A Phase 2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Myelofibrosis

Official 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 and Essential Thrombocytopenia)

Summary:

Phase 1 Part (Complete): Open-label, sequential dose escalation study of pelabresib in patients with previously treated Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasms, and Myelofibrosis. Phase 2 Part: Open-label study of CPI-0610 with and without Ruxolitinib in patients with Myelofibrosis. CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.

Trial Description

Primary Outcome:

• Phase 2 (Cohorts 1B and 2B and Arm 3): Evaluate the spleenic response
• Phase 2 (Cohorts 1A and 2A): Evaluate the RBC (Red Blood Cell) transfusion independence rate
• Phase 2 (Arm 4): Evaluate the complete hematological response rate

• Phase 2 (Arms 1, 2, and 3): Evaluate the duration of the spleenic response by imaging
• Phase 2 (all arms): Evaluate the change in patient reported outcomes
• Phase 2 (all arms): area under the curve (AUC)
• Phase 2 (all arms): maximum observed plasma concentration (Cmax)

View this trial on ClinicalTrials.gov