Efficacy and Safety Study of NeuVax™ (Nelipepimut-S or E75) Vaccine to Prevent Breast Cancer Recurrence (PRESENT)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Galena Biopharma, Inc.
ClinicalTrials.gov Identifier:
First received: November 20, 2011
Last updated: April 14, 2015
Last verified: April 2015

Purpose of this trial:

  1. To assess the efficacy and safety of NeuVax™ administered with adjuvant Leukine® (sargramostim, GM-CSF).
  2. To evaluate and compare the disease free survival (DFS) in the vaccinated and control subjects.

Condition Intervention Phase
Breast Cancer With Low to Intermediate HER2 Expression
Biological: NeuVax™ vaccine
Biological: Leukine® (sargramostim, GM-CSF) and water for injection
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: PRESENT: Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer With Low to Intermediate HER2 Expressions With NeuVax™Treatment

Resource links provided by NLM:

Further study details as provided by Galena Biopharma, Inc.:

Primary Outcome Measures:
  • Comparison of DFS in vaccine treated patients and control patients [ Time Frame: 36 months ] [ Designated as safety issue: No ]
    The primary objective is to compare the DFS in subjects with operable early-stage, node-positive breast cancer who receive standard of care multimodality therapy plus NeuVax™ as the treatment group or standard of care multimodality therapy plus the vaccine adjuvant, Leukine® as the control group.

Secondary Outcome Measures:
  • Assessment of DFS and OS at 3, 5 and 10 years in vaccine and control groups, respectively; assessment of safety [ Time Frame: 3 through 10 years ] [ Designated as safety issue: Yes ]

    The secondary objectives are to evaluate the 2 groups of subjects for:

    • 5- and 10-year DFS
    • 3-year OS
    • 5- and 10-year OS
    • Time to recurrence (TTR), time to local recurrence (TTLR), time to distant recurrence (TTDR), time to bone metastases (TTBM)
    • Overall safety profile and adverse events (AEs)

Estimated Enrollment: 700
Study Start Date: November 2011
Estimated Study Completion Date: April 2025
Estimated Primary Completion Date: April 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NeuVax™
NeuVax™ in WFI solution with Leukine®
Biological: NeuVax™ vaccine
E75 peptide acetate (HER2/neu p366-379) in WFI (1.5 mg/mL) mixed with 250 micrograms Leukine® (sargramostim, GM-CSF), will be administered in intradermal injections, in four divided doses (4 sites on upper thigh) once a month, for six consecutive months. At the end of the six months, NeuVax™ vaccine boosters will be given the same way once every six months for the next five times (totaling 36 months).
Other Name: E75 peptide acetate , WFI, sargramostim, GM-CSF
Active Comparator: Leukine®
Leukine® with WFI
Biological: Leukine® (sargramostim, GM-CSF) and water for injection
Leukine® 250 micrograms in water for injection, will be administered in intradermal injections, in four divided doses (4 sites on upper thigh) once a month, for six consecutive months. At the end of the six months, boosters inoculations will be given the same way once every six months for the next five times (totaling 36 months).
Other Name: Leukine®

Detailed Description:

This is a multicenter, multinational, prospective, randomized, double-blind, controlled Phase 3 study.

The subjects eligible for this trial have an early stage node-positive breast cancer. Their tumors express low or intermediate levels of the HER2 protein. NeuVax™ will be administered after completion of front-line, standard of care therapy (surgery, radiation therapy, and chemotherapy) and can be given concomitantly with physician prescribed endocrine treatment.

NeuVax™ is the immmunodominant nonapeptide derived from the extracellular domain of the HER2 protein, a well-established target for therapeutic intervention in breast carcinoma. The nelipepimut sequence stimulates specific CD8+ cytotoxic T lymphocytes (CTL) following binding to HLA-A2/A3 molecules on antigen presenting cells (APC). These activated specific CTLs recognize, neutralize and destroy through cell lysis HER2 expressing cancer cells, including occult cancer cells and micrometastatic foci. The nelipepimut immune response can also generate CTLs to other immunogenic peptides through inter- and intra-antigenic epitope spreading. Based on a successful Phase 2 trial, which achieved its primary endpoint of DFS, the Food and Drug Administration (FDA) granted NeuVax a Special Protocol Assessment (SPA) for its Phase 3 PRESENT (Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer with Low to Intermediate HER2 Expression with NeuVax Treatment) study.

The active portion of the study will last three years (36 months). The follow-up will last from 5 to 10 years.


