A trial looking at midostaurin for mast cell leukaemia or systemic mastocytosis

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

Cancer type:

Acute leukaemia
Blood cancers




Phase 2

This trial is looking at a drug called midostaurin (also known as PKC412) for people who have mast cell leukaemia or a condition called systemic mastocytosis.

Mast cell leukaemia is a very rare type of acute leukaemia Open a glossary item. In this type of leukaemia there are too many of a type of white blood cell called ‘mast cells’ in the blood or bone marrow Open a glossary item. Systemic mastocytosis is another condition where there are too many mast cells in different parts of the body which can cause a lot of different symptoms. Some people who have systemic mastocytosis go on to develop mast cell leukaemia. Doctors don’t yet know the best way to treat either of these conditions.

Midostaurin is a new drug that may slow down the growth of mast cells. A number of proteins (enzymes Open a glossary item) are involved when cells divide. One of these enzymes is called kinase C. We know from earlier research that midostaurin can block kinase C and stop it working. If kinase C doesn’t work, the cells won’t be able to divide and the number of mast cells may drop. Midostaurin is one of a group of drugs called kinase inhibitors.

The aims of this trial are to

  • Find out if midostaurin helps people with mast cell leukaemia or systemic mastocytosis
  • Learn more about the side effects
  • Understand more about how midostaurin works in the body

Who can enter

You can enter this trial if you

  • Have mast cell leukaemia or aggressive systemic mastocytosis (the trial doctor can advise you about this)
  • Are well enough to take part in the trial (performance status 0, 1, 2 or 3)
  • Have satisfactory blood test results
  • Are at least 18 years old
  • Are willing to use reliable contraception during the trial and for 3 months after if there is any chance you or your partner could become pregnant

You cannot enter this trial if you

  • Have already had 3 or more different types of treatment for systemic mastocytosis after which the condition has got worse
  • Have had chemotherapy, interferon, another treatment that targets cancer cells, or any other experimental drug in the last month
  • Have already had midostaurin
  • Have had blood growth factors in the last 2 weeks
  • Have had surgery in the last 2 weeks
  • Have certain heart or lung problems (the trial doctors can advise you about this)
  • Have any other serious medical condition including (but not limited to) diabetes or infection that cannot be controlled with medication
  • Are known to be HIV positive or have active hepatitis
  • Are pregnant or breast feeding

Trial design

This is an international study that will recruit about 60 people in a number of different countries. Everybody taking part will have midostaurin. It comes as a capsule. To begin with, you take 4 capsules twice a day. But the trial doctors may change the dose, depending on any side effects you have.

You take the tablets after food in the morning and the evening. You must swallow the tablets whole with water. You cannot chew them. As long as you don't have bad side effects, you can carry on taking midostaurin for as long as it helps you.

The trial team will ask you to fill in a diary for a couple of days before each hospital visit. In this, you write down what time you take the tablets, how many you take and whether or not you are sick.

They will also ask you to fill in some questionnaires throughout your treatment. These will ask about any side effects you have had and about how you have been feeling. This is called a quality of life study.

Hospital visits

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

You will stay in hospital for 3 days when you start treatment, and then you go to hospital

  • Each week during the first month of treatment
  • Every 2 weeks during the second month
  • Then, once a month for up to a year

After a year, you will have hospital appointments every 3 months for as long as you are having midostaurin. You will have regular blood tests, urine tests, ECGs, scans and X-rays throughout your treatment.

When you finish treatment, you will see the trial doctors again within a month. Then a research nurse will contact you by phone every month until the last person joining the trial has finished treatment. After that they will contact you every 3 months.

Side effects

Midostaurin is a new treatment, so there may be side effects we don’t know about yet. Having mast cell leukaemia or mastocytosis puts you at high risk of having an allergic reaction to the drug. Because of this, you have to stay in hospital for a few days when you first start treatment so the doctors can keep a close eye on you.

Other possible side effects of midostaurin include

  • Lung problems
  • Sickness
  • Loss of appetite or indigestion
  • Fatigue
  • Headache
  • Diarrhoea and wind
  • Dehydration Open a glossary item

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

Professor Reilly

Supported by

Experimental Cancer Medicine Centre (ECMC)

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 5010

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

Wendy took part in a new trial studying the possible side effect of hearing loss

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"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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