A trial of ruxolitinib and BKM120 for myelofibrosis

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

Cancer type:

Blood cancers




Phase 1

This trial is looking at 2 drugs called ruxolitinib (pronounced ruck-sol-it-in-ib) and BKM120 to treat 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). 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.

The trial is looking at a combination of 2 drugs called ruxolitinib (also known as INC424) and BKM120. They are both types of biological therapy.

Ruxolitinib is a cancer growth blocker. It stops signals that cancer cells use to divide and grow. Doctors can already use ruxolitinib to treat myelofibrosis.

BKM120 blocks a group of proteins called PI3K. Blocking PI3K proteins may also help to treat myelofibrosis. BKM120 is being looked at in trials for different types of cancer.

The main aims of the trial are to

  • Find the highest safe doses of ruxolitinib and BKM120 that you can have at the same time for myelofibrosis
  • Learn more about the side effects of this drug combination

Who can enter

You may be able to enter this trial if you

  • Have either primary or secondary myelofibrosis that is classed as intermediate or high risk and is causing symptoms
  • Have an enlarged spleen Open a glossary item that your doctor can feel when they examine you
  • Have 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 at least 18 years old
  • Are willing to use reliable contraception during the trial and for up to 6 months afterwards if there is any chance you or your partner could become pregnant

As well as the above, if you’ve already had ruxolitinib or a similar drug (a JAK2 inhibitor), you can only take part if your spleen didn’t reduce in size by at least half or other symptoms didn’t improve. Or if the treatment did help you, the benefit has now been lost, for example you spleen is now getting bigger.

You cannot enter this trial if you

  • Have already had a drug that blocks PI3K or a protein called AKT - your doctor can advise you about this
  • Have already had ruxolitinib or another JAK2 inhibitor and you had bad side effects
  • Could have a stem cell transplant
  • Have had radiotherapy to your spleen in the last year
  • Have had major surgery or a serious injury in the last 2 weeks (or earlier if you have not fully recovered)
  • Have not recovered from the side effects of earlier myelofibrosis treatment unless they are very mild - apart from hair loss, tiredness (fatigue) or sickness
  • Have liver or kidney problems
  • Have had problems with bleeding or take any other 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 taken certain types of medication to prevent fits (seizures) in the last 2 weeks – the trial team can advise you about this
  • 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)
  • Are taking any medication that affects an enzyme called CYP3A4 - your doctor 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 certain heart problems, or take medication that can affect your heart – the trial doctors can advise you about this
  • Have any problem with your digestive system Open a glossary item that could affect how you absorb tablets or capsules
  • Have had any other cancer in the last 3 years, apart from non melanoma skin cancer that was successfully treated or cervical cancer that was cured with surgery
  • Have hepatitis A, B or C
  • Are known to be HIV positive
  • Have had mental health problems in the past, or have moderate to severe depression or severe anxiety – doctors use questionnaires to check this
  • Have problems with alcohol or drug addiction that the trial doctors think could affect you taking part
  • Have any other medical condition that the trial doctors thinks could affect you taking part
  • Are pregnant or breastfeeding

Trial design

This phase 1 trial will recruit more than 60 people. Everybody taking part has both ruxolitinib and BKM120.

You take ruxolitinib tablets twice a day. You take BKM120 capsules once a day. The trial team will explain exactly how and when to take them.

The first patients taking part will have the lowest doses of both drugs. If they don’t have any serious side effects, the next few patients will have a higher dose of one drug and then the next few will have a higher dose of the other. And so on, until they find the best doses of both drugs to give together. This is called a dose escalation study.

When the researchers have found the highest safe doses, the rest of the people joining the trial will have these doses of the drugs.

As long as you don’t have bad side effects, you have treatment for 24 weeks (nearly 6 months). The trial team will then check to see whether the treatment is helping you. If it is, you can have another 24 weeks of treatment.

The trial team will ask you to fill out a questionnaire before you start treatment, every 4 weeks during treatment and when you finish treatment. The questionnaire will ask about side effects and how you’ve been feeling.  This is called a quality of life study. They will also ask you to fill in some questionnaires that ask about your mood and whether you feel depressed.

Hospital visits

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

  • Physical examination
  • Blood tests
  • Urine test
  • Heart trace (ECG Open a glossary item)
  • Heart scan (echocardiogram Open a glossary item or MUGA scan Open a glossary item)
  • Bone marrow test

Depending on when you join the trial, you may also have a CT scan or MRI scan.

You go to hospital at least 9 times in the first 4 weeks of treatment, 4 times in the next 4 weeks and then once every 4 weeks after that.

You have regular blood tests and ECGs. You have another bone marrow test after about 6 months of treatment. Depending on when you join the trial, you may have 2 more CT or MRI scans during treatment.

When you finish treatment, you see the trial team again a month later. You have a physical examination, blood tests and an ECG. You have another bone marrow test if it is more than 6 weeks since you had one. You may also have a CT or MRI scan.

Side effects

Having ruxolitinib and BKM120 together is a new drug combination and there may be side effects we don’t know about yet.

The possible side effects of ruxolitinib include

  • A drop in blood cells causing an increased risk of infection, bleeding or bruising, tiredness and breathlessness
  • Diarrhoea
  • Feeling or being sick
  • Tiredness (fatigue)
  • A build of fluid causing swelling in your hands or feet
  • Change to your heart rhythm
  • Infections in your  lungs or urinary system Open a glossary item
  • Headache
  • Shortness of breath
  • Cough
  • Pain in your arms or legs
  • Changes to the way your liver works
  • High levels of cholesterol in your blood
  • Shingles Open a glossary item
  • Night sweats
  • Weight loss or weight gain
  • Wind (flatulence)

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

If you need to stop taking ruxolitinib, your doctors will reduce the dose gradually if possible. They may give you steroids Open a glossary item to reduce the withdrawal effects.

In other trials, the most common side effects of BKM120 have been

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

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:

Oracle 10694

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.”

Last reviewed:

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