  1. Primary efficacy endpoint:

    • 3-year DFS
  2. Secondary efficacy endpoints:

    • 5- and 10-year DFS
    • 3-year OS
    • 5- and 10-year OS
    • Safety profile, and adverse events (AEs)
    • Patterns of recurrence to include Time to recurrence (TTR), time to local recurrence (TTLR), time to distant recurrence (TTDR), time to bone metastases (TTBM)

Safety Assessments:

Subjects will be assessed at every study visit for the safety endpoints, AEs,vital signs, physical examinations and laboratory data; yearly follow-up of survival will include imaging studies, ECGs, MUGA or ECHO scans and concomitant medications.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Pathological diagnosis of invasive adenocarcinoma of the breast
  • Breast cancer completely excised, or patient receiving neoadjuvant therapy prior to surgery
  • One of these 2 surgical treatments and axillary staging with sentinel lymph node dissection or axillary dissection level I/II:

    1. Total mastectomy-patients with a positive sentinel lymph node must have undergone a completion axillary dissection level I/II
    2. BCS (lumpectomy)-patients with a positive sentinel lymph node must have undergone a completion axillary dissection level I/II unless they had clinically node negative T1-T2 tumors and fewer than 3 involved lymph nodes
  • Node-positive disease
  • Primary tumor stage T1-3 at initial diagnosis
  • HER2 negative (HER2 1+ by IHC or HER2 2+ by IHC/FISH)
  • HLA-A2 or HLA-A3 haplotype
  • Completed NCCN approved neo-adjuvant/adjuvant chemotherapy or both
  • Completed radiation therapy
  • No evidence of disease
  • Able and willing (or have legal representative) to understand the study and provide consent

Key Exclusion Criteria:

  • Bilateral breast malignancy or suspicious mass in opposite breast
  • Inflammatory breast malignancy
  • History of prior breast cancer, ductal carcinoma in situ
  • Prior trastuzumab therapy
  • New York Heart Association Stage 3 or 4 cardiac disease
  • Sensory/motor neuropathy ≥ Grade 2
  • Autoimmune diseases or immune deficiency disease
  • Subjects on chronic steroid therapy, other immunosuppressive therapy
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01479244

