Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera

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What is this study about?

This clinical trial is focused on studying a condition called Polycythemia Vera, a rare blood disorder where the body produces too many red blood cells. This can lead to complications such as blood clots. The study is testing a new treatment called Rusfertide, also known by its code name PTG-300. Rusfertide is given as an injection under the skin and is designed to help control the number of red blood cells in the body.

The purpose of the study is to evaluate how safe and effective Rusfertide is in helping patients with Polycythemia Vera maintain control over their hematocrit levels, which is the proportion of red blood cells in the blood. Participants in the study will receive either Rusfertide or a placebo, which looks like the treatment but does not contain the active substance. The study will last for a period of time, during which participants will receive regular injections and have their blood monitored to see how well the treatment is working.

Throughout the study, the main goal is to see if Rusfertide can reduce the need for phlebotomies, which are procedures to remove blood from the body to lower red blood cell levels. The study will also look at other factors, such as changes in fatigue levels and overall well-being. This research aims to provide more information about the potential benefits of Rusfertide for people living with Polycythemia Vera.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age, diagnosis of polycythemia vera, and recent medical history related to blood tests and treatments.

2 initial assessment

An initial assessment is conducted to establish baseline health metrics. This includes measuring hematocrit levels, white blood cell count, and platelet count.

3 randomization and treatment assignment

Participants are randomly assigned to receive either rusfertide or a placebo. The dosage of rusfertide may vary, including options such as 10mg, 20mg, 30mg, 45mg, or 60mg, administered via subcutaneous injection.

4 treatment phase

During the treatment phase, participants receive their assigned medication regularly. The primary goal is to maintain hematocrit control without the need for phlebotomy, which is a procedure to remove blood to reduce iron levels.

5 monitoring and evaluation

Participants are monitored for their response to the treatment from Week 20 through Week 32. The effectiveness is measured by the absence of phlebotomy eligibility, which is determined by specific hematocrit levels.

6 secondary assessments

Secondary assessments include evaluating the mean number of phlebotomies required, maintaining hematocrit levels below 45%, and assessing changes in fatigue and other symptoms using standardized questionnaires.

7 completion of study participation

Upon completion of the study period, participants undergo a final evaluation to assess the overall impact of the treatment on their condition.

Who Can Join the Study?

  • Men and women who are 18 years or older can participate.
  • Participants must have a diagnosis of Polycythemia Vera, which is a condition where the body makes too many red blood cells.
  • Participants should have needed phlebotomies (a procedure to remove blood) because of high hematocrit levels, which is the percentage of red blood cells in the blood. Specifically, they should have had:
    • At least 3 phlebotomies in the last 28 weeks or at least 5 in the last year.
    • The last phlebotomy should have been within 3 months before joining the study.
    • No phlebotomy in the 6 days before joining the study.
  • Before joining the study, blood test results should show:
    • Hematocrit less than 45%.
    • White blood cells between 4,000 and 20,000 per microliter.
    • Platelets between 100,000 and 1,000,000 per microliter.
  • If participants are on cytoreductive therapy (treatment to reduce the number of blood cells), they must be on a stable treatment plan for Polycythemia Vera.
  • If participants are only treated with phlebotomy, they must have stopped any cytoreductive therapy 2 to 6 months before the study starts.

Who Cannot Join the Study?

