Ongoing Clinical Trials for Polycythemia Vera
Polycythemia vera is a rare blood disorder where the bone marrow produces too many red blood cells, causing the blood to thicken. Currently, 10 clinical trials are actively recruiting or ongoing across Europe, testing various treatment approaches including new medications and combination therapies. These trials are being conducted in multiple countries including Germany, France, Spain, Italy, Poland, and several others.
Clinical trial locations
- Austria
- Belgium
- Bulgaria
- Croatia
- Czechia
- Denmark
- France
- Comparison of ruxolitinib versus hydroxycarbamide or interferon alfa as first treatment for high-risk polycythemia vera patients
- Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera
- Study on Hydroxycarbamide Resistance in Patients with Polycythemia Vera Identified by AI Predictors
- Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera
- Germany
- Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera
- Study of Gandotinib for Patients with Myeloproliferative Neoplasms, Myelofibrosis, Essential Thrombocythemia, or Polycythemia Vera
- Study of SLN124 for Patients with Polycythemia Vera
- Study on Hydroxycarbamide Resistance in Patients with Polycythemia Vera Identified by AI Predictors
- Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies
- Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera
- Hungary
- Ireland
- Italy
- Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera
- Study on the Safety of Bomedemstat for Patients with Myeloproliferative Neoplasms Who Participated in a Previous Bomedemstat Study
- Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies
- Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera
- Netherlands
- Poland
- Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera
- Study of SLN124 for Patients with Polycythemia Vera
- Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies
- Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera
- Study on the Effects of Sapablursen for Patients with Phlebotomy Dependent Polycythemia Vera
- Portugal
- Spain
- Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera
- Study of Gandotinib for Patients with Myeloproliferative Neoplasms, Myelofibrosis, Essential Thrombocythemia, or Polycythemia Vera
- Study of SLN124 for Patients with Polycythemia Vera
- Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera
- Sweden
Comparison of ruxolitinib versus hydroxycarbamide or interferon alfa as first treatment for high-risk polycythemia vera patients
This trial is being conducted in France and compares three different treatment approaches for patients who have been recently diagnosed with high-risk disease. Participants must be at least 18 years old and have a confirmed diagnosis within the last 15 years according to World Health Organization criteria.
Who can join: To be eligible, patients must be classified as high-risk, meaning they have elevated white blood cell counts above 11 x 10⁹/l and either be over 60 years old, have a history of blood clots or major bleeding, have very high platelet counts above 1000 x 10⁹/l, or have high blood pressure or diabetes requiring medication. Patients can continue blood-thinning medications and phlebotomy procedures, and may have received up to one type of cell-reducing therapy for less than 10 years without showing resistance or intolerance.
Who cannot join: The trial excludes patients under 18 years old, those previously treated with ruxolitinib or other JAK inhibitors, and individuals with active bleeding disorders, severe heart problems, significant liver or kidney disease, or uncontrolled infections. Pregnant or breastfeeding women, those with recent cancer history, and patients with unstable medical conditions are also excluded.
Study focus: The trial aims to determine which treatment is most effective at preventing serious complications such as major blood clots, severe bleeding, and disease progression to other blood disorders. Participants will be randomly assigned to receive either ruxolitinib tablets, hydroxycarbamide capsules, or peginterferon alfa-2a injections. The study will monitor patients for up to 96 months with regular check-ups.
Medications being tested: Ruxolitinib is a JAK inhibitor that helps control blood cell production by blocking specific proteins. Hydroxycarbamide slows down blood cell production in the bone marrow. Peginterferon alfa-2a is a protein-based medication that helps regulate blood cell production and supports the immune system.
Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera
This trial is being conducted across 11 European countries including Bulgaria, Poland, Croatia, Netherlands, Austria, Italy, Ireland, France, Hungary, Spain, and Germany. It is designed to compare the effectiveness and safety of two treatments for patients with the JAK2V617F mutation who are at high risk due to age or previous blood clots.
Who can join: Patients must be at least 18 years old with a confirmed diagnosis within the last 3 years according to WHO 2016 criteria. They must have the JAK2V617F-positive genetic change and be at high risk, meaning they are either 60 years or older or have experienced blood clots previously. Patients must need treatment and have normalized hematocrit levels below 45% at randomization, with good performance status and zero peripheral blood blast count.
