Clinical trial • Phase III • Oncology|Rare Disease

RUSFERTIDE for Polycythemia vera

Phase III trial of RUSFERTIDE for Polycythemia vera.

Overview

Trial Therapeutic Area
Oncology|Rare Disease
Trial Disease
Polycythemia vera
Trial Stage
Phase III
Drug Modality
Peptide/protein/enzyme
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
29-02-2024
First CTIS Authorization Date
10-04-2024

Trial design

Randomised, placebo to match ptg-300 for injection (formulation (lyophilized powder) based upon that of ptg-300 for injection, with the drug substance omitted).-controlled Phase III trial across 52 sites in Spain, Portugal, Belgium and others.

Randomised
Yes
Comparator
Placebo to Match PTG-300 for Injection (Formulation (lyophilized powder) based upon that of PTG-300 for Injection, with the drug substance omitted).
Target Sample Size
148
Trial Duration For Participant
224

Eligibility

Recruits 148 Vulnerable population is selected in the CTIS record (isVulnerablePopulationSelected = true). Subject information and informed consent forms (L1_SIS and ICF documents) are listed in multiple versions and languages. No explicit assent procedures or detailed consent/assent handling text is provided in the record beyond the inclusion criterion that participants must be aged 18 or older (or the country-specific minimum age of consent)..

Vulnerable Population
Vulnerable population is selected in the CTIS record (isVulnerablePopulationSelected = true). Subject information and informed consent forms (L1_SIS and ICF documents) are listed in multiple versions and languages. No explicit assent procedures or detailed consent/assent handling text is provided in the record beyond the inclusion criterion that participants must be aged 18 or older (or the country-specific minimum age of consent).

Inclusion criteria

  • {"criterion_text":"- 1 - Male and female subjects aged 18 (or the minimum country specific age of consent if >18) years or older.\n- 2 - Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera\n- 3 - Phlebotomy requiring defined as ALL of the following: a. At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization, and b. Last phlebotomy due to inadequate hematocrit control within 3 months before randomization, and c. No phlebotomy within 6 days prior to randomization (do not include day of phlebotomy and day of randomization in the 6-day count). Note: Phlebotomies performed within an 8-day period will be counted as a single phlebotomy\n- 4 - CBC values immediately prior to randomization: a. Hematocrit <45%, b. WBC 4000/µL to 20,000/µL (inclusive) and c. Platelets 100,000/µL to 1,000,000/µL (inclusive).\n- 5 - Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen\n- 6 - Subjects treated with phlebotomy alone at randomization must have stopped cytoreductive therapy 2 to 6 months before screening."}

Exclusion criteria

  • {"criterion_text":"- 1 - Clinically meaningful laboratory abnormalities at Screening\n- 2 - Subjects who require phlebotomy at hematocrit levels lower than 45%\n- 3 - Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.\n- 4 - Active or chronic bleeding within 2 months prior to randomization.\n- 5 - History of invasive malignancies within the last 5 years, except a) localized cured cancer (e.g. prostate cancer and cervical cancer). b) localized cured in situ or stage 1 squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or in situ melanoma of the skin.\n- 6 - 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 screening unless the cancer is adequately treated before randomization.\n- 7 - Received busulfan, pipobroman or 32Phosphorus within 7 months prior to screening."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of subjects achieving a response starting at Week 20 through Week 32 (inclusive) who receive rusfertide compared to placebo. A response is defined as absence of phlebotomy eligibility. Phlebotomy eligibility is defined as either: • a confirmed hematocrit ≥45% and that is at least 3% higher than the baseline hematocrit (value immediately prior to randomization at Week 0); or • a hematocrit ≥48%.","definition_or_measurement_approach":"Response defined as absence of phlebotomy eligibility. Phlebotomy eligibility defined as either: a confirmed hematocrit ≥45% and at least 3% higher than baseline hematocrit (value immediately prior to randomization at Week 0), or a hematocrit ≥48%. Endpoint measured as proportion of subjects achieving response between Week 20 and Week 32 inclusive, comparing rusfertide to placebo."}

