A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera
- Registration Number
- NCT05210790
- Lead Sponsor
- Protagonist Therapeutics, Inc.
- Brief Summary
The study is designed to evaluate the safety and efficacy of rusfertide in subjects with polycythemia vera (PV) in maintaining hematocrit control and in improving symptoms of PV.
- Detailed Description
Phase 3 study in approximately 250 subjects previously diagnosed with polycythemia vera (PV) who require phlebotomy on a routine basis. There is a 32-week period during which rusfertide or placebo will be added-on to each subject's ongoing therapy for polycythemia vera which may include phlebotomy only or phlebotomy plus stable doses of either of hydroxyurea, interferon and/or ruxolitinib. All subjects who successfully complete the double blind 32-week portion of the study will receive rusfertide for 124 weeks. Approximately 6 and 12 months after their last dose of rusfertide, subjects will have a post-study contact (e.g. by phone) for safety.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 250
All subjects must meet ALL of the following inclusion criteria to be enrolled. There are additional inclusion criteria.
-
Male and female subjects aged 18 (or the country specific minimum age of consent >18) years or older.
-
Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera.
-
At least 3 phlebotomies due to inadequate hematocrit control in 6 months before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization.
-
CBC values immediately prior to randomization:
- Hematocrit <45%,
- WBC 4000/ฮผL to 20,000/ฮผL (inclusive), and
- Platelets 100,000/ฮผL to 1,000,000/ฮผL (inclusive)
-
Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen.
-
Subjects treated with phlebotomy alone at randomization must have stopped cytoreductive therapy 2 to 6 months before screening.
Main
Subjects must meet NONE of the following exclusion criteria to be enrolled. There are additional exclusion criteria.
- Clinically meaningful laboratory abnormalities at Screening.
- Subjects who require phlebotomy at hematocrit levels lower than 45%.
- Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.
- Active or chronic bleeding within 2 months prior to randomization.
- History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer.
- Subjects with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during screen unless the cancer is adequately treated before randomization.
- Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Rusfertide Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label) Placebo Placebo Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label) Rusfertide Rusfertide Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)
- Primary Outcome Measures
Name Time Method Proportion of subjects achieving a response who receive rusfertide compared to placebo. Week 20 through Week 32 Response is defined as absence of phlebotomy eligibility.
- Secondary Outcome Measures
Name Time Method Proportion of subjects with HCT values <45% for rusfertide and placebo. Week 0 to Week 32 Comparison of mean number of phlebotomies between rusfertide and placebo. Week 0 to Week 32 Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo. Week 32 Comparison of mean change from baseline in total MFSAF total score. Week 32
Trial Locations
- Locations (171)
University of Pittsburgh Medical Center
๐บ๐ธPittsburgh, Pennsylvania, United States
Sharp Memorial Hospital
๐บ๐ธSan Diego, California, United States
Good Samaritan Infusion Center - Thomas
๐บ๐ธCincinnati, Ohio, United States
