Active not recruitingPHASE2INTERVENTIONAL

Long-term Study Evaluating the Effect of Givinostat in Patients With Chronic Myeloproliferative Neoplasms

This long-term study is for people in the UK who have specific chronic blood conditions called Myeloproliferative Neoplasms, such as Polycythemia Vera or Myelofibrosis. It aims to understand the long-term safety, how well patients tolerate, and the effectiveness of a medicine called givinostat. Participants will continue taking givinostat, either by itself or with other medicines, at a dose they have handled well before. Regular check-ups, blood tests, and heart tests will help doctors see how patients are doing. The study will continue for several years, possibly until givinostat becomes widely available, to carefully track its effects and ensure ongoing patient benefit.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Italfarmaco

Enrolment target

Start

01 Mar 2013

Estimated completion

01 Dec 2026

Chronic Myeloproliferative Neoplasms

What is this study about?

This research study is looking at a medicine called givinostat to understand how it works over a long time in people who have certain chronic blood conditions. These conditions are part of a group called Myeloproliferative Neoplasms, and they include illnesses like Polycythemia Vera, Essential Thrombocythemia, and Myelofibrosis. The main goal of this study is to see how safe givinostat is, how well people can take it without too many side effects, and if it continues to help their condition.

This study is for patients who have already been taking givinostat in earlier research studies or through a special access program. They will continue with the same treatment plan they've been on, meaning the same dose they've tolerated well, either givinostat on its own or in combination with other medicines. The study is designed to be ongoing, potentially lasting several years, to gather a lot of information on the medicine's long-term effects. This helps doctors and researchers get a full picture of givinostat's benefits and any potential challenges over time.

Regular visits will involve check-ups, blood tests, and heart scans (ECGs). Doctors will carefully watch how you respond to the treatment and adjust the givinostat dose if needed for your safety. This detailed monitoring helps to ensure that the treatment is working effectively and that any side effects are managed promptly. The study is important because it contributes to our understanding of how givinostat can help people with these conditions in the long run.

Key takeaways

This is a long-term study for people already taking givinostat for certain blood conditions.
It aims to check the safety and how well givinostat works over a long period.
You'll have regular visits for check-ups, blood tests, and heart tests.
You must have tolerated givinostat well and shown benefit from it before.
Participation could last several years, but you can leave anytime.
Study contributes to understanding givinostat's long-term effects.

Who may be eligible?

To join this study, you must already be taking givinostat and have found it helpful, meaning you've shown a good response to the treatment. You also need to have tolerated it well without severe problems. This means you would have participated in an earlier research study for these conditions or received givinostat through a special access program.

Participants must be adults, aged 18 or older, and have one of the specific chronic blood conditions (Myeloproliferative Neoplasms). Your general health should be good enough to participate, and you'll need to be able to understand and sign a consent form, agreeing to take part.

There are also reasons why you might not be able to join. For example, if you are pregnant or breastfeeding, or if you have certain heart conditions or other serious health issues that could make taking the medicine unsafe. Your doctor will carefully review all these points with you.

Quick self-check

Are you 18 years old or older?
Do you have Polycythemia Vera, Essential Thrombocythemia, or Myelofibrosis?
Have you previously taken givinostat in another study or special access program?
Did you tolerate givinostat well and find it helpful?
Are you not pregnant or breastfeeding?
Do you not have certain serious heart conditions?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you take part in this study, you will continue with your current givinostat treatment, at the dose you have tolerated best so far. This might be givinostat by itself or with other medicines you were previously taking it with. You will need to attend appointments every three months. At each visit, doctors will perform check-ups, take blood samples for lab tests, and conduct an ECG (a test to check your heart's electrical activity). These regular assessments help the study doctors monitor your safety, how well you are tolerating the treatment, and if it is still benefiting you. The overall duration of your participation could be several years, potentially until givinostat becomes available as a standard medicine. However, you are always free to stop taking part in the study whenever you wish, without giving a reason.

Potential risks and benefits

The potential benefits of continuing in this study include ongoing access to givinostat, which you have previously found helpful for your condition, and careful monitoring of your health by medical professionals. Potential risks are mainly related to possible side effects from givinostat, which will be closely watched at every visit. There's also the chance the treatment might stop working as well over time or that new side effects could develop. You have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

Locations (15)

Charite Research Organization GmbH
Berlin, Germany
Universitaetsklinikum Freiburg, Innere Medizin I - Haematologie und Onkologie
Freiburg im Breisgau, Germany
Azienda Ospedaliero-Universitaria Policlinico Consorziale, Bari
Bari, Italy
Istituto Tumori Giovanni Paolo II IRCCS Ospedale Oncologico di Bari
Bari, Italy
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, Italy
Azienda Ospedaliero-Universitaria Careggi, Florence
Florence, Italy
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico UOS Oncoematologia anziano
Milan, Italy
Azienda Unità Sanitaria Locale - Presidio Ospedaliero "Spirito Santo", Pescara
Pescara, Italy
Fondazione I.R.C.C.S.-Policlinico San Matteo, Pavia
Pavia, Italy
Azienda Ospedaliera "Bianchi-Melacrino-Morelli"
Reggio Calabria, Italy
Ospedale San Bortolo, Vicenza
Vicenza, Italy
Azienda Ospedaliera Universitaria Università degli Studi "Federico II", Naple
Naples, Italy

+3 more sites — see the official record for the full list.

Common questions

What kind of blood conditions is this study for?

This study is for specific chronic blood conditions like Polycythemia Vera, Essential Thrombocythemia, and Myelofibrosis.

Do I need to be taking givinostat already to join?

Yes, this study is only for patients who have already been taking givinostat in earlier studies or through a special program and have found it helpful.

How often will I need to visit the clinic?

You will have a study visit approximately every three months for check-ups and tests.

Can I stop taking part in the study if I change my mind?

Yes, you can choose to leave the study at any point without it affecting your medical care.

Will taking part cost me anything?

The study should not cost you anything directly. All study-related treatments and tests are provided.