Breast Ductal Carcinoma In SituPhase 2NCT02636582
Nelipepimut-S Plus GM-CSF Vaccine Therapy in Treating Patients With Breast Cancer
A Phase 2 study testing Laboratory Biomarker Analysis, Nelipepimut-S Plus GM-CSF Vaccine and Sargramostim in people with Breast Ductal Carcinoma In Situ, run by National Cancer Institute (NCI) at 4 sites in United States.
Trial summary
This phase II trial studies how well nelipepimut-S plus GM-CSF vaccine therapy or sargramostim works in treating patients with breast cancer. Vaccines made from peptide or antigen and/or a person's white blood cells mixed with tumor proteins may help the body build an effective immune response to kill tumor cells that… Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Breast Ductal Carcinoma In Situ
- What is being tested?
- Laboratory Biomarker Analysis, Nelipepimut-S Plus GM-CSF Vaccine and Sargramostim
- Type of treatment
- Vaccines, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 4 sitesUnited States
- Study dates
- 14 Jun 2016to 17 May 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must be pre- or post-menopausal
- Participants must have a diagnosis of DCIS made by core needle biopsy
- Participants must be human leukocyte antigen (HLA)-A2 positive
- Eastern Cooperative Oncology Group (ECOG) performance status must be 0 or 1 (Karnofsky >= 60%)
- Clinical chemistry less than 2 x normal upper limit of normal range
- Platelets >= 100,000/mm^3
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- Hemoglobin >= 10 g/dL
- Blood urea nitrogen < 2 x upper limit of normal (ULN)
- Alkaline phosphatase < 2 x ULN
- Lactate dehydrogenase < 2 x ULN
- Creatinine < 2 x ULN
- Bilirubin < 2 x ULN
- Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) < 2 x ULN
- A normal ejection fraction, as defined by the participant's institution; only limited echocardiograms (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
- Willingness to comply with all study interventions and follow-up procedures
- The ability to understand and willingness to sign a written informed consent document
Who cannot join
- 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; 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
- Any autoimmune disease or other medical condition that, in the opinion of the investigator, would compromise the subject's safety
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- 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
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
M D Anderson Cancer Center
Houston, Texas, 77030
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
About this study
Official title: VADIS Trial: Phase II Trial of Nelipeimut-S Peptide Vaccine in Women With DCIS of the Breast
This phase II trial studies how well nelipepimut-S plus GM-CSF vaccine therapy or sargramostim works in treating patients with breast cancer. Vaccines made from peptide or antigen and/or a person's white blood cells mixed with tumor proteins may help the body build an effective immune response to kill tumor cells that express breast cancer antigens. It is not yet known whether nelipepimut-S plus GM-CSF vaccine or sargramostim is more effective in treating patients with breast cancer.
Read the detailed description
PRIMARY OBJECTIVE:
- Evaluate for nelipepimut-S-specific cytotoxic T lymphocyte (CTL; cluster of differentiation [CD]8+ T cell) response in patients receiving NeuVax (nelipepimut-S plus GM-CSF [sargramostim]) compared to patients receiving GM-CSF alone (control).
SECONDARY OBJECTIVES:
- Toxicity profile and frequency of adverse events in women with ductal carcinoma in situ (DCIS) of the breast receiving nelipepimut-S vaccine as compared to women receiving GM-CSF alone.
II. Presence of DCIS at resection. III. Difference in HER2 expression in the biopsy and the surgical specimen excised post-vaccination.
IV. Histologic responses:
IVa. Degree of lymphocyte infiltration determined on hematoxylin and eosin (H\&E) stained slides and immune infiltration as determined by multiplex immunofluorescence staining for markers including but not limited to CD3, CD4 and CD8.
IVb. Immune infiltrates in normal tissue maximally distant from the tumor (in mastectomy samples).
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive nelipepimut-S plus GM-CSF vaccine intradermally (ID) on days 0 and 14 and then undergo surgery on day 28.
ARM II: Patients receive sargramostim ID on days 0 and 14 and then undergo surgery on day 28.
After completion of study treatment, patients are followed up at 1 and 3 months.
