MedPath

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

Phase 3
Completed
Conditions
Breast Cancer With Low to Intermediate HER2 Expression
Interventions
Biological: Leukine® (sargramostim, GM-CSF) and water for injection
Biological: NeuVax™ vaccine
Registration Number
NCT01479244
Lead Sponsor
Galena Biopharma, Inc.
Brief 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.

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:

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
758
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Leukine®Leukine® (sargramostim, GM-CSF) and water for injectionLeukine® with WFI
NeuVax™NeuVax™ vaccineNeuVax™ in WFI solution with Leukine®
Primary Outcome Measures
NameTimeMethod
Comparison of DFS in vaccine treated patients and control patients36 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.

Secondary Outcome Measures
NameTimeMethod
Assessment of DFS and OS at 3, 5 and 10 years in vaccine and control groups, respectively; assessment of safety3 through 10 years

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)

Trial Locations

Locations (194)

University Hospital Hradec Kralove, Clinic of Oncology and Radiotherapy

🇨🇿

Hradec Kralove, Czech Republic

Borsod-Abauj-Zemplen County Hospital and University Educational Hospital, Center for Clinical Oncology and Radiology

🇭🇺

Miskolc, Hungary

Nizhny Novgorod Regional Oncology Center

🇷🇺

Nizhny Novgorod, Russian Federation

Orel Oncology Center

🇷🇺

Orel, Russian Federation

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

Alabama Oncology

🇺🇸

Bessemer, Alabama, United States

Physicians Pain Specialists of Alabama, P.C.

🇺🇸

Mobile, Alabama, United States

Pain Centers Nationwide

🇺🇸

Peoria, Arizona, United States

East Valley Hematology and Oncology Medical Group

🇺🇸

Burbank, California, United States

Valley Pain Consultants

🇺🇸

Scottsdale, Arizona, United States

Coastal Pain Research

🇺🇸

Carlsbad, California, United States

Navajo Road Pain Management Center

🇺🇸

El Cajon, California, United States

California Cancer Associates for Research and Excellence, Inc.

🇺🇸

Fresno, California, United States

California Cancer Associates For Research and Excellence

🇺🇸

Fresno, California, United States

St. Jude Medical Center, Virginia K. Crosson Cancer Center

🇺🇸

Fullerton, California, United States

Interventional Pain Management

🇺🇸

Irvine, California, United States

University of Southern California/Norris Cancer Hospital

🇺🇸

Los Angeles, California, United States

South Orange County Surgical Medical Group

🇺🇸

Laguna Hills, California, United States

Alexander Ford, MD

🇺🇸

Los Angeles, California, United States

Cancer Care Institute

🇺🇸

Los Angeles, California, United States

Pete J. Ruane, MD, Inc.

🇺🇸

Los Angeles, California, United States

Cedars-Sinai Medical Center Samuel Oschin Comprehensive Cancer Institute

🇺🇸

Los Angeles, California, United States

Pain Institute of Santa Monica

🇺🇸

Santa Monica, California, United States

Stamford Hospital, Bennett Cancer Center

🇺🇸

Stamford, Connecticut, United States

Randy Scharlach, MD

🇺🇸

Woodland Hills, California, United States

Holy Cross Hospital-Michael & Dianne Bienes Comprehensive Cancer Center

🇺🇸

Fort Lauderdale, Florida, United States

Red Rocks Center for Rehabilitation

🇺🇸

Golden, Colorado, United States

Western Connecticut Health Network, Danbury Hospital

🇺🇸

Danbury, Connecticut, United States

Innovative Medical Research of South Florida, Inc.

