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/μL to 20,000/μL (inclusive), and
- Platelets 100,000/μL to 1,000,000/μL (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
🇺🇸Pittsburgh, Pennsylvania, United States
Sharp Memorial Hospital
🇺🇸San Diego, California, United States
Good Samaritan Infusion Center - Thomas
🇺🇸Cincinnati, Ohio, United States
AP-HP Hopital Saint-Louis
🇫🇷Paris, France
City of Hope Medical Center, Duarte
🇺🇸Duarte, California, United States
Infirmary Cancer Care
🇺🇸Mobile, Alabama, United States
Fondazione IRCCS Policlinico San Matteo
🇮🇹Pavia, Italy
Centro de Estudios de Investigacion SIYODE
🇲🇽Madero, Mexico
Albert Schweitzer Ziekenhuis
🇳🇱Dordrecht, Netherlands
Hospital Quironsalud de Zaragoza
🇪🇸Zaragoza, Spain
Hospital Germans Trias i Pujol
🇪🇸Barcelona, Spain
UOC Ematologia e Centro Trapianti AST Pesaro Urbino, Stabilimento Muraglia
🇮🇹Pesaro, Italy
Hospital Universitario Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Erasmus MC
🇳🇱Rotterdam, Netherlands
Fondazione Policlinico Univ. A . Gemelli IRCCS
🇮🇹Roma, Italy
Churchill Hospital
🇬🇧Oxford, United Kingdom
Hospital Universitari i Politecnic La Fe
🇪🇸Valencia, Spain
Northwick Park Hospital
🇬🇧Harrow, United Kingdom
MICS Centrum Medyczne Bydgoszcz
🇵🇱Bydgoszcz, Poland
Hospital Clinic Barcelona
🇪🇸Barcelona, Spain
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
Hospital Universitario Ramon y Cajal
🇪🇸Madrid, Spain
Hospital del Mar
🇪🇸Barcelona, Spain
Gazi University Medical Faculty
🇹🇷Yenimahalle, Ankara, Turkey
Barnet Hospital
🇬🇧Barnet, United Kingdom
The Royal London Hospital
🇬🇧London, United Kingdom
Hillingdon Hospital
🇬🇧Uxbridge, Middlesex, United Kingdom
Pilgrim Hospital
🇬🇧Boston, United Kingdom
Mission Cancer & Blood
🇺🇸Des Moines, Iowa, United States
University of Kansas Cancer Center
🇺🇸Kansas City, Kansas, United States
Rocky Mountain Cancer Centers - Boulder
🇺🇸Boulder, Colorado, United States
Ege University Hospital Department of Hematology
🇹🇷Bornova, Izmir, Turkey
University of California, San Francisco
🇺🇸San Francisco, California, United States
Memorial Sloan Kettering Cancer Center - BASKING RIDGE
🇺🇸Basking Ridge, New Jersey, United States
ZNA Stuivenberg
🇧🇪Antwerp, Belgium
Cancer Care Specialists
🇺🇸Reno, Nevada, United States
Medizinische Universitaet Graz
🇦🇹Graz, Austria
Centro de Estudios Clinicos SAGA
🇨🇱Providencia, Santiago, Chile
Biocenter Centro Clinico de Investigacion
🇨🇱Concepción, Chile
UOC Ematologia - ASST Spedail Civill Brescia
🇮🇹Brescia, Italy
Klinika Hematologii I Transplantologii - Uniwersyteckie Centrum Kliniczne
🇵🇱Gdańsk, Poland
Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi Oddział
🇵🇱Łódź, Poland
Hospital de Braga
🇵🇹Braga, Portugal
Karadeniz Technical University Medical Faculty Farabi Hospital
🇹🇷Trabzon, Ortahisar, Turkey
Sanko Universitesi tip Fakultesi Ic Hastaliklari Anabilim Dali
🇹🇷Gaziantep, Sehitkamil, Turkey
Marmara University Pendik Training & Research Hospital
🇹🇷Istanbul, Turkey
Erciyes Universitesi Tip Fakultesi Hastanesi, Dedeman Hospital
🇹🇷Kayseri, Turkey
The Alfred Hospital
🇦🇺Melbourne, Victoria, Australia
Shaare Zedek Medical Center
🇮🇱Jerusalem, Israel
HSHS St. Mary's Hospital
🇺🇸Green Bay, Wisconsin, United States
ONCOResearch Lerchenfeld GmbH
🇩🇪Hamburg, Germany
East Carolina University
🇺🇸Greenville, North Carolina, United States
Sunnybrook Health Sciences Centre
🇨🇦Toronto, Ontario, Canada
Krankenhaus der Elisabethinen Linz
🇦🇹Linz, Austria
Medizinische Universitaet Wien
🇦🇹Wien, Austria
Klinikum Wels-Grieskirchen Gmb
🇦🇹Wels, Austria
Centro Medico Inmunocel
