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

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ClinicalTrials.gov Identifier: NCT03594630
Recruitment Status : Not yet recruiting
First Posted : July 20, 2018
Last Update Posted : June 24, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

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.

Condition or disease Intervention/treatment Phase
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 Drug: Chemotherapy Other: Patient Observation Other: Questionnaire Administration Procedure: Resection of Rectum Early Phase 1

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Organ Preservation With Active Surveillance After Chemoradiation in Rectal Cancer (OPTION)
Estimated Study Start Date : October 2019
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : October 2025

Arm Intervention/treatment
Active Comparator: Group I (surgical resection)
Participants who have achieved clinical complete response undergo standard surgical resection.
Other: Questionnaire Administration
Ancillary studies

Procedure: Resection of Rectum
Undergo surgical resection
Other Name: 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.
Drug: Chemotherapy
Receive chemotherapy
Other Names:
  • Chemo
  • Chemotherapy (NOS)
  • Chemotherapy, Cancer, General

Other: Patient Observation
Receive active surveillance
Other Names:
  • Active Surveillance
  • deferred therapy
  • expectant management
  • observation
  • Watchful Waiting

Other: Questionnaire Administration
Ancillary studies

Procedure: Resection of Rectum
Undergo surgical resection
Other Name: Proctectomy




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.


Secondary Outcome Measures :
  1. 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.

  2. Overall survival (OS) [ Time Frame: Up to 5 years ]
    Overall survival will be estimated using Kaplan-Meier method.

  3. Regression-free survival (RFS) [ Time Frame: Up to 5 years ]
    Regression-free survival will be estimated using Kaplan-Meier method.

  4. Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ Time Frame: Up to 5 years ]
  5. Surgical success rates [ Time Frame: Up to 5 years ]
  6. Radiographic Findings [ Time Frame: Up to 5 years ]
    MRI performed.

  7. Pathologic Findings [ Time Frame: Up to 5 years ]
    Blood and tissue collected at various time points.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03594630


Contacts
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Contact: George J. Chang 713-792-6940 gchang@mdanderson.org

Locations
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United States, Texas
M D Anderson Cancer Center Not yet recruiting
Houston, Texas, United States, 77030
Contact       gchang@mdanderson.org   
Sponsors and Collaborators
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Investigators
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Principal Investigator: George Chang M.D. Anderson Cancer Center

Additional Information:
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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT03594630     History of Changes
Other Study ID Numbers: 2016-0549
NCI-2018-01142 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2016-0549 ( Other Identifier: M D Anderson Cancer Center )
P30CA016672 ( U.S. NIH Grant/Contract )
First Posted: July 20, 2018    Key Record Dates
Last Update Posted: June 24, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Adenocarcinoma
Rectal Neoplasms
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Colorectal Neoplasms
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Intestinal Diseases
Rectal Diseases