🇺🇸

Aventura, Florida, United States

Pain Management Associates of CT, PC

🇺🇸

Stamford, Connecticut, United States

Cancer Specialists of North Florida (ICON)

🇺🇸

Jacksonville, Florida, United States

Florida Cancer Research Institute (Florida Cancer Care)

🇺🇸

Plantation, Florida, United States

Florida Cancer Care

🇺🇸

Plantation, Florida, United States

Northeast Georgia Cancer Care

🇺🇸

Athens, Georgia, United States

Interventional Spine and Pain Management

🇺🇸

Atlanta, Georgia, United States

Northwestern University Feinberg School of Medicine, Lynn Sage Comprehensive Breast Center

🇺🇸

Chicago, Illinois, United States

Central Georgia Cancer Care

🇺🇸

Macon, Georgia, United States

Cancer Treatment Centers of America

🇺🇸

Tulsa, Oklahoma, United States

Pain Management Institute

🇺🇸

Overland Park, Kansas, United States

Mary Bird Perkins Cancer Center

🇺🇸

Baton Rouge, Louisiana, United States

Physiatry Consultants

🇺🇸

Bay City, Michigan, United States

Michigan Interventional Pain Center

🇺🇸

Brownstown, Michigan, United States

Lahey Clinic Department of Hematology and Oncology

🇺🇸

Burlington, Massachusetts, United States

William Beaumont Hospital Cancer Center

🇺🇸

Royal Oak, Michigan, United States

Glenn Saperstein, D.O., LLC

🇺🇸

Saginaw, Michigan, United States

Advanced Physical Medicine

🇺🇸

St. Clair Shores, Michigan, United States

Weill Cornell Medical College

🇺🇸

New York, New York, United States

Eastchester Cancer Care Center

🇺🇸

Bronx, New York, United States

Upper East Side Pain Medicine, P.C.

🇺🇸

New York, New York, United States

Fountain Medical Group

🇺🇸

New York, New York, United States

Comprehensive Pain Management

🇺🇸

Syosset, New York, United States

APWI

🇺🇸

Williamsville, New York, United States

Akron General Medical Center

🇺🇸

Akron, Ohio, United States

J. Fred Stoner, MD

🇺🇸

New Castle, Pennsylvania, United States

Gettysburg Cancer Center

🇺🇸

Gettysburgh, Pennsylvania, United States

Conemaugh Memorial Hospital-Memorial Medical Center

🇺🇸

Johnstown, Pennsylvania, United States

University of Pittsburgh Medical Center (UPMC) Cancer Center

🇺🇸

Pittsburgh, Pennsylvania, United States

Jerrold Rosenberg, MD

🇺🇸

Providence, Rhode Island, United States

Progressive Pain Solutions

🇺🇸

Wind Gap, Pennsylvania, United States

The West Clinic

🇺🇸

Memphis, Tennessee, United States

PRIDE

🇺🇸

Dallas, Texas, United States

The West Clinic Comprehensive Breast Center

🇺🇸

Germantown, Tennessee, United States

UT Southwestern Medical Center / Simmons Comprehensive Cancer Center

🇺🇸

Dallas, Texas, United States

Brooke Army Medical Center (now SAMMC)

