Breast Cancer With Low to Intermediate HER2 ExpressionPhase 3PRESENTNCT01479244
Efficacy and Safety Study of NeuVax™ (Nelipepimut-S or E75) Vaccine to Prevent Breast Cancer Recurrence
A Phase 3 study testing NeuVax™ vaccine and Leukine® (sargramostim, GM-CSF) and water for injection in people with Breast Cancer With Low to Intermediate HER2 Expression, run by Galena Biopharma, Inc. at 197 sites in 13 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Breast Cancer With Low to Intermediate HER2 Expression
- What is being tested?
- NeuVax™ vaccine and Leukine® (sargramostim, GM-CSF) and water for injection
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 758 participants
- Where
- 197 sitesin 13 countries
- Study dates
- Nov 2011to 21 Sep 2016
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- 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:
- Total mastectomy-patients with a positive sentinel lymph node must have undergone a completion axillary dissection level I/II
- 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
Show all 13Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
197 sites in 13 countries.
197 sites in 13 countries
United States81 sites
Akron General Medical Center
Akron, Ohio, 44302
Northeast Georgia Cancer Care
Athens, Georgia, 30607
Interventional Spine and Pain Management
Atlanta, Georgia, 30309
University of Colorado Cancer Center, Anschutz Medical Campus
Aurora, Colorado, 80045
Innovative Medical Research of South Florida, Inc.
Aventura, Florida, 33180
Mary Bird Perkins Cancer Center
Baton Rouge, Louisiana, 70809
Physiatry Consultants
Bay City, Michigan, 48796
Alabama Oncology
Bessemer, Alabama, 35022
Center of Cancer and Blood Disorders (VEEDA network)
Bethesda, Maryland, 20817
St. Alexius Medical Center, Mid Dakota Clinic, PC
Bismarck, North Dakota, 58501
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Michigan Interventional Pain Center
Brownstown, Michigan, 48183
East Valley Hematology and Oncology Medical Group
Burbank, California, 91505
Lahey Clinic Department of Hematology and Oncology
Burlington, Massachusetts, 01805
Coastal Pain Research
Carlsbad, California, 92009
Northwestern University Feinberg School of Medicine, Lynn Sage Comprehensive Breast Center
Chicago, Illinois, 60611
PRIDE
Dallas, Texas, 75235
UT Southwestern Medical Center / Simmons Comprehensive Cancer Center
Dallas, Texas, 75390
Western Connecticut Health Network, Danbury Hospital
Danbury, Connecticut, 06810
Navajo Road Pain Management Center
El Cajon, California, 92020
Virginia Cancer Specialists
Fairfax, Virginia, 22031
Holy Cross Hospital-Michael & Dianne Bienes Comprehensive Cancer Center
Fort Lauderdale, Florida, 33308
Brooke Army Medical Center (now SAMMC)
Fort Sam Houston, Texas, 78234
California Cancer Associates for Research and Excellence, Inc.
Fresno, California, 93720
California Cancer Associates For Research and Excellence
Fresno, California, 93720
St. Jude Medical Center, Virginia K. Crosson Cancer Center
Fullerton, California, 92801
The West Clinic Comprehensive Breast Center
Germantown, Tennessee, 38138
Red Rocks Center for Rehabilitation
Golden, Colorado, 80401
Gettysburg Cancer Center
Hillsdale, Pennsylvania, 17325
Baylor College of Medicine
Houston, Texas, 77030
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Interventional Pain Management
Irvine, California, 92618
Cancer Specialists of North Florida (ICON)
Jacksonville, Florida, 32256
Conemaugh Memorial Hospital-Memorial Medical Center
Johnstown, Pennsylvania, 15905
South Orange County Surgical Medical Group
Laguna Hills, California, 92653
University of Southern California/Norris Cancer Hospital
Los Angeles, California, 90033
Alexander Ford, MD
Los Angeles, California, 90035
Cancer Care Institute
Los Angeles, California, 90036
Pete J. Ruane, MD, Inc.
Los Angeles, California, 90036
Cedars-Sinai Medical Center Samuel Oschin Comprehensive Cancer Institute
Los Angeles, California, 90048
James Graham Brown Cancer Center, University of Louisville
Louisville, Kentucky, 40202
Central Georgia Cancer Care
Macon, Georgia, 31201
The West Clinic
Memphis, Tennessee, 38120
Medical College of Wisconsin Department of Surgery Froedtert Hospital and Cancer Center
Milwaukee, Wisconsin, 52336
Physicians Pain Specialists of Alabama, P.C.
