A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

NCT05210790 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PTG-300-11
• Study Type: interventional
• Target: 293
• Locations: 19 countries
• Sites: 178

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.

Conditions

Polycythemia Vera

Keywords

Polycythemia Vera; PV

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects achieving a response who receive rusfertide compared to placebo.

  Response is defined as absence of phlebotomy eligibility.

  Week 20 through Week 32

Secondary Outcomes (4)

• Comparison of mean number of phlebotomies between rusfertide and placebo.

  Week 0 to Week 32

• Proportion of subjects with HCT values <45% for 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

Study Arms (2)

Rusfertide

EXPERIMENTAL

Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)

Drug: Rusfertide

Placebo

EXPERIMENTAL

Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)

Drug: Placebo; Drug: Rusfertide

Interventions

Placebo DRUG

Placebo

Placebo

Rusfertide DRUG

Experimental drug

Placebo; Rusfertide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Protagonist Therapeutics, Inc.: lead

Study Sites (184)

Infirmary Cancer Care

Mobile, Alabama, 36607, United States

Location

Palo Verde Hematology-Oncology

Glendale, Arizona, 85304, United States

Location

City of Hope Medical Center, Duarte

Duarte, California, 91010, United States

Location

California Cancer Associates for Research and Excellence - Fresno

Fresno, California, 93720, United States

Location

Marin Cancer Care

Greenbrae, California, 94904, United States

Location

City of Hope Medical Center, Lenner

Irvine, California, 92618, United States

Location

University of California, San Diego (UCSD) - Moores Cancer Center

La Jolla, California, 92037, United States

Location

University of California, Los Angeles (UCLA) - Medical Center

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Pomona Valley Hospital Medical Center

Pomona, California, 91767, United States

Location

Sharp Memorial Hospital

San Diego, California, 92123, United States

Location

University of California, San Francisco

San Francisco, California, 94117, United States

Location

City of Hope Medical Center, Upland

Upland, California, 91786, United States

Location

Rocky Mountain Cancer Centers

Aurora, Colorado, 80012, United States

Location

University of Colorado - Aurora Cancer Center

Aurora, Colorado, 80045, United States

Location

Rocky Mountain Cancer Centers - Boulder

Boulder, Colorado, 80303, United States

Location

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, 80907, United States

Location

Rocky Mountain Cancer Centers

Colorado Springs, Colorado, 80923, United States

Location

Rocky Mountain Cancer Centers

Denver, Colorado, 80218, United States

Location

Rocky Mountain Cancer Centers

Lakewood, Colorado, 80228, United States

Location

Rocky Mountain Cancer Centers

Littleton, Colorado, 80120, United States

Location

Rocky Mountain Cancer Centers

Lone Tree, Colorado, 80124, United States

Location

Yale University School of Medicine - Yale Cancer Center

New Haven, Connecticut, 06510, United States

Location

BRCR Medical Center

Plantation, Florida, 33322, United States

Location

BayCare Suncoast Medical Clinic - Hematology/Oncology

St. Petersburg, Florida, 33705, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

Northwestern Medicine Lake Forest Hospital

Lake Forest, Illinois, 60045, United States

Location

Northwest Oncology and Hematology

Rolling Meadows, Illinois, 60008, United States

Location

Orchard Healthcare Research Inc

Skokie, Illinois, 60046, United States

Location

Fort Wayne Medical Oncology and Hematology

Fort Wayne, Indiana, 46845, United States

Location

Mission Cancer & Blood

Des Moines, Iowa, 50314, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Kansas Cancer Center

Kansas City, Kansas, 66160, United States

Location

Pontchartrain Cancer Center

Covington, Louisiana, 70433, United States

Location

Pontchartrain Cancer Center

Hammond, Louisiana, 70403, United States

Location

American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders)

Bethesda, Maryland, 20817, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Cancer Care Specialists

