A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera

Polycythemia Vera Clinical Trial

— VERIFY  

Official title:

A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients With Polycythemia Vera

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05210790
• Other study ID #: PTG-300-11
• Status: Recruiting
• Phase: Phase 3
• Start date: April 1, 2022
• Est. completion date: June 2025

Study information

• Verified date: March 2024
• Source: Protagonist Therapeutics, Inc.
• Contact: Study Director
• Phone: 1-888-899-1543
• Email: ptgxclintrials[@]ptgx-inc.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: June 2025
• Est. primary completion date: February 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Main Inclusion Criteria: 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:

1. Hematocrit <45%,

2. WBC 4000/µL to 20,000/µL (inclusive), and

3. 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 Exclusion Criteria: 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.

Related Conditions & MeSH terms

• Polycythemia
• Polycythemia Vera

Intervention

Drug:
• Placebo
Placebo
• Rusfertide
Experimental drug

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Protagonist Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Belgium,  Canada,  Chile,  Czechia,  France,  Germany,  Hong Kong,  Hungary,  Israel,  Italy,  Mexico,  Netherlands,  Poland,  Portugal,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Comparison of mean number of phlebotomies between rusfertide and placebo.		Week 0 to Week 32
Secondary	Proportion of subjects with HCT values <45% for rusfertide and placebo.		Week 0 to Week 32
Secondary	Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo.		Week 32
Secondary	Comparison of mean change from baseline in total MFSAF total score.		Week 32