Mobile, Alabama, 36606
J. Fred Stoner, MD
New Castle, Pennsylvania, 16101
Fountain Medical Group
New York, New York, 10021
Weill Cornell Medical College
New York, New York, 100665
Upper East Side Pain Medicine, P.C.
New York, New York, 36607
Cancer Treatment Centers of America
Newnan, Georgia, 30265
MD Anderson Center-Orlando Health
Orlando, Florida, 32806
Pain Management Institute
Overland Park, Kansas, 66210
Pain Centers Nationwide
Peoria, Arizona, 83582
University of Pittsburgh Medical Center (UPMC) Cancer Center
Pittsburgh, Pennsylvania, 15232
Florida Cancer Research Institute (Florida Cancer Care)
Plantation, Florida, 33317
Florida Cancer Care
Plantation, Florida, 33324
OHSU Knight Cancer Institute
Portland, Oregon, 97239
Jerrold Rosenberg, MD
Providence, Rhode Island, 02904
William Beaumont Hospital Cancer Center
Royal Oak, Michigan, 48071
Glenn Saperstein, D.O., LLC
Saginaw, Michigan, 48604
Advanced Physical Medicine
Saint Clair Shores, Michigan, 48080
Cancer Care Centers of South Texas
San Antonio, Texas, 78229
Clinical Trials of Texas, Inc
San Antonio, Texas, 78229
Moores UC San Diego Cancer Center (UCSD)
San Diego, California, 92093
University of California, San Francisco (UCSF), Hellen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
Pain Institute of Santa Monica
Santa Monica, California, 90404
Valley Pain Consultants
Scottsdale, Arizona, 85254
Swedish Cancer Institute Research
Seattle, Washington, 98104
Stamford Hospital, Bennett Cancer Center
Stamford, Connecticut, 06904
Pain Management Associates of CT, PC
Stamford, Connecticut, 06905
Comprehensive Pain Management
Syosset, New York, 11791
Multicare Health System-Research Institute
Tacoma, Washington, 98405
H.Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
Eastchester Cancer Care Center
The Bronx, New York, 10469
Cancer Treatment Centers of America
Tulsa, Oklahoma, 74133
Cancer Center of Kansas
Wichita, Kansas, 67214
APWI
Williamsville, New York, 14221
Progressive Pain Solutions
Wind Gap, Pennsylvania, 18091
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157
Randy Scharlach, MD
Woodland Hills, California, 91367
Russia30 sites
Arkhangelsk Clinical Oncology Center
Arkhangelsk, 163045
Regional Oncology Center
Irkutsk, 664035
Ivanovo Regional Oncology Center
Ivanovo, 153040
Republican Clinical Oncoogy Center
Kazan', Tatarstan Republic, 420029
Clinical Oncology Center #1, Department of Healthcare of Krasnodar Territory Administration
Krasnodar, 350040
Krasnoyarsk Kryzhanovsky Regional Oncology Center
Krasnoyarsk, 660133
Kursk Regional Clinical Oncology Center
Kursk, 305035
N.N. Blokhin Russian Cancer Research Center
Moscow, 115478
N.N. Blokhin Russian Cancer Reserach Center
Moscow, 115478
Clinical Diagnostics Center
Nizhny Novgorod, 603006
Nizhny Novgorod Regional Oncology Center
Nizhny Novgorod, 603081
Orenburg Regional Clinical Oncology Center
Orenburg, 460051
Orel Oncology Center
Oryol, 302020
Pyatigorsk Oncology Center
Pyatigorsk, Stavropol Kray, 357502
Medical Institution of Ryazan Region
Ryazan, 390011
Leningrad Regional Oncology Center
Saint Petersburg, 191104
St. Petersburg Clinical Hospital
Saint Petersburg, 194017
Mechnikov North-Western State Medical University
Saint Petersburg, 195067
Petrov Research Institute of Oncology
Saint Petersburg, 197758
Municipal Clinical Oncology Center
Saint Petersburg, 198255
Samara Regional Clinical Oncology Center
Samara, 443031
Republican Oncology Center
Saransk, Respublika Mordoviya, 430032
Oncology Center #2, Department of Healthcare of Krasnodar Territory Administration
Sochi, Krasnodarskiy Kray, 354057