Study focus: The trial will run for 48 weeks, during which researchers will monitor how well each treatment controls the disease and check for side effects. The main goal is to see if patients can maintain healthy red blood cell levels without needing phlebotomy procedures. The study will also examine white blood cell counts, platelet levels, and spleen size using imaging techniques.
Medications being tested: Givinostat is a histone deacetylase inhibitor being studied for its ability to control blood cell overproduction in patients with the JAK2V617F mutation. Hydroxyurea is a well-established antimetabolite medication that interferes with DNA synthesis in rapidly dividing cells and has been a standard treatment for many years.
Study on the Safety of Bomedemstat for Patients with Myeloproliferative Neoplasms Who Participated in a Previous Bomedemstat Study
This trial is taking place in Italy and is an extension study for patients who previously participated in bomedemstat research. It focuses on monitoring the long-term safety of the medication in patients with various myeloproliferative neoplasms, including essential thrombocythemia and myelofibrosis.
Who can join: Patients must have previously been part of a bomedemstat clinical trial and have been receiving the medication for at least six months with good tolerance and benefit according to their doctor’s assessment. They must be able to start treatment immediately without a break from medication and be capable of swallowing pills and following at-home dosing instructions.
Study focus: The extension study will continue to monitor participants until February 2035 to ensure the treatment remains safe and to observe any potential side effects. For those with essential thrombocythemia, the study will examine how long the treatment continues to work effectively and observe if there is any progression to more serious conditions like myelodysplastic syndrome or acute myeloid leukemia.
Medications being tested: Bomedemstat is an enzyme inhibitor taken orally in hard capsule form. It works by inhibiting an enzyme called LSD1, which plays a role in regulating blood cell production. The medication is currently under investigation specifically for its safety and efficacy in treating myeloproliferative neoplasms.
Study on the Safety and Effectiveness of Peginterferon Alfa-2a and Ruxolitinib for Newly Diagnosed Polycythemia Vera Patients
This trial is being conducted in Denmark and tests a combination therapy approach for newly diagnosed patients. The study evaluates the safety of using two medications together over a 24-month period.
Who can join: Participants must be 18 years or older with a confirmed diagnosis according to WHO 2016 criteria. They must have a negative tuberculosis test from within the last month and show biochemical evidence of active disease, which can include high red blood cell levels, white blood cell counts greater than 10 billion per liter, or platelet counts above 400 billion per liter. Patients may also have hypermetabolic symptoms such as significant weight loss, night sweats, or fever, as well as severe itching, enlarged spleen, or a history of blood clots.
Study focus: The primary objective is to evaluate the safety of combining peginterferon alfa-2a, given as a subcutaneous injection, with ruxolitinib, taken as 5 mg tablets. The treatment will continue for 24 months with regular monitoring at specified intervals including 3, 6, 8, 9, 12, 18, and 24 months to assess safety and effectiveness. The study aims to determine the proportion of patients achieving complete hematological remission.
Medications being tested: Peginterferon alfa-2a is an immunomodulatory agent administered via injection that works by modulating the immune system and inhibiting the proliferation of abnormal blood cells. Ruxolitinib is a Janus kinase inhibitor taken orally that works by inhibiting specific enzymes involved in signaling pathways leading to blood cell overproduction.
Study of Gandotinib for Patients with Myeloproliferative Neoplasms, Myelofibrosis, Essential Thrombocythemia, or Polycythemia Vera
This trial is being conducted in Austria, Spain, and Germany and focuses on testing gandotinib for patients with various myeloproliferative neoplasms. The study includes patients with myelofibrosis, essential thrombocythemia, and the condition under discussion.
Who can join: Patients must have a WHO-confirmed diagnosis and have stopped all previous approved therapies for at least 14 days, though hydroxyurea on a stable dose is permitted. Patients must be reliable, willing to follow study procedures, and agree to use medically approved birth control. Additional requirements include adequate organ function and an ECOG performance status of 0, 1, or 2, indicating good daily functioning ability. Patients must be 18 years or older and have given written informed consent.
