A trial of INC424 for myelofibrosis

Cancer type:

Blood cancers




Phase 1

This trial is looking at INC424 for people with myelofibrosis and a low platelet count.

Myelofibrosis (pronounced my-eh-lo-fy-bro-sis) is a rare blood disorder. It is a condition that causes scarring of the bone marrow Open a glossary item. A small number of people with myelofibrosis go on to develop acute myeloid leukaemia.

More about this trial

Myelofibrosis can develop without having had any other condition. This is called primary myelofibrosis (PMF) or chronic idiopathic myelofibrosis. It can also develop in people who have polycythaemia vera or thrombocythaemia. This is called secondary myelofibrosis. This trial is for people with either primary or secondary myelofibrosis who also have a low number of blood cells called platelets. This is described as having a low platelet count.

We know from earlier trials that a drug called INC424 can help people with myelofibrosis. But people who had a low platelet count were not able to take part in these studies. This is because a drop in the number of platelets is a side effect of INC424.

In this trial, researchers will test lower doses of INC424. The aim of the trial is to find a safe dose of INC424 for people who have myelofibrosis and who already have a low platelet count.

Who can enter

You can enter this trial if you

  • Have either primary or secondary myelofibrosis that is considered to be at least intermediate risk and is causing symptoms
  • Have an enlarged spleen Open a glossary item that your doctor can feel when they examine you
  • Have a low platelet count
  • Have other satisfactory blood test results
  • Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • Have finished any other treatment for myelofibrosis at least a week ago
  • Are willing to use a reliable form of contraception during the trial and for 4 weeks afterwards if there is any chance you or your partner could become pregnant
  • Are at least 18 years old

You cannot enter this trial if you

  • Have had a very low platelet count in the last month (the trial doctor can advise you about this)
  • Have had to have platelets in the last 2 weeks
  • Have been having growth factors for blood cells such as EPO or GCSF for a month or more
  • May be able to have a stem cell transplant to treat your myelofibrosis (your doctor can discuss this with you)
  • Have had radiotherapy to your spleen in the last year
  • Have had another experimental drug in the last month, or earlier if there is any chance some of the drug could still be in your body a month later
  • Have had any other cancer in the last 5 years, apart from carcinoma in situ of the cervix or non melanoma skin cancer that was successfully treated and there has been no sign of it coming back in the last 3 years
  • Have had problems with bleeding or blood clotting in the past, or regularly take any medication that can affect how your blood clots (you can take part if you take aspirin, as long as it is a low dose - the trial doctors can advise you about this)
  • Have problems that mean your liver or kidneys are not working very well (the trial doctors can advise you about this)
  • Have an infection that needs treatment (if you are taking antibiotics you can join the trial once you have finished the course)
  • Have hepatitis A, B or C
  • Are known to be HIV positive
  • Have any condition that could affect how you absorb tablets
  • Have a heart condition that is a cause for concern, or any other condition that the trial doctors thinks could affect you taking part in this study
  • Have already had treatment with another drug similar to INC424
  • Take any other medication that blocks an enzyme called CYP3A4 (your doctor can advise you about this)
  • Are pregnant or breastfeeding

Trial design

This international trial aims to recruit 62 people in different countries. Everybody taking part will have INC424 tablets each day. The trial doctor will explain exactly how and when to take them.

The study is in 2 parts. In part 1, the first few people have a low dose of INC424. If they don’t have any bad side effects, the next few people will have a higher dose. And so on, until they find the best dose to give. This is called a ‘dose escalation study’. The researchers want to find a ‘safe starting dose’. This means that people taking part may be able to have their dose of INC424 increased later in the trial.

In part 2, a larger number of people will have the highest safe starting dose that was found in part 1. People in part 2 of the study may also be able to have their dose increased later on.

7 days before starting treatment until you finish, you use a small electronic device to record any symptoms you have. The device is simple to use. You give a score of 1 to 10 for the 7 most common symptoms of myelofibrosis. The trial team will show you how to use the device as well as giving you written instructions. You have to take the device back to the hospital each time you see the trial team. They can then take off (download) the information.

During the first 4 weeks of treatment, you also fill in a diary twice a day. In this, you record if you have taken your tablets.

You fill out a questionnaire before you start treatment, a number of times during the trial, and when you finish treatment. The questionnaire will ask about side effects you have had and how you have been feeling. This is called a quality of life study.

As long as you don't have bad side effects, you take INC424 tablets for 24 weeks. If they are helping, you can then carry on taking them for up to 24 weeks after the last person has joined the trial. At this point, if INC424 is not licensed in the UK, the study will be extended so you can continue to take it. You will be able to discuss your treatment options with your doctor.

Hospital visits

You will see the trial team and have some tests before you start treatment. The tests include

  • Physical examination
  • Blood tests
  • Heart trace (ECG Open a glossary item)

You go to hospital

  • Twice a week for the first 4 weeks of treatment
  • Once a week in the next 4 weeks
  • Once every 4 weeks for the next 4 months
  • Once every 12 weeks for the rest of your treatment - you also need to have blood tests every 6 weeks

Some visits last about 2 hours and you must not eat or drink anything apart from water for at least 8 hours beforehand. At other visits, you just have a blood test, so these are much quicker and you can eat and drink as normal beforehand.

After you finish treatment, you go back and see the trial team about a month later.

Side effects

AS INC424 is still quite a new drug, there may be some side effects we don’t know about yet. The known side effects include

  • A drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
  • Diarrhoea
  • Feeling sick
  • Tiredness (fatigue)
  • Swelling of your hands or feet
  • Headache
  • Cough or shortness of breath
  • Heart murmur
  • Pain in your arms and legs

There may be some risks associated with stopping INC424 suddenly. These include feeling anxious, difficulty sleeping and weakness. Also, symptoms of myelofibrosis may come back.



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.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Claire Harrison

Supported by

Experimental Cancer Medicine Centre (ECMC)

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:


Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Charlie took part in a trial to try new treatments

A picture of Charlie

“I think it’s really important that people keep signing up to these type of trials to push research forward.”

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