Vaccine Therapy in Treating Patients With Newly Diagnosed Advanced Colon Polyps
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ClinicalTrials.gov Identifier: NCT02134925 |
Recruitment Status :
Active, not recruiting
First Posted : May 9, 2014
Results First Posted : May 18, 2018
Last Update Posted : January 26, 2022
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Condition or disease | Intervention/treatment | Phase |
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Colorectal Adenoma Colorectal Adenoma With Severe Dysplasia Colorectal Tubulovillous Adenoma | Other: Laboratory Biomarker Analysis Biological: MUC1 Peptide-Poly-ICLC Vaccine Other: Quality-of-Life Assessment Other: Saline | Phase 2 |
PRIMARY OBJECTIVES:
I. To compare the immunogenicity at week 12 of a MUC1 peptide vaccine with adjuvant (MUC1 peptide-poly-ICLC adjuvant vaccine) (administered at 0, 2, and 10 weeks) in participants with a history of an advanced adenoma, randomized to receive MUC1 peptide vaccine versus placebo.
SECONDARY OBJECTIVES:
I. To evaluate the ability of the vaccine to elicit a long-term memory response.
II. To compare the adenoma recurrence rate from surveillance exams occurring at least 1 year and up to 3 years after week 0 vaccine administration - MUC1 versus placebo.
III. To compare the adenoma recurrence rates between MUC1 and placebo by excluding the following types of adenomas: participants with adenomas =< 5 mm; participants with adenomatous tissue which may represent residual adenoma at the site of the previous advanced adenoma; participants with adenomatous tissue detected in the same segment of the bowel as the previous advanced adenoma.
IV. To assess adverse events to the MUC1 peptide vaccine in comparison to placebo during Parts I and II.
V. To assess patient reported injection site reaction events from the Vaccine Report Card.
TERTIARY OBJECTIVES:
I. To compare the anti-MUC1 antibody titer at the time of surveillance colonoscopy for the purpose of evaluating the anti-MUC1 antibody response in relation to adenoma recurrence.
II. To evaluate MUC1 expression on baseline advanced adenomas and on recurrent adenomas detected at surveillance colonoscopy.
III. To evaluate levels of circulating myeloid derived suppressor cells (MDSC) in the vaccinated and the placebo group and correlate with anti-MUC1 antibody levels and adenoma recurrence.
IV. To establish a biospecimen repository archive including live cells, plasma, and germline deoxyribonucleic acid (DNA) for future immunologic (e.g. MUC1-specific T cells) and other assays (systems biology approach to detect differences between responders and non-responders), testing not currently accommodated within the budget of this trial.
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
ARM I: Participants receive MUC1 peptide-poly-ICLC adjuvant vaccine subcutaneously (SC) in weeks 0, 2 and 10 and a booster injection in week 53.
ARM II: Participants receive saline SC in weeks 0, 2, and 10 and a booster injection in week 53.
After completion of treatment, patients are followed up every 6 months for up to 3 years.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 110 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Double (Participant, Investigator) |
Primary Purpose: | Prevention |
Official Title: | Randomized, Double-Blind, Placebo-Controlled Trial of MUC1 Vaccine in Patients With Newly Diagnosed Advanced Adenomas |
Actual Study Start Date : | June 23, 2014 |
Actual Primary Completion Date : | January 27, 2017 |
Arm | Intervention/treatment |
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Experimental: Arm I (MUC1 peptide-poly-ILCLC adjuvant vaccine)
Participants receive MUC1 peptide-poly-ICLC adjuvant vaccine SC in weeks 0, 2 and 10 and a booster injection in week 53.
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Other: Laboratory Biomarker Analysis
Correlative studies Biological: MUC1 Peptide-Poly-ICLC Vaccine Given SC Other: Quality-of-Life Assessment Ancillary studies
Other Name: Quality of Life Assessment |
Placebo Comparator: Arm II (saline)
Participants receive saline SC in weeks 0, 2, and 10 and a booster injection in week 53.
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Other: Laboratory Biomarker Analysis
Correlative studies Other: Quality-of-Life Assessment Ancillary studies
Other Name: Quality of Life Assessment Other: Saline Given SC
Other Names:
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- Change in Anti-MUC1 Immunoglobulin G (IgG) Levels as Determined by Enzyme-linked Immunosorbent Assay (ELISA) [ Time Frame: Week 0 to week 12 ]The ratio of the week 12 to week 0 IgG levels will be calculated and compared between the MUC1 vaccine and placebo. The Wilcoxon Rank-Sum test will be used. For all measurements of response (i.e. the primary endpoint), the 95% confidence intervals will also be provided.