AP-HP Hopital Saint-Louis
๐ซ๐ทParis, France
City of Hope Medical Center, Duarte
๐บ๐ธDuarte, California, United States
Infirmary Cancer Care
๐บ๐ธMobile, Alabama, United States
Fondazione IRCCS Policlinico San Matteo
๐ฎ๐นPavia, Italy
Centro de Estudios de Investigacion SIYODE
๐ฒ๐ฝMadero, Mexico
Albert Schweitzer Ziekenhuis
๐ณ๐ฑDordrecht, Netherlands
Hospital Quironsalud de Zaragoza
๐ช๐ธZaragoza, Spain
Hospital Germans Trias i Pujol
๐ช๐ธBarcelona, Spain
UOC Ematologia e Centro Trapianti AST Pesaro Urbino, Stabilimento Muraglia
๐ฎ๐นPesaro, Italy
Hospital Universitario Vall d'Hebron
๐ช๐ธBarcelona, Spain
Hospital Universitario 12 de Octubre
๐ช๐ธMadrid, Spain
Erasmus MC
๐ณ๐ฑRotterdam, Netherlands
Fondazione Policlinico Univ. A . Gemelli IRCCS
๐ฎ๐นRoma, Italy
Churchill Hospital
๐ฌ๐งOxford, United Kingdom
Hospital Universitari i Politecnic La Fe
๐ช๐ธValencia, Spain
Northwick Park Hospital
๐ฌ๐งHarrow, United Kingdom
MICS Centrum Medyczne Bydgoszcz
๐ต๐ฑBydgoszcz, Poland
Hospital Clinic Barcelona
๐ช๐ธBarcelona, Spain
Hospital General Universitario Gregorio Maranon
๐ช๐ธMadrid, Spain
Hospital Universitario Ramon y Cajal
๐ช๐ธMadrid, Spain
Hospital del Mar
๐ช๐ธBarcelona, Spain
Gazi University Medical Faculty
๐น๐ทYenimahalle, Ankara, Turkey
Barnet Hospital
๐ฌ๐งBarnet, United Kingdom
The Royal London Hospital
๐ฌ๐งLondon, United Kingdom
Hillingdon Hospital
๐ฌ๐งUxbridge, Middlesex, United Kingdom
Pilgrim Hospital
๐ฌ๐งBoston, United Kingdom
Mission Cancer & Blood
๐บ๐ธDes Moines, Iowa, United States
University of Kansas Cancer Center
๐บ๐ธKansas City, Kansas, United States
Rocky Mountain Cancer Centers - Boulder
๐บ๐ธBoulder, Colorado, United States
Ege University Hospital Department of Hematology
๐น๐ทBornova, Izmir, Turkey
University of California, San Francisco
๐บ๐ธSan Francisco, California, United States
Memorial Sloan Kettering Cancer Center - BASKING RIDGE
๐บ๐ธBasking Ridge, New Jersey, United States
ZNA Stuivenberg
๐ง๐ชAntwerp, Belgium
Cancer Care Specialists
๐บ๐ธReno, Nevada, United States
Medizinische Universitaet Graz
๐ฆ๐นGraz, Austria
Centro de Estudios Clinicos SAGA
๐จ๐ฑProvidencia, Santiago, Chile
Biocenter Centro Clinico de Investigacion
๐จ๐ฑConcepciรณn, Chile
UOC Ematologia - ASST Spedail Civill Brescia
๐ฎ๐นBrescia, Italy
Klinika Hematologii I Transplantologii - Uniwersyteckie Centrum Kliniczne
๐ต๐ฑGdaลsk, Poland
Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi Oddziaล
๐ต๐ฑลรณdลบ, Poland
Hospital de Braga
๐ต๐นBraga, Portugal
Karadeniz Technical University Medical Faculty Farabi Hospital
๐น๐ทTrabzon, Ortahisar, Turkey
Sanko Universitesi tip Fakultesi Ic Hastaliklari Anabilim Dali
๐น๐ทGaziantep, Sehitkamil, Turkey
Marmara University Pendik Training & Research Hospital
๐น๐ทIstanbul, Turkey
Erciyes Universitesi Tip Fakultesi Hastanesi, Dedeman Hospital
๐น๐ทKayseri, Turkey
The Alfred Hospital
๐ฆ๐บMelbourne, Victoria, Australia
Shaare Zedek Medical Center
๐ฎ๐ฑJerusalem, Israel
HSHS St. Mary's Hospital
๐บ๐ธGreen Bay, Wisconsin, United States
ONCOResearch Lerchenfeld GmbH
๐ฉ๐ชHamburg, Germany
East Carolina University
๐บ๐ธGreenville, North Carolina, United States
Sunnybrook Health Sciences Centre
๐จ๐ฆToronto, Ontario, Canada
Krankenhaus der Elisabethinen Linz
๐ฆ๐นLinz, Austria
Medizinische Universitaet Wien
๐ฆ๐นWien, Austria
Klinikum Wels-Grieskirchen Gmb
๐ฆ๐นWels, Austria
Centro Medico Inmunocel
๐จ๐ฑLas Condes, Santiago, Chile
Centre Hospitalier d'Argenteuil
๐ซ๐ทArgenteuil, France
Centre Hospitalier de Beziers
๐ซ๐ทBรฉziers, France
Princess Margaret Hospital