Study focus: The study evaluates how effective gandotinib is in treating these blood cancers. Participants will take the medication as an oral capsule once daily. Some participants may receive a placebo for comparison. The study monitors treatment response over time to assess how well the medication manages the conditions and explores its potential side effects.
Medications being tested: Gandotinib is a kinase inhibitor currently under investigation in clinical trials. It is taken once daily and aims to reduce the number of abnormal blood cells and improve overall health in patients with myeloproliferative neoplasms by targeting specific pathways involved in blood cell production.
Study of SLN124 for Patients with Polycythemia Vera
This trial is being conducted in Bulgaria, Poland, Spain, Italy, and Germany. It tests a new treatment called SLN124, which is designed to target specific genetic material in the body and is given as a subcutaneous injection.
Who can join: Male and female patients aged 18 or older who are not receiving cytoreductive therapy must have stopped such treatment for at least 24 weeks and recovered from side effects. Those on stable doses of hydroxyurea, interferon, busulfan, or ruxolitinib for at least 12 weeks may participate. Patients must have had a recent skin examination and have an ECOG score of 0, 1, or 2. They must have had at least 3 phlebotomies in the last 6 months or 5 or more in the last 12 months with documented high blood cell levels. Women of childbearing potential must have negative pregnancy tests and all participants must agree to use effective birth control methods.
Study focus: The study is conducted in two phases. The first phase assesses safety and tolerability of different SLN124 doses. The second phase compares SLN124 with a placebo to determine if it helps maintain normal hematocrit levels without requiring phlebotomies. The trial examines whether SLN124 can help manage the condition by reducing the need for frequent blood removal procedures.
Medications being tested: SLN124 is a therapeutic agent administered through subcutaneous injections. It works by targeting specific genetic pathways to control red blood cell production. The medication is currently under investigation for its effects on blood disorders and is classified as a therapeutic agent targeting genetic pathways.
Study on Hydroxycarbamide Resistance in Patients with Polycythemia Vera Identified by AI Predictors
This trial is being conducted in Germany and France and uses artificial intelligence to identify patients who might be resistant to hydroxycarbamide before they start treatment. The study monitors patients for up to 15 months.
Who can join: Patients must be at least 18 years old with a confirmed diagnosis according to specific medical guidelines and an ECOG score of 2 or less. They must have no previous treatment with blood cell production-reducing drugs and no phlebotomy in the last 14 days. Eligibility for hydroxycarbamide treatment includes being high-risk (age 60 or older and/or history of blood clots) or low-risk with signs of disease worsening or increasing risk of blood clots and bleeding. Female participants of childbearing potential must have a negative pregnancy test within 72 hours before the first treatment dose.
Study focus: The study evaluates how patients respond to hydroxycarbamide, particularly looking at resistance or intolerance. Advanced techniques including artificial intelligence help identify patients more likely to benefit from the medication and those who might need alternative treatments. Researchers observe how many patients develop resistance or intolerance within the first 6 to 9 months and throughout the entire 15-month period.
Medications being tested: Hydroxycarbamide, also known as hydroxyurea, is an antineoplastic agent taken orally in capsule or tablet form. It works by inhibiting an enzyme called ribonucleotide reductase, which is essential for DNA synthesis, thereby slowing down blood cell production and reducing the risk of complications.
Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies
This trial is being conducted in Sweden, Poland, Germany, and Italy. It evaluates the long-term safety of treatments for patients who have previously participated in studies involving ruxolitinib, either alone or in combination with other drugs.
Who can join: Patients must be currently enrolled in a Novartis or Incyte-sponsored study and receiving ruxolitinib alone or combined with panobinostat, siremadlin, or rineterkib. They must have met all requirements of their original study and be benefiting from the treatment as determined by the investigator. Both male and female patients from different age groups, including children, teenagers, and adults, may participate.
Study focus: The open-label, multi-center study gathers long-term safety information on these medications and their combinations. Participants continue their treatment as they have been doing in previous studies while being monitored for frequency and severity of side effects or adverse events. The study assesses participants regularly to determine any clinical benefits from continued treatment.
