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

NCT03594630 | Recruiting Early Phase 1 FDA Drug

• 2 other identifiers: 2016-0549NCI-2018-01142
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (3)

• 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.

  At 12 months

• 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.

  At 12 months

• Time to surgery or death

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

  Up to 12 months

Secondary Outcomes (7)

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

  Up to 3 years

• Overall survival (OS)

  Up to 5 years

• Regression-free survival (RFS)

  Up to 5 years

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

  Up to 5 years

• Surgical success rates

  Up to 5 years

Study Arms (2)

Group I (surgical resection)

ACTIVE COMPARATOR

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

Other: Questionnaire Administration; Procedure: Resection of Rectum

Group II (active surveillance)

EXPERIMENTAL

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; Other: Patient Observation; Other: Questionnaire Administration; Procedure: Resection of Rectum

Interventions

Chemotherapy DRUG

Receive chemotherapy

Also known as: Chemo, Chemotherapy (NOS), Chemotherapy, Cancer, General

Group II (active surveillance)

Patient Observation OTHER

Receive active surveillance

Also known as: Active Surveillance, deferred therapy, expectant management, observation, Watchful Waiting

Group II (active surveillance)

Questionnaire Administration OTHER

Ancillary studies

Group I (surgical resection); Group II (active surveillance)

Resection of Rectum PROCEDURE

Undergo surgical resection

Also known as: Proctectomy

Group I (surgical resection); Group II (active surveillance)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Rectal Neoplasms

Interventions

Drug Therapy; Watchful Waiting; Observation; Proctectomy

Condition Hierarchy (Ancestors)

Colorectal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Intestinal Diseases; Rectal Diseases

Intervention Hierarchy (Ancestors)

Therapeutics; Outcome Assessment, Health Care; Outcome and Process Assessment, Health Care; Quality of Health Care; Health Services Administration; Methods; Investigative Techniques; Surgical Procedures, Colorectal; Digestive System Surgical Procedures; Surgical Procedures, Operative

Study Officials

• George Chang

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

George J. Chang

CONTACT

713-792-6940; gchang@mdanderson.org

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2018
• First Posted: July 20, 2018
• Study Start: March 13, 2024
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: November 6, 2025

Record last verified: 2025-11

Locations

US (1)