"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial of azacitidine and ruxolitinib for blood disorders called myeloproliferative neoplasms (PHAZAR)
This trial is for people with advanced myeloproliferative neoplasms that include thrombocythaemia, polycythaemia vera or myelofibrosis.
More about this trial
Myeloproliferative neoplasms (MPN) are a rare group of disorders of the bone marrow that cause an increase in the number of blood cells. They are also called myeloproliferative disorders.
Researchers are looking for new ways to treat people with these blood disorders. In this trial, they want to see if 2 drugs called azacitidine and ruxolitinib can help. Both drugs are already used on their own to treat this group of people. But researchers want to see how well they work when given together.
Ruxolitinib is a
The aims of the trial are to
- find a safe dose of azactidine and ruxolitinib to have together
- see how well treatment works
- find out more about the side effects
Who can enter
The following bullet points list the entry conditions for this trial. If you are unsure about any of these speak with your doctor or the trial team. They will be able to advise you.
You may be able to join this trial if all of the following apply. You
- Have a myeloproliferative neoplasm that includes essential thrombocythaemia, polycythaemia vera or myelofibrosis and you have a lot of immature cells called blasts in your blood and
bone marrow. This means your blood disorder is in accelerated or blast phase.
- Need treatment for your blood disorder
- Have satisfactory liver and kidney blood test results
- Are well enough to be up and about for at least some of the day, even if you need help looking after yourself (performance status 0, 1, 2 or 3)
- Are at least 16 years old
- Are willing to use reliable contraception during treatment and for 3 months afterwards if there is any chance you or your partner could become pregnant
You cannot join this trial if any of these apply. You
- Have significant heart problems
- Have had growth factors to increase the number of red blood cells in your blood such as
EPOin the month before joining this trial
- Have had growth factors to increase the number of platelets in your blood in the two weeks before joining this trial
- Have taken any medication that blocks an enzyme called CYP3A4 in the 7 days before joining this trial
- Have already had treatment for the accelerated or blast phase of your myeloproliferative disorder, including a stem cell transplant
- Have had a drug called 5-azacitidine in the past
- Can’t have azacitidine or ruxolitinib for any reason
- Have had treatment as part of a clinical trial in the last 2 weeks
- Have a very bad infection
- Are known to have HIV
- Have an active hepatitis A, hepatitis B or hepatitis C infection
- Have any other medical condition or mental health problem that the trial team think could affect you taking part
- Are pregnant or breastfeeding
This is a phase 1 trial. The trial team need about 34 people to take part.
Everyone taking part will have azacitidine and ruxolitinib. The first few people will have a low dose of ruxolitinib. If they don’t have any serious side effects, the next few people will have a higher dose. And so on, until they find the best dose. This is called a dose escalation study.
Everyone has the same dose of azacitidine.
While having treatment you continue to have the same dose of ruxolitinib and azacitidine you started on unless your side effects are bad and your doctor decides to reduce the dose.
You have treatment in cycles. Each cycle lasts 4 weeks.
You have azacitidine as an injection under the skin. You have it for 7 days at the beginning of each treatment cycle. You go to hospital to have your injection daily from Monday to Friday and again on the following Monday and Tuesday. Each visit will take between 1 and 3 hours.
Ruxolitinib is a tablet. You take it twice every day. You start ruxolitinib on the day you start having azacitidine.
As long as the side effects aren’t too bad, you continue having treatment as long as it is helping you.
You have a bone marrow test every 3 months while you are having treatment.
Quality of life
Everybody taking part will be asked to fill out a questionnaire before starting treatment, at set times during the trial and after finishing treatment. The questionnaire will ask about any side effects you have had and about how you have been feeling. This is called a
The researchers want to find out more about the effect of treatment on your MPN. To help them do this, they will ask for the following samples,
- extra blood samples
- a sample of the bone marrow (
biopsy) taken when you have a bone marrow test
- saliva (spit) and toe nail clippings
- a sample of bone marrow if your MPN gets worse.
Not all patients will be suitable to have the treatment in this trial. The trial team will ask 30 of these people who have advanced MPN to take part in a small study. They want to collect important information about people with MPN to better understand these disorders in the future. If you would like to take part the trial team will ask you
- to complete 3 short quality of life questionnaires when you join the study and then 3 and 6 months later.
- to have a sample of the bone marrow when you have your routine bone marrow tests done
- for blood samples and for a sample of spit (saliva) and toe nail clippings
If you don’t want to give these samples or complete the questionnaires, you don’t have to. You can still take part in the observation study.
You see the doctors and have some tests before you start treatment. The tests include
- A physical examination
- Blood tests
- Bone marrow test
- Heart trace (
You go to hospital to have azacitidine. You should not need to stay overnight.
When you stop treatment you see the trial team a month later for a check up. Your doctor will talk to you about further treatment options. The trial team will see how you are getting on about a year after stopping trial treatment.
As azacitidine and ruxolitinib is a new treatment combination, there might be some side effects we don’t know about yet. The trial team will monitor you during the time you have treatment and you will be given a phone number to call them if you are worried about anything.
The most common side effects of azacitidine and ruxolitinib include
- a drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
- feeling or being sick
- diarrhoea or constipation
- joint, muscle, chest or stomach pain
- tiredness (fatigue)
- fevers (temperature)
- loss of appetite
- shortness of breath
- skin rash, itchiness, bruising
- nose bleeds or blood in the urine
- high levels of cholesterol in the blood
- raised levels of liver enzymes in the blood
- high blood pressure
Having azacitidine may cause an injection site reaction including itching, pain, redness, bleeding, bruising or swelling.
You may also be more likely to develop shingles with ruxolitinib and should contact your doctor if you have any symptoms such as pain and spots that look like small blisters.
How to join a clinical trial
Dr Mark Drummond
University of Birmingham