๐จ๐ฆToronto, Ontario, Canada
HCL Centre Hospitalier Lyon Sud
๐ซ๐ทPierre-Bรฉnite, France
Uniklinik RWTH Aachen - Klinik fuer Haematologie, Onkologie, Haemostaseologie und Stammzelltransplantation (Med. Klinik IV)
๐ฉ๐ชAachen, Germany
Monmouth Medical Center
๐บ๐ธLong Branch, New Jersey, United States
Charite Universitaetsmedizin Berlin
๐ฉ๐ชBerlin, Germany
Rutgers Cancer Institute of New Jersey
๐บ๐ธNew Brunswick, New Jersey, United States
Rocky Mountain Cancer Centers
๐บ๐ธLone Tree, Colorado, United States
BRCR Medical Center
๐บ๐ธPlantation, Florida, United States
Memorial Sloan Kettering Cancer Center - MONMOUTH
๐บ๐ธMiddletown, New Jersey, United States
Kepler Universitaets Klinikum GmbH
๐ฆ๐นLinz, Austria
AP-HP Hospital Necker-Enfants Malades
๐ซ๐ทParis, Cedex 15, France
Sourasky Medical Center
๐ฎ๐ฑTel Aviv, Israel
Azienda Ospedaliero - Universitaria Careggi
๐ฎ๐นFirenze, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Ematologia (Padiglione Marcora),
๐ฎ๐นMilano, Italy
Gaziantep Universitesi Sahinbey Arastirma ve Uygulama Hastanesi
๐น๐ทGaziantep, Turkey
HSHS St. Vincent Hospital
๐บ๐ธGreen Bay, Wisconsin, United States
Memorial Sloan Kettering Cancer Center - BERGEN
๐บ๐ธMontvale, New Jersey, United States
Northwestern Memorial Hospital
๐บ๐ธChicago, Illinois, United States
Jewish General Hospital
๐จ๐ฆMontrรฉal, Quebec, Canada
CHU Angers
๐ซ๐ทAngers, Cedex 09, France
InVo-Institut fuer Versorgungsforschung in der Onkologie GbR
๐ฉ๐ชKoblenz, Germany
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
๐ฉ๐ชMainz, Germany
Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII
๐ฎ๐นBergamo, Italy
Szpital Specjalistyczny w Brzozowie Podkarpacki Osrodek Onkologiczny im.ks.B.Markiewicza
๐ต๐ฑBrzozรณw, Poland
Wojewodzkie Wielospecjalistyczne Centrum, Onkologii i Traumatologii im. M Kopernika w Lodzi
๐ต๐ฑLodz, Poland
Centrum Medyczne Melita Medical
๐ต๐ฑWrocลaw, Poland
CHULN - Hospital de Santa Maria
๐ต๐นLisboa, Portugal
Ankara Universitesi Tip Fakultesi Hematologi Bilim dali Ankara
๐น๐ทBalkiraz, Ankara, Turkey
Pontchartrain Cancer Center
๐บ๐ธHammond, Louisiana, United States
Monmouth Medical Center Southern Campus
๐บ๐ธLakewood, New Jersey, United States
CHU Amiens Picardie - Hopital Sud
๐ซ๐ทAmiens, France
Queen Mary Hospital
๐ญ๐ฐHong Kong, Hong Kong
AOU San Luigi Gonzaga Orbassano
๐ฎ๐นTorino, Italy
AOU Citta della Salute e della Scienza di Torino
๐ฎ๐นTorino, Italy
Azienda ASST-SETTELAGHI, Ospedale di Circolo e Fond. Macchi di Varese Ospedael Vecchio
๐ฎ๐นVarese, Italy
Promed P.Lach R.Glowacki Spolka Jawna
๐ต๐ฑKrakow, Poland
MICS Centrum Medyczne Torun
๐ต๐ฑToruล, Poland
Pamukkale University Hospitals
๐น๐ทPamukkale, Denizli, Turkey
Universitaetsmedizin Greifswald
๐ฉ๐ชGreifswald, Germany
Debreceni Egyetem Klinikai Kozpont
๐ญ๐บDebrecen, Hungary
IRCCS Istituto Romagnolo per lo Studio dei Tumori "Dino Amadon"
๐ฎ๐นMeldola, Forli-Cesena, Italy
SUPERARE Centro de Infusion S.A. de C.V.
๐ฒ๐ฝMexico City, Mexico
Boca Clinical Trials Mexico S.C. - Ciudad de Mexico
๐ฒ๐ฝMexico City, Mexico
MTZ Clinical Research Powered by Pratia
๐ต๐ฑWarszawa, Poland
Conde Hospital Garcia de Orta
๐ต๐นAlmada, Portugal
Instituto Portugues de Oncologia do Porto Francisco Gentil, EPE
๐ต๐นPorto, Portugal
Oregon Health and Science University (OHSU) Knight Cancer Institute
๐บ๐ธPortland, Oregon, United States
University of Iowa Hospitals and Clinics
๐บ๐ธIowa City, Iowa, United States
Oncology Associates of Oregon
๐บ๐ธEugene, Oregon, United States
One Clinical Research
๐ฆ๐บNedlands, Perth, Australia
Wiener Gesundheitsverbund-Klinik Hietzing 5-Med.
