Clinical Trial TitleA phase 2 study of INCB050465 in combination with ruxolitinib in subjects with myelofibrosis.
Clinical Trial Protocol Description:
Myelofibrosis (MF) belongs to a group of blood cancers known as myeloproliferative neoplasms in which the bone marrow cells that produce the body’s blood cells develop and function abnormally. The result is excessive fibrous (scar) tissue formation in the bone marrow, which can lead to severe anemia, weakness, fatigue and enlarged spleen and liver. The cancer develops when a mutation occurs in the DNA of a single hematopoietic (blood forming) stem cell. As the mutated blood cell replicates and divides, it passes along the mutation to new cells. As a result, it becomes more difficult for the bone marrow to create normal blood cells and blood cell production may move to the spleen (causing enlargement) or to other areas of the body.
This a phase 2 study using the combination of the PIK3Kd inhibitor, INCB050465, and ruxolitinib (a JAK1 and JAK2 inhibitor), for subjects with primary myelofibrosis or secondary MF, either post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis whose disease response was inadequate with treatment with ruxolitinib alone.
Clinical Trial Eligibility Criteria:
In order to participate you must meet the following criteria:
- Have a diagnosis of primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis.
- Have been treated with ruxolitinib for 6 months or longer with a stable dose for 8 weeks or longer.
- Have evidence of inadequate response to ruxolitinib, such as: a palpable spleen of greater than 10 centimeters below the left edge of the eighth through tenth ribs in the middle of the chest; have a palpable enlarged spleen of 5-10 centimeters below the left edge of the eighth through tenth ribs in the middle of the chest and have active symptoms of MF at screening;
- Are fully active, able to carry on all pre-disease activities without restriction; or restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, such as, light house work, office work; or ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours.
You will be excluded from the study if any of the following criteria apply to you:
- Have received any experimental drug therapy for MF or any other standard drug (such as danazol, hydroxyurea, etc.), with the exception of ruxolitinib within 6 months of starting study (combination) therapy and/or lack of recovery from all side effects from previous therapy (except ruxolitinib).
- Are not able to swallow food or you have any condition of the upper gastrointestinal tract that precludes administration of oral medications.
- Are unwilling to be transfused with blood components.
- Have inadequate liver function at screening and baseline visits.
- Have inadequate renal function at screening and baseline visits.
This is a partial list of inclusion and exclusion criteria.