  Hide Study Locations
United States, Alabama
Alabama Oncology
Bessemer, Alabama, United States, 35022
United States, California
St. Jude Medical Center, Virginia K. Crosson Cancer Center
Fullerton, California, United States, 92801
South Orange County Surgical Medical Group
Laguna Hills, California, United States, 92653
Cedars-Sinai Medical Center Samuel Oschin Comprehensive Cancer Institute
Los Angeles, California, United States, 90048
University of Southern California/Norris Cancer Hospital
Los Angeles, California, United States, 90033
Moores UC San Diego Cancer Center (UCSD)
San Diego, California, United States, 92093
University of California, San Francisco (UCSF), Hellen Diller Family Comprehensive Cancer Center
San Francisco, California, United States, 94115
United States, Colorado
University of Colorado Cancer Center, Anschutz Medical Campus
Aurora, Colorado, United States, 80045
United States, Florida
Holy Cross Hospital-Michael & Dianne Bienes Comprehensive Cancer Center
Fort Lauderdale, Florida, United States, 33308
Cancer Specialists of North Florida (ICON)
Jacksonville, Florida, United States, 32256
MD Anderson Center-Orlando Health
Orlando, Florida, United States, 32806
Florida Cancer Research Institute (Florida Cancer Care)
Plantation, Florida, United States, 33317
H.Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, United States, 33612
United States, Georgia
Northeast Georgia Cancer Care
Athens, Georgia, United States, 30607
Central Georgia Cancer Care
Macon, Georgia, United States, 31201
United States, Illinois
Northwestern University Feinberg School of Medicine, Lynn Sage Comprehensive Breast Center
Chicago, Illinois, United States, 60611
United States, Kansas
Cancer Center of Kansas
Wichita, Kansas, United States, 67214
United States, Kentucky
James Graham Brown Cancer Center, University of Louisville
Louisville, Kentucky, United States, 40202
United States, Louisiana
Mary Bird Perkins Cancer Center
Baton Rouge, Louisiana, United States, 70809
United States, Maryland
Center of Cancer and Blood Disorders (VEEDA network)
Bethesda, Maryland, United States, 20817
United States, Massachusetts
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
Lahey Clinic Department of Hematology and Oncology
Burlington, Massachusetts, United States, 01805
United States, Michigan
William Beaumont Hospital Cancer Center
Royal Oak, Michigan, United States, 48071
United States, New York
Eastchester Cancer Care Center
Bronx, New York, United States, 10469
Weill Cornell Medical College
New York, New York, United States, 100665
United States, North Dakota
St. Alexius Medical Center, Mid Dakota Clinic, PC
Bismarck, North Dakota, United States, 58501
United States, Oregon
OHSU Knight Cancer Institute
Portland, Oregon, United States, 97239
United States, Pennsylvania
Conemaugh Memorial Hospital-Memorial Medical Center
Johnstown, Pennsylvania, United States, 15905
University of Pittsburgh Medical Center (UPMC) Cancer Center
Pittsburgh, Pennsylvania, United States, 15232
United States, Tennessee
The West Clinic
Memphis, Tennessee, United States, 38120
United States, Texas
UT Southwestern Medical Center / Simmons Comprehensive Cancer Center
Dallas, Texas, United States, 75390
Brooke Army Medical Center (now SAMMC)
Ft. Sam Houston, Texas, United States, 78234
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Clinical Trials of Texas, Inc
San Antonio, Texas, United States, 78229
United States, Virginia
Virginia Cancer Specialists
Fairfax, Virginia, United States, 22031
United States, Washington
Swedish Cancer Institute Research
Seattle, Washington, United States, 98104
Multicare Health System-Research Institute
Tacoma, Washington, United States, 98405
United States, Wisconsin
Medical College of Wisconsin Department of Surgery Froedtert Hospital and Cancer Center
Milwaukee, Wisconsin, United States, 52336
Central Onco Hospital
Plovdiv, Bulgaria, 4002
Sofia, Bulgaria, 1303
Complex Oncology Center, Veliko Tarnovo
Tarnovo, Bulgaria, 5000
Sveta Marina
Varna, Bulgaria, 9010
Canada, Alberta
Cross Cancer Institute
Edmonton, Alberta, Canada, T6G1Z2
Canada, Ontario
Ottawa Hospital Cancer Centre
Ottawa, Ontario, Canada, K1H 8L6
Health Sciences North Cancer Centre
Sudbury, Ontario, Canada, P3E 5J1
Sunnybrook Research Institute
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Hospital Charles LeMorne
Greenfield Park, Quebec, Canada, J4V 2H1
Czech Republic