Tambov Regional Clinical Oncology Center
Tambov, 392013
Research Institute of Oncology
Tomsk, 634028
Bashkir State Medical University
Ufa, 450000
Regional Clinical Oncology Center
Velikiy Novgorod, 173016
Volgograd Regional Oncology Center #3
Volzhskiy, 404130
Yaroslavl Regional Oncology Center
Yaroslavl, 150054
Sverdlovsk Regional Oncology Center
Yekaterinburg, 620036
Ukraine17 sites
Chernivtsi Regional Clinical Oncology Center, Day Care Unit
Chernivtsi, 58013
Dnipropetrovsk City Multispecialty Clinical Hospital #4/Dnipropetrovsk Regional Council, Department of Chemotherapy
Dnipropetrovsk, 49102
Donetsk City Oncology Center
Donetsk, 83087
Donetsk Regional Antitumor Center, Oncosurgery Department #2
Donetsk, 83092
Kharkiv Regional Clinical Oncology Center, Day Care Unit
Kharkiv, Kyivskyi District, 61070
State Institution: S.P. Grigoriev Institute of Medical Radiology Under the Academy of Medical Sciences of Ukraine
Kharkiv, 61024
Public Institution: Kiev Regional Oncology Hospital within Kyiv Regional Council
Kiev, 04107
Kirovohrad Regional Oncology Center, Department of Mammology
Kirovohrad, 25011
Kryvyi Rih Oncology Center/Dnipropetrovsk Regional Council, Department of Chemotherapy
Kryvyi Rih, Dnipropetrovsk Oblast, 50000
Kyiv City Clinical Oncology Center, Department of Chemotherapy
Kyiv, 03115
Lviv State Regional Treatment and Diagnostics Oncology Center, Department of Chemotherapy
Lviv, 79031
Mariupol City Interdistrict Oncology Center, Surgery Department #2
Mariupol, Donetsk Oblast, 87500
Odesa Regional Clinical Hospital, Center for Mastology
Odesa, 65000
Clinical Diagnostics Center
Simferopol, Autonomous Republic of Crimea, 95000
Zakarpattia Regional Clinical Oncology Center, Department of Chemotherapy
Uzhhorod, 88014
Vinnytsia Regional Clinical Oncology Center
Vinnytsia, 21029
Public Institution Zaporizhia Regional Clinical Oncology Center within Zaporizhia Regional Council
Zaporizhia, 69040
Germany13 sites
Charité Campus Mitte Universitaetsmedizin-Berlin Brustzentrum/Studienzentrum
Berlin, 10117
Brustzentrum Klinikum Bremen-Mitte gGmbH
Bremen, 28177
Universitätsklinikum Erlangen, Frauenklinik
Erlangen, D-91054
Kliniken Essen Mitte, Evang. Huyssens Stiftung/Knappschaft GmbH, Klinik für Senologie/Brustzentrum
Essen, D-45136
Universitätsklinikum Frankfurt, Klinik für Frauenheilkunde und Geburtshilfe
Frankfurt, 60590
Nationales Centrum für Tumorerkrankungen (NCT), Gynäkologische Onkologie
Heidelberg, 69120
Universitätsklinikum Schleswig-Holstein, Campus Kiel
Kiel, 24105
Universität Marburg
Marburg, 35043
Rotkreuzklinikum München gGmbH, Frauenklinik
München, 80637
Universitätsklinikum Münster, Klinik und Poliklinik für Frauenheilkunde und Geburtshilfe
Münster, 48149
Klinikum Stuttgart, Krankenhaus Bad Cannstatt, Frauenklinik
Stuttgart, 70374
Klinikum Mutterhaus der Borromaeerinnen
Trier, 54290
Gemeinschaftspraxis für Innere Medizin, Haematologie und internistische Onkologie Dr. R Schlag/Dr. Schoettker
Würzburg, D-97080
United Kingdom12 sites
Leeds Teaching Hospitals Trust, St. James University Hospital
Leeds, West Yorkshire, LS9 7TF
Leicester Royal Infirmary
Leicester, LE1 5WW
The Royal Marsden NHS Foundation Trust
London, SW3 6JJ
Macclesfield District General Hospital
Macclesfield, Cheshire, SK10 3BL
James Cook University Hospital
Middlesbrough, Middlesborough, TS4 3BW
Academic Unit of Oncology, Nottingham University Hospital-City Campus
Nottingham, NG7 2UH
Plymouth Oncology Centre, Derriford Hospital
Plymouth, Plymouth, PL6 8DH
Weston Park Hospital
Sheffield, S10 2SJ
Southampton General Hospital
Southampton, SO16 6YD