๐ฆ๐นVienna, Austria
Gemeinschaftspraxis Haematologie - Onkologie - Hauptstelle
๐ฉ๐ชDresden, Germany
MD Anderson Cancer Center
๐บ๐ธHouston, Texas, United States
University of Colorado - Aurora Cancer Center
๐บ๐ธAurora, Colorado, United States
Moffitt Cancer Center
๐บ๐ธTampa, Florida, United States
American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
๐บ๐ธBethesda, Maryland, United States
University College London Hospital
๐ฌ๐งLondon, United Kingdom
Royal Free Hospital, Royal Free London NHSFT
๐ฌ๐งLondon, United Kingdom
University of Michigan
๐บ๐ธAnn Arbor, Michigan, United States
Fakultni nemocnice Olomouc
๐จ๐ฟOlomouc, Czechia
Palo Verde Hematology-Oncology
๐บ๐ธGlendale, Arizona, United States
Marin Cancer Care
๐บ๐ธGreenbrae, California, United States
California Cancer Associates for Research and Excellence - Fresno
๐บ๐ธFresno, California, United States
City of Hope Medical Center, Lenner
๐บ๐ธIrvine, California, United States
University of California, Los Angeles (UCLA) - Medical Center
๐บ๐ธLos Angeles, California, United States
University of California, San Diego (UCSD) - Moores Cancer Center
๐บ๐ธLa Jolla, California, United States
Stanford University
๐บ๐ธPalo Alto, California, United States
Pomona Valley Hospital Medical Center
๐บ๐ธPomona, California, United States
City of Hope Medical Center, Upland
๐บ๐ธUpland, California, United States
BayCare Suncoast Medical Clinic - Hematology/Oncology
๐บ๐ธSaint Petersburg, Florida, United States
Northwestern Medicine Lake Forest Hospital
๐บ๐ธLake Forest, Illinois, United States
Northwest Oncology and Hematology
๐บ๐ธRolling Meadows, Illinois, United States
Orchard Healthcare Research Inc
๐บ๐ธSkokie, Illinois, United States
Fort Wayne Medical Oncology and Hematology
๐บ๐ธFort Wayne, Indiana, United States
New York Cancer and Blood Specialists
๐บ๐ธRiverhead, New York, United States
Roswell Park Comprehensive Cancer Center
๐บ๐ธBuffalo, New York, United States
Memorial Sloan Kettering Cancer Center - COMMACK
๐บ๐ธCommack, New York, United States
Memorial Sloan Kettering Cancer Center - WESTCHESTER
๐บ๐ธHarrison, New York, United States
Ichan School of Medicine at Mount Sinai
๐บ๐ธNew York, New York, United States
Memorial Sloan Kettering Cancer Center - KOCH
๐บ๐ธNew York, New York, United States
New York Presbyterian Hospital/Weill Cornell Medical Center
๐บ๐ธNew York, New York, United States
Memorial Sloan Kettering Cancer Center - NASSAU
๐บ๐ธUniondale, New York, United States
Good Samaritan Infusion Center - Kenwood
๐บ๐ธCincinnati, Ohio, United States
Gabrail Cancer Center
๐บ๐ธCanton, Ohio, United States
TriHealth Cancer Institute - Good Samaritan Hospital
๐บ๐ธCincinnati, Ohio, United States
The Ohio State University
๐บ๐ธColumbus, Ohio, United States
Ohio Health Research Institute
๐บ๐ธColumbus, Ohio, United States
Thomas Jefferson University Hospital
๐บ๐ธPhiladelphia, Pennsylvania, United States
University of Washington - Fred Hutchinson Cancer Center
๐บ๐ธSeattle, Washington, United States
Texas Oncology San Antonio
๐บ๐ธSan Antonio, Texas, United States
Royal Adelaide Hospital
๐ฆ๐บAdelaide, South Australia, Australia
Campus Gasthuisberg
๐ง๐ชLeuven, Belgium
University Hospital Gent
๐ง๐ชGent, Belgium
Fakultni nemocnice Brno
๐จ๐ฟBrno, Czechia
Vseobecna fakultni nemocnice v Praze
๐จ๐ฟPrague, Czechia
CHU Poitiers
๐ซ๐ทPoitiers, Cedex, France
Semmelweis Egyetem
๐ญ๐บBudapest, Hungary
Markhot Ferenc Oktatokorhaz es Rendelointezet
๐ญ๐บEger, Hungary
Rambam Health Care Campus
๐ฎ๐ฑHaifa, Israel
Hadassah Medical Center Ein Karem
๐ฎ๐ฑJerusalem, Israel
Rabin Medical Center - Davidoff Hematology Center
๐ฎ๐ฑPetach Tikva, Israel
Meir Medical Center
๐ฎ๐ฑKfar Saba, Israel
Shamir Medical Center (Assaf Harofe)
๐ฎ๐ฑZerifin, Israel
IRCCS Azienda Ospedaliero-Universtaria di Bologna
๐ฎ๐นBologna, Italy
Community Cancer Trials of Utah
๐บ๐ธOgden, Utah, United States
Duke University Medical Center
๐บ๐ธDurham, North Carolina, United States
Yale University School of Medicine - Yale Cancer Center
๐บ๐ธNew Haven, Connecticut, United States
Montefiore Einstein Center for Cancer Care
๐บ๐ธBronx, New York, United States
Wilmot Cancer Institute, University of Rochester
๐บ๐ธRochester, New York, United States
The University of Alberta - Edmonton
๐จ๐ฆEdmonton, Alberta, Canada
Montefiore Medical Center
๐บ๐ธBronx, New York, United States