Masaryk Memorial Cancer Institute, Comprehensive Cancer Care Clinic
Brno, Czech Republic, 656 53
University Hospital Hradec Kralove, Clinic of Oncology and Radiotherapy
Hradec Kralove, Czech Republic, 500 05
University Hospital Olomouc, Clinic of Oncology
Olomouc, Czech Republic, 775 20
General University Hospital in Prague, Clinic of Oncology
Prague 2, Czech Republic, 128 08
Institut de Cancérologie de l'Ouest Siet Paul Papin
Angers, Cedex 9, France, 49933
Centre Oscar Lambret
Cédex, Lille, France, 59020
Institut de Cancérologie de l'Ouest/René Gauducheau
St Herblain, Nantes Cedex, Loire-Atlantique, France, 44805
Charité Campus Mitte Universitaetsmedizin-Berlin Brustzentrum/Studienzentrum
Berlin, Germany, 10117
Brustzentrum Klinikum Bremen-Mitte gGmbH
Bremen, Germany, 28177
Universitätsklinikum Erlangen, Frauenklinik
Erlangen, Germany, D-91054
Kliniken Essen Mitte, Evang. Huyssens Stiftung/Knappschaft GmbH, Klinik für Senologie/Brustzentrum
Essen, Germany, D-45136
Universitätsklinikum Frankfurt, Klinik für Frauenheilkunde und Geburtshilfe
Frankfurt, Germany, 60590
Nationales Centrum für Tumorerkrankungen (NCT), Gynäkologische Onkologie
Heidelberg, Germany, 69120
Universitätsklinikum Schleswig-Holstein, Campus Kiel
Kiel, Germany, 24105
Universität Marburg
Marburg, Germany, 35043
Rotkreuzklinikum München gGmbH, Frauenklinik
Muenchen, Germany, 80637
Universitätsklinikum Münster, Klinik und Poliklinik für Frauenheilkunde und Geburtshilfe
Münster, Germany, 48149
Klinikum Stuttgart, Krankenhaus Bad Cannstatt, Frauenklinik
Stuttgart, Germany, 70374
Klinikum Mutterhaus der Borromaeerinnen
Trier, Germany, 54290
Gemeinschaftspraxis für Innere Medizin, Haematologie und internistische Onkologie Dr. R Schlag/Dr. Schoettker
Wuerzburg, Germany, D-97080
Hetenyi Geza Hospital, Department of Oncology
Szolnok, Jasz-Nagykun-Szolnok, Hungary, H-5000
Semmelweis University, Department of Diagnostic Radiology and Oncotherapy
Budapest, Hungary, H-1083
Petz Aladar County Teaching Hospital, Center for Oncoradiology
Gyor, Hungary, H-9024
Kaposi Mor County Teaching Hospital, Center for Clinical Oncology
Kaposvar, Hungary, 7400
Borsod-Abauj-Zemplen County Hospital and University Educational Hospital, Center for Clinical Oncology and Radiology
Miskolc, Hungary, 3526
Sheba Medical Centre
Tel Hasomer, Ramat Gan, Israel, 52621
Rambam Health Care Campus
Haifa, Israel, 31096
Rabin Medical Centre
Petach Tikva, Israel, 49100
Kaplan Medical Centre
Rehovot, Israel, 76100
Provincial Specialist Hospital in Wroclaw, Department of Chemotherapy
Kamienskiego, Wroclaw, Poland, 51-124
Polish Red Cross Marine Hospital, Department of Chemotherapy
Gdynia, Poland, 81-519
Nicolaus Copernicus Memorial Provincial Specialist Hospital in Lodz, Department of Chemotheraphy
Lodz, Poland, 93-513
Greater Poland Cancer Center, Department of Chemotherapy
Poznan, Poland, 61-866
Warsaw, Poland, 04-125
Non-Public Health Care Facility MAGODENT
Warsaw, Poland, 04-125
"Gavril Curteanu" Municipal Clinical Hospital
Oradea, Bihor, Romania, 410469
Medisprof SRL, Department of Medical Oncology
Cluj-Napoca, Cluj, Romania, 400058
Oncology Center "Sf. Nectarie", Department of Medical Oncology
Craiova, Dolj County, Romania, 200347
Dr. Constantin Opris County Emergency Hospital, Department of Oncology
Baia Mare, Maramures, Romania, 430031
Coltea Clinical Hospital, Oncology Department
Bucharest, Romania, 030171
Bucharest Universtiy Emergency Hosptial, Oncology Department
Bucharest, Romania, 050098
Filantropia Clinical Hospital, Obstetrics and Gynaecology Department
Bucharest, Romania, 011171
"Prof. Dr. Ion Chiricuta" Institute of Oncology
Cluj-Napoca, Romania, 400015
Iasi Regional Oncology Institute, Department of Medical Oncology
Iasi, Romania, 700483
Oncomed SRL, Department of Medical Oncology
Timisoara, Romania, 300239
Russian Federation
Republican Oncology Center
Saransk, Republic of Mordovia, Russian Federation, 430032
Republican Clinical Oncoogy Center
Kazan, Republic of Tatarstan, Russian Federation, 420029
Pyatigorsk Oncology Center
Pyatigorsk, Stavropol Region, Russian Federation, 357502
Arkhangelsk Clinical Oncology Center
Arkhangelsk, Russian Federation, 163045
Sverdlovsk Regional Oncology Center
Ekaterinburg, Russian Federation, 620036
Regional Oncology Center
Irkutsk, Russian Federation, 664035
Ivanovo Regional Oncology Center
Ivanovo, Russian Federation, 153040
Clinical Oncology Center #1, Department of Healthcare of Krasnodar Territory Administration