🇨🇱Las Condes, Santiago, Chile
Centre Hospitalier d'Argenteuil
🇫🇷Argenteuil, France
Centre Hospitalier de Beziers
🇫🇷Béziers, France
Princess Margaret Hospital
🇨🇦Toronto, Ontario, Canada
HCL Centre Hospitalier Lyon Sud
🇫🇷Pierre-Bénite, France
Uniklinik RWTH Aachen - Klinik fuer Haematologie, Onkologie, Haemostaseologie und Stammzelltransplantation (Med. Klinik IV)
🇩🇪Aachen, Germany
Monmouth Medical Center
🇺🇸Long Branch, New Jersey, United States
Charite Universitaetsmedizin Berlin
🇩🇪Berlin, Germany
Rutgers Cancer Institute of New Jersey
🇺🇸New Brunswick, New Jersey, United States
Rocky Mountain Cancer Centers
🇺🇸Lone Tree, Colorado, United States
BRCR Medical Center
🇺🇸Plantation, Florida, United States
Memorial Sloan Kettering Cancer Center - MONMOUTH
🇺🇸Middletown, New Jersey, United States
Kepler Universitaets Klinikum GmbH
🇦🇹Linz, Austria
AP-HP Hospital Necker-Enfants Malades
🇫🇷Paris, Cedex 15, France
Sourasky Medical Center
🇮🇱Tel Aviv, Israel
Azienda Ospedaliero - Universitaria Careggi
🇮🇹Firenze, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Ematologia (Padiglione Marcora),
🇮🇹Milano, Italy
Gaziantep Universitesi Sahinbey Arastirma ve Uygulama Hastanesi
🇹🇷Gaziantep, Turkey
HSHS St. Vincent Hospital
🇺🇸Green Bay, Wisconsin, United States
Memorial Sloan Kettering Cancer Center - BERGEN
🇺🇸Montvale, New Jersey, United States
Northwestern Memorial Hospital
🇺🇸Chicago, Illinois, United States
Jewish General Hospital
🇨🇦Montréal, Quebec, Canada
CHU Angers
🇫🇷Angers, Cedex 09, France
InVo-Institut fuer Versorgungsforschung in der Onkologie GbR
🇩🇪Koblenz, Germany
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
🇩🇪Mainz, Germany
Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII
🇮🇹Bergamo, Italy
Szpital Specjalistyczny w Brzozowie Podkarpacki Osrodek Onkologiczny im.ks.B.Markiewicza
🇵🇱Brzozów, Poland
Wojewodzkie Wielospecjalistyczne Centrum, Onkologii i Traumatologii im. M Kopernika w Lodzi
🇵🇱Lodz, Poland
Centrum Medyczne Melita Medical
🇵🇱Wrocław, Poland
CHULN - Hospital de Santa Maria
🇵🇹Lisboa, Portugal
Ankara Universitesi Tip Fakultesi Hematologi Bilim dali Ankara
🇹🇷Balkiraz, Ankara, Turkey
Pontchartrain Cancer Center
🇺🇸Hammond, Louisiana, United States
Monmouth Medical Center Southern Campus
🇺🇸Lakewood, New Jersey, United States
CHU Amiens Picardie - Hopital Sud
🇫🇷Amiens, France
Queen Mary Hospital
🇭🇰Hong Kong, Hong Kong
AOU San Luigi Gonzaga Orbassano
🇮🇹Torino, Italy
AOU Citta della Salute e della Scienza di Torino
🇮🇹Torino, Italy
Azienda ASST-SETTELAGHI, Ospedale di Circolo e Fond. Macchi di Varese Ospedael Vecchio
🇮🇹Varese, Italy
Promed P.Lach R.Glowacki Spolka Jawna
🇵🇱Krakow, Poland
MICS Centrum Medyczne Torun
🇵🇱Toruń, Poland
Pamukkale University Hospitals
🇹🇷Pamukkale, Denizli, Turkey
Universitaetsmedizin Greifswald
🇩🇪Greifswald, Germany
Debreceni Egyetem Klinikai Kozpont
🇭🇺Debrecen, Hungary
IRCCS Istituto Romagnolo per lo Studio dei Tumori "Dino Amadon"
🇮🇹Meldola, Forli-Cesena, Italy
SUPERARE Centro de Infusion S.A. de C.V.
🇲🇽Mexico City, Mexico
Boca Clinical Trials Mexico S.C. - Ciudad de Mexico
🇲🇽Mexico City, Mexico
MTZ Clinical Research Powered by Pratia
🇵🇱Warszawa, Poland
Conde Hospital Garcia de Orta
🇵🇹Almada, Portugal
Instituto Portugues de Oncologia do Porto Francisco Gentil, EPE
🇵🇹Porto, Portugal
Oregon Health and Science University (OHSU) Knight Cancer Institute
🇺🇸Portland, Oregon, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
Oncology Associates of Oregon
🇺🇸Eugene, Oregon, United States
One Clinical Research
🇦🇺Nedlands, Perth, Australia
Wiener Gesundheitsverbund-Klinik Hietzing 5-Med.