Reno, Nevada, 89511, United States

Location

Memorial Sloan Kettering Cancer Center - BASKING RIDGE

Basking Ridge, New Jersey, 07920, United States

Location

Monmouth Medical Center Southern Campus

Lakewood, New Jersey, 08701, United States

Location

Monmouth Medical Center

Long Branch, New Jersey, 07740, United States

Location

Memorial Sloan Kettering Cancer Center - MONMOUTH

Middletown, New Jersey, 07748, United States

Location

Memorial Sloan Kettering Cancer Center - BERGEN

Montvale, New Jersey, 07645, United States

Location

Rutgers Cancer Institute of New Jersey

New Brunswick, New Jersey, 08901, United States

Location

New York Cancer and Blood Specialists

Babylon, New York, 11702, United States

Location

Roswell Park Comprehensive Cancer Center

Buffalo, New York, 14263, United States

Location

Memorial Sloan Kettering Cancer Center - COMMACK

Commack, New York, 11725, United States

Location

Memorial Sloan Kettering Cancer Center - WESTCHESTER

Harrison, New York, 10604, United States

Location

New York Cancer and Blood Specialists

New Hyde Park, New York, 11042, United States

Location

Memorial Sloan Kettering Cancer Center - KOCH

New York, New York, 10021, United States

Location

New York Cancer and Blood Specialists

New York, New York, 10028, United States

Location

Ichan School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

New York Presbyterian Hospital/Weill Cornell Medical Center

New York, New York, 10065, United States

Location

New York Cancer and Blood Specialists

Patchogue, New York, 11772, United States

Location

New York Cancer and Blood Specialists

Port Jefferson Station, New York, 11776, United States

Location

New York Cancer and Blood Specialists

Riverhead, New York, 11901, United States

Location

Wilmot Cancer Institute, University of Rochester

Rochester, New York, 14642, United States

Location

Montefiore Einstein Center for Cancer Care

The Bronx, New York, 10461, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

New York Cancer and Blood Specialists

The Bronx, New York, 10469, United States

Location

Memorial Sloan Kettering Cancer Center - NASSAU

Uniondale, New York, 11553, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

East Carolina University

Greenville, North Carolina, 27858, United States

Location

Gabrail Cancer Center

Canton, Ohio, 44718, United States

Location

TriHealth Cancer Institute - Good Samaritan Hospital

Cincinnati, Ohio, 45220, United States

Location

Good Samaritan Infusion Center - Kenwood

Cincinnati, Ohio, 45236, United States

Location

Good Samaritan Infusion Center - Thomas

Cincinnati, Ohio, 45242, United States

Location

The Ohio State University

Columbus, Ohio, 43210, United States

Location

Ohio Health Research Institute

Columbus, Ohio, 43214, United States

Location

Oncology Associates of Oregon

Eugene, Oregon, 97401, United States

Location

Oregon Health and Science University (OHSU) Knight Cancer Institute

Portland, Oregon, 97210, United States

Location

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15232, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Texas Oncology San Antonio

San Antonio, Texas, 78240, United States

Location

Community Cancer Trials of Utah

Ogden, Utah, 84405, United States

Location

University of Washington - Fred Hutchinson Cancer Center

Seattle, Washington, 98019, United States

Location

HSHS St. Vincent Hospital

Green Bay, Wisconsin, 54301, United States

Location

HSHS St. Mary's Hospital

Green Bay, Wisconsin, 54303, United States

Location

One Clinical Research

Nedlands, Perth, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Medizinische Universitaet Graz

Graz, 8036, Austria

Location

Kepler Universitaets Klinikum GmbH

Linz, 4021, Austria

Location

Krankenhaus der Elisabethinen Linz

Linz, A-4020, Austria

Location

Medizinische Universitaet Wien

Vienna, 1090, Austria

Location

Wiener Gesundheitsverbund-Klinik Hietzing 5-Med.

Vienna, 1130, Austria

Location

Klinikum Wels-Grieskirchen Gmb

Wels, 4600, Austria

Location

ZNA Stuivenberg

Antwerp, 2060, Belgium

Location

University Hospital Gent

Ghent, 9000, Belgium

Location

Campus Gasthuisberg

Leuven, 3000, Belgium

Location

The University of Alberta - Edmonton

Edmonton, Alberta, T6G 2G3, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Princess Margaret Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada

Location

Centro Medico Inmunocel

Las Condes, Santiago Metropolitan, 7580206, Chile

Location

Centro de Estudios Clinicos SAGA

Providencia, Santiago Metropolitan, 7500653, Chile

Location

Biocenter Centro Clinico de Investigacion

Concepción, 4070196, Chile

Location

Fakultni nemocnice Brno

Brno, Czechia

Location

Fakultni nemocnice Olomouc

Olomouc, Czechia

Location

Vseobecna fakultni nemocnice v Praze

Prague, 128 00, Czechia

Location

CHU Angers

Angers, Cedex 09, France

Location

AP-HP Hospital Necker-Enfants Malades

Paris, Cedex 15, 75743, France

Location

CHU Poitiers

Poitiers, Cedex, 86000, France

Location

CHU Amiens Picardie - Hopital Sud

Amiens, France

Location

Centre Hospitalier d'Argenteuil

Argenteuil, 95107, France

Location

Centre Hospitalier de Beziers

Béziers, 34500, France

Location

AP-HP Hopital Saint-Louis

Paris, 75010, France

Location

HCL Centre Hospitalier Lyon Sud

Pierre-Bénite, France

Location

Uniklinik RWTH Aachen - Klinik fuer Haematologie, Onkologie, Haemostaseologie und Stammzelltransplantation (Med. Klinik IV)

Aachen, 52074, Germany

Location

Charite Universitaetsmedizin Berlin

Berlin, 10117, Germany

Location

Gemeinschaftspraxis Haematologie - Onkologie - Hauptstelle

Dresden, 01307, Germany

Location

Universitaetsmedizin Greifswald

Greifswald, 17489, Germany

Location

ONCOResearch Lerchenfeld GmbH

Hamburg, 22081, Germany

Location

InVo-Institut fuer Versorgungsforschung in der Onkologie GbR

Koblenz, 56068, Germany

Location

Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz

Mainz, 55131, Germany

Location

Queen Mary Hospital

Hong Kong, Hong Kong

Location

Semmelweis Egyetem

Budapest, 1085, Hungary

Location

Debreceni Egyetem Klinikai Kozpont

Debrecen, 4032, Hungary

Location

Markhot Ferenc Oktatokorhaz es Rendelointezet

Eger, 3300, Hungary

Location

Rambam Health Care Campus

Haifa, 3109601, Israel

Location

Hadassah Medical Center Ein Karem

Jerusalem, 121000, Israel

Location

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

Location

Meir Medical Center

Kfar Saba, 4428164, Israel

Location

Rabin Medical Center - Davidoff Hematology Center

Petah Tikva, 4941492, Israel

Location

Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Shamir Medical Center (Assaf Harofe)

Ẕerifin, 70300, Israel

Location

IRCCS Istituto Romagnolo per lo Studio dei Tumori "Dino Amadon"

Meldola, Forli-Cesena, 47014, Italy

Location

Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII

Bergamo, 24127, Italy

Location

IRCCS Azienda Ospedaliero-Universtaria di Bologna

Bologna, 40138, Italy

Location

UOC Ematologia - ASST Spedail Civill Brescia

Brescia, 25123, Italy

Location

Azienda Ospedaliero - Universitaria Careggi

Florence, 50134, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Ematologia (Padiglione Marcora),

Milan, 35-20122, Italy

Location

Fondazione IRCCS Policlinico San Matteo

Pavia, Italy

Location

UOC Ematologia e Centro Trapianti AST Pesaro Urbino, Stabilimento Muraglia

Pesaro, 61122, Italy

Location

Fondazione Policlinico Univ. A . Gemelli IRCCS

Roma, 00168, Italy

Location

AOU San Luigi Gonzaga Orbassano

Torino, 10043, Italy

Location

AOU Citta della Salute e della Scienza di Torino

Torino, 10126, Italy

Location

Azienda ASST-SETTELAGHI, Ospedale di Circolo e Fond. Macchi di Varese Ospedael Vecchio

Varese, 21100, Italy

Location

Centro de Estudios de Investigacion SIYODE

Madero, 89450, Mexico

Location

SUPERARE Centro de Infusion S.A. de C.V.

