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

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

10 criteria

  • 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 trial?

5 criteria

  • 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.
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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.

What is already known about the treatment

Rusfertide – Rusfertide is administered through subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials, specifically in a Phase 3 study, to assess its safety and effectiveness in treating polycythemia vera, a condition characterized by an overproduction of red blood cells. The main therapeutic indication for rusfertide is to help maintain control of hematocrit levels, which is the proportion of blood volume occupied by red blood cells. At the molecular level, rusfertide acts as a hepcidin mimetic, which means it mimics the action of hepcidin, a natural hormone that regulates iron levels in the body. This medication falls under the pharmacological classification of hepcidin mimetics.

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-509750-58-00Protocol codeVERIFYEstimated enrolment250 patientsSponsorTakeda Development Center Americas Inc.

sourced from the EU Clinical Trials Register and site verification

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