This study will evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics of the orally administered lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat, in participants with polycythemia vera (PV). At Week 36 of dosing, participants will be assessed for eligibility to receive additional treatment through Week 52. Participants deriving clinical benefit and safely tolerating bomedemstat will qualify for continued treatment at the Investigator's discretion.
With Amendment 3, after all ongoing participants have reached 52 weeks of treatment, eligible participants may transition to a bomedemstat extension study if available. With Amendment 4, all secondary PK and patient reported outcome measures were designated as exploratory.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Oral tablet
BRCR Global ( Site 0120)
Plantation, Florida, United States
Hematology Oncology of the North Shore ( Site 0104)
Skokie, Illinois, United States
University of Michigan Comprehensive Cancer Center ( Site 0008)
Ann Arbor, Michigan, United States
Comprehensive Cancer Centers of Nevada - Peak ( Site 0118)
Las Vegas, Nevada, United States
Duke University Medical Center ( Site 0016)
Durham, North Carolina, United States
Ohio State University Comprehensive Cancer Center ( Site 0103)
Columbus, Ohio, United States
OHSU Knight Cardiovascular Institute Cardiology Clinic - South Waterfront ( Site 0102)
Portland, Oregon, United States
Huntsman Cancer Hospital at the University of Utah ( Site 0119)
Salt Lake City, Utah, United States
Sunshine Coast Hematology and Oncology Clinic (Site 0506)
Sunshine Coast, Queensland, Australia
Monash Medical Centre ( Site 0006)
Clayton, Victoria, Australia
...and 7 more locations
Number of participants with adverse events (AEs)
An AE is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants with one or more AEs will be reported.
Time frame: Up to ~56 weeks
Number of participants who discontinued study intervention due to AEs
An AE is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants who discontinue study intervention due to AEs will be reported.
Time frame: Up to ~52 weeks
Number of participants with sustained change from baseline of hematocrit to <45% without concomitant phlebotomy at Week 36
Hematocrit will be analyzed by taking blood samples from participants at designated time points during the study. The number of participants who have achieved a sustained change from baseline of hematocrit to \<45% without concomitant phlebotomy at Week 36 will be reported.
Time frame: Baseline through Week 36
Duration of reduction of hematocrit to <45% without phlebotomy
Hematocrit will be analyzed by taking blood samples from participants at designated time points during the study. The duration of a reduction in hematocrit to \<45% without phlebotomy up to Week 36 will be reported.
Time frame: Baseline through Week 36
Number of participants with platelet count ≤ 450 x 10^9/L at Week 36
Platelet count will be analyzed by taking blood samples from participants at designated time points during the study. The percentage of participants who have a platelet count ≤450 X 10\^9/L will be reported.
Time frame: Baseline through Week 36
Duration of platelet count ≤ 450 x 10^9/L in participants at Week 36
Platelet count will be analyzed by taking blood samples from participants at designated time points during the study. The duration of the reduction of platelet count of ≤450 X 10\^9/L in participants will be reported.
Time frame: Baseline through Week 36
Number of participants with white blood cell (WBC) count of <10 x 10^9/L at Week 36
WBC count will be analyzed by taking blood samples from participants at designated time points during the study. The percentage of participants who have a WBC count of \<10 X 10\^9/L will be reported.
Time frame: Baseline through Week 36
Duration of white blood cell (WBC) count <10 x 10^9/L in participants at Week 36
WBC count will be analyzed by taking blood samples from participants at designated time points during the study. The duration of the reduction of WBC count \<10 X 10\^9/L in participants will be reported.
Time frame: Baseline through Week 36
Number of participants with thrombotic events
Thrombotic events are defined as: new or recurrent acute myocardial infarction; unstable angina; stroke; transient ischemic attack (TIA); deep venous thrombosis (DVT); pulmonary embolism (PE); thrombotic digital ischemia; other thrombotic events such as peripheral limb ischemia or Budd-Chiari syndrome that are assessed to be due to underlying PV; other vascular occlusive events such as symptoms of cardiac, abdominal or peripheral limb ischemia supported by objective evidence of vessel disease and/or ischemia. The number of participants with thrombotic events will be reported.
Time frame: Baseline through Week 36
Number of participants with major hemorrhagic events
Hemorrhagic events are defined as: Major Bleeding (MB) Events such as fatal bleeding, and/or symptomatic bleeding in a critical area or organ such as intracranial, intraspinal, intraocular, retroperitoneal, intra-articular or pericardial, or intramuscular with compartment syndrome, and/or bleeding causing a fall in hemoglobin level of 2 g/dL or more, or leading to transfusion of 2 or more units of whole blood or red cells; Clinically Relevant Non-Major Bleeding (CRNMB) Events Leading to hospitalization or increased level of care or clinically important, prompting a face-to-face medical evaluation. The number of participants with hemorrhagic events will be reported.
Time frame: Baseline through Week 36
Number of participants with a reduction