- Conditions
- Breast Ductal Carcinoma In Situ
- Being tested
- Laboratory Biomarker Analysis, Nelipepimut-S Plus GM-CSF Vaccine, Sargramostim, Surgical Procedure
- Medical subject headings
- Carcinoma, Intraductal, Noninfiltrating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laboratory Biomarker AnalysisOther
Correlative studies
Nelipepimut-S Plus GM-CSF VaccineDrug
Also called E75 Plus GM-CSF, E75 Vaccine Plus GM-CSF, HLA A2/A3-Restricted HER-2/neu Peptide Vaccine Plus GM-CSF, Nelipepimut-S Plus Sargramostim
Given ID
SargramostimBiological
Also called 23-L-Leucinecolony-Stimulating Factor 2, DRG-0012, Leukine, Prokine
Given ID
Surgical ProcedureProcedure or surgery
Also called Operation, Surgery, Surgery Type, Surgery, NOS
Undergo surgery
| Study group | Role | What they receive |
|---|---|---|
| Arm I (nelipepimut-S plus GM-CSF vaccine) | Experimental | Patients receive nelipepimut-S plus GM-CSF vaccine ID on days 0 and 14 and then undergo surgery on day 28. Receives: Other: Laboratory Biomarker Analysis; Drug: Nelipepimut-S Plus GM-CSF Vaccine; Procedure: Surgical Procedure |
| Arm II (sargramostim) | Active comparator | Patients receive sargramostim ID on days 0 and 14 and then undergo surgery on day 28. Receives: Other: Laboratory Biomarker Analysis; Biological: Sargramostim; Procedure: Surgical Procedure |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Percent of Nelipepimut-S-specific Cytotoxic T Lymphocyte Response (E75)
Change from baseline in the Mean percent of Nelipepimut-S-specific cytotoxic T lymphocyte response one-month post-surgical resection. Wilcoxon rank sum test exact P-value was used.
Measured: One-Month post-surgical resection from baseline
Secondary outcomes (5)
Intra-tumoral Tumor-infiltrating Lymphocytes (iTILs) Within the Basement Membrane
Change from baseline in the iTILs at surgical resection. Wilcoxon rank sum test exact P-value was used.
Measured: Baseline to surgical resection, up to 5 weeks
Intra-tumoral Tumor-infiltrating Lymphocytes (iTILs)
Change from baseline in the iTILs at surgical resection. Wilcoxon rank sum test exact P-value was used.
Measured: Baseline to surgical resection, up to 5 weeks
Number of Participants With HER2 Expression in Biopsy and Resection Specimens
Difference in HER2 Expression in Biopsy and Resection Specimens. Differences were assessed using a Fisher's exact test.
Measured: Baseline to surgical resection, up to 5 weeks
Number of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 4.03
The number of serious and non serious adverse events for both arms. Graded According to national Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 4.03.
Measured: 3-6 months after surgery
Number of Participants With Presence of Ductal Carcinoma in Situ (DCIS)
Presence of DCIS with Invasive Cancer.
Measured: At resection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2015 (estimated)
- Study start14 Jun 2016
- Primary completion22 Jul 2019
- Study completion17 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Breast Ductal Carcinoma In SituStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Powel H Brown, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02636582
- Sponsor's protocol ID
- NCI-2015-02189
- Other IDs
- NCI-2015-02189, N01-CN-2012-00034, 2016-0164, MDA2014-04-02, N01CN00034, P30CA016672
- Status last verified
- Nov 2023
- First submitted
- 18 Dec 2015
- Last updated
- 5 Dec 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- VADIS Trial: Phase II Trial of Nelipeimut-S Peptide Vaccine in Women With DCIS of the Breast
- ClinicalTrials.gov ID
- NCT02636582
- Acronym / sponsor ID
- NCI-2015-02189
- Conditions
- Breast Ductal Carcinoma In Situ
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 14 Jun 2016 (actual)
- Primary completion
- 22 Jul 2019 (actual)
- Study completion
- 17 May 2023 (actual)
- First posted
- 22 Dec 2015 (estimated)
- Results
- Posted 16 May 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well nelipepimut-S plus GM-CSF vaccine therapy or sargramostim works in treating patients with breast cancer. Vaccines made from peptide or antigen and/or a person's white blood cells mixed with tumor proteins may help the body build an effective immune response to kill tumor cells that express breast cancer antigens. It is not yet known whether nelipepimut-S plus GM-CSF vaccine or sargramostim is more effective in treating patients with breast cancer.
Detailed description
PRIMARY OBJECTIVE:
- Evaluate for nelipepimut-S-specific cytotoxic T lymphocyte (CTL; cluster of differentiation [CD]8+ T cell) response in patients receiving NeuVax (nelipepimut-S plus GM-CSF [sargramostim]) compared to patients receiving GM-CSF alone (control).
