A trial of pacritinib for myelofibrosis (PERSIST- 1)

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

Cancer type:

Blood cancers




Phase 3

This trial is comparing a drug called pacritinib with other available treatments for myelofibrosis.

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

Doctors can treat myelofibrosis with chemotherapy, biological therapies and a number of other drugs. If you don’t have symptoms, it may be best not to have any treatment, but just to have regular check ups and blood tests. This is called watchful waiting.

Researchers are looking for new ways to treat myelofibrosis. In this trial, they are looking at a drug called pacritinib.

Pacritinib is a type of biological therapy. It is a cancer growth blocker. It stops signals that cancer cells use to divide and grow.

The aim of this study is to see if pacritinib helps people with myelofibrosis more than other available treatments.

Who can enter

You may be able to enter this trial if you

  • Have either primary or secondary myelofibrosis that is considered to be at least intermediate risk and is causing symptoms
  • Have an enlarged spleen Open a glossary item that your doctor can feel when they examine you
  • Don’t need complete care (performance status 0, 1, 2 or 3)
  • Have satisfactory blood test results
  • Are at least 18 years old
  • Are willing to use reliable contraception if there is any chance you or your partner could become pregnant

You cannot enter this trial if you

  • Have had radiotherapy to your spleen in the last 6 months
  • Have had treatment with radioactive phosphorus (32P) in the last year
  • Have already had a drug called a JAK2 inhibitor – the trial doctor can confirm this for you
  • Have had medication that can affect a body protein called CYP3A4 in the last week
  • Have had drugs to encourage the production of red blood cells in the last 4 weeks
  • Have had drugs to encourage the production of blood cells called platelets in the last 2 weeks
  • Have had any other treatment for myelofibrosis in the last 2 weeks, or in the last 4 weeks if it was an experimental drug
  • Have any problems with your digestive system Open a glossary item that could affect how you absorb drugs
  • Have had a stem cell transplant using cells from a donor, or you would be able to have this type of transplant
  • Have had surgery to remove your spleen (a splenectomy) or are going to have this type of operation
  • Have certain bowel problems such as long term problems with diarrhoea or constipation
  • Have had a heart attack in the last 6 months or have certain other heart problems – the trial team can advise you about this
  • Have another serious medical condition or mental health problem that the trial team think could affect you taking part
  • Have had any other cancer in the last 3 years, unless it was a very early stage and has been successfully treated – the trial team can advise you about this
  • Are known to be HIV positive
  • Have hepatitis A, B or C
  • Are pregnant or breastfeeding

Trial design

This phase 3 trial will recruit about 322 people. It is a randomised trial. The people taking part are put into treatment groups by a computer. Neither you nor your doctor will be able to decide which group you are in.

People in one group have pacritinib. People in the other group have the treatment that they and their doctor think is best for them. For some people, the best available treatment may be watchful waiting.

There will be twice as many people in the pacritinib group as in the best available treatment group.

PERSIST- 1 trial diagram

If you are in the pacritinib group, you take 4 capsules every day. If you are in the other group, your treatment will be whatever you and your doctor decide is best for you.

The trial team will give you an electronic device called an eDiary that you use each day to answer questions about any symptoms you have. You start using this 7 to 10 days before you start treatment and carry on until you stop treatment, or until you’ve been having it for at least 6 months.

The trial team will also ask you to fill out a questionnaire

  • Before you start treatment
  • Every 8 weeks for the first 6 months of treatment
  • Every 3 months during the rest of your treatment
  • 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.

As long as you don’t have bad side effects, you can carry on your treatment plan for as long as it is helping you.

If you are in the best available treatment group and your myelofibrosis gets worse, or you have had this treatment for at least 6 months, you may be able to start having pacritinib.

Hospital visits

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

You also have a bone marrow test if you haven’t had this done in the last 6 months.

A member of the trial team will ring you during the first few days of treatment to see how you are.

You go to hospital to see the trial doctor

  • Every week for the first 4 weeks of treatment
  • Once every 4 weeks for the next 5 months
  • Then every 3 months for as long as you carry on having treatment in the trial or your disease doesn’t get worse

You have a blood test each time. If you are in the pacritinib group, you may have some extra blood tests at 4 of your hospital visits. On these occasions, you have to stay at the hospital for up to 6 hours after taking the capsules.

You have a heart scan after 2 weeks and 3 weeks of treatment. If you are in the pacritinib group, you have an extra heart trace on each of these days – 4 hours after you take pacritinib.

Everybody taking part has a CT or MRI scan every 3 months. You have a bone marrow test after having about 6 months of treatment.

When you finish the treatment, you see the trial team and have a physical examination, blood tests and a CT or MRI scan. The trial team will then contact you by phone every 6 months to see how you are. They will do this for up to 3 years.

If you are in the pacritinib group and you stop taking the capsules before your myelofibrosis gets worse, you carry on seeing the trial team regularly until your disease does start to get worse.

Side effects

As pacritinib is a new drug, there may be side effects we don’t know about yet. In other trials, the most common side effects were

If you are in the best available treatment group, your doctor will talk to you about the possible side effects of any treatment you have.

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

Cell Therapeutics Inc (CTI)
National Institute for Health Research Cancer Research Network (NCRN)

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 10767

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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