A Phase I/II Study of the Drug CPI-0610 in Patients with Myelofibrosis


Full Title

A Phase 1/2 Study of CPI-0610, a Small Molecule Inhibitor of BET Proteins: Phase 1 (Dose Escalation of CPI-0610 in Patients with Hematological Malignancies) and Phase 2 (Dose Expansion of CPI- 0610 with and without Ruxolitinib in Patients with Myelofibrosis)


Myelofibrosis (MF) is a bone marrow disorder that disrupts the normal production of blood cells. The purpose of this study is to assess the safety and effectiveness of the investigational drug CPI-0610 given alone or in combination with ruxolitinib (a drug called a JAK inhibitor) in patients with MF.

Laboratory studies have shown that CPI-0610 can shrink or stabilize tumors. CPI-0610 prevents a group of proteins called BET proteins from attaching to certain cancer-causing genes, which may “turn off” the genes and stop them from making new cancer cells.

Patients who have taken a JAK inhibitor in the past will take CPI-0610 alone. Those who are currently taking a JAK inhibitor or who have never taken one will receive treatment with both CPI-0610 and ruxolitinib. Both medications are taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have myelofibrosis.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Raajit Rampal at 212-639-2194.





Disease Status

Newly Diagnosed & Relapsed/Refractory