RecruitingPHASE3INTERVENTIONAL

Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera

This research study is looking at two medicines, givinostat and hydroxyurea, for a blood condition called polycythemia vera (PV). PV causes your body to make too many red blood cells. The study wants to find out if givinostat works better and is safer than hydroxyurea, which is a common treatment. It’s for patients who have a higher chance of developing blood clots because of their PV and have a particular gene change (JAK2V617F). The study is in its third phase, meaning the treatments have already been tested in smaller groups. We want to understand if givinostat can offer a better way to manage PV and its symptoms.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Italfarmaco

Enrolment target

220

Start

26 Mar 2024

Estimated completion

01 Jul 2026

Polycythemia Vera

What is this study about?

Polycythemia vera (PV) is a type of blood disorder where your body makes too many blood cells, especially red blood cells. This can make your blood thicker, increasing the risk of serious problems like blood clots, which can lead to strokes or heart attacks. In some cases, PV can also turn into other more serious blood conditions over time. Many patients with PV have a specific change in a gene called JAK2V617F, which is a key driver of the disease.

Currently, one common treatment for PV is a medicine called hydroxyurea. However, for many patients, hydroxyurea doesn't fully control their symptoms or reduce their long-term risks effectively. Doctors are always looking for better ways to treat PV, especially for patients who are at a higher risk of complications, such as those over 60 years old or those who have had a blood clot before.

This study is comparing hydroxyurea with a new medicine called givinostat. Givinostat belongs to a group of drugs that have shown promise in slowing down the growth of unhealthy cells. Early studies have suggested that givinostat might be helpful for patients with PV caused by the JAK2V617F gene change and seems to have an acceptable safety profile. The main goal of this study is to see if givinostat is more effective than hydroxyurea at managing PV and keeping you healthy.

Key takeaways

This study compares givinostat with hydroxyurea for high-risk polycythemia vera.
It's for people with a specific JAK2 gene change.
The aim is to find out if givinostat is safer and more effective.
Participation involves taking one of the two medicines and regular check-ups.
You'll be closely monitored for both benefits and side effects.
You can stop participating at any time.

Who may be eligible?

To join the main part of this study, you must be an adult aged 18 or over and have been diagnosed with polycythemia vera (PV) caused by the JAK2V617F gene change. You'd be considered at a higher risk of blood clots, which means you're either 60 years old or older, or you've had a blood clot in the past three years. You also need to be currently needing treatment for your PV, for example, if your red blood cell count, white blood cell count, or platelet count is too high.

There are also some reasons why you might *not* be able to join. For example, if you've had certain heart problems recently, have other serious health conditions like issues with your liver or kidneys, or have very high levels of fats in your blood. If you've previously had problems taking hydroxyurea (meaning it didn't work or caused severe side effects), you also wouldn't be able to join.

If you successfully complete the main part of the study, you might then be able to continue into an extended treatment phase. This allows patients who benefited from givinostat, or those who didn't fully respond to hydroxyurea, to keep taking givinostat long-term to gather more information.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with polycythemia vera (PV)?
Does your PV have the JAK2V617F gene change?
Are you considered at 'high risk' for blood clots (e.g., over 60 or had a clot before)?
Are you currently needing treatment for your PV (e.g., high blood counts)?
Have you *not* had severe heart problems or other serious health issues recently?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be given either givinostat or hydroxyurea. Neither you nor your doctor will get to choose which medicine you receive, as this is decided randomly, like flipping a coin. You will be closely monitored throughout the study with regular hospital visits, blood tests, and check-ups to see how the treatment is working and if you experience any side effects. The main part of the treatment will last for 48 weeks (about 11 months).

After this main period, if you meet certain criteria, you might be able to continue receiving givinostat in an 'extended treatment phase'. This extra phase would allow doctors to collect more information on the long-term effects and safety of givinostat. The total duration of your participation, including any extended phase, would be discussed with you, but typically clinical trials involve regular follow-ups for a significant period.

Potential risks and benefits

By taking part in this study, you might benefit from a new treatment that could potentially be more effective than standard care for your polycythemia vera. You'll also receive very close medical attention and monitoring. However, there are potential risks, as any medication can have side effects. Givinostat might have different side effects compared to hydroxyurea, and some side effects could be serious. You will be fully informed about all known side effects before you decide to join. Remember, taking part in any clinical trial is entirely voluntary. You have the right to withdraw from the study at any time without giving a reason, and it will not affect your future medical care.

Locations (90)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Alabama at Birmingham
Verified postcode
Birmingham, United States· Not yet recruiting
Emad Ibrahim, MD, Inc
Verified postcode
Redlands, United States· Recruiting
US Oncology Inc
Verified postcode
Englewood, United States· Recruiting
American Oncology Partners of Maryland, PA
Verified postcode
Bethesda, United States· Recruiting
Icahn School of Medicine at Mount Sinai
Verified postcode
New York, United States· Recruiting
University of North Carolina at Chapel Hill
Verified postcode
Chapel Hill, United States· Recruiting
The Cleveland Clinic Foundation
Verified postcode
Cleveland, United States· Recruiting
Oncology Associates of Oregon, P.C.
Verified postcode
Eugene, United States· Recruiting
MD Anderson Cancer Center
Verified postcode
Houston, United States· Recruiting
Texas Oncology (Webster) - USOR
Verified postcode
Houston, United States· Recruiting
Texas Oncology-Denison Cancer Center
Verified postcode
Sherman, United States· Recruiting
University of Utah - Huntsman Cancer Institute - PPDS
Verified postcode
Salt Lake City, United States· Recruiting

Common questions

What is polycythemia vera (PV)?

It's a rare blood disorder where your body makes too many red blood cells, which can make your blood thicker and increase your risk of blood clots.

What does 'high-risk' PV mean?

It means you have a higher chance of developing serious complications like blood clots. This usually applies if you are over 60 or have had a blood clot before.

What is a 'JAK2V617F mutation'?

This is a specific change in a gene that is often found in people with PV and helps drive the disease.

Is givinostat a new medicine?

Yes, it's an investigational medicine being tested in this study to see if it's a better treatment option for PV than existing ones.

Can I choose which treatment I get?

No, in this type of study, the treatment you receive (either givinostat or hydroxyurea) is chosen randomly by a computer, not by you or your doctor.

How to find out more

Maurizio Caserini

patientadvocacy@italfarmacogroup.com +39 02 6443 1 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.