Title: CMML: A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Ruxolitinib for the treatment of Chronic Myelomonocytic Leukemia (CMML) and Cataloging the Molecular Consequences of JAK2 Inhibition in Chronic Myelomonocytic Leukemia (CMML): A Correlative Study Identifying Targetable CMML Sub-Clones by Leveraging GM-CSF Dependent pSTAT Hypersensitivity
PI: Dr. Gail Roboz
Research Nurse: Tania Curcio, RN
Phone: (212) 746-2571
Sponsor: Moffitt Cancer Center
This clinical trial is for men and women with chronic myelomonocytic leukemia (CMML). The study is evaluating a drug called ruxolitinib. Ruxolitinib has been approved by the Food and Drug Administration (FDA) to treat patients with myelofibrosis, a type of bone marrow cancer.
Chronic Myelomonocytic Leukemia is a cancer that shares features of a myelodyslastic syndrome (MDS), cancer in which the bone marrow does not make enough healthy blood cells, and myeloproliferative neoplasm (MPN), a cancer of blood cell overproduction. In CMML, too many blood cells called myelocytes and monocytes are produced, but they never mature. These immature blood cells are called blasts. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.
The study drug, ruxolitinib, targets the JAK1 and JAK2 pathways. JAK1 and JAK2 are present in CMML. By targeting the JAK1 and JAK2 pathway, ruxolitnib could potentially help control the overproduction of blood cells.
The study has 2 parts (participants will only be enrolled in 1 part).
In Part 1, different groups of participants will receive increasing dose levels of Ruxolitinib to find the dose that has the most effect while still being tolerated by participants. Part 2 will study how the maximum tolerated dose (determined in Part 1) affects the cancer.
Participants will take Ruxolitinib twice daily in 28 day cycles. Participants will continue on study treatment for as long as they are responding to therapy and not experiencing unacceptable side effects, but not past Week 48 of treatment.
- Men and women age 18 and older
- Diagnosis of CMML
- Must be willing to come to clinic for study visits
- Detailed eligibility reviewed when you contact the study team
Disease Status: CMML
Study Status: Open
Expiration: May 3, 2017