
"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 looking at the drugs ruxolitinib and panobinostat as treatment for myelofibrosis.
Myelofibrosis (pronounced my-eh-lo-fy-bro-sis) is a rare blood disorder. It is a condition that causes scarring of the . 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 essential thrombocythaemia. This is called secondary myelofibrosis. This trial is for people with either primary or secondary myelofibrosis.
The trial is looking at a combination of 2 drugs called ruxolitinib (also known as INC424) and panobinostat (also known as LBH589). They are both types of biological therapy.
Ruxolitinib is a cancer growth blocker. It stops signals that cancer cells use to divide and grow. Doctors can already use ruxolitinib to treat myelofibrosis.
Panobinostat is a drug that blocks that cells need to grow and divide. Researchers are testing it in clinical trials for different types of cancer.
This is the first time that the combination of these 2 drugs has been tested in people. The aims of the trial are to
You may be able to enter this trial if
You cannot enter this trial if you
Everybody taking part in the trial has both ruxolitinib and panobinostat. The researchers want to find the highest safe doses of ruxolitinib and panobinostat that you can have at the same time. They want to learn more about the side effects of this drug combination and what happens to the drugs in your body.
You have 4 week (1 month) cycles of treatment.
You take ruxolitinib tablets twice each day. You take panobinostat capsules on 3 days in the 1st and 3rd week of each treatment cycle. The trial team will give you more information about exactly how and when to take the drugs.
As long as you don’t have bad side effects, you can carry on having the trial treatment for as long as it helps you.
You see the trial team and have some tests before you start treatment. The tests include
The trial team will also ask you to wear a small device for 24 hours to record your heart beat. This is called a .
You go to hospital 8 times in the 1st cycle of treatment. At some of these visits you have more heart traces. You also have regular blood tests before and after you take the trial drugs. These blood samples help the researchers to learn more about happens to the drugs in your body. This is called . They will also look for substances called
that can show what effect the drugs are having on your disease.
You go to hospital once a week during the 2nd and 3rd cycles of treatment. And from then on, you go to hospital once every 4 weeks. You have blood tests at each visit. You have a bone marrow test after the 6th and 12th cycles of treatment. You have an MRI scan or CT scan every 12 weeks.
When you finish treatment, you have a physical examination, a heart trace, blood tests and a urine test. If you stop the treatment within the first year, you have a bone marrow test and an MRI or CT scan if you haven’t had either of these in the previous 6 weeks.
The possible side effects of ruxolitinib include
As panobinostat is a new drug and this is the first time it is being tested alongside ruxolitinib, there may be side effects we don’t know about yet. The most common side effects that are known include
The trial team will talk to you about all the possible side effects before you agree to join the trial.
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Dr Claire Harrison
Novartis
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.”