Secondary endpoints

  • {"endpoint_text":"- 1 - Mean number of phlebotomies between Week 0 through Week 32 (inclusive).","definition_or_measurement_approach":"Mean count of phlebotomy procedures per subject during Week 0 through Week 32 (inclusive)."}
  • {"endpoint_text":"- 2 - Proportion of subjects with all hematocrit values <45% between Week 0 through Week 32 (inclusive).","definition_or_measurement_approach":"Proportion of subjects whose hematocrit measurements are all <45% during Week 0 through Week 32 (inclusive)."}
  • {"endpoint_text":"- 3 - Mean change from baseline at end of Part 1a (Week 32) in the Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 8a total T-score","definition_or_measurement_approach":"Mean change from baseline to Week 32 in PROMIS Fatigue Short Form 8a total T-score."}
  • {"endpoint_text":"- 4 - Mean change from baseline at end of Part 1a (Week 32) in the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF)","definition_or_measurement_approach":"Mean change from baseline to Week 32 in MFSAF v4.0 score."}
  • {"endpoint_text":"- 5 - Mean change from baseline at end of Part 1a (Week 32) in the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF)","definition_or_measurement_approach":"Mean change from baseline to Week 32 in MPN-SAF score."}

Recruitment

Planned Sample Size
148
Recruitment Window Months
55
Consent Approach
Informed consent to be obtained from participants (participants must be aged 18 or older, or meet the country-specific minimum age of consent). Subject information and informed consent forms (L1_SIS and ICF) are available in multiple languages and country-specific versions (documents listed include Main ICF and Pregnancy/Partner ICF versions and country-specific ICFs). Languages and country-specific ICFs present in the dossier include English, Dutch, French, German, Italian, Spanish, Polish, Hungarian, Czech, Portuguese and others as indicated by document listings. No assent procedures for minors are provided (minors are not eligible).

Methods

  • K1 recruitment arrangements documents submitted per country (country-specific K1 files listed for PL, NL, ES, IT, HU, DE, CZ, BE, AT, FR).
  • K2 recruitment materials: Brochure/Trifold, Flyer/Participant Flyer, Dear Colleague Letter, Participant Journey, Patiro documents used as recruitment channels.
  • Patient Recruitment responsibilities assigned to Medpace Finland Oy (listed under sponsor third parties).

Geography

Total Number Of Sites
52
Total Number Of Participants
102

Spain

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
27-11-2025
Processing Time Days
623
Number Of Sites
8
Number Of Participants
18

Sites

Site Name
Institut Catala D'oncologia
Department Name
Hematology
Contact Person Name
Blanca Xicoy
Contact Person Email
bxicoy@iconcologia.net
Site Name
Hospital Quironsalud Zaragoza
Department Name
Hematology
Contact Person Name
Pilar Giraldo
Contact Person Email
giraldocastellano@gmail.com
Site Name
Hospital Clinic De Barcelona
Department Name
Hematology
Contact Person Name
Alberto Alvarez
Contact Person Email
aalvar@clinic.cat
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Hematology and Hemotherapy
Contact Person Name
Santiago Osorio
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Hematology
Contact Person Name
Valentin Garcia
Contact Person Email
jvalentingg@gmail.com
Site Name
Hospital Del Mar
Department Name
Clinic Hematology
Contact Person Name
Patricia Velez
Contact Person Email
patricia.velez.tenza@psmar.cat
Site Name
Hospital Universitari Vall D Hebron
Department Name
Hematology
Contact Person Name
Maria Laura Fox
Contact Person Email
mlfox@vhebron.net
Site Name
Hospital Universitario 12 De Octubre
Department Name
Hematology
Contact Person Name
Rosa Ayala
Contact Person Email
rayaladiaz12@gmail.com

