Clinical trials on Polycythemia vera

Polycythemia vera is a rare blood disorder characterized by an increased production of red blood cells in the bone marrow. This overproduction leads to thickening of the blood, which can cause a range of health issues and complications. The exact cause of polycythemia vera is unknown, but it is linked to a mutation in the JAK2 gene in most patients. The disease progresses slowly and symptoms may include headaches, dizziness, fatigue, and an itching sensation, particularly after a hot bath. As the blood becomes thicker, the risk of clotting and other complications increases.


  • Increased Blood Viscosity: This can lead to impaired blood flow, increasing the risk of blood clots. These clots can result in serious conditions such as stroke or heart attack.
  • Enlarged Spleen (Splenomegaly): The spleen may become enlarged as it works harder to filter the excessive red blood cells, leading to abdominal pain and fullness.
  • Hypertension: Thickened blood can raise blood pressure, stressing the cardiovascular system.
  • Bleeding Problems: Despite the increased number of cells, bleeding and bruising can occur due to abnormalities in blood platelets.
  • Iron Deficiency: Overproduction of red blood cells can deplete the body’s iron stores, causing symptoms of iron deficiency anemia.
  • Gout: Increased cell turnover may lead to elevated uric acid levels, causing painful joint inflammation.
  • Myelofibrosis: In advanced cases, the bone marrow may become scarred, leading to anemia and other blood disorders.
  • Risk of Acute Leukemia: There is a small but significant risk of transformation into acute myeloid leukemia or other blood cancers over time.

Treatment Methods

  • Phlebotomy: Regular removal of blood (similar to blood donation) to decrease the red blood cell mass and reduce blood viscosity.
  • Hydroxyurea: A chemotherapy drug used to suppress the bone marrow’s production of blood cells.
  • Ruxolitinib: A JAK2 inhibitor specifically approved for treating polycythemia vera, especially in patients with an inadequate response to hydroxyurea.
  • Interferon Alfa: Used to stimulate the immune system to counteract the overproduction of blood cells.
  • Aspirin: Low-dose aspirin may be prescribed to reduce the risk of blood clots and improve blood flow.


The prognosis for individuals with polycythemia vera varies based on the severity of the disease and the effectiveness of treatment. With appropriate management, many patients maintain a good quality of life and a near-normal life expectancy. Treatment can significantly reduce the risk of complications such as thrombosis and lessen symptoms. However, untreated polycythemia vera can lead to severe health issues, including life-threatening blood clots and progression to more serious blood disorders like myelofibrosis or acute leukemia. Early diagnosis and ongoing medical care are crucial to managing the disease effectively and improving long-term outcomes.
  • CT-EU-00116351

    Comparing ruxolitinib, hydroxycarbamide, and Interferon as first-line treatments for high-risk Polycythemia Vera

    The study, known as MITHRIDATE, investigates the effectiveness of the drug Ruxolitinib compared to either Hydroxycarbamide or Interferon Alpha for patients with high-risk Polycythemia Vera, a type of blood disorder. This Phase III clinical trial aims to determine which treatment is more effective in managing the disease without leading to additional health complications.

    Patients in the trial are assigned to receive either Ruxolitinib or the best available therapy, chosen from Hydroxycarbamide or Interferon Alpha, as decided by the overseeing doctors. The course of the study involves regular monitoring and assessments to evaluate the impact of these treatments on the disease’s progression and patients’ overall health and quality of life. The study is conducted in a controlled environment to ensure reliable results.

    • Interferon-Alpha
    • Hydroxycarbamide
    • Ruxolitinib
  • A study of sapablursen for patients with Polycythemia Vera requiring frequent blood removal

    This study evaluates the drug sapablursen (also known as ISIS 702843 or IONIS-TMPRSS6-LRx) in patients with polycythemia vera, a condition where the body makes too many red blood cells. The main purpose is to see if sapablursen can reduce the need for phlebotomy—a process of drawing blood to lower blood cell count—and improve patients’ quality of life. The study has four phases: screening, initial treatment, extended treatment, and post-treatment, lasting a total of about 96 weeks. Participants will receive sapablursen through subcutaneous injections. There is no placebo involved in this study.

    • sapablursen
  • Study of SLN124 for treatment of Polycythemia Vera

    This here clinical trial is aimin’ to study a new drug called SLN124 for folks with Polycythemia Vera, which is a type of blood disorder. SLN124 is a special kind of molecule that targets a specific messenger in the body that’s involved in this condition.

    The trial has two parts. First, they’ll be testin’ different doses of SLN124 to find the right amount that’s safe and well-tolerated. Thereafter, they’ll have a bigger group of patients, with some gettin’ the real SLN124 and others gettin’ a placebo. This second part will be double-blind, meanin’ neither the patients nor the doctors know who’s gettin’ the real drug.

    The main things they’ll be lookin’ at are any side effects or safety issues with SLN124, and how well it works at reducin’ the need for phlebotomies, which are those blood removal procedures that Polycythemia Vera patients often need. They’ll be keepin’ a close eye on folks for almost a year to check how the drug is workin’ and make sure it’s safe.

    • SLN124
    • placebo
  • Studying effects of ruxolitinib and panobinostat on myelofibrosis and polycythemia vera

    This is a study to check the long-term safety of two drugs – Ruxolitinib alone or Ruxolitinib combined with Panobinostat. In that trial, patients can take part if they’ve previously been involved in a study by Novartis or Incyte, and the treatment was beneficial. All participants will receive either ruxolitinib monotherapy or ruxolitinib in combination with panobinostat, at the same dose/schedule that they were taking in the parent study. Researchers will look at participant’s general health and well-being. They’ll also note down any side effects or health problems that appear during taking the drugs.

    • panobinostat
    • Ruxolitinib
  • Examining rusfertide treatment for polycythemia vera

    The study being described is a Phase 3 clinical trial to assess the safety and effectiveness of the drug Rusfertide in patients with Polycythemia Vera (PV), a type of blood disorder characterized by an increase in red blood cells. The purpose of the study is to determine whether Rusfertide can help control hematocrit levels and improve symptoms related to PV more effectively than a placebo. The study involves an initial period where patients are given either Rusfertide or a placebo in addition to their standard treatments, followed by an extended phase where all participants receive Rusfertide. The entire study spans several weeks, including follow-up contacts for safety assessments.

    • Rusfertide
  • Examining hydroxyurea treatment resistance in people with Polycythemia Vera

    This clinical trial aims to investigate the factors that predict hydroxyurea (HU) treatment failure in adult patients with polycythemia vera. Polycythemia vera is a blood disorder that affects the body’s red blood cells and can lead to life-threatening complications such as blood clots and strokes. HU is a common treatment for the condition, but some patients may develop resistance to the medication. In this open-label, researchers will evaluate HU resistance in patients with polycythemia vera. The study includes three periods: screening, an observation period for HU resistance, and a follow-up period. Eligible participants will receive HU treatment for up to 15 months. The trial aims to enroll 300 adult patients with polycythemia vera.

    • Hydroxyurea