A trial of PLX2853 with ruxolitinib for myelofibrosis (PROMise)
Cancer type:
Status:
Phase:
This trial is adding PLX2853 to ruxolitinib. It is for people with myelofibrosis who are already taking ruxolitinib but it isn’t working too well for them.
Cancer Research UK supports this trial.
More about this trial
Ruxolitinib is a 
called a cancer growth blocker. It works by blocking substances that trigger cancer cells to grow and divide. Doctors use it to treat myelofibrosis.
For some people ruxolitinib is good for controlling the symptoms of myelofibrosis and reducing the size of the . For most people it doesn’t work so well. Researchers are looking for other treatments to improve the outcome for people with myelofibrosis.
PLX 2853 is a new targeted drug that is also a cancer growth blocker. It blocks certain activities in the cancer cells that they need to grow and divide. By blocking these activities PLX2853 might help to control or slow the growth of cancer cells.
Researchers think that adding PLX2853 to ruxolitinib might help reduce the size of the spleen and better control myelofibrosis.
To find this out they need to do a clinical trial. Before doing a bigger trial they need to know the best dose of PLX2853 to take with ruxolitinib.
In this trial you continue to take ruxolitinib as usual and you also take PLX2853.
The aims of this trial are to find:
- the best dose of PLX2853 to take with ruxolitinib
- out how well the combination works to reduce the size of the spleen
- out what the side effects are
- out how it affects
quality of life
Who can enter
The following bullet points are a summary of 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 all of the following apply. You:
- have myelofibrosis that is intermediate risk 2 or high risk. Your doctor will know what risk group your myelofibrosis is.
- have had at least 24 weeks of ruxolitinib and your spleen is more than 5cm from the bottom of your chest (ribcage). Your doctor will know this.
- have satisfactory blood test results
- are having the same dose of ruxolitinib for at least 4 weeks before joining the trial
- are willing to use reliable contraception during the trial and for a time after if there is any chance you or your partner could become pregnant
- can look after yourself but can’t work (performance status 0, 1 or 2)
- are at least 16 years old
Who can’t take part
You cannot join this trial if any of these apply. You:
- have ongoing side effects from previous treatment. This is apart from mild side effects and moderate hair loss, hot flushes, low sex drive and nerve pain.
- have had treatment with a drug that works the same way as PLX2853 (a bromodomain inhibitor) such as OTX-015 and CPI-0610. Your doctor will know if you had one of these drugs or similar ones.
- have an active infection of hepatitis A, hepatitis B or hepatitis C or any ongoing infection affecting your whole body that needs treatment
- have a
heart problem such as a heart attack or chest pain that the trial team or your doctor thinks could affect you taking part - have high blood pressure that isn’t controlled by medication
- have a problem with
blood clots such as a deep vein thrombosis (DVT) or a blood clot in the lung within 3 months of joining the trial - are taking medication such as warfarin or heparin to prevent blood clots
- had a
stroke within 3 months of joining the trial - are taking a high dose of vitamin B17 (biotin) or supplements that contain a high dose of vitamin B17. You might be able to join if you lower your dose to the recommended daily dose. Your doctor will know what the recommended daily dose is.
- can’t swallow tablets or capsules
- have a problem with your
digestive system that affects how well your body absorbs tablets or capsules. This includes feeling or being sick if it is a major problem. - are taking part in another clinical trial using an experimental drug
- are pregnant or breastfeeding
Trial design
This is a phase 1 trial. It is taking place in the UK. The team need up to 60 people to take part.
Everyone has ruxolitinib and PLX2853. You continue to have the same dose of ruxolitinib that you were having before joining the trial.
PLX2853 is a tablet you take once a day every day. Your doctor will tell how many tablets to take. You take them on an empty stomach. You should not eat at least 2 hours before taking the tablets and for 1 hour afterwards. You may have a low fat snack after taking the tablets if you are feeling or being sick.
You continue taking ruxolitinib and PLX2853 for 24 weeks as long as it is helping and the side effects aren’t too bad. After this if it is working you might be able to continue having PLX2853. Your doctor will talk to you about this.
After the trial you continue with your ruxolitinib as usual.
Research samples
You give blood samples at the start of the trial, during and at the end. Where possible you have these at the same time as your routine blood tests.
You have a bone marrow test at the start of the trial and then at:
- 6 months
- 1 year and then every year
You don’t have to agree to have the test at 1 year or every year after. This won’t affect you taking part in the rest of the trial.
Quality of life
You fill in a questionnaire when you start treatment and then:
- every 4 weeks while having PLX2853
- then every year
The questions ask about:
- your general health
- what daily activities you can do
- any side effects
This is a quality of life questionnaire.
Hospital visits
You see the doctor to have some tests before you can take part. These include:
- a
physical examination - blood tests
- ultrasound of the spleen
- bone marrow test
During treatment with PLX2853 you:
- see the doctor every 3 weeks for bloods and to see how you are
- have an ultrasound of the spleen every 6 months
- have a bone marrow test at 6 months
After 24 weeks if you are still taking PLX2853 you see the doctor every 12 weeks.
When you stop taking PLX2853 you see the doctor within a month to see how you are. You then see the doctor every year.
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.
The common side effects of PLX2853 include:
- diarrhoea
- feeling or being sick
- tiredness and lack of energy (fatigue)
- loss of appetite and changes to taste
- a drop in blood cells causing tiredness, an increased risk of bruising, bleeding and infection
- tummy (abdominal) pain and swelling
- pain when swallowing
- sore throat, sore mouth and or dry mouth
- a change to how the liver works
- cough
- nose bleeds
- muscle weakness
- skin rash
- sweating a lot
indigestion - high blood pressure
Your doctor or a member of the trial team will talk to you about the side effects of PLX2853 before you agree to take part.
We have information about ruxolitinib.
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 Adam Mead
Supported by
Cancer Research UK
University of Birmingham
Other information
This is Cancer Research UK trial number CRUK/18/008.
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