🇺🇸

Ft. Sam Houston, Texas, United States

Virginia Cancer Specialists

🇺🇸

Fairfax, Virginia, United States

Multicare Health System-Research Institute

🇺🇸

Tacoma, Washington, United States

Central Onco Hospital

🇧🇬

Plovdiv, Bulgaria

Serdika

🇧🇬

Sofia, Bulgaria

Tsaritsa Yoanna - ISUL

🇧🇬

Sofia, Bulgaria

Complex Oncology Center, Veliko Tarnovo

🇧🇬

Tarnovo, Bulgaria

Cross Cancer Institute

🇨🇦

Edmonton, Alberta, Canada

Sveta Marina

🇧🇬

Varna, Bulgaria

Health Sciences North Cancer Centre

🇨🇦

Sudbury, Ontario, Canada

Sunnybrook Research Institute

🇨🇦

Toronto, Ontario, Canada

Hospital Charles LeMorne

🇨🇦

Greenfield Park, Quebec, Canada

Ottawa Hospital Cancer Centre

🇨🇦

Ottawa, Ontario, Canada

Masaryk Memorial Cancer Institute, Comprehensive Cancer Care Clinic

🇨🇿

Brno, Czech Republic

Institut de Cancérologie de l'Ouest Siet Paul Papin

🇫🇷

Angers, Cedex 9, France

University Hospital Olomouc, Clinic of Oncology

🇨🇿

Olomouc, Czech Republic

General University Hospital in Prague, Clinic of Oncology

🇨🇿

Prague 2, Czech Republic

Centre Oscar Lambret

🇫🇷

Cédex, Lille, France

Charité Campus Mitte Universitaetsmedizin-Berlin Brustzentrum/Studienzentrum

🇩🇪

Berlin, Germany

Centre Antoine Lacassagne

🇫🇷

Nice, France

Institut de Cancérologie de l'Ouest/René Gauducheau

🇫🇷

St Herblain, Nantes Cedex, Loire-Atlantique, France

Universitätsklinikum Frankfurt, Klinik für Frauenheilkunde und Geburtshilfe

🇩🇪

Frankfurt, Germany

Universität Marburg

🇩🇪

Marburg, Germany

Brustzentrum Klinikum Bremen-Mitte gGmbH

🇩🇪

Bremen, Germany

Universitätsklinikum Erlangen, Frauenklinik

🇩🇪

Erlangen, Germany

Nationales Centrum für Tumorerkrankungen (NCT), Gynäkologische Onkologie

🇩🇪

Heidelberg, Germany

Kliniken Essen Mitte, Evang. Huyssens Stiftung/Knappschaft GmbH, Klinik für Senologie/Brustzentrum

🇩🇪

Essen, Germany

Universitätsklinikum Schleswig-Holstein, Campus Kiel

🇩🇪

Kiel, Germany

Rotkreuzklinikum München gGmbH, Frauenklinik

🇩🇪

Muenchen, Germany

Klinikum Stuttgart, Krankenhaus Bad Cannstatt, Frauenklinik

🇩🇪

Stuttgart, Germany

Gemeinschaftspraxis für Innere Medizin, Haematologie und internistische Onkologie Dr. R Schlag/Dr. Schoettker

🇩🇪

Wuerzburg, Germany

Klinikum Mutterhaus der Borromaeerinnen

🇩🇪

Trier, Germany

Semmelweis University, Department of Diagnostic Radiology and Oncotherapy

🇭🇺

Budapest, Hungary

Universitätsklinikum Münster, Klinik und Poliklinik für Frauenheilkunde und Geburtshilfe

🇩🇪

Münster, Germany

Petz Aladar County Teaching Hospital, Center for Oncoradiology

🇭🇺

Gyor, Hungary

Kaposi Mor County Teaching Hospital, Center for Clinical Oncology

🇭🇺

Kaposvar, Hungary

Hetenyi Geza Hospital, Department of Oncology

🇭🇺

Szolnok, Jasz-Nagykun-Szolnok, Hungary

Szent Margit Hospital, V. Department of Internal Medicine, Oncology

🇭🇺

Budapest, Hungary

Sheba Medical Centre

🇮🇱

Tel Hasomer, Ramat Gan, Israel

Kaplan Medical Centre

🇮🇱

Rehovot, Israel

Rambam Health Care Campus

🇮🇱

Haifa, Israel

Rabin Medical Centre

🇮🇱

Petach Tikva, Israel

Polish Red Cross Marine Hospital, Department of Chemotherapy

🇵🇱

Gdynia, Poland

Provincial Specialist Hospital in Wroclaw, Department of Chemotherapy

🇵🇱

Kamienskiego, Wroclaw, Poland

Greater Poland Cancer Center, Department of Chemotherapy

🇵🇱

Poznan, Poland

Nicolaus Copernicus Memorial Provincial Specialist Hospital in Lodz, Department of Chemotheraphy