SECONDARY OBJECTIVES:
- Toxicity profile and frequency of adverse events in women with ductal carcinoma in situ (DCIS) of the breast receiving nelipepimut-S vaccine as compared to women receiving GM-CSF alone.
II. Presence of DCIS at resection. III. Difference in HER2 expression in the biopsy and the surgical specimen excised post-vaccination.
IV. Histologic responses:
IVa. Degree of lymphocyte infiltration determined on hematoxylin and eosin (H\&E) stained slides and immune infiltration as determined by multiplex immunofluorescence staining for markers including but not limited to CD3, CD4 and CD8.
IVb. Immune infiltrates in normal tissue maximally distant from the tumor (in mastectomy samples).
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive nelipepimut-S plus GM-CSF vaccine intradermally (ID) on days 0 and 14 and then undergo surgery on day 28.
ARM II: Patients receive sargramostim ID on days 0 and 14 and then undergo surgery on day 28.
After completion of study treatment, patients are followed up at 1 and 3 months.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (nelipepimut-S plus GM-CSF vaccine) | Experimental | Other: Laboratory Biomarker Analysis, Drug: Nelipepimut-S Plus GM-CSF Vaccine, Procedure: Surgical ProcedurePatients receive nelipepimut-S plus GM-CSF vaccine ID on days 0 and 14 and then undergo surgery on day 28. |
| Arm II (sargramostim) | Active comparator | Other: Laboratory Biomarker Analysis, Biological: Sargramostim, Procedure: Surgical ProcedurePatients receive sargramostim ID on days 0 and 14 and then undergo surgery on day 28. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Participants must be pre- or post-menopausal
- Participants must have a diagnosis of DCIS made by core needle biopsy
- Participants must be human leukocyte antigen (HLA)-A2 positive
- Eastern Cooperative Oncology Group (ECOG) performance status must be 0 or 1 (Karnofsky >= 60%)
- Clinical chemistry less than 2 x normal upper limit of normal range
Key exclusion criteria (15 listed)
- 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; 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
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 |
|---|---|---|
| Laboratory Biomarker AnalysisCorrelative studies | Other | Arm I (nelipepimut-S plus GM-CSF vaccine), Arm II (sargramostim) |
| Nelipepimut-S Plus GM-CSF VaccineAlso: E75 Plus GM-CSF, E75 Vaccine Plus GM-CSF, HLA A2/A3-Restricted HER-2/neu Peptide Vaccine Plus GM-CSF, Nelipepimut-S Plus SargramostimGiven ID | Drug | Arm I (nelipepimut-S plus GM-CSF vaccine) |
| SargramostimAlso: 23-L-Leucinecolony-Stimulating Factor 2, DRG-0012, Leukine, ProkineGiven ID | Biological | Arm II (sargramostim) |
| Surgical ProcedureAlso: Operation, Surgery, Surgery Type, Surgery, NOSUndergo surgery | Procedure or surgery | Arm I (nelipepimut-S plus GM-CSF vaccine), Arm II (sargramostim) |
Primary outcome
Mean Percent of Nelipepimut-S-specific Cytotoxic T Lymphocyte Response (E75)
Time frame: One-Month post-surgical resection from baseline
Change from baseline in the Mean percent of Nelipepimut-S-specific cytotoxic T lymphocyte response one-month post-surgical resection. Wilcoxon rank sum test exact P-value was used.
5 secondary outcomes
Intra-tumoral Tumor-infiltrating Lymphocytes (iTILs) Within the Basement Membrane
Time frame: Baseline to surgical resection, up to 5 weeks
Intra-tumoral Tumor-infiltrating Lymphocytes (iTILs)
Time frame: Baseline to surgical resection, up to 5 weeks
Number of Participants With HER2 Expression in Biopsy and Resection Specimens
Time frame: Baseline to surgical resection, up to 5 weeks
Number of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 4.03
Time frame: 3-6 months after surgery
Number of Participants With Presence of Ductal Carcinoma in Situ (DCIS)
Time frame: At resection
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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. This study is not recruiting at the moment.
Powel H Brown principal investigator
M.D. Anderson Cancer Center
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Laboratory Biomarker Analysis, Nelipepimut-S Plus GM-CSF Vaccine and Sargramostim in people with Breast Ductal Carcinoma In Situ, run by National Cancer Institute (NCI) at 4 sites 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 16 inclusion and 15 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: finished, as of the sponsor's last update on 5 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 May 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Percent of Nelipepimut-S-specific Cytotoxic T Lymphocyte Response (E75) (time frame: One-Month post-surgical resection from baseline). 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 5 Dec 2023.