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Organ Preservation in Early Rectal Cancer Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03548961
Recruitment Status : Recruiting
First Posted : June 7, 2018
Last Update Posted : July 1, 2019
Information provided by (Responsible Party):
Fox Chase Cancer Center

Brief Summary:
This is a single arm phase II study of neoadjuvant chemotherapy followed by local excision and post-operative chemoradiotherapy in patients with early stage, low rectal adenocarcinoma. After completion of pre-treatment tests/procedures (including pelvic MRI/ERUS; MRI is mandatory at baseline and other imaging is encouraged) and confirmation of eligibility, systemic therapy with FOLFOX will be administered for 12 weeks. 2 to 4 weeks after the chemotherapy, restaging of the primary tumor will be done to evaluate response to therapy (Pelvic MRI and /or sigmoidoscopy). Patients with disease progression or inadequate response to chemotherapy to allow local excision will continue with evaluation and treatment per the current standard of care (chemoradiation followed by TME). These patients will be considered failures for the primary endpoint of the study. Patients who respond to the neoadjuvant chemotherapy will proceed with local excision (open, TEMS or TAMIS), 6-12 weeks after the completion of neoadjuvant chemotherapy, followed by 5-FU based chemoradiotherapy 4-12 weeks after local excision. Patients with positive margins at the time of local excision will also be treated as per standard of care and will be considered as failures. Number of patients who can undergo successful local excision with this approach will define the success of the strategy. After chemoradiation therapy post local excision, patients will be followed closely every 3 months for the first 3 years and then every 2 months for the next 2 years (history/physical, CEA and pelvic MRI). Patients who are deemed failures for the primary end-point will be followed as per standard of care, off-study.

Condition or disease Intervention/treatment Phase
Rectal Cancer Drug: FOLFOX regimen Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 19 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: GI-116: Phase II Study of Organ Preservation in Early Rectal Cancer Patients
Actual Study Start Date : May 11, 2018
Estimated Primary Completion Date : March 1, 2022
Estimated Study Completion Date : March 1, 2026

Arm Intervention/treatment
Experimental: Neoadjuvant chemotherapy Drug: FOLFOX regimen
FOLFOX regimen will be administered for 6 cycles. Each cycle is 14 days followed by restaging, local surgery and concurrent chemo radiation therapy

Primary Outcome Measures :
  1. number of patients whose tumor can be resected by local excision with negative margins [ Time Frame: 3 months ]
    Patients who undergo neoadjuvant chemotherapy, and are able to achieve a local excision in early stage lower rectal adenocarcinoma with negative margins

Secondary Outcome Measures :
  1. Number of patients with improved bowel function in patients treated with organ preserving approach for early stage low rectal cancer before and after therapy [ Time Frame: 3 years ]
    Bowel function will be assessed by Bowel Function Index- Functional Assessment of Chronic Illness Therapy (FACIT) version 4 survey to be completed by patients. The survey measures- Physical well-being, social/family well-being, emotional well-being, functional well-being

  2. Number of patients with improved sexual function treated with organ preserving approach for early stage low rectal cancer before and after therapy [ Time Frame: 3 years ]
    Sexual function in women will be assessed by Female Sexual Function Index (FSFI) 2000 and in men will be assessed by International Index of Erectile Function (IIEF) questionnaire respectively to be completed by patients. The questions ask about the patients' sexual feelings and responses.

  3. Number of patients with improved health-related quality of life treated with organ preserving approach for early stage low rectal cancer before and after therapy [ Time Frame: 3 years ]
    Quality of life will be assessed by Patient Reported Outcome Measurement Information System-10 (PROMIS-10) questionnaire to be completed by patients. This questionnaire will have 10 questions.PROMIS Global-10 allows each of the individual items to be examined separately to provide specific information about perceptions of physical function, pain, fatigue, emotional distress, social health and general perceptions of health.

  4. Overall survival (OS) of patients [ Time Frame: 5 years ]
    OS will be assessed from start of treatment for disease that the patient is still alive

  5. Disease-free survival (DFS) of patients [ Time Frame: 5 years ]
    DFS will be assessed as the length of time that patient survived without any sign of symptoms of that cancer

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

Inclusion criteria

  1. Histologically proven adenocarcinoma of the lower rectum (lower border ≤6 cm from anal verge as assessed by pelvic MRI).
  2. Clinical stage T1N0, T2N0, T3N0; high risk T1 and low risk T3 stage patients are also allowed. Clinical staging should be estimated based on the combination of the following assessments: physical exam by the primary surgeon, CT Chest/Abdomen/Pelvis or PET/CT along with Pelvic MRI and Endoscopic Rectal Ultrasound (ERUS). If a pelvic MRI is performed, it is acceptable to perform CT of the chest/abdomen, omitting CT imaging of the pelvis.
  3. No prior therapy for rectal cancer
  4. Age > 18 years.
  5. ECOG performance status 0 or 1
  6. Patients must have normal organ and marrow function as defined below

    • Leukocytes > 3,000/mcL
    • Absolute neutrophil count > 1,500/mcL
    • Platelets > 100,000/mcL
    • Total bilirubin < 1.5 times ULN
    • AST/ALT (SGOT/SGPT) < 3 times institutional normal limits
    • Creatinine < 1.5 times ULN OR
    • Creatinine clearance > 60 Ml/min/1.73 m2 for patients with creatinine levels above institutional normal
  7. Ability to understand and willingness to sign a written informed consent and HIPAA consent document

Exclusion Criteria:

  1. Patients with contraindication to use FOLFOX chemotherapy and pelvic radiation.
  2. Low risk T1 tumors that fulfill all of the following - size<4 cm, lack of lymphovascular invasion and well differentiated histology, are excluded
  3. High risk T3 tumors that fulfill any of the following - circumferential tumor, extension into mesorectal fascia > 5mm, prediction of positive circumferential resection margin, are also excluded.
  4. T4, node positive or advanced rectal adenocarcinoma. Node positivity defined as nodes greater than 1cm in short axis with loss of uniform cortex/fatty hilum
  5. Patients receiving other investigational agents
  6. Patients who have had chemotherapy (for other malignancies) within 3 years prior to registration
  7. Patients with any prior pelvic radiation therapy
  8. Prior malignancies requiring systemic therapy within the last 3 years (as prior therapy can increase toxicity of current chemo regimen, those patients should be excluded).
  9. History of allergic reactions attributed to compound of similar chemical or biologic composition to the agents used in this study
  10. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  11. Known HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with chemotherapeutic drugs. In addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy.
  12. Pregnant or breast feeding.

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 identifier (NCT number): NCT03548961

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Contact: Namrata Vijayvergia, MD 215214-1515

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United States, Pennsylvania
Fox Chase Cancer Center Recruiting
Philadelphia, Pennsylvania, United States, 19111
Contact: Namrata Vijayvergia, MD    215-214-1515   
Sponsors and Collaborators
Fox Chase Cancer Center
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Responsible Party: Fox Chase Cancer Center Identifier: NCT03548961    
Other Study ID Numbers: GI-116
18-1013 ( Other Identifier: FCCC )
First Posted: June 7, 2018    Key Record Dates
Last Update Posted: July 1, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Rectal Neoplasms
Colorectal Neoplasms
Intestinal Neoplasms
Gastrointestinal Neoplasms
Digestive System Neoplasms
Neoplasms by Site
Digestive System Diseases
Gastrointestinal Diseases
Intestinal Diseases
Rectal Diseases