Mexico City, ZC 03100, Mexico

Location

Boca Clinical Trials Mexico S.C. - Ciudad de Mexico

Mexico City, ZC01120, Mexico

Location

Albert Schweitzer Ziekenhuis

Dordrecht, 3318 AT, Netherlands

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

Szpital Specjalistyczny w Brzozowie Podkarpacki Osrodek Onkologiczny im.ks.B.Markiewicza

Brzozów, 36-200, Poland

Location

MICS Centrum Medyczne Bydgoszcz

Bydgoszcz, 85-065, Poland

Location

Klinika Hematologii I Transplantologii - Uniwersyteckie Centrum Kliniczne

Gdansk, 80-214, Poland

Location

Promed P.Lach R.Glowacki Spolka Jawna

Krakow, 31-513, Poland

Location

Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi Oddział

Lodz, 93-510, Poland

Location

Wojewodzkie Wielospecjalistyczne Centrum, Onkologii i Traumatologii im. M Kopernika w Lodzi

Lodz, 93-513, Poland

Location

MICS Centrum Medyczne Torun

Torun, 87-100, Poland

Location

MTZ Clinical Research Powered by Pratia

Warsaw, 02-172, Poland

Location

Centrum Medyczne Melita Medical

Wroclaw, 50-449, Poland

Location

Conde Hospital Garcia de Orta

Almada, 2801-951, Portugal

Location

Hospital de Braga

Braga, 4710-243, Portugal

Location

CHULN - Hospital de Santa Maria

Lisbon, 1649-035, Portugal

Location

Instituto Portugues de Oncologia do Porto Francisco Gentil, EPE

Porto, 4200-072, Portugal

Location

Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Clinic Barcelona

Barcelona, 08036, Spain

Location

Hospital Germans Trias i Pujol

Barcelona, 08916, Spain

Location

Hospital del Mar

Barcelona, 8003, Spain

Location

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain

Location

Hospital Quironsalud de Zaragoza

Zaragoza, Spain

Location

Ankara Universitesi Tip Fakultesi Hematologi Bilim dali Ankara

Balkiraz, Ankara, 06620, Turkey (Türkiye)

Location

Gazi University Medical Faculty

Yenimahalle, Ankara, 06560, Turkey (Türkiye)

Location

Pamukkale University Hospitals

Pamukkale, Denizli, 20160, Turkey (Türkiye)

Location

Ege University Hospital Department of Hematology

Bornova, İzmir, 35100, Turkey (Türkiye)

Location

Karadeniz Technical University Medical Faculty Farabi Hospital

Trabzon, Ortahisar, 61080, Turkey (Türkiye)

Location

Sanko Universitesi tip Fakultesi Ic Hastaliklari Anabilim Dali

Gaziantep, Sehitkamil, 27090, Turkey (Türkiye)

Location

Gaziantep Universitesi Sahinbey Arastirma ve Uygulama Hastanesi

Gaziantep, 27410, Turkey (Türkiye)

Location

Marmara University Pendik Training & Research Hospital

Istanbul, 34899, Turkey (Türkiye)

Location

Erciyes Universitesi Tip Fakultesi Hastanesi, Dedeman Hospital

Kayseri, 38039, Turkey (Türkiye)

Location

Hillingdon Hospital

Uxbridge, Middlesex, U38 3NN, United Kingdom

Location

Barnet Hospital

Barnet, EN5 3DJ, United Kingdom

Location

Pilgrim Hospital

Boston, PE21 9QS, United Kingdom

Location

Northwick Park Hospital

Harrow, HA1 3UJ, United Kingdom

Location

The Royal London Hospital

London, E1 1BB, United Kingdom

Location

University College London Hospital

London, NW1 2PG, United Kingdom

Location

Royal Free Hospital, Royal Free London NHSFT

London, NW3 2QG, United Kingdom

Location

Churchill Hospital

Oxford, OX3 7LE, United Kingdom

Location

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, Double-blind (Part 1a): Subjects will be randomized in a blinded fashion to 32 weeks of rusfertide or placebo added-on to each subject's ongoing treatment for polycythemia vera. Open-label (Part 1b + Part 2): Open-label treatment phase during which all subjects who complete Part 1a successfully will receive rusfertide for 124 weeks.

Study Record Dates

• First Submitted: January 14, 2022
• First Posted: January 27, 2022
• Study Start: April 1, 2022
• Primary Completion: February 21, 2025
• Study Completion (Estimated): June 1, 2027
• Last Updated: August 7, 2025

Record last verified: 2025-08

Locations

AU (3); CA (4); DE (7); NL (2); PT (4); HK (1); FR (8); IL (7); IT (12); HU (3); ES (9); PL (9); CZ (3); TU (9); GB (8); CL (3); ME (3); BE (3); US (80)