Portugal

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
11-04-2024
Processing Time Days
28
Number Of Sites
4
Number Of Participants
2

Sites

Site Name
CCAB Centro Clinico Academico Braga Associacao
Department Name
Medical Oncology
Contact Person Name
Herlander Marques
Contact Person Email
herlandermarques@hotmail.com
Site Name
Instituto Portugues De Oncologia Do Porto Francisco Gentil E.P.E.
Department Name
Clinical Hematology
Contact Person Name
Isabel Oliveira
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Immunohemotherapy
Contact Person Name
Maria Blanca Polo Guerrero
Site Name
Hospital Garcia De Orta E.P.E.
Department Name
Hemato-oncology
Contact Person Name
Inês Conde
Contact Person Email
conde.ines@gmail.com

Belgium

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
02-12-2025
Processing Time Days
628
Number Of Sites
3
Number Of Participants
5

Sites

Site Name
Universiteit Gent
Department Name
Haematology
Contact Person Name
Dominiek Mazure
Contact Person Email
dominiek.mazure@uzgent.be
Site Name
UZ Leuven
Department Name
Haematology
Contact Person Name
Andrzej Mital
Contact Person Email
timothy.devos@uzleuven.be
Site Name
Het Ziekenhuisnetwerk Antwerpen
Department Name
Haematology
Contact Person Name
Nikki Charlotta Paul Granacher
Contact Person Email
nikki.granacher@zna.be

Netherlands

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
17-11-2025
Processing Time Days
613
Number Of Sites
2
Number Of Participants
8

Sites

Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Hematology
Contact Person Name
Peter te Boekhorst
Contact Person Email
p.teboekhorst@erasmusmc.nl
Site Name
Albert Schweitzer Ziekenhuis
Department Name
Internal Medicine
Contact Person Name
Peter Westerweel
Contact Person Email
wetenschap@asz.nl

Italy

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
26-01-2026
Processing Time Days
683
Number Of Sites
9
Number Of Participants
21

Sites

Site Name
Careggi University Hospital
Department Name
Hematology
Contact Person Name
Alessandro Maria Vannucchi
Contact Person Email
a.vannucchi@unifi.it
Site Name
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
Department Name
Medical Oncology
Contact Person Name
Alessandro Lucchesi
Site Name
Azienda Ospedaliera Ospedali Riuniti Marche Nord
Department Name
Complex Unit of Hematology and Bone Marrow Transplantation Center
Contact Person Name
Alessandro Isidori
Site Name
Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
Department Name
Hematology
Contact Person Name
Palandri Francesca
Contact Person Email
francesca.palandri@unibo.it
Site Name
Azienda Ospedaliero-Universitaria San Luigi Gonzaga
Department Name
Hematology
Contact Person Name
Alessandro Morotti
Contact Person Email
alessandro.morotti@unito.it
Site Name
Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII
Department Name
Oncology and hemato-oncology
Contact Person Name
Federico Lussana
Contact Person Email
flussana@asst-pg23.it
Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
Hematology
Contact Person Name
Elisa Rumi
Contact Person Email
elisa.rumi@unipv.it
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Hematology
Contact Person Name
Valerio De Stefano
Contact Person Email
valerio.destefano@unicatt.it
Site Name
Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino
Department Name
Hematology
Contact Person Name
Giulia Benevolo

Hungary

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
16-12-2025
Processing Time Days
642
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Heves Varmegyei Markhot Ferenc Oktatokorhaz Es Rendelointezet
Department Name
Belgyógyászati-Infektológiai Centrum
Contact Person Name
Balazs TAJTI
Contact Person Email
tajtibalazs1985@gmail.com
Site Name
University Of Debrecen
Department Name
Belgyógyászati Klinika B épület Hematológia
Contact Person Name
Arpad ILLES
Contact Person Email
illes.arpad@med.unideb.hu

Germany

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
17-12-2025
Processing Time Days
643
Number Of Sites
6
Number Of Participants
10