Krasnodar, Russian Federation, 350040
Krasnoyarsk Kryzhanovsky Regional Oncology Center
Krasnoyarsk, Russian Federation, 660133
Kursk Regional Clinical Oncology Center
Kursk, Russian Federation, 305035
N.N. Blokhin Russian Cancer Research Center
Moscow, Russian Federation, 115478
N.N. Blokhin Russian Cancer Reserach Center
Moscow, Russian Federation, 115478
Clinical Diagnostics Center
Nizhny Novgorod, Russian Federation, 603006
Nizhny Novgorod Regional Oncology Center
Nizhny Novgorod, Russian Federation, 603081
Orel Oncology Center
Orel, Russian Federation, 302020
Orenburg Regional Clinical Oncology Center
Orenburg, Russian Federation, 460051
Medical Institution of Ryazan Region
Ryazan, Russian Federation, 390011
Samara Regional Clinical Oncology Center
Samara, Russian Federation, 443031
Petrov Research Institute of Oncology
St Petersburg, Russian Federation, 197758
Leningrad Regional Oncology Center
St. Petersburg, Russian Federation, 191104
Mechnikov North-Western State Medical University
St. Petersburg, Russian Federation, 195067
Municipal Clinical Oncology Center
St. Petersburg, Russian Federation, 198255
St. Petersburg Clinical Hospital
St. Petersburg, Russian Federation, 194017
Tambov Regional Clinical Oncology Center
Tambov, Russian Federation, 392013
Research Institute of Oncology
Tomsk, Russian Federation, 634028
Bashkir State Medical University
Ufa, Russian Federation, 450000
Regional Clinical Oncology Center
Veliky Novgorod, Russian Federation, 173016
Volgograd Regional Oncology Center #3
Volzhskiy, Russian Federation, 404130
Yaroslavl Regional Oncology Center
Yaroslavl, Russian Federation, 150054
Kryvyi Rih Oncology Center/Dnipropetrovsk Regional Council, Department of Chemotherapy
Kryvyi Rih, Dnipropetrovsk Region, Ukraine, 50000
Mariupol City Interdistrict Oncology Center, Surgery Department #2
Mariupol, Donetsk Region, Ukraine, 87500
Kharkiv Regional Clinical Oncology Center, Day Care Unit
Kharkiv, Kyivskyi District, Ukraine, 61070
Chernivtsi Regional Clinical Oncology Center, Day Care Unit
Chernivtsi, Ukraine, 58013
Dnipropetrovsk City Multispecialty Clinical Hospital #4/Dnipropetrovsk Regional Council, Department of Chemotherapy
Dnipropetrovsk, Ukraine, 49102
Public Institution: Kiev Regional Oncology Hospital within Kyiv Regional Council
Kiev, Ukraine, 04107
Kirovohrad Regional Oncology Center, Department of Mammology
Kirovohrad, Ukraine, 25011
Lviv State Regional Treatment and Diagnostics Oncology Center, Department of Chemotherapy
Lviv, Ukraine, 79031
Odesa Regional Clinical Hospital, Center for Mastology
Odesa, Ukraine, 65000
Zakarpattia Regional Clinical Oncology Center, Department of Chemotherapy
Uzhhorod, Ukraine, 88014
Vinnytsia Regional Clinical Oncology Center
Vinnytsia, Ukraine, 21029
United Kingdom
Macclesfield District General Hospital
Macclesfield, Cheshire, United Kingdom, SK10 3BL
The Christie NHS Foundation Trust
Withington, Manchester, United Kingdom, M20 4BX
The Royal Marsden NHS Foundation Trust
Sutton, Surrey, United Kingdom, SM2 5PT
Leeds Teaching Hospitals Trust, St. James University Hospital
Leeds, West Yorkshire, United Kingdom, LS9 7TF
Leicester Royal Infirmary
Leicester, United Kingdom, LE1 5WW
The Royal Marsden NHS Foundation Trust
London, United Kingdom, SW3 6JJ
James Cook University Hospital
Middlesborough, United Kingdom, TS4 3BW
Academic Unit of Oncology, Nottingham University Hospital-City Campus
Nottingham, United Kingdom, NG7 2UH
Plymouth Oncology Centre, Derriford Hospital
Plymouth, United Kingdom, PL6 8DH
Weston Park Hospital
Sheffield, United Kingdom, S10 2SJ
Southampton General Hospital
Southampton, United Kingdom, SO16 6YD
University Hospital of North Staffordshire
Stoke on Trent, United Kingdom, ST4 6QG
Sponsors and Collaborators
Galena Biopharma, Inc.
Principal Investigator: Elizabeth A Mittendorf, MD,FACS M.D. Anderson Cancer Center
  More Information

Additional Information:
Responsible Party: Galena Biopharma, Inc.
ClinicalTrials.gov Identifier: NCT01479244     History of Changes
Other Study ID Numbers: NeuVax™ PH3-01, BB-IND 009187, 2011-005219-98
Study First Received: November 20, 2011
Last Updated: April 14, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Galena Biopharma, Inc.:
low or intermediate
breast cancer
node-positive tumors

Additional relevant MeSH terms:
Breast Neoplasms
Breast Diseases
Neoplasms by Site
Skin Diseases

ClinicalTrials.gov processed this record on November 27, 2015