- Adenoma Recurrence Rate [ Time Frame: At least one year and up to 3 years ]The secondary endpoint for Part 3 will evaluate the adenoma recurrence rate from surveillance exams. The rate is defined as the percentage of participants with adenoma recurrence.
- Booster Response [ Time Frame: At week 55 ]The key secondary endpoint for Part 2 will assess the booster response at week 55 vs. week 52 for the vaccine as compared to placebo. The IgG ratios are summarized according to the following categories : <1, 1-<1.5, 1.5-<2, and >=2 (1-year response rate).
- Participant-reported Injection Site Reactions - Redness at the Injection Site, Swelling/Induration, Itching at Site, and Skin Warmth at Site [ Time Frame: Up to 55 weeks ]Participant-reported injection site reaction information is collected by the use of a participant-completed Vaccine Report Card. Participant-reported injection site reactions will be compared between study arms and are summarized below for redness at the injection site, swelling/induration, itching at site, and skin warmth at site.
- Participant-reported Injection Site Reactions - Pain at the Injection Site Without Touching, and Tenderness (Pain at the Injection Site With Touch) [ Time Frame: Up to 55 weeks ]Participant-reported injection site reaction information is collected by the use of a participant-completed Vaccine Report Card. Participant-reported injection site reactions will be compared between study arms and are summarized below for pain at the injection site without touching, and tenderness (pain at the injection site with touch).
- Number of Patients With at Least a 2-Fold Increase in the IgG Ratio [ Time Frame: At 12 weeks ]The frequency and percentage of patients with at least a 2-fold increase in the IgG Ratio will be calculated and compared between the MUC1 vaccine and placebo. The Fisher's exact test will be used.
- Anti-MUC1 Antibody Titer by ELISA [ Time Frame: At approximately week 156 ]Comparisons between MUC1 and placebo will be performed using a two-sample t-test or Wilcoxon rank sum test, as appropriate. All categorical variables will be analyzed using chi-square tests or Fisher's exact test.
- Change in Levels of Circulating MDSC in Peripheral Blood Mononuclear Cells by Flow Cytometry [ Time Frame: Baseline to up to 3 years ]MDSC levels will be correlated with anti-MUC1 antibody levels and adenoma recurrence. Descriptive statistics and simple scatter plots will be generated to review the continuous biomarker data. In addition, for continuous biomarker values, the actual and percent change in the level of each of the biomarkers from baseline to post-baseline time points will be explored within each arm using Wilcoxon signed rank tests, and paired sample t-tests.
- Change in MUC1 Expression [ Time Frame: Baseline to up to 3 years ]Descriptive statistics and simple scatter plots will be generated to review the continuous biomarker data. In addition, for continuous biomarker values, the actual and percent change in the level of each of the biomarkers from baseline to post-baseline time points will be explored within each arm using Wilcoxon signed rank tests, and paired sample t-tests.
- Establishment of a Biospecimen Repository Archive Including Live Cells, Plasma, and Germline DNA for Future Immunologic and Other Assays [ Time Frame: Up to 3 years ]
- Incidence of Adverse Events as Graded by the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0. [ Time Frame: Up to 3 years ]

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Ages Eligible for Study: | 40 Years to 70 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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History of at least one of the following conditions in the previous 12 months:
- Colorectal adenoma(s) >= 1 cm in maximal diameter
- Colorectal adenoma(s) with villous or tubulovillous histology
- Colorectal adenoma(s) with high grade (severe) dysplasia
- Presumptive evidence that all adenomatous lesions, including qualifying advanced adenoma, have been completely removed
- Ability to understand and the willingness to sign a written informed consent document
- Willingness to undergo screening tests and procedures
- Willingness to provide blood samples for toxicity monitoring and research purposes
- Not pregnant or nursing; note: a negative (serum or urine) pregnancy test must be documented =< 7 days prior to registration/randomization for women of childbearing potential
- Willingness to employ adequate contraception through week 53 of the study; note: women of childbearing potential and men must agree to use adequate contraception (hormonal, barrier method of birth control, abstinence) prior to study entry and for the period of active vaccination (through week 53); should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her physician immediately
- Eastern Cooperative Oncology Group (ECOG) performance status =< 1 (Karnofsky >= 70%)
- Hemoglobin greater than 90% of the lower limit of institutional normal
- Platelets >= 100 B/L (10^9/L)
- White blood cell (WBC) > 2.5 B/L (10^9/L)
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]), alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 1.5 x institutional upper limit of normal
- Alkaline phosphatase =< 1.5 x institutional upper limit of normal
- Total bilirubin =< 1.5 x institutional upper limit of normal
- Blood urea nitrogen (BUN) =< 1.5 x institutional upper limit of normal
- Creatinine =< 1.5 x institutional upper limit of normal
- Antinuclear antibody (ANA) test result excludes overt autoimmune disease; note: test result may be reported in any of the following formats: =< 1:160, negative, or < 1.0
Exclusion Criteria:
- History of any colorectal cancer
- History of other malignancy =< 5 years prior to the registration/randomization evaluation, with the exception of basal cell or squamous cell skin cancer
- Presence of an active acute or chronic infection or uncontrolled illness including, but not limited to unstable congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Acquired immunosuppressive diseases such as active human immunodeficiency virus (HIV) infection or congenital diseases of immunity
- History of heritable cancer syndrome (familial adenomatous polyposis [FAP], hereditary nonpolyposis colorectal cancer [HNPCC])
- History of auto-immune disease such as, but not restricted to, inflammatory bowel disease, systemic lupus erythematosus, rheumatoid arthritis, ankylosing spondylitis, scleroderma, multiple sclerosis, Hashimoto's thyroiditis, or Grave's disease
- Current or planned use of immunomodulators including: infliximab, 6-MP (mercaptopurine), methotrexate, cyclosporine, or other immunomodulatory drugs
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to the study agent
- Pregnant women
- Breastfeeding women
- Diagnosis of nonalcoholic steatohepatitis (NASH) and a NAFLD (nonalcoholic fatty liver disease) activity score (NAS) >= 5; NOTES and EXCEPTIONS: NAS is based on findings from a liver biopsy; participants with NAS of =< 2 are eligible for enrollment; participants with NAS of 3-4 must be discussed with the principal investigator and Division of Cancer Prevention (DCP) before enrollment to consider other risk factors (i.e., obesity, alcohol intake); participants with a prior diagnosis of NASH and no available NAS must be discussed with the principal investigator and DCP before enrollment to considered risk factors (i.e., obesity, alcohol intake)
- Receiving any other investigational agent =< 3 months prior to registration/randomization, except innocuous agents with no known interaction with the study agent (e.g., standard dose multivitamins or topical agents for limited skin conditions)
- Any use of oral corticosteroids =< 12 weeks prior to registration/randomization

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): NCT02134925
United States, Massachusetts | |
Massachusetts General Hospital Cancer Center | |
Boston, Massachusetts, United States, 02114 | |
United States, Minnesota | |
Mayo Clinic in Rochester | |
Rochester, Minnesota, United States, 55905 | |
United States, Missouri | |
Kansas City Veterans Affairs Medical Center | |
Kansas City, Missouri, United States, 64128 | |
United States, Pennsylvania | |
Thomas Jefferson University Hospital | |
Philadelphia, Pennsylvania, United States, 19107 | |
Fox Chase Cancer Center | |
Philadelphia, Pennsylvania, United States, 19111 | |
University of Pittsburgh Cancer Institute (UPCI) | |
Pittsburgh, Pennsylvania, United States, 15232 | |
Puerto Rico | |
University of Puerto Rico | |
San Juan, Puerto Rico, 00936 |
Principal Investigator: | Robert E Schoen | Mayo Clinic |
Documents provided by National Cancer Institute (NCI):
Responsible Party: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT02134925 |
Obsolete Identifiers: | NCT02886000 |
Other Study ID Numbers: |
NCI-2014-01080 NCI-2014-01080 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ) HHSN261201200042I N01-CN-2012-00042 MAY2013-01-01 ( Other Identifier: Mayo Clinic in Rochester ) MAY2013-01-01 ( Other Identifier: DCP ) N01CN00042 ( U.S. NIH Grant/Contract ) P30CA015083 ( U.S. NIH Grant/Contract ) |
First Posted: | May 9, 2014 Key Record Dates |
Results First Posted: | May 18, 2018 |
Last Update Posted: | January 26, 2022 |
Last Verified: | January 2022 |
Adenoma Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms |
Poly ICLC Interferon Inducers Immunologic Factors Physiological Effects of Drugs |