Sites

Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
III. Med. Klinik
Contact Person Name
Daniel Sasca
Site Name
Universitaetsmedizin Greifswald KöR
Department Name
Hematology and Oncology
Contact Person Name
Adrian Schwarzer
Site Name
Universitaetsklinikum Aachen AöR
Department Name
Medizinische Klinik IV
Contact Person Name
Steffen Koschmieder
Contact Person Email
skoschmieder@ukaachen.de
Site Name
OncoResearch Lerchenfeld GmbH
Department Name
Praxis
Contact Person Name
Thomas Wolff
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Hämatologie und Onkologie und Tumorimmunologie
Contact Person Name
Philipp David Immanuel Le Coutre
Contact Person Email
philipp.lecoutre@charite.de
Site Name
Gemeinschaftspraxis Haematologie Onkologie
Department Name
Gemeinschaftspraxis
Contact Person Name
Thomas Illmer
Contact Person Email
buero@onkologie-dresden.net

Czechia

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
13-01-2026
Processing Time Days
671
Number Of Sites
3
Number Of Participants
12

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
I. interní klinika – Hematologické ambulance
Contact Person Name
Anna Jonášová
Contact Person Email
anna.jonasova@vfn.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Interní hematologická a onkologická klinika
Contact Person Name
Jiří Mayer
Contact Person Email
mayer.jiri@fnbrno.cz
Site Name
University Hospital Olomouc
Department Name
Hemato-onkologická klinika
Contact Person Name
Antonín Hluší
Contact Person Email
antonin.hlusi@fnol.cz

Austria

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
22-12-2025
Processing Time Days
648
Number Of Sites
4
Number Of Participants
6

Sites

Site Name
Klinik Hietzing
Department Name
5. Med. Abteilung
Contact Person Name
Daniel Lechner-Radner
Site Name
Ordensklinikum Linz GmbH
Department Name
Hematology and Oncology
Contact Person Name
Veronika Buxhofer-Ausch
Site Name
Kepler Universitaetsklinikum GmbH
Department Name
Internal Medicine 3
Contact Person Name
Clemens Schmitt
Site Name
Medical University Of Graz
Department Name
internal medicine
Contact Person Name
Albert Wölfler
Contact Person Email
albert.woelfler@medunigraz.at

France

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
757
Number Of Sites
5
Number Of Participants
7

Sites

Site Name
Centre Hospitalier Victor Dupouy
Department Name
Hematology
Contact Person Name
Annalisa Andreoli
Site Name
Centre Hospitalier Regional D'Angers
Department Name
Hematology
Contact Person Name
Françoise Boyer
Contact Person Email
frboyer-perrard@chu-angers.fr
Site Name
Hopital Saint Louis
Department Name
Hematology
Contact Person Name
Jean-Jacques Kiladjian
Contact Person Email
jean-jacques.kiladjian@aphp.fr
Site Name
Centre Hospitalier De Beziers
Department Name
Hematology
Contact Person Name
Yuriy Drebit
Contact Person Email
yuriy.drebit@ch-beziers.fr
Site Name
Centre Hospitalier Lyon Sud
Department Name
Hematology
Contact Person Name
Vincent Alcazer
Contact Person Email
Vincent.alcazer@chu-lyon.fr

Poland

Earliest CTIS Part Ii Submission Date
14-03-2024
Latest Decision Or Authorization Date
05-12-2025
Processing Time Days
631
Number Of Sites
6
Number Of Participants
10

