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

Primary Purpose

Rectal Adenocarcinoma, Stage II Rectal Cancer AJCC v8, Stage IIA Rectal Cancer AJCC v8

Status

Not yet recruiting

Phase

Early Phase 1

Locations

United States

Study Type

Interventional

Intervention

Chemotherapy

Patient Observation

Questionnaire Administration

Resection of Rectum

About this trial

This is an interventional treatment trial for Rectal Adenocarcinoma

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

M D Anderson Cancer Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Experimental

Arm Label

Group I (surgical resection)

Group II (active surveillance)

Arm Description

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

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.

Outcomes

Primary Outcome Measures

Overall organ preservation rate

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.

Local tumor regrowth rate

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.

Time to surgery or death

The study will use Kaplan-Meier methods to estimate probability for deferral patients respectively.

Secondary Outcome Measures

Decision quality assessment determined by European Organization for Treatment and Research of Cancer Quality of Life Questionnaire (EORTC-QLQ30+CR29)

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.

Overall survival (OS)

Overall survival will be estimated using Kaplan-Meier method.

Regression-free survival (RFS)

Regression-free survival will be estimated using Kaplan-Meier method.

Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0

Surgical success rates

Radiographic Findings

MRI performed.

Pathologic Findings

Blood and tissue collected at various time points.

Full Information

NCT ID

NCT03594630

First Posted

June 20, 2018

Last Updated

September 1, 2023

Sponsor

M.D. Anderson Cancer Center

Collaborators

National Cancer Institute (NCI)

1. Study Identification

Unique Protocol Identification Number

NCT03594630

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2023

Overall Recruitment Status

Not yet recruiting

Study Start Date

October 31, 2023 (Anticipated)

Primary Completion Date

October 31, 2024 (Anticipated)

Study Completion Date

October 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

M.D. Anderson Cancer Center

Collaborators

National Cancer Institute (NCI)

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Rectal Adenocarcinoma, Stage II Rectal Cancer AJCC v8, Stage IIA Rectal Cancer AJCC v8, Stage IIB Rectal Cancer AJCC v8, Stage IIC Rectal Cancer AJCC v8, Stage III Rectal Cancer AJCC v8, Stage IIIA Rectal Cancer AJCC v8, Stage IIIB Rectal Cancer AJCC v8, Stage IIIC Rectal Cancer AJCC v8

7. Study Design

Primary Purpose

Treatment

Study Phase

Early Phase 1

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

120 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Group I (surgical resection)

Arm Type

Active Comparator

Arm Description

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

Arm Title

Group II (active surveillance)

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Chemotherapy

Other Intervention Name(s)

Chemo, Chemotherapy (NOS), Chemotherapy, Cancer, General

Intervention Description

Receive chemotherapy

Intervention Type

Other

Intervention Name(s)

Patient Observation

Other Intervention Name(s)

Active Surveillance, deferred therapy, expectant management, observation, Watchful Waiting

Intervention Description

Receive active surveillance

Intervention Type

Other

Intervention Name(s)

Questionnaire Administration

Intervention Description

Ancillary studies

Intervention Type

Procedure

Intervention Name(s)

Resection of Rectum

Other Intervention Name(s)

Proctectomy

Intervention Description

Undergo surgical resection

Primary Outcome Measure Information:

Title

Overall organ preservation rate

Description

Time Frame

At 12 months

Title

Local tumor regrowth rate

Description

Time Frame

At 12 months

Title

Time to surgery or death

Description

The study will use Kaplan-Meier methods to estimate probability for deferral patients respectively.

Time Frame

Up to 12 months

Secondary Outcome Measure Information:

Title

Decision quality assessment determined by European Organization for Treatment and Research of Cancer Quality of Life Questionnaire (EORTC-QLQ30+CR29)

Description

Time Frame

Up to 3 years

Title

Overall survival (OS)

Description

Overall survival will be estimated using Kaplan-Meier method.

Time Frame

Up to 5 years

Title

Regression-free survival (RFS)

Description

Regression-free survival will be estimated using Kaplan-Meier method.

Time Frame

Up to 5 years

Title

Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0

Time Frame

Up to 5 years

Title

Surgical success rates

Time Frame

Up to 5 years

Title

Radiographic Findings

Description

MRI performed.

Time Frame

Up to 5 years

Title

Pathologic Findings

Description

Blood and tissue collected at various time points.

Time Frame

Up to 5 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

George J. Chang

Phone

713-792-6940

gchang@mdanderson.org

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

George Chang

Organizational Affiliation

M.D. Anderson Cancer Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

M D Anderson Cancer Center

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Contact:

gchang@mdanderson.org

12. IPD Sharing Statement

Links:

URL

http://www.mdanderson.org

Description

MD Anderson Cancer Center

Learn more about this trial

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

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