A trial of panobinostat and ruxolitinib 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 the drugs ruxolitinib and panobinostat as treatment for 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 essential 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 panobinostat (also known as LBH589). 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.

Panobinostat is a drug that blocks enzymes Open a glossary item that cells need to grow and divide. Researchers are testing it in clinical trials for different types of cancer.

This is the first time that the combination of these 2 drugs has been tested in people. The aims of the trial are to

  • Find the highest safe doses of the drugs that you can have at the same time
  • See what happens to the drugs in your body
  • Learn more about the side effects

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 or abnormal blood test results
  • Your spleen Open a glossary item is enlarged by at least 5cm
  • Your doctor thinks your blood test results are satisfactory
  • You are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • You finished any other treatment for myelofibrosis at least a week ago (or longer if there is any chance some of the drug could still be in your body)
  • You are able to swallow tablets and capsules
  • You are at least 18 years old
  • You are willing to use reliable contraception during the trial and for 3 months afterwards if there is any chance you or your partner could become pregnant

You cannot enter this trial if you

  • Are taking part in another trial
  • Could have a stem cell transplant
  • Are having treatment with growth factors for blood cells such as EPO or GCSF
  • Have had radiotherapy to your spleen in the last year
  • Are known to be very sensitive to either of the drugs in the trial, or had bad side effects when taking ruxolitinib, panobinostat, or similar drugs in the past
  • 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 another medical condition that puts you at an increased risk of bleeding – the trial doctors can advise you about this
  • Have had a heart attack in the last 6 months or have high blood pressure or diabetes that can’t be controlled with medication
  • Have certain other heart problems, or take medication that can affect your heart – the trial doctors can advise you about this
  • Have had an experimental drug for any other medical condition in the last 4 weeks
  • 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 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, prostate cancer that is controlled or any other cancer if it was very early stage (carcinoma in situ Open a glossary item)
  • Have hepatitis A, B or C
  • Are HIV positive
  • Have any other medical condition that the trial doctors thinks could affect you taking part in this study
  • Are pregnant or breastfeeding

Trial design

Everybody taking part in the trial has both ruxolitinib and panobinostat. The researchers want to find the highest safe doses of ruxolitinib and panobinostat that you can have at the same time. They want to learn more about the side effects of this drug combination and what happens to the drugs in your body.

You have 4 week (1 month) cycles of treatment.

You take ruxolitinib tablets twice each day. You take panobinostat capsules on 3 days in the 1st and 3rd week of each treatment cycle. The trial team will give you more information about exactly how and when to take the drugs.

As long as you don’t have bad side effects, you can carry on having the trial treatment for as long as it helps you.

Hospital visits

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

The trial team will also ask you to wear a small device for 24 hours to record your heart beat. This is called a holter monitor Open a glossary item.

You go to hospital 8 times in the 1st cycle of treatment. At some of these visits you have more heart traces. You also have regular blood tests before and after you take the trial drugs. These blood samples help the researchers to learn more about happens to the drugs in your body. This is called pharmacokinetics Open a glossary item. They will also look for substances called biomarkers Open a glossary item that can show what effect the drugs are having on your disease.

You go to hospital once a week during the 2nd and 3rd cycles of treatment. And from then on, you go to hospital once every 4 weeks. You have blood tests at each visit. You have a bone marrow test after the 6th and 12th cycles of treatment. You have an MRI scan or CT scan every 12 weeks.

When you finish treatment, you have a physical examination, a heart trace, blood tests and a urine test. If you stop the treatment within the first year, you have a bone marrow test and an MRI or CT scan if you haven’t had either of these in the previous 6 weeks.

Side effects

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
  • Tiredness (fatigue)
  • A build of fluid causing swelling (oedema) in your hands or feet
  • Change to your heart rhythm
  • Headache
  • Pain in your joints  or in your arms or legs
  • Weight gain
  • Feeling or being sick
  • Cough
  • Breathing difficulties
  • Infections in your airways or lungs
  • High temperature (fever)
  • Tummy (abdominal) pain
  • Confusion
  • Problems sleeping

As panobinostat is a new drug and this is the first time it is being tested alongside ruxolitinib, there may be side effects we don’t know about yet. The most common side effects that are known include

  • A drop in the number of platelets causing bleeding problems
  • Feeling or being sick
  • Diarrhoea
  • Loss of appetite
  • Tiredness (fatigue)

The trial team will talk to you about all the possible side effects before you agree to join the trial.

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

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Last review date

CRUK internal database number:

Oracle 10689

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

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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