Medications being tested: Ruxolitinib is a JAK inhibitor administered orally that works by inhibiting Janus kinase enzymes controlling blood cell production. Panobinostat is a histone deacetylase inhibitor taken as a capsule that affects gene expression and cancer cell death. Siremadlin is an MDM2 inhibitor administered orally that promotes cancer cell death by activating the p53 tumor suppressor pathway. Rineterkib is a kinase inhibitor taken orally that interferes with cancer cell growth and survival pathways.
Study on the Effects of Rusfertide (PTG-300) for Patients with Polycythemia Vera
This trial is being conducted across 11 European countries including Spain, Italy, France, Poland, Austria, Belgium, Netherlands, Portugal, Czechia, Germany, and Hungary. It tests a new treatment called rusfertide, given as a subcutaneous injection.
Who can join: Men and women aged 18 or older with a confirmed diagnosis must have needed at least 3 phlebotomies in the last 28 weeks or at least 5 in the last year, with the last procedure within 3 months before joining. Blood tests should show hematocrit less than 45%, white blood cells between 4,000 and 20,000 per microliter, and platelets between 100,000 and 1,000,000 per microliter. Patients on cytoreductive therapy must be on a stable treatment plan, while those treated only with phlebotomy must have stopped cytoreductive therapy 2 to 6 months before the study.
Study focus: The study evaluates how safe and effective rusfertide is in helping patients maintain control over hematocrit levels. Participants receive either rusfertide or a placebo with regular injections and blood monitoring throughout the study period. The main goal is to see if rusfertide can reduce the need for phlebotomies while also examining changes in fatigue levels and overall well-being.
Medications being tested: Rusfertide is a hepcidin mimetic administered through subcutaneous injection. It mimics the action of hepcidin, a natural hormone that regulates iron levels in the body. By controlling iron levels, rusfertide may help manage red blood cell production and maintain healthier blood thickness, reducing the risk of complications.
Study on the Effects of Sapablursen for Patients with Phlebotomy Dependent Polycythemia Vera
This trial is being conducted in Poland and tests a treatment called sapablursen, given as a subcutaneous injection. The study focuses specifically on patients who require frequent phlebotomy procedures to manage their condition.
Who can join: Patients must have a confirmed diagnosis according to modified WHO 2016 criteria and be phlebotomy dependent, meaning they require regular blood removal. Patients do not need to be on cytoreductive therapy, but if they have been on such therapy before, it must have been stopped at least 3 months before the study. If currently on cytoreductive therapy, the dose must have been stable for at least 3 months. Both men and women can participate.
Study focus: The study lasts for 37 weeks, with a focus on the last 20 weeks to assess treatment effectiveness. It aims to see if sapablursen can reduce the number of times patients need phlebotomy. Throughout the study, various health checks are conducted, including blood tests and heart monitoring, to ensure participant safety. The study may include an optional extension period of up to 73 weeks to evaluate long-term effects.
Medications being tested: Sapablursen is an antisense oligonucleotide administered through injections. It is currently in the experimental phase with ongoing research to determine its safety and efficacy. The medication works by binding to RNA molecules and altering gene expression to reduce the need for phlebotomy by targeting specific molecular pathways involved in blood cell production.
Summary
The 10 ongoing clinical trials for polycythemia vera demonstrate diverse research approaches across Europe. Germany leads with the most trials at 6, followed by Poland with 5, and Spain and Italy with 4 each. France hosts 4 trials as well. This concentration reflects strong research infrastructure in these countries.
Several trials focus on comparing established treatments with newer options. Ruxolitinib appears in multiple studies, both as a single agent and in combination therapies, reflecting its important role in current treatment approaches. Traditional treatments like hydroxycarbamide and peginterferon alfa-2a are being evaluated against newer medications such as givinostat, bomedemstat, and rusfertide.
Many trials specifically target high-risk patients or those who have experienced resistance to standard treatments. The studies generally range from 24 weeks to several years, with some extension studies monitoring long-term safety until 2035. A common goal across multiple trials is reducing the need for phlebotomy procedures while maintaining safe blood cell levels.
The variety of mechanisms being tested includes JAK inhibitors, histone deacetylase inhibitors, hepcidin mimetics, and antisense oligonucleotides. This diversity suggests researchers are exploring multiple pathways to improve treatment outcomes. Several trials use advanced approaches such as artificial intelligence to predict treatment response, representing innovation in patient selection and personalized medicine.
