Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

Study Comparing Givinostat and Hydroxyurea for Patients with High-Risk Polycythemia Vera

Fast replyRegistered drug
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

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, which is a type of blood cancer that causes the body to produce too many red blood cells. This can lead to complications such as blood clots. The study is specifically looking at patients who have a mutation known as JAK2V617F-positive and are considered high-risk due to factors like age or previous blood clots. The trial will compare the effectiveness and safety of a medication called Givinostat against another treatment known as Hydroxyurea.

The purpose of the study is to determine which treatment is more effective and safer for patients with high-risk Polycythemia Vera. Participants in the study will receive either Givinostat or Hydroxyurea for a period of 48 weeks. During this time, doctors will monitor the patients to see how well the treatments work in controlling the disease and to check for any side effects. The study will also involve regular check-ups and tests to ensure the safety and well-being of the participants.

Throughout the study, the goal is to see if patients can maintain a healthy level of red blood cells without needing additional procedures like phlebotomy, which is a process to remove blood from the body. The study will also look at other factors such as white blood cell counts, platelet levels, and spleen size, which can be measured using imaging techniques like MRI or CT scans. The results will help determine the best treatment option for managing high-risk Polycythemia Vera.

The research process

The trial runs in 5 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, the patient will be randomly assigned to receive either givinostat or hydroxyurea. This is a phase 3 study aimed at assessing the effectiveness and safety of these treatments in patients with a specific type of blood disorder known as Polycythemia Vera.

  2. Step 2

    Treatment administration

    The patient will receive givinostat in the form of capsules, which are taken orally. The available dosages are 100 mg, 75 mg, and 50 mg. The specific dosage and frequency will be determined by the study protocol.

    Alternatively, if assigned to the hydroxyurea group, the patient will take this medication orally as well. The dosage and frequency will be specified according to the study guidelines.

  3. Step 3

    Treatment duration

    The treatment will continue for a period of 48 weeks. During this time, the patient will be monitored for response to the treatment and any side effects.

  4. Step 4

    Response assessment

    At the end of the 48-week period, the patient's response to the treatment will be evaluated. This includes checking blood counts and spleen size through imaging tests like MRI or CT scans.

    The primary goal is to achieve specific blood count levels and normal spleen size without the need for additional procedures like phlebotomy.

  5. Step 5

    Safety and tolerability evaluation

    Throughout the study, the safety and tolerability of the treatment will be closely monitored. This involves regular check-ups and reporting any side effects experienced during the trial.

Who can join the trial?

14 criteria

  • Patients must be able to provide informed consent and be willing to sign an informed consent form (ICF).
  • Patients must be 18 years of age or older.
  • Patients must have a diagnosis of **Polycythemia Vera (PV)** confirmed according to the 2016 World Health Organization (WHO) criteria no more than 3 years before signing the ICF.
  • Patients must have **JAK2V617F-positive** disease, which means they have a specific genetic change related to their condition.
  • Patients with PV must be at high risk of **thrombosis** (blood clots) at screening, which means they are either 60 years or older or have had a blood clot before.
  • Patients must need treatment at the time of screening.
  • Patients must have a **normalized Hematocrit (HCT)**, which means their HCT level is less than 45% at randomization. HCT is a measure of the proportion of blood that is made up of red blood cells.
  • Patients must have an **ECOG performance status** of 2 or less at screening. This is a scale used to assess how a patient's disease is affecting their daily living abilities, with lower numbers indicating better function.
  • Patients must have a **peripheral blood blast count** of 0% at screening. This means there are no immature blood cells in their blood.
  • Female patients must be either postmenopausal, sterilized, or if they can have children and are sexually active, they must use a highly effective method of contraception.
  • Female patients who can have children must agree to use highly effective contraception during the study and for at least 6 months after the last dose of study treatment if they received hydroxyurea.
  • Male patients must use condoms and ensure that they or their female partner(s) use a highly effective method of contraception during the study and for at least 1 year after the last dose of study treatment if they received hydroxyurea.
  • Male patients must agree not to donate sperm during the study and for at least 1 year following the last study drug administration if they received hydroxyurea.
  • Patients must be willing and able to comply with the requirements of the study.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Givinostat

    is a medication being studied for its effectiveness and safety in treating patients with a condition called polycythemia vera. This condition is characterized by an overproduction of red blood cells. Givinostat works by inhibiting certain enzymes that may play a role in the disease process, potentially helping to control the symptoms and progression of polycythemia vera.

  • Hydroxyurea

    is a medication commonly used to treat polycythemia vera. It helps to reduce the number of red blood cells produced by the body, which can help manage symptoms and reduce the risk of complications associated with the disease. In this study, hydroxyurea is being used as a comparison to evaluate the effectiveness of givinostat.

What is already known about the treatment

  • Givinostat

    Givinostat is administered orally in tablet form. It is currently being studied in clinical trials for its effectiveness in treating high-risk Polycythemia Vera, particularly in patients with the JAK2V617F mutation. The medication works by inhibiting certain enzymes called histone deacetylases, which play a role in regulating gene expression and can affect the growth of abnormal blood cells. Givinostat is classified as a histone deacetylase inhibitor and is being evaluated for its potential to manage symptoms and reduce complications associated with this blood disorder.

  • Hydroxyurea

    Hydroxyurea is taken orally, usually in capsule form. It is a well-established medication used in the treatment of high-risk Polycythemia Vera, among other conditions. Hydroxyurea works by interfering with the DNA synthesis of rapidly dividing cells, which helps to control the overproduction of blood cells. It is classified as an antineoplastic agent and is commonly used to manage symptoms and reduce the risk of complications in patients with this condition.

Investigated diseases

Polycythemia Vera – Polycythemia Vera is a rare blood disorder characterized by the overproduction of red blood cells, which can lead to increased blood viscosity and volume. This condition is often associated with a mutation in the JAK2 gene, specifically JAK2V617F, which contributes to the abnormal growth of blood cells. As the disease progresses, patients may experience symptoms such as headaches, dizziness, and an increased risk of blood clots. Over time, the excessive production of blood cells can also lead to an enlarged spleen and other complications. The disease is considered high-risk when there is a significant likelihood of developing serious complications.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502276-23-00Protocol codeDSC/08/2357/32Estimated enrolment293 patientsSponsorItalfarmaco S.p.A.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).