A trial comparing ruxolitinib with best available treatment for polycythaemia vera (MITHRIDATE)
Cancer type:
Status:
Phase:
This trial is comparing ruxolitinib with the best available treatment for people with high risk polycythaemia vera.
You have high risk 
if you have, or have had, a high count and at least 1 of the following. You:
- are over 60 years old
- have had a blood clot or bleeding problem
- have, or have had, a high count of blood cells called
platelets
More about this trial
The best available treatment for people with polycythaemia vera is hydroxycarbamide or peginterferon alfa 2a. But these treatments can stop working. Or you might have side effects that affect your . In these cases your doctor will try other treatments.
Ruxolitinib is a called a cancer growth blocker. It blocks signals that cancer cells use to divide and grow.
Research shows that ruxolitinib can help people with polycythaemia vera when their initial treatment stops working. Or when their side effects affect their .
In this trial researchers want people to have ruxolitinib as their first treatment. They will compare it with hydroxycarbamide and peginterferon alfa 2a to find out:
- how well ruxolitinib works as the first treatment
- more about the side effects
- how it affects quality of life
Who can enter
The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you.
Who can take part
You may be able to join this trial if you have, or have had, a high white blood cells count and one of the following apply. You:
- are over 60 years old
- have had a blood clot or bleeding problem
- have, or have had, a high count of blood cells called platelets
- high blood pressure or diabetes that needs medication to control it
And all of the following apply. You:
- have had a diagnosis of polycythaemia vera in the past 15 years
- have the JAK-2 gene change (
mutation ) - have had either hydroxycarbamide or interferon alpha for less than 10 years
- have satisfactory blood test results
- can look after yourself but might not be able to work (performance status 0, 1 or 2)
- are willing to use reliable contraception during treatment and for at least 6 months after if there is any chance you or your partner could become pregnant
- are at least 18 years old
- are able to give informed consent
Who can’t take part
You cannot join this trial if any of these apply. You:
- have had both hydroxycarbamide and peginterferon alfa 2a
- stopped treatment because it wasn’t working (
resistant ) or the side effects were very bad - have already had ruxolitinib
- had polycythaemia vera that has changed to myelofibrosis
- had a low count of the blood cells called platelets and, or a type of white blood cell called
neutrophils in the past year - have had heart problems such as a heart attack in the past 6 months, an irregular heartbeat or angina that isn’t controlled by medication
- have an active infection including hepatitis B, hepatitis C, HIV,
autoimmune disease affecting the liver (autoimmune hepatitis) or tuberculosis (TB) - have a skin cancer (either a
melanoma or a non melanoma skin cancer ) that isn't controlled. Your doctor will know if it is controlled or not. - are allergic to any of the treatments or their ingredients
- have an allergy or sensitivity to lactose. You have a hereditary condition called galactose intolerance, total lactase deficiency or glucose-galactose malabsorption
- have any other medical condition or mental health problem that the trial doctor thinks could affect you taking part
- are pregnant or breastfeeding
- aren’t able to give informed consent
Trial design
This is a phase 3 trial. The trial team need 586 people, with 293 people from both France and the UK to join.
It is a randomised trial. Neither you nor your doctor can choose which group you are in. The 2 groups are:
- ruxolitinib
- best available treatment
When you agree to join the trial your doctor talks to you about hydroxycarbamide and interferon alpha. They decide which might work best for you in case you are in the best available treatment group.
Ruxolitinib is a tablet. You take them twice a day with a full glass of water. Your doctor tells you how many to take.
You continue to have ruxolitinib for 3 years. You might be able to take it for longer if it is working and the side effects aren’t too bad.
Hydroxycarbamide are capsules. You take them with a full glass of water. You doctor tells you how many to take and when.
You have peginterferon alfa 2a as an injection under the skin (subcutaneous). You have it in the tummy (abdomen), thigh or top of your arm. A nurse can teach you to give yourself the injection at home. Or they can teach a family member. You must keep interferon alpha in the fridge. Your doctor tells you how much you need.
You continue to have hydroxycarbamide or peginterferon alfa 2a as long as they are working and the side effects aren’t too bad.
You have a diary to write down when you take your medication and how much. You must do this and bring the diary with you to every appointment at the hospital.
Quality of life
Researchers want to know how the treatment affects your quality of life. They ask you to fill in 3 questionnaires:
- every day for a week before starting treatment
- then at week 12
- week 24
- week 36
- a year
- 15 months
- 1 ½ years
- 2 years
- 2 ½ years
- 3 years
The questions ask about:
- your general health
- any symptoms
- any side effects
- your daily activity
This is a quality of life study.
Samples for research
You give samples at the start and throughout the trial. These include:
- blood samples
bone marrow samples - urine samples
- nail clippings
Researchers use these samples to find out more about polycythaemia vera and its treatment. They also look for substances () in the samples that might tell them how well treatment is working.
Hospital visits
You see the doctor to have tests before taking part. These tests include:
- a physical examination
- blood tests
- bone marrow sample if it is necessary. Your doctor will tell you if this is so.
- ultrasound of the
spleen
You see the doctor for blood tests and to see how you are at:
- 2 weeks
- 1 month
- 2 months
- 3 months
- 6 months
- 9 months
- 1 year
You then see the doctor:
- every 3 months for the next 2 years
- every year till the end of the trial
You have an ultrasound of your spleen at year 1. You have a bone marrow sample at year 1 and year 3.
Side effects
The trial team monitor you during treatment and afterwards. Contact your advice line or tell your doctor or nurse if any side effects are bad or not getting better.
When taking ruxolitinib there are certain medications, complementary therapies, foods and drinks you shouldn’t take. Your doctor will tell more about this.
The most common side effects of ruxolitinib can include:
- a drop in blood cells causing breathlessness, tiredness, an increased risk of infection and an increased risk of bruising and bleeding
- headaches and dizziness
- weight gain or loss
- feeling tired and lacking energy (fatigue)
- a change to the way your liver or pancreas works
- constipation
- wind (flatulence)
- an increased risk of getting a
non melanoma skin cancer . Your doctor will check your skin for signs of this. You need to cover up and use suncream to protect your skin when you go outside. - high blood pressure (hypertension)
- high cholesterol and triglycerides (fats) in the blood
Peginterferon alfa 2a can affect the . It may cause inflammation in different parts of the body which can cause serious side effects. They could happen during treatment, or some months after treatment has finished. Rarely, these side effects could be life threatening.
If you have any of these side effects, you should tell the doctor or nurse as soon as possible that you are on, or have been on, an .
We have information about:
- hydroxycarbamide and its side effects
- peginterferon alfa 2a and its side effects
Your trial doctor will talk to you about the possible side effects before you agree to take part in the trial.
Location
Recruitment start:
Recruitment end:
How to join a clinical trial
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Chief Investigator
Professor Claire Harrison
Supported by
Novartis
University of Birmingham
MPN Voice
Institut National du Cancer
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