🇦🇹Vienna, Austria
Gemeinschaftspraxis Haematologie - Onkologie - Hauptstelle
🇩🇪Dresden, Germany
MD Anderson Cancer Center
🇺🇸Houston, Texas, United States
University of Colorado - Aurora Cancer Center
🇺🇸Aurora, Colorado, United States
Moffitt Cancer Center
🇺🇸Tampa, Florida, United States
American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)
🇺🇸Bethesda, Maryland, United States
University College London Hospital
🇬🇧London, United Kingdom
Royal Free Hospital, Royal Free London NHSFT
🇬🇧London, United Kingdom
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Fakultni nemocnice Olomouc
🇨🇿Olomouc, Czechia
Palo Verde Hematology-Oncology
🇺🇸Glendale, Arizona, United States
Marin Cancer Care
🇺🇸Greenbrae, California, United States
California Cancer Associates for Research and Excellence - Fresno
🇺🇸Fresno, California, United States
City of Hope Medical Center, Lenner
🇺🇸Irvine, California, United States
University of California, Los Angeles (UCLA) - Medical Center
🇺🇸Los Angeles, California, United States
University of California, San Diego (UCSD) - Moores Cancer Center
🇺🇸La Jolla, California, United States
Stanford University
🇺🇸Palo Alto, California, United States
Pomona Valley Hospital Medical Center
🇺🇸Pomona, California, United States
City of Hope Medical Center, Upland
🇺🇸Upland, California, United States
BayCare Suncoast Medical Clinic - Hematology/Oncology
🇺🇸Saint Petersburg, Florida, United States
Northwestern Medicine Lake Forest Hospital
🇺🇸Lake Forest, Illinois, United States
Northwest Oncology and Hematology
🇺🇸Rolling Meadows, Illinois, United States
Orchard Healthcare Research Inc
🇺🇸Skokie, Illinois, United States
Fort Wayne Medical Oncology and Hematology
🇺🇸Fort Wayne, Indiana, United States
New York Cancer and Blood Specialists
🇺🇸Riverhead, New York, United States
Roswell Park Comprehensive Cancer Center
🇺🇸Buffalo, New York, United States
Memorial Sloan Kettering Cancer Center - COMMACK
🇺🇸Commack, New York, United States
Memorial Sloan Kettering Cancer Center - WESTCHESTER
🇺🇸Harrison, New York, United States
Ichan School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Memorial Sloan Kettering Cancer Center - KOCH
🇺🇸New York, New York, United States
New York Presbyterian Hospital/Weill Cornell Medical Center
🇺🇸New York, New York, United States
Memorial Sloan Kettering Cancer Center - NASSAU
🇺🇸Uniondale, New York, United States
Good Samaritan Infusion Center - Kenwood
🇺🇸Cincinnati, Ohio, United States
Gabrail Cancer Center
🇺🇸Canton, Ohio, United States
TriHealth Cancer Institute - Good Samaritan Hospital
🇺🇸Cincinnati, Ohio, United States
The Ohio State University
🇺🇸Columbus, Ohio, United States
Ohio Health Research Institute
🇺🇸Columbus, Ohio, United States
Thomas Jefferson University Hospital
🇺🇸Philadelphia, Pennsylvania, United States
University of Washington - Fred Hutchinson Cancer Center
🇺🇸Seattle, Washington, United States
Texas Oncology San Antonio
🇺🇸San Antonio, Texas, United States
Royal Adelaide Hospital
🇦🇺Adelaide, South Australia, Australia
Campus Gasthuisberg
🇧🇪Leuven, Belgium
University Hospital Gent
🇧🇪Gent, Belgium
Fakultni nemocnice Brno
🇨🇿Brno, Czechia
Vseobecna fakultni nemocnice v Praze
🇨🇿Prague, Czechia
CHU Poitiers
🇫🇷Poitiers, Cedex, France
Semmelweis Egyetem
🇭🇺Budapest, Hungary
Markhot Ferenc Oktatokorhaz es Rendelointezet
🇭🇺Eger, Hungary
Rambam Health Care Campus
🇮🇱Haifa, Israel
Hadassah Medical Center Ein Karem
🇮🇱Jerusalem, Israel
Rabin Medical Center - Davidoff Hematology Center
🇮🇱Petach Tikva, Israel
Meir Medical Center
🇮🇱Kfar Saba, Israel
Shamir Medical Center (Assaf Harofe)
🇮🇱Zerifin, Israel
IRCCS Azienda Ospedaliero-Universtaria di Bologna
🇮🇹Bologna, Italy
Community Cancer Trials of Utah
🇺🇸Ogden, Utah, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Yale University School of Medicine - Yale Cancer Center
🇺🇸New Haven, Connecticut, United States
Montefiore Einstein Center for Cancer Care
🇺🇸Bronx, New York, United States
Wilmot Cancer Institute, University of Rochester
🇺🇸Rochester, New York, United States
The University of Alberta - Edmonton
🇨🇦Edmonton, Alberta, Canada
Montefiore Medical Center
🇺🇸Bronx, New York, United States