Rectal AdenocarcinomaEarly Phase 1NCT03594630
Active Surveillance and Chemotherapy Before Surgery in Treating Participants With Stage II-III Rectal Cancer
A Early Phase 1 study testing Chemotherapy, Patient Observation and Questionnaire Administration in people with Rectal Adenocarcinoma and Stage II Rectal Cancer AJCC v8, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Rectal Adenocarcinoma, Stage II Rectal Cancer AJCC v8, Stage IIA Rectal Cancer AJCC v8
- What is being tested?
- Chemotherapy, Patient Observation and Questionnaire Administration
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Mar 2024to 31 Oct 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
M D Anderson Cancer Center
Houston, Texas, 77030
gchang@mdanderson.org
About this study
Official title: 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.
Read the detailed description
PRIMARY OBJECTIVES:
- 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:
- 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.
- 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:
- 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.
- 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
- Being tested
- Chemotherapy, Patient Observation, Questionnaire Administration, Resection of Rectum
- Medical subject headings
- Rectal Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ChemotherapyDrug
Also called Chemo, Chemotherapy (NOS), Chemotherapy, Cancer, General
Receive chemotherapy
Patient ObservationOther
Also called Active Surveillance, deferred therapy, expectant management, observation
Receive active surveillance
Questionnaire AdministrationOther
Ancillary studies
Resection of RectumProcedure or surgery
Also called Proctectomy
Undergo surgical resection
| Study group | Role | What they receive |
|---|---|---|
| Group I (surgical resection) | Active comparator | Participants who have achieved clinical complete response undergo standard surgical resection. Receives: 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. Receives: Drug: Chemotherapy; Other: Patient Observation; Other: Questionnaire Administration; Procedure: Resection of Rectum |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: At 12 months
Time to surgery or death
The study will use Kaplan-Meier methods to estimate probability for deferral patients respectively.
Measured: 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)
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.
Measured: Up to 3 years
Overall survival (OS)
Overall survival will be estimated using Kaplan-Meier method.
Measured: Up to 5 years
Regression-free survival (RFS)
Regression-free survival will be estimated using Kaplan-Meier method.
Measured: Up to 5 years
Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Measured: Up to 5 years
Surgical success rates
Measured: Up to 5 years
Radiographic Findings
MRI performed.
Measured: Up to 5 years
Pathologic Findings
Blood and tissue collected at various time points.
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2018
- Study start13 Mar 2024
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rectal AdenocarcinomaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- George Chang, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03594630
- Sponsor's protocol ID
- 2016-0549
- Other IDs
- NCI-2018-01142, 2016-0549
- Status last verified
- May 2026
- First submitted
- 20 Jun 2018
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Organ Preservation With Active Surveillance After Chemoradiation in Rectal Cancer (OPTION)
- ClinicalTrials.gov ID
- NCT03594630
- Acronym / sponsor ID
- 2016-0549
- 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
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 13 Mar 2024 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 20 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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:
- 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.
- 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:
- 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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group I (surgical resection) | Active comparator | Other: Questionnaire Administration, Procedure: Resection of RectumParticipants who have achieved clinical complete response undergo standard surgical resection. |
| Group II (active surveillance) | Experimental | Drug: Chemotherapy, Other: Patient Observation, Other: Questionnaire Administration, Procedure: Resection of RectumParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ChemotherapyAlso: Chemo, Chemotherapy (NOS), Chemotherapy, Cancer, GeneralReceive chemotherapy | Drug | Group II (active surveillance) |
| Patient ObservationAlso: Active Surveillance, deferred therapy, expectant management, observationReceive active surveillance | Other | Group II (active surveillance) |
| Questionnaire AdministrationAncillary studies | Other | Group I (surgical resection), Group II (active surveillance) |
| Resection of RectumAlso: ProctectomyUndergo surgical resection | Procedure or surgery | Group I (surgical resection), Group II (active surveillance) |
Primary outcome
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.
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.
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.
7 secondary outcomes
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
Overall survival (OS)
Time frame: Up to 5 years
Regression-free survival (RFS)
Time frame: Up to 5 years
Incidence of adverse events graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Time frame: Up to 5 years
Surgical success rates
Time frame: Up to 5 years
Radiographic Findings
Time frame: Up to 5 years
Pathologic Findings
Time frame: Up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
George Chang principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Chemotherapy, Patient Observation and Questionnaire Administration in people with Rectal Adenocarcinoma and Stage II Rectal Cancer AJCC v8, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact George J. Chang (713-792-6940, gchang@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall organ preservation rate (time frame: At 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2026.