🇵🇱

Lodz, Poland

Non-Public Health Care Facility MAGODENT

🇵🇱

Warsaw, Poland

Dr. Constantin Opris County Emergency Hospital, Department of Oncology

🇷🇴

Baia Mare, Maramures, Romania

Magodent

🇵🇱

Warsaw, Poland

"Gavril Curteanu" Municipal Clinical Hospital

🇷🇴

Oradea, Bihor, Romania

Medisprof SRL, Department of Medical Oncology

🇷🇴

Cluj-Napoca, Cluj, Romania

Oncology Center "Sf. Nectarie", Department of Medical Oncology

🇷🇴

Craiova, Dolj County, Romania

Filantropia Clinical Hospital, Obstetrics and Gynaecology Department

🇷🇴

Bucharest, Romania

Coltea Clinical Hospital, Oncology Department

🇷🇴

Bucharest, Romania

"Prof. Dr. Ion Chiricuta" Institute of Oncology

🇷🇴

Cluj-Napoca, Romania

Bucharest Universtiy Emergency Hosptial, Oncology Department

🇷🇴

Bucharest, Romania

Iasi Regional Oncology Institute, Department of Medical Oncology

🇷🇴

Iasi, Romania

Oncomed SRL, Department of Medical Oncology

🇷🇴

Timisoara, Romania

Oncology Center #2, Department of Healthcare of Krasnodar Territory Administration

🇷🇺

Sochi, Krasnodar, Russian Federation

Republican Oncology Center

🇷🇺

Saransk, Republic of Mordovia, Russian Federation

Republican Clinical Oncoogy Center

🇷🇺

Kazan, Republic of Tatarstan, Russian Federation

Arkhangelsk Clinical Oncology Center

🇷🇺

Arkhangelsk, Russian Federation

Pyatigorsk Oncology Center

🇷🇺

Pyatigorsk, Stavropol Region, Russian Federation

Regional Oncology Center

🇷🇺

Irkutsk, Russian Federation

Sverdlovsk Regional Oncology Center

🇷🇺

Ekaterinburg, Russian Federation

Ivanovo Regional Oncology Center

🇷🇺

Ivanovo, Russian Federation

Clinical Oncology Center #1, Department of Healthcare of Krasnodar Territory Administration

🇷🇺

Krasnodar, Russian Federation

Krasnoyarsk Kryzhanovsky Regional Oncology Center

🇷🇺

Krasnoyarsk, Russian Federation

Kursk Regional Clinical Oncology Center

🇷🇺

Kursk, Russian Federation

N.N. Blokhin Russian Cancer Research Center

🇷🇺

Moscow, Russian Federation

N.N. Blokhin Russian Cancer Reserach Center

🇷🇺

Moscow, Russian Federation

Clinical Diagnostics Center

🇺🇦

Simferopol, Autonomous Republic of Crimea, Ukraine

Orenburg Regional Clinical Oncology Center

🇷🇺

Orenburg, Russian Federation

Medical Institution of Ryazan Region

🇷🇺

Ryazan, Russian Federation

Samara Regional Clinical Oncology Center

🇷🇺

Samara, Russian Federation

Petrov Research Institute of Oncology

🇷🇺

St Petersburg, Russian Federation

Leningrad Regional Oncology Center

🇷🇺

St. Petersburg, Russian Federation

St. Petersburg Clinical Hospital

🇷🇺

St. Petersburg, Russian Federation

Mechnikov North-Western State Medical University

🇷🇺

St. Petersburg, Russian Federation

Municipal Clinical Oncology Center

🇷🇺

St. Petersburg, Russian Federation

Tambov Regional Clinical Oncology Center

🇷🇺

Tambov, Russian Federation

Research Institute of Oncology

🇷🇺

Tomsk, Russian Federation

Bashkir State Medical University

🇷🇺

Ufa, Russian Federation

Regional Clinical Oncology Center

🇷🇺

Veliky Novgorod, Russian Federation

Volgograd Regional Oncology Center #3

🇷🇺

Volzhskiy, Russian Federation

Kryvyi Rih Oncology Center/Dnipropetrovsk Regional Council, Department of Chemotherapy

🇺🇦

Kryvyi Rih, Dnipropetrovsk Region, Ukraine

Yaroslavl Regional Oncology Center

🇷🇺

Yaroslavl, Russian Federation

Chernivtsi Regional Clinical Oncology Center, Day Care Unit

🇺🇦

Chernivtsi, Ukraine

Mariupol City Interdistrict Oncology Center, Surgery Department #2

🇺🇦

Mariupol, Donetsk Region, Ukraine

Kharkiv Regional Clinical Oncology Center, Day Care Unit

🇺🇦

Kharkiv, Kyivskyi District, Ukraine

Dnipropetrovsk City Multispecialty Clinical Hospital #4/Dnipropetrovsk Regional Council, Department of Chemotherapy

