
Last year in the UK over 60,000 cancer patients enrolled on clinical trials aimed at improving cancer treatments and making them available to all.
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This trial is looking at ruxolitinib (also known as INC424) and a drug called sonidegib (also known as LDE225) 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) 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 who also have an enlarged .
The trial is looking at a combination of 2 drugs called ruxolitinib and sonidegib. 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.
Sonidegib is a new drug that blocks a specific type of cell signalling called the hedgehog pathway.
The aims of this trial are to
You may be able to enter this trial if
You cannot enter this trial if you
This phase 1/2 trial is in 2 parts and will recruit about 80 people all together. Everybody taking part has ruxolitinib and sonidegib.
In part 1, the researchers are looking for the highest safe doses of ruxolitinib and sonidegib that you can have at the same time for myelofibrosis. The first few patients taking part will have low doses of both drugs. If they don’t have any serious side effects, the next few patients will have higher doses. And so on, until they find the best doses to give. This is called a dose escalation study.
In part 2, the researchers want to see how well the treatment works. Everybody joining this part of the trial has the highest safe doses found in part 1 of the trial.
Ruxolitinib is a tablet that you take twice a day. Sonidegib is a capsule that you take once a day. The trial team will give you a diary to keep at home. Each day you write down what time you eat in the morning and when you take the tablets and capsules.
As long as you don’t have any bad side effects and the treatment is helping, you can carry on taking the trial drugs for at least 2 years. After this time if the drugs are helping then you can take part in an extension study. This means you continue to take both drugs but you see the trial team less frequently.
The trial team will ask you to fill out a questionnaire before you start treatment, a number of times during treatment and after you finish treatment. The questionnaire will ask about side effects and how you’ve been feeling. This is called a quality of life study.
You see the trial team and have some tests before you start the trial treatment. The tests include
You see the trial team 12 times during the first 3 months of treatment. After that, you see them every 4 weeks for up to 2 years. If you continue treatment for longer than 2 years you see the doctors every 3 months. You have blood tests at each visit and an ECG at some visits. You may have more scans or X-rays if your doctor thinks they are necessary.
You have a bone marrow test
When you finish the trial treatment, you will have another hospital visit to have a physical examination, blood tests, a CT or MRI scan and an ECG. And a member of the trial team will contact you by phone a month later to see how you are.
As sonidegib is a new drug, there may be side effects we don’t know about yet. In trials so far, the most common side effects have been
Sonidegib can also affect the way your muscles, kidneys, liver and pancreas work. You will have regular blood tests to monitor for any changes.
The most common side effects of ruxolitinib for people with myelofibrosis include
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Prof Claire Harrison
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
Novartis
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
Last year in the UK over 60,000 cancer patients enrolled on clinical trials aimed at improving cancer treatments and making them available to all.