Sites

Site Name
Medicover Integrated Clinical Services Sp. z o.o. (Bydgoszcz)
Department Name
Centrum Medyczne Bydgoszcz
Contact Person Name
Jarosław Czyż
Contact Person Email
aleksandra.robak@medicover.com
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Hematologii i Transplantologii
Contact Person Name
Andrzej Mital
Contact Person Email
hematologia@uck.gda.pl
Site Name
Szpital Specjalistyczny W Brzozowie Podkarpacki Osrodek Onkologiczny Im.Ks.B.Markiewicza
Department Name
Oddział Hematologii Onkologicznej z Pododdziałem Transplantologii Klinicznej
Contact Person Name
Jacek Krzanowski
Site Name
Medicover Integrated Clinical Services Sp. z o.o. (Torun)
Department Name
Centrum Medyczne Toruń
Contact Person Name
Marcin Rymko
Contact Person Email
ewa.rymko@medicover.com
Site Name
Melita Medical Sp. z o.o.
Contact Person Name
Justyna Rybka
Contact Person Email
b.lapuszynski@melitamedical.pl
Site Name
Wojewodzkie Wielospecjalistyczne Centrum Onkologii I Traumatologii Im M.Kopernika W Lodzi
Department Name
Oddział Hematologii Ogólnej i Chorób Wewnętrznych
Contact Person Name
Tadeusz Robak
Contact Person Email
robaktad@csk.umed.lodz.pl

Sponsor

Primary sponsor

Full Name
Protagonist Therapeutics Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Suvoda LLC
Responsibilities
sponsorDuties codes: 3
Name
Medpace Finland Oy
Responsibilities
Multiple sponsorDuties including code 15 with value 'Patient Recruitment' and other duty codes (1,2,5,6,7,8)
Name
Syneos Health Inc.
Responsibilities
Safety Database and Management
Name
Icon Laboratory Services Inc.
Responsibilities
sponsorDuties codes: 4 (laboratory services)
Name
Charles River Laboratories Inc.
Responsibilities
sponsorDuties codes: 4 (laboratory services)
Name
Intrinsic Lifesciences LLC
Responsibilities
sponsorDuties codes: 4

Third parties

  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"sponsorDuties codes: 3","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Intrinsic Lifesciences LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"Safety Database and Management (sponsorDuties code: 15; value provided: 'Safety Database and Management')","organisation_type":"Pharmaceutical company"}
  • {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"sponsorDuties codes: 1,15 (value 'Patient Recruitment' provided),2,5,6,7,8","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Charles River Laboratories Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Rusfertide 10mg
Active Substance
RUSFERTIDE
Modality
Peptide/protein/enzyme
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
1
Orphan Designation
Yes
Dose Levels
10 mg | 20 mg | 30 mg | 45 mg | 60 mg
Maximum Dose
90 mg (maxDailyDoseAmount)
Investigational Product Name
Rusfertide 20mg
Active Substance
RUSFERTIDE
Modality
Peptide/protein/enzyme
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
1
Orphan Designation
Yes
Dose Levels
10 mg | 20 mg | 30 mg | 45 mg | 60 mg
Maximum Dose
90 mg (maxDailyDoseAmount)
Investigational Product Name
Rusfertide 30mg
Active Substance
RUSFERTIDE
Modality
Peptide/protein/enzyme
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
1
Orphan Designation
Yes
Dose Levels
10 mg | 20 mg | 30 mg | 45 mg | 60 mg
Maximum Dose
90 mg (maxDailyDoseAmount)
Investigational Product Name
Rusfertide 45mg
Active Substance
RUSFERTIDE
Modality
Peptide/protein/enzyme
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
1
Orphan Designation
Yes
Dose Levels
10 mg | 20 mg | 30 mg | 45 mg | 60 mg
Maximum Dose
90 mg (maxDailyDoseAmount)
Investigational Product Name
Rusfertide 60mg
Active Substance
RUSFERTIDE
Modality
Peptide/protein/enzyme
Routes Of Administration
SUBCUTANEOUS USE
Route
Subcutaneous
Authorisation Status
1
Orphan Designation
Yes
Dose Levels
10 mg | 20 mg | 30 mg | 45 mg | 60 mg
Maximum Dose
90 mg (maxDailyDoseAmount)
Investigational Product Name
Placebo to Match PTG-300 for Injection
Modality
Other
Combination Treatment
Yes

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