not having surgery within 12 months. The study will use Kaplan-Meier methods to estimate probability of local tumor regrowth at 12 months for all patients and for deferral patients respectively.
At 12 months
Time to surgery or death
Time Frame: Up to 12 months
The study will use Kaplan-Meier methods to estimate probability for deferral patients respectively.
Up to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Time Frame: Up to 5 years
Up to 5 years
Decision quality assessment determined by European Organization for Treatment and Research of Cancer Quality of Life Questionnaire (EORTC-QLQ30+CR29)
Time Frame: Up to 3 years
The aspects of the patient's decision making process will be documented and studied to assess what criteria, including the impact of patient-provider communication, seem to be driving the patients' decision to delay surgery or not. The decision evaluation questions such as decision quality survey, satisfaction with decision and anticipated regret questionnaire will be documented and summarized. Descriptive statistics will be used to summarize continuous variables and frequency and percentage will be used to tabulate categorical variables. The study will summarize quality of life measures, toxicity, surgical success rates, clinical, radiographic and pathologic findings, and exploratory comparisons among patient cohorts will be done.
Up to 3 years
Overall survival (OS)
Time Frame: Up to 5 years
Overall survival will be estimated using Kaplan-Meier method.
Up to 5 years
Regression-free survival (RFS)
Time Frame: Up to 5 years
Regression-free survival will be estimated using Kaplan-Meier method.
Up to 5 years
Surgical success rates
Time Frame: Up to 5 years
Up to 5 years
Radiographic Findings
Time Frame: Up to 5 years
MRI performed.
Up to 5 years
Pathologic Findings
Time Frame: Up to 5 years
Blood and tissue collected at various time points.
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: George Chang, M.D. Anderson Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

October 31, 2024

Primary Completion (Estimated)

October 31, 2026

Study Completion (Estimated)

October 31, 2026

Study Registration Dates

First Submitted

June 20, 2018

First Submitted That Met QC Criteria

July 10, 2018

First Posted (Actual)

July 20, 2018

Study Record Updates

Last Update Posted (Actual)

March 12, 2024

Last Update Submitted That Met QC Criteria

March 7, 2024

Last Verified

September 1, 2023

More Information

Terms related to this study

Other Study ID Numbers

  • 2016-0549 (Other Identifier: M D Anderson Cancer Center)
  • NCI-2018-01142 (Registry Identifier: CTRP (Clinical Trial Reporting Program))

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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