University Hospital of North Staffordshire
Stoke-on-Trent, ST4 6QG
The Royal Marsden NHS Foundation Trust
Sutton, Surrey, SM2 5PT
The Christie NHS Foundation Trust
Withington, Manchester, M20 4BX
Romania10 sites
Dr. Constantin Opris County Emergency Hospital, Department of Oncology
Baia Mare, Maramures, 430031
Filantropia Clinical Hospital, Obstetrics and Gynaecology Department
Bucharest, 011171
Coltea Clinical Hospital, Oncology Department
Bucharest, 030171
Bucharest Universtiy Emergency Hosptial, Oncology Department
Bucharest, 050098
Medisprof SRL, Department of Medical Oncology
Cluj-Napoca, Cluj, 400058
"Prof. Dr. Ion Chiricuta" Institute of Oncology
Cluj-Napoca, 400015
Oncology Center "Sf. Nectarie", Department of Medical Oncology
Craiova, Dolj, 200347
Iasi Regional Oncology Institute, Department of Medical Oncology
Iași, 700483
"Gavril Curteanu" Municipal Clinical Hospital
Oradea, Bihor County, 410469
Oncomed SRL, Department of Medical Oncology
Timișoara, 300239
Hungary6 sites
Szent Margit Hospital, V. Department of Internal Medicine, Oncology
Budapest, H-1032
Semmelweis University, Department of Diagnostic Radiology and Oncotherapy
Budapest, H-1083
Petz Aladar County Teaching Hospital, Center for Oncoradiology
Győr, H-9024
Kaposi Mor County Teaching Hospital, Center for Clinical Oncology
Kaposvár, 7400
Borsod-Abauj-Zemplen County Hospital and University Educational Hospital, Center for Clinical Oncology and Radiology
Miskolc, 3526
Hetenyi Geza Hospital, Department of Oncology
Szolnok, Jász-Nagykun-Szolnok, H-5000
Poland6 sites
Polish Red Cross Marine Hospital, Department of Chemotherapy
Gdynia, 81-519
Provincial Specialist Hospital in Wroclaw, Department of Chemotherapy
Kamienskiego, Wroclaw, 51-124
Nicolaus Copernicus Memorial Provincial Specialist Hospital in Lodz, Department of Chemotheraphy
Lodz, 93-513
Greater Poland Cancer Center, Department of Chemotherapy
Poznan, 61-866
Magodent
Warsaw, 04-125
Non-Public Health Care Facility MAGODENT
Warsaw, 04-125
Bulgaria5 sites
Central Onco Hospital
Plovdiv, 4002
Serdika
Sofia, 1303
Tsaritsa Yoanna - ISUL
Sofia, 1527
Sveta Marina
Varna, 9010
Complex Oncology Center, Veliko Tarnovo
Veliko Tarnovo, 5000
Canada5 sites
Cross Cancer Institute
Edmonton, Alberta, T6G1Z2
Health Sciences North Cancer Centre
Greater Sudbury, Ontario, P3E 5J1
Hospital Charles LeMorne
Greenfield Park, Quebec, J4V 2H1
Ottawa Hospital Cancer Centre
Ottawa, Ontario, K1H 8L6
Sunnybrook Research Institute
Toronto, Ontario, M4N 3M5
Czechia4 sites
Masaryk Memorial Cancer Institute, Comprehensive Cancer Care Clinic
Brno, 656 53
University Hospital Hradec Kralove, Clinic of Oncology and Radiotherapy
Hradec Králové, 500 05
University Hospital Olomouc, Clinic of Oncology
Olomouc, 775 20
General University Hospital in Prague, Clinic of Oncology
Prague, 128 08
France4 sites
Institut de Cancérologie de l'Ouest Siet Paul Papin
Angers, Cedex 9, 49933
Centre Oscar Lambret
Cédex, Lille, 59020
Centre Antoine Lacassagne
Nice, 06189
Institut de Cancérologie de l'Ouest/René Gauducheau
Saint Herblain, Nantes Cedex, Loire-Atlantique, 44805
Israel4 sites
Rambam Health Care Campus
Haifa, Haifa District, 31096
Rabin Medical Centre
Petah Tikva, 49100
Kaplan Medical Centre
Rehovot, 76100
Sheba Medical Centre
Tel Hasomer, Ramat Gan, 52621
About this study
Official title: PRESENT: Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer With Low to Intermediate HER2 Expressions With NeuVax™Treatment
Purpose of this trial:
- To assess the efficacy and safety of NeuVax™ administered with adjuvant Leukine® (sargramostim, GM-CSF).
