A trial looking at INC424 for myelofibrosis

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

Cancer type:

Blood cancers

Myelofibrosis

Status:

Closed

Phase:

Phase 2

This is a trial to learn more about a drug called INC424 for myelofibrosis.

Myelofibrosis (pronounced my-eh-lo-fy-bro-sis) is a rare blood disorder 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.

We know from earlier trials that a type of biological therapy called INC424 can help people with myelofibrosis. But researchers want to learn more about how well it works and how it affects peoples’ quality of life Open a glossary item .

The aims of the trial are to

See how 2 different doses of INC424 help people with myelofibrosis
Learn more about the side effects

Who can enter

You may be able to enter this trial if you

Have myelofibrosis that is considered to be high or intermediate risk
Have recovered from the side effects of any other treatment
Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
Have satisfactory blood test results
Are willing to use a reliable form of contraception during the trial and for 4 weeks afterwards if there is any chance you could become pregnant
Are at least 18 years old

You cannot enter this trial if you

Are already taking part in trials called COMFORT-I or COMFORT-II, which are also looking at INC424
Would be able to take part in any other current trial of INC424
Could have a stem cell transplant using someone else’s cells and you have a suitable donor
Have had growth factors for blood cells such as EPO or GCSF in the last 2 weeks
Are having any other treatment for myelofibrosis
Are taking aspirin (unless it is a low dose) or steroids
Are taking any medication that affects an enzyme called CYP3A4 (your doctor can advise you about this)
Have had any type of cancer in the last 3 years apart from non melanoma skin cancer
Have ever had a very low platelet count, unless this was a side effect of treatment
Have had an experimental drug as part of another clinical trial in the last month
Have any condition that could affect how you absorb tablets
Have an infection that needs treatment (if you are taking antibiotics you can join the trial once you have finished the course)
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 hepatitis A, B or C
Are known to be HIV positive
Are known to be very sensitive to INC424 or similar drugs
Are pregnant or breastfeeding

Trial design

This phase 2 trial will recruit about 33 people in the UK. Everybody taking part will have INC424 tablets.

The researchers are looking at 2 different doses of INC424. The dose you have will depend on your platelet count. During the trial, the doctors may increase or reduce your dose, depending on how well the treatment is working and whether you have side effects.

You take INC424 tablets every day. The trial team will explain more about how and when to take your tablets. As long as you don’t have bad side effects, you can carry on taking them for as long as they help you, or until the last person joining the study has had nearly a year of treatment, whichever comes first.

The trial team will ask you to fill out a questionnaire before you start treatment and then at each hospital visit during your treatment. The questionnaires 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 team and have some tests before you start treatment. The tests include

Physical examination
Blood tests
Heart trace (ECG )

You go to hospital

Every 4 weeks for the first 3 months of treatment
Once every 3 months for the rest of your treatment

Each visit lasts 2 or 3 hours and you must not eat or drink anything (apart from water) for 8 hours beforehand. You have blood tests each time.

After you finish treatment, you go back and see the trial team 4 weeks later.

Side effects

As INC424 is quite a new drug, there may be 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
Tiredness (fatigue)
Diarrhoea
Headache

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

If you need to stop taking INC424, the dose will be reduced gradually if possible. The doctors may give you steroids to reduce the withdrawal effects.