A trial of givinostat for polycythaemia vera

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Polycythaemia

Status:

Closed

Phase:

Phase 1/2

This trial is looking at a drug called givinostat to treat a blood disorder called polycythaemia vera.

Polycythaemia vera is a myeloproliferative disorder Open a glossary item . This means a condition in which the bone marrow produces too many blood cells. In polycythaemia, this is too many red blood cells.

More about this trial

Givinostat is a drug that blocks proteins (enzymes Open a glossary item ) in the body called histone deacetylases (pronounced dee-as-et-isle-azes). Cells need these to grow and divide. Blocking them may stop cancer growing. Drugs that block these enzymes are called histone deacetylase inhibitors or HDAC inhibitors.

We know from research that givinostat can help people with myeloproliferative disorders such as polycythaemia.

The aims of this trial are to find out

The best dose of givinostat to treat polycythaemia
How well givinostat works for people with polycythaemia
What the side effects are

Who can enter

This trial is in 2 parts. You can join both parts of the trial if all of following apply.

You have polycythaemia vera
You have a change in a gene (mutation ) called the JAK2 gene, which is a cause of polycythaemia
Your blood test results for polycythaemia show that it is not under control (your doctor can tell you this)
Your other blood test results are satisfactory
You are willing to use reliable contraception for a month before starting treatment, during treatment and for 3 months afterwards
You are at least 18 years old

To join part 1 of the trial you must also be well enough to carry out all your normal activities, apart from heavy physical work (performance status of 0 or 1) .

To join part 2 of the trial you must also be well enough to be up and about for at least half the day (performance status 0, 1 or 2).

You cannot join this trial if any of these apply. You

Have already had treatment for your polycythaemia apart from aspirin or having blood taken
Have had a drug that works in a similar to givinostat (your doctor can tell you about this)
Are allergic to givinostat or its ingredients
Have certain heart problems (the trial team can advise you about this)
Have an infection that needs treatment
Have had another cancer
Have another medical condition that the trial team think could affect you taking part
Have had an experimental drug or used a device as part of another clinical trial in the past month
Are pregnant or breastfeeding

Trial design

This trial is in 2 parts. The trial team need 24 people to join the 1st part and 28 people to join the 2nd part. Everyone will have givinostat.

In the 1st part of the trial, the first few people taking part will have a low dose of givinostat. If they don’t have any serious side effects, the next few patients will have a higher dose. And so on, until they find the best dose to give. This is called a dose escalation study.

In the 2nd part of the trial, the researchers want to find out how well givinostat works for people with polycythaemia. You have the dose that was found to be best in the first part.

Givinostat is a capsule. Your doctor will tell you how many to take and how often. You can continue taking givinostat for 6 months as part of the trial.

After 6 months, you may be able to continue taking givinostat if your doctor feels it is helping you and the side effects aren’t too bad. Your doctor will talk to you about this.

The trial team will ask you to fill out a questionnaire before you start treatment, then after 3 months and 6 months. The questionnaire will ask about side effects and how you’ve been feeling. This is called a quality of life study.

In both parts of the trial, the researchers will ask for extra blood samples. They will use these to find out what happens to givinostat in your body and how it works.

If you are in the 2nd part of the trial, the researchers will ask for a sample of your bone marrow before you start treatment and after 6 months. You don’t have to agree to this. You can still take part in the trial.

Hospital visits

You see the doctor to have some tests before taking part. These tests include

A physical examination
Blood tests
Urine test
Heart trace (ECG )
CT scan or MRI scan

During treatment you see the doctor every 4 weeks for the same tests. You have another CT scan or MRI scan at 6 months.

At the end of the trial your doctor will tell you how often they want to see you.

Side effects

The most common side effects of givinostat include

Tummy (abdominal) pain
Feeling of fullness in the tummy (bloating)
Constipation or diarrhoea
Indigestion
Feeling or being sick
Loss of appetite
Not enough fluid in your body (dehydration )
Changes to the way your heart works
A drop in the blood cells that help the blood to clot causing an increased risk of bruising and bleeding
Feeling of burning when passing urine
Headache