A trial of ruxolitinib and sonidegib (LDE225) for myelofibrosis

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 bone marrow. 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 spleen.

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

• Find the highest safe doses of ruxolitinib and sonidegib that you can have at the same time for myelofibrosis

• See if having these 2 drugs together helps people with myelofibrosis

• Learn more about the side effects of this drug combination and what happens to the drugs in your body