A trial looking at ruxolitinib before a stem cell transplant for myelofibrosis (MPD-RC114)

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

Cancer type:

Blood cancers




Phase 2

This trial is looking at treatment with a drug called ruxolitinib before a stem cell transplant for people with myelofibrosis.

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.

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.

More about this trial

Doctors may recommend that people with intermediate or high risk myelofibrosis have a stem cell transplant to try to cure their disease.

Ruxolitinib is a cancer growth blocker. It stops signals that cancer cells use to divide and grow.

Doctors are always trying to improve existing treatment. They think that ruxolitinib treatment may be useful for patients before their stem cell transplant. It may improve people’s health and fitness and reduce the side effects of the transplant. Although ruxolitinib can already be used to treat myelofibrosis, it has not been used in this way before.
The aims of this trial are to

  • Find out if having ruxolitinib before a stem cell transplant will improve treatment for myelofibrosis
  • Learn more about the side effects of having ruxolitnib before a stem cell transplant

Who can enter

You may be able to join this trial if all of the following apply.

  • You have either primary or secondary myelofibrosis that is considered to be intermediate or high risk myelofibrosis
  • You have satisfactory blood test results
  • You are going to have a stem cell transplant and there is a donor who stem cells closely march your own. The donor may be related (for example a brother or sister) or unrelated. You are unable to take part in this trial if your donor is your identical twin
  • Your kidneys, liver, heart and lungs are working normally
  • You are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • You are aged between 18 and 70 years
  • You are willing to use reliable contraception during treatment if there is any chance that you or your partner could become pregnant

You cannot join this trial if any of these apply.

  • You have already had treatment with a drug that targets and stops the action of the JAK2 gene (except ruxolitinib)
  • You have had had treatment with ruxolitinib for longer than 6 months. You may be able to take part if you have had less than 6 months of ruxolitinib treatment and your myelofibrosis is stable (your doctor can explain this to you in more detail)
  • You are allergic to the drugs used in this trial or similar drugs
  • You have had any treatment for your myelofibrosis, including any treatment in a trial, in the last 2 weeks
  • You have an infection that needs treatment
  • You have already had a transplant using the bone marrow or stem cells from a donor (allogeneic transplant Open a glossary item) for any blood disorder
  • You are due to have a stem cell transplant using cord blood
  • You are still having side effects from any treatment of your myelofibrosis
  • You have scarring of the liver (cirrhosis)
  • You have had any other cancer in the last 5 years apart from non melanoma skin cancer
  • You are HIV positive or have active hepatitis A, B or C
  • You have any other serious medical condition that the trial team think could affect you taking part
  • You need to have treatment with medication that affects body substances called CYP3A4 enzymes (your doctor can advise you about this)
  • You are pregnant or breastfeeding

Trial design

This is a phase 2 trial. The researchers need 88 people to join across different countries and 20 people to join in the UK.

Everyone has treatment with ruxolitinib tablets. You take these every day for 62 days. The number of tablets you take each day will depend on the amount of platelets circulating in your blood. You have a blood test once or twice a week to measure the number of platelets and the trial doctor will tell you how many tablets to take.
Your dose of ruxolitinib will be reduced gradually during the last few days of treatment and stopped 2 days before you are due to start preparation for your transplant. You have chemotherapy (and sometimes other types of drugs) to destroy your own stem cells Open a glossary item. You then have your transplant using the stem cells of your donor. You have further treatment after your transplant.

You have a transplant as part of your standard care whether you take part in this trial or not. Your doctor will explain what your transplant involves in more detail. You stay in hospital for about a month and will be looked after in a special single room (in isolation) for some of this time.

The researchers will also take a small sample of blood and blood marrow on 3 separate occasions during the first 2 years following your transplant. This is in addition to the samples taken as part of your routine care. Where possible the bone marrow and blood will be taken at the same time as routine tests.

The researcher will carry out various tests on these samples. For example, they will look for biomarkers Open a glossary item to find out why treatment might work for some people and not for others.

The trial team will ask you to fill out a questionnaire before you start treatment, 3 times during ruxolitinib treatment, then a further 7 times spread over 4 years following your transplant. The questionnaire will ask about side effects and how you’ve been feeling.  This is called a quality of life study.

Hospital visits

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

  • Physical examination
  • Heart trace (ECG Open a glossary item)
  • Heart scan (echocardiogram Open a glossary item)
  • CT scan
  • Measurement of your spleen Open a glossary item (the doctor will do this using their hands or an ultrasound scan)
  • Urine test to check that your kidneys are working properly
  • Tests to check that your lungs are healthy (lung function tests Open a glossary item)
  • Blood tests
  • Bone marrow test
  • 2 toenail clippings (or fingernail clippings) to be used for various tests including looking at DNA Open a glossary item
  • Pregnancy test (if appropriate)

You have some of the above tests again before and during your transplant.

During treatment with ruxolitinib you visit hospital at least once every week for 8 weeks. You have blood tests and see a doctor who will check how you are. At some of these appointments the doctor will examine you and check your weight, blood pressure and heart rate.

After your transplant you have appointments with the trial team

  • 3 times over the first 4 months
  • Every 6 months for the next year and a half
  • At 2, 3 and 4 years

You have blood tests and see your doctor who will examine you and ask about any side effects.

You have 4 further bone marrow tests in the 2 years following your transplant.

The trial team see and collect information about you for up to 4 years following your transplant. After that, you continue to be monitored by your consultant as part of your routine care.

Side effects

The most common side effects of ruxolitinib include

  • drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
  • Diarrhoea
  • Feeling or being sick
  • Tiredness (fatigue)
  • A build up of fluid causing swelling in your hands and feet
  • Headache
  • Cough
  • Change to your heart rhythm
  • Pain in your arms and legs

The most common side effects of a stem cell transplant include

  • A drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
  • Feeling or being sick
  • Diarrhoea
  • Sore mouth

The trial team will give you more information about your stem cell transplant and the side effects of the treatment you have before and after your transplant. We have more information about the side effects of stem cell transplants.

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 Adam Mead

Supported by

NIHR Clinical Research Network: Cancer
Oxford University Hospitals NHS Trust 

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

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

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