A Phase 3, Randomized, Double-blind, Active-Control Study of Pelabresib (CPI-0610) and Ruxolitinib vs. Placebo and Ruxolitinib in JAKi Treatment Naive MF Patients
- Study HIC#:2000030483
- Last Updated:11/15/2022
A Phase 3, randomized, blinded study comparing pelabresib (CPI-0610) and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients that have not been previously treated with Janus kinase inhibitors (JAKi). Pelabresib is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
- Start Date08/10/2022
- End Date09/01/2022
Trial Purpose and Description
Primary Outcome Measures :
- Splenic response at Week 24 [ Time Frame: 24 weeks of treatment ]The primary endpoint of the study is splenic response, defined as a ≥35% reduction from baseline in spleen volume as measured by magnetic resonance imaging (MRI) or computerized tomography (CT), at Week 24.
Secondary Outcome Measures :
- Total Symptom Score response at Week 24 [ Time Frame: 24 weeks of treatment ]The key secondary endpoint of the study is Total Symptom Score response, defined as a ≥50% decrease from baseline in Total Symptom Score as measured by the Myelofibrosis Symptom Assessment Form v4.0, at Week 24. A higher Total Symptom Score indicates a higher disease burden and thus a worse outcome.
- Aged ≥ 18 years
- Confirmed diagnosis of myelofibrosis (primary, post-polycythemia vera, or post essential thrombocythemia)
- Adequate hematologic, renal, and hepatic function
- Have at least 2 symptoms with an average score ≥ 3 or an average total score of ≥ 10 over the 7-day period prior to randomization using the MFSAF v4.0
- Prognostic risk-factor score of Intermediate-1 or higher per Dynamic International Prognostic Scoring System (DIPSS) scoring system
- Spleen volume of ≥ 450 cm^3
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Splenectomy or splenic irradiation in the previous 6 months
- Chronic or active conditions and/or concomitant medication use that would prohibit treatment
- Had prior treatment with any JAKi or BET inhibitor for treatment of a myeloproliferative neoplasm