- To evaluate and compare the disease free survival (DFS) in the vaccinated and control subjects.
Read the 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.
Endpoints:
- Primary efficacy endpoint:
- 3-year DFS
- 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.
- Being tested
- NeuVax™ vaccine, Leukine® (sargramostim, GM-CSF) and water for injection
- Keywords
- HER2, low or intermediate, breast cancer, node-positive tumors
- Medical subject headings
- Breast Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NeuVax™ vaccineBiological
Also called E75 peptide acetate , WFI, sargramostim, GM-CSF
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).
Leukine® (sargramostim, GM-CSF) and water for injectionBiological
Also called Leukine®
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).
| Study group | Role | What they receive |
|---|---|---|
| NeuVax™ | Experimental | NeuVax™ in WFI solution with Leukine® Receives: Biological: NeuVax™ vaccine |
| Leukine® | Active comparator | Leukine® with WFI Receives: Biological: Leukine® (sargramostim, GM-CSF) and water for injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparison of DFS in vaccine treated patients and control patients
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.
Measured: 36 months
Secondary outcomes (1)
Assessment of DFS and OS at 3, 5 and 10 years in vaccine and control groups, respectively; assessment of safety
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)
Measured: 3 through 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2011 (estimated)
- Study startNov 2011
- Primary completion21 Sep 2016
- Study completion21 Sep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Breast Cancer With Low to Intermediate HER2 ExpressionStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 758 participants (actual)
Sponsor and investigators
- Lead sponsor
- Galena Biopharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Elizabeth A Mittendorf, MD,FACS, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01479244
- Sponsor's protocol ID
- NeuVax™ PH3-01, BB-IND 009187
- Other IDs
- 2011-005219-98
- Acronym
- PRESENT
- Status last verified
- Feb 2017
- First submitted
- 20 Nov 2011
- Last updated
- 27 Feb 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Amin A, Benavides LC, Holmes JP, Gates JD, Carmichael MG, Hueman MT, Mittendorf EA, Storrer CE, Jama YH, Craig D, Stojadinovic A, Ponniah S, Peoples GE. Assessment of immunologic response and recurrence patterns among patients with clinical recurrence after vaccination with a preventive HER2/neu peptide vaccine: from US Military Cancer Institute Clinical Trials Group Study I-01 and I-02. Cancer Immunol Immunother. 2008 Dec;57(12):1817-25. doi: 10.1007/s00262-008-0509-2. Epub 2008 Apr 8.
PubMed 18392824Mittendorf EA, Clifton GT, Holmes JP, Clive KS, Patil R, Benavides LC, Gates JD, Sears AK, Stojadinovic A, Ponniah S, Peoples GE. Clinical trial results of the HER-2/neu (E75) vaccine to prevent breast cancer recurrence in high-risk patients: from US Military Cancer Institute Clinical Trials Group Study I-01 and I-02. Cancer. 2012 May 15;118(10):2594-602. doi: 10.1002/cncr.26574. Epub 2011 Oct 11.
PubMed 21989902Mittendorf EA, Lu B, Melisko M, Price Hiller J, Bondarenko I, Brunt AM, Sergii G, Petrakova K, Peoples GE. Efficacy and Safety Analysis of Nelipepimut-S Vaccine to Prevent Breast Cancer Recurrence: A Randomized, Multicenter, Phase III Clinical Trial. Clin Cancer Res. 2019 Jul 15;25(14):4248-4254. doi: 10.1158/1078-0432.CCR-18-2867. Epub 2019 Apr 29.
