
"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This trial is for people with advanced myeloproliferative neoplasms that include thrombocythaemia, polycythaemia vera or myelofibrosis.
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 . It is a cancer growth blocker. It stops signals that cancer cells use to divide and grow. Azacitidine is
.
The aims of the trial are to
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
You cannot join this trial if any of these apply. You
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
Research samples
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,
Observation study
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
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
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
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.
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Dr Mark Drummond
Bloodwise
Celgene
Novartis
University of Birmingham
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”