🇺🇦

Dnipropetrovsk, Ukraine

Donetsk City Oncology Center

🇺🇦

Donetsk, Ukraine

Donetsk Regional Antitumor Center, Oncosurgery Department #2

🇺🇦

Donetsk, Ukraine

State Institution: S.P. Grigoriev Institute of Medical Radiology Under the Academy of Medical Sciences of Ukraine

🇺🇦

Kharkiv, Ukraine

Public Institution: Kiev Regional Oncology Hospital within Kyiv Regional Council

🇺🇦

Kiev, Ukraine

Kirovohrad Regional Oncology Center, Department of Mammology

🇺🇦

Kirovohrad, Ukraine

Lviv State Regional Treatment and Diagnostics Oncology Center, Department of Chemotherapy

🇺🇦

Lviv, Ukraine

Kyiv City Clinical Oncology Center, Department of Chemotherapy

🇺🇦

Kyiv, Ukraine

Odesa Regional Clinical Hospital, Center for Mastology

🇺🇦

Odesa, Ukraine

Zakarpattia Regional Clinical Oncology Center, Department of Chemotherapy

🇺🇦

Uzhhorod, Ukraine

Vinnytsia Regional Clinical Oncology Center

🇺🇦

Vinnytsia, Ukraine

Public Institution Zaporizhia Regional Clinical Oncology Center within Zaporizhia Regional Council

🇺🇦

Zaporizhia, Ukraine

Macclesfield District General Hospital

🇬🇧

Macclesfield, Cheshire, United Kingdom

The Royal Marsden NHS Foundation Trust

🇬🇧

London, United Kingdom

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

The University of Texas MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

University of Texas MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Swedish Cancer Institute Research

🇺🇸

Seattle, Washington, United States

James Cook University Hospital

🇬🇧

Middlesborough, United Kingdom

Leicester Royal Infirmary

🇬🇧

Leicester, United Kingdom

Academic Unit of Oncology, Nottingham University Hospital-City Campus

🇬🇧

Nottingham, United Kingdom

Plymouth Oncology Centre, Derriford Hospital

🇬🇧

Plymouth, United Kingdom

Weston Park Hospital

🇬🇧

Sheffield, United Kingdom

Southampton General Hospital

🇬🇧

Southampton, United Kingdom

University Hospital of North Staffordshire

🇬🇧

Stoke on Trent, United Kingdom

Moores UC San Diego Cancer Center (UCSD)

🇺🇸

San Diego, California, United States

Cancer Care Centers of South Texas

🇺🇸

San Antonio, Texas, United States

Clinical Trials of Texas, Inc

🇺🇸

San Antonio, Texas, United States

Cancer Center of Kansas

🇺🇸

Wichita, Kansas, United States

The Christie NHS Foundation Trust

🇬🇧

Withington, Manchester, United Kingdom

University of California, San Francisco (UCSF), Hellen Diller Family Comprehensive Cancer Center

🇺🇸

San Francisco, California, United States

OHSU Knight Cancer Institute

🇺🇸

Portland, Oregon, United States

Leeds Teaching Hospitals Trust, St. James University Hospital

🇬🇧

Leeds, West Yorkshire, United Kingdom

St. Alexius Medical Center, Mid Dakota Clinic, PC

🇺🇸

Bismarck, North Dakota, United States

University of Colorado Cancer Center, Anschutz Medical Campus

🇺🇸

Aurora, Colorado, United States

H.Lee Moffitt Cancer Center and Research Institute

🇺🇸

Tampa, Florida, United States

MD Anderson Center-Orlando Health

🇺🇸

Orlando, Florida, United States

Wake Forest Baptist Health

🇺🇸

Winston-Salem, North Carolina, United States

James Graham Brown Cancer Center, University of Louisville

🇺🇸

Louisville, Kentucky, United States

Center of Cancer and Blood Disorders (VEEDA network)

🇺🇸

Bethesda, Maryland, United States

Medical College of Wisconsin Department of Surgery Froedtert Hospital and Cancer Center

🇺🇸

Milwaukee, Wisconsin, United States

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