  • Patients who do not have a diagnosis of Polycythemia Vera cannot participate. Polycythemia Vera is a condition where the body makes too many red blood cells.
  • Patients who are not within the specified age range cannot participate. The age range includes adults and older adults.
  • Patients who are not part of the specified clinical trial group cannot participate. This group includes individuals with certain characteristics needed for the study.
  • Both male and female patients are eligible, but those who do not meet other criteria cannot participate.
  • Patients who are considered part of a vulnerable population, such as those who may not be able to give informed consent, cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Centre Hospitalier Victor Dupouy Argenteuil France
Universitaetsklinikum Aachen AöR Aachen Germany
Centre Hospitalier Lyon Sud Pierre Benite France
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital Universitario 12 De Octubre Madrid Spain
Hospital Del Mar Barcelona Spain
Instituto Portugues De Oncologia Do Porto Francisco Gentil E.P.E. Porto Portugal
CCAB Centro Clinico Academico Braga Associacao Braga Portugal
Albert Schweitzer Ziekenhuis Dordrecht The Netherlands
Szpital Specjalistyczny W Brzozowie Podkarpacki Osrodek Onkologiczny Im.Ks.B.Markiewicza Brzozow Poland
Hospital Quironsalud Zaragoza Zaragoza Spain
Azienda Ospedaliera Ospedali Riuniti Marche Nord Pesaro Italy
OncoResearch Lerchenfeld GmbH Hamburg Germany
University Of Debrecen Debrecen Hungary
Melita Medical sp. z o.o. Wroclaw Poland
Azienda Ospedaliero-Universitaria San Luigi Gonzaga Orbassano Italy
Fondazione IRCCS Policlinico San Matteo Pavia Italy
Hospital Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
Kepler Universitaetsklinikum GmbH Linz Austria
Gasthuiszusters Antwerpen Antwerp Belgium
Markhot Ferenc Oktatokorhaz Es Rendelointezet Eger Hungary
Klinik Hietzing Vienna Austria
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
University Hospital Olomouc Olomouc Czechia
Universitair Ziekenhuis Gent Gent Belgium
Owbigezlbzkuer Lydn Gzzl Linz Austria
Caakgl Hcgqyzgiapx Rntrqhgf Dapuehhmwejbyq Angers France
Wfmeoveolop Wicrcytpfikvrrgibeql Clkmlgf Ojtzjnbbc I Tcjfbhoewfkfd Ij Mmrzcvbytgi W Lthpv Lodz Poland
Masdfgvad Iajlhcqfev Cimbsuhq Sjqhqstk Sch z oyyd Warsaw Poland
Iuucnvke Roimoyztf Pft Ln Sozecd Drv Tywifn Dofo Akljlve Iisc Spudcm Meldola Italy
Acsugnlutf Pdxahstw Hykwsadn Db Pywoe Paris France
Etropmk Uouenogyfmfu Mbmfrhv Crbkzjf Rvppkyldu (yltdvnn Moz Rotterdam The Netherlands
Aqgfiao Oetroazddkn Ujdnyrjxhvtjl Cpnlbiifjxtd Dyiro Scexdn E Djhbc Sjuncle Dy Tquima Turin Italy
Amyeqjv Usogs Szwtozjom Lzmxsl Dk Blycitp Bologna Italy
Uzlkvebuevefxd Cmuuhyt Klovegvqg Gdansk Poland
Aoaflyv Ohaevcqzvwh Plhm Gxorxehh Xsbum Bergamo Italy
Ixvzautn Cnoduq Dmnvdrymqllxfqcgn L'hospitalet De Llobregat Spain
Hhijorcx Vmlj dpdlrcnf Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
21.09.2022
Belgium Belgium
Not recruiting
21.09.2022
Czechia Czechia
Not recruiting
21.09.2022
France France
Not recruiting
21.09.2022
Germany Germany
Not recruiting
21.09.2022
Hungary Hungary
Not recruiting
21.09.2022
Italy Italy
Not recruiting
21.09.2022
Poland Poland
Not recruiting
21.09.2022
Portugal Portugal
Not recruiting
21.09.2022
Spain Spain
Not recruiting
21.09.2022
The Netherlands The Netherlands
Not recruiting
21.09.2022

Trial locations

Investigated drugs:

Rusfertide (PTG-300) is a medication being studied for its ability to help control hematocrit levels in patients with polycythemia vera. Polycythemia vera is a condition where the body produces too many red blood cells, leading to thicker blood and an increased risk of blood clots. Rusfertide acts as a hepcidin mimetic, which means it mimics the action of hepcidin, a natural hormone that helps regulate iron levels in the body. By controlling iron levels, rusfertide may help manage the production of red blood cells and maintain a healthier blood thickness, reducing the risk of complications associated with polycythemia vera.

Investigated diseases:

Polycythemia Vera – Polycythemia Vera is a rare blood disorder characterized by the overproduction of red blood cells in the bone marrow. This excess of red blood cells causes the blood to thicken, which can lead to slower blood flow and an increased risk of clotting. Symptoms may include headaches, dizziness, itchiness, and a ruddy complexion. Over time, the condition can cause complications such as an enlarged spleen and increased risk of blood clots. The disease progresses slowly, and individuals may experience varying degrees of symptoms. Regular monitoring and management are essential to control the condition and prevent complications.

Trial ID:
2023-509750-58-00
Protocol code:
VERIFY
NCT ID:
NCT05210790
Trial Phase:
Therapeutic confirmatory (Phase III)

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