PubMed 31036542
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PRESENT: Prevention of Recurrence in Early-Stage, Node-Positive Breast Cancer With Low to Intermediate HER2 Expressions With NeuVax™Treatment
- ClinicalTrials.gov ID
- NCT01479244
- Acronym / sponsor ID
- PRESENT / NeuVax™ PH3-01, BB-IND 009187
- Keywords
- HER2, low or intermediate, breast cancer, node-positive tumors
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 758 participants (actual)
- Lead sponsor
- Galena Biopharma, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2011
- Primary completion
- 21 Sep 2016 (actual)
- Study completion
- 21 Sep 2016 (actual)
- First posted
- 24 Nov 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Purpose of this trial:
- To assess the efficacy and safety of NeuVax™ administered with adjuvant Leukine® (sargramostim, GM-CSF).
- To evaluate and compare the disease free survival (DFS) in the vaccinated and control subjects.
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.
Endpoints:
- Primary efficacy endpoint:
- 3-year DFS
- 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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NeuVax™ | Experimental | Biological: NeuVax™ vaccineNeuVax™ in WFI solution with Leukine® |
| Leukine® | Active comparator | Biological: Leukine® (sargramostim, GM-CSF) and water for injectionLeukine® with WFI |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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:
- Node-positive disease
- Primary tumor stage T1-3 at initial diagnosis
Key exclusion criteria (8 listed)
- 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
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 |
|---|---|---|
| NeuVax™ vaccineAlso: E75 peptide acetate , WFI, sargramostim, GM-CSFE75 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). | Biological | NeuVax™ |
| Leukine® (sargramostim, GM-CSF) and water for injectionAlso: Leukine®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). | Biological | Leukine® |
Primary outcome
Comparison of DFS in vaccine treated patients and control patients
Time frame: 36 months
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.
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2017.
- Background: PubMed 18392824Amin A, Benavides LC, Holmes JP, Gates JD, Carmichael MG, Hueman MT, Mittendorf EA, Storrer CE, Jama YH, Craig D, Stojadinovic A, Ponniah S, Peoples GE. Assessment of immunologic response and recurrence patterns among patients with clinical recurrence after vaccination with a preventive HER2/neu peptide vaccine: from US Military Cancer Institute Clinical Trials Group Study I-01 and I-02. Cancer Immunol Immunother. 2008 Dec;57(12):1817-25. doi: 10.1007/s00262-008-0509-2. Epub 2008 Apr 8.
- Background: PubMed 21989902Mittendorf EA, Clifton GT, Holmes JP, Clive KS, Patil R, Benavides LC, Gates JD, Sears AK, Stojadinovic A, Ponniah S, Peoples GE. Clinical trial results of the HER-2/neu (E75) vaccine to prevent breast cancer recurrence in high-risk patients: from US Military Cancer Institute Clinical Trials Group Study I-01 and I-02. Cancer. 2012 May 15;118(10):2594-602. doi: 10.1002/cncr.26574. Epub 2011 Oct 11.
- Derived from this study: PubMed 31036542Mittendorf EA, Lu B, Melisko M, Price Hiller J, Bondarenko I, Brunt AM, Sergii G, Petrakova K, Peoples GE. Efficacy and Safety Analysis of Nelipepimut-S Vaccine to Prevent Breast Cancer Recurrence: A Randomized, Multicenter, Phase III Clinical Trial. Clin Cancer Res. 2019 Jul 15;25(14):4248-4254. doi: 10.1158/1078-0432.CCR-18-2867. Epub 2019 Apr 29.
- Sponsor websiteLink provided by the sponsor.
- Vaccine siteLink provided by the sponsor.
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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.
Elizabeth A Mittendorf, MD,FACS principal investigator
M.D. Anderson Cancer Center
197 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NeuVax™ vaccine and Leukine® (sargramostim, GM-CSF) and water for injection in people with Breast Cancer With Low to Intermediate HER2 Expression, run by Galena Biopharma, Inc. at 197 sites in 13 countries.
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 13 inclusion and 8 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 27 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 197 sites in United States, Russia, Ukraine, Germany, United Kingdom and 8 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Comparison of DFS in vaccine treated patients and control patients (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01479244, which also lists 3 linked publications.
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 27 Feb 2017.