The following datasets contain the data available for EPPT MDA2014-04-02. The description and documentation for each file is listed below. SAS7bdat and CSV versions of the actual data will be available to CDAS projects approved to use this study's data.

Analysis Datasets

Raw Datasets

These 42 datasets contain the raw form data received, excluding PII.

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Trial Summary

This randomized 2:1 phase II trial studies the safety and effectiveness of NeuVax (nelipepimut-S + GM-CSF) or GM-CSF alone in women with DCIS of the breast. The change (if any) in in the number of nelipepimut-S-cytotoxic T lymphocytes (CTL) is to be evaluated.

Randomized trial with two arms:

• Arm I: NeuVax (1000 ug nelipepimut-S + 250 ug GM-CSF).
• Arm II: GM-CSF alone (250 ug).

Target Randomizable Enrollment: 48

Enrollment Statistics

Actual Registration: 45

• 13 people randomized (13 of 45 registered)
  • 9 in Arm I: NeuVax (9 of 13 randomized)
    • 9 participants completed study
  • 4 in Arm II: GM-CSF (4 of 13 randomized)
    • 3 participants completed study
    • 1 participant did not complete study due to disease progression
• 32 people not randomized (32 of 45 registered)

Final Analysis Population: 13

Eligibility Criteria

Inclusion Criteria

• Participants must be female ≥18 years of age, pre- or post-menopausal. The minimum age of 18 was chosen as breast cancer is extremely rare in women less than 18 years. Because no dosing or adverse event data are currently available on the use of NeuVax in participants <18 years of age, children are excluded from this study but will be eligible for future pediatric trials, if applicable.
• Participants must have a diagnosis of DCIS made by core needle biopsy
• Participants must be HLA-A2 positive.
• ECOG performance status must be 0 or 1 (Karnofsky ≥ 60%).
• Clinical chemistry less than 2x normal upper limit of normal range; adequate kidney and liver function as measured by creatinine, bilirubin, and liver enzymes (see below):
  • Platelets ≥100,000/mm3
  • Hemoglobin ≥10 g/dL
  • Blood Urea Nitrogen <2 x ULN
  • Alkaline Phosphatase <2 x ULN
  • Lactate Dehydrogenase <2 x ULN
  • Creatinine <2 x ULN
  • Bilirubin <2 x ULN
  • AST (SGOT)/ALT (SGPT) <2 x ULN (ULN = upper limit of normal as defined by the participating institutions laboratory).
• A normal ejection fraction, as defined by the participant's institution (see footnote 1 below).
• Willingness to comply with all study interventions and follow-up procedures.
• The ability to understand and willingness to sign a written informed consent document.

Only limited ECHOs will be used as cardiac evaluation. No other tests are allowed. ECHO is to be done only in HLA-A2 positive participants. If ECHO has been done within 30 days prior to randomization and results showing a normal ejection fraction have been obtained prior to randomization, an additional ECHO is not needed at Baseline.

Exclusion Criteria

• Invasive breast cancer. Areas of microinvasion or suspicious for microinvasion on the core biopsy is allowed.
• History of prior breast cancer treated within the past two years. Patients completing all breast cancer-specific treatment over two years prior to the current diagnosis are eligible.
• History of prior ductal carcinoma in situ (DCIS) treated within the past two years. Patients completing all treatment for a previous diagnosis of DCIS over two years prior to the current diagnosis are eligible.
• Prior lobular carcinoma in situ (LCIS) is allowed.
• Pregnant, unwilling to use adequate contraception during study treatment duration or breastfeeding (see footnote 1 below).
• Any autoimmune disease or other medical condition that, in the opinion of the investigator, would compromise the subject's safety.
• Immune deficiency diseases such as immunoglobulin deficiency or immunosuppressive therapy that might interfere with appropriate immune response.
• Known history of or known active infection with human immunodeficiency virus (HIV), hepatitis B or hepatitis C.
• Patients on chronic steroid therapy or other immunosuppressive therapy except for topical or inhaled steroids known to have low systemic absorption.
• Patients with a known hypersensitivity to GM-CSF, yeast-derived products, or any component of the GM-CSF product (e.g., mannitol).
• Concurrent treatment with other investigational agent.
• History of non-breast malignancy within 5 years prior to randomization, except curatively treated superficial bladder cancer, carcinoma in situ of the cervix (Stage 0 - 1), and basal cell or squamous cell carcinoma of the skin.
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to NeuVax.
• 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.
• No recent or planned immunotherapy.

The effects of NeuVax on the developing human fetus are unknown. For this reason and because NeuVax may be teratogenic, pregnant women will be excluded. All heterosexually active women who may become pregnant must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation OR be post-menopausal defined as any one of the following 1) prior hysterectomy, 2) absence of menstrual period for 1 year in the absence of prior chemotherapy or 3) absence of menstrual period for 2 years in women with a prior history of chemotherapy exposure who were pre-menopausal prior to chemotherapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her study physician immediately.

The Schema is a timeline of the study. It indicates start/end points, visits expected, major testing to be done, and any other information that is crucial to understanding how the study was completed.

Results/Findings:

45 patients were registered; 7 withdrew consent, 1 opted for surgery at an external facility, 20 were ineligible due to negative HLA-A2, and 4 failed screening for other reasons, leaving 13 patients enrolled. The 13 patients were randomized (2:1) into treatment groups, with nine patients receiving NPS+GM-CSF and four patients receiving GM-CSF alone. The two groups were well-matched for age; however, the GM-CSF alone group had higher percentages of African American (50% vs. 22%) and Hispanic (25% vs. 11%) patients as compared to the NPS+GM-CSF group. In general, vaccination was well-tolerated with similar treatment-related toxicity profiles in the NPS+GM-CSF vs GM-GSF groups (Grade 1 - 93.3% vs. 89.3%, Grade 2 - 6.7% vs. 10.7%, respectively). The mean NPS-specific CTL% in the NPS+GM-CSF group at 1-month post-op was double that of the GM-CSF alone group (0.10 +/- 0.12% vs. 0.05 +/- 0.08, p=0.70). In addition, between baseline pre-vaccination and 1-month post-op, the NPS+GM-CSF group experienced an 11-fold increase in percentage of NPS-specific CTL (0.01 +/- 0.02% vs. 0.11 +/- 0.12%) as compared to only a 2.25-fold increase of NPS-specific CTL in the GM-CSF alone group (0.04 +/- 0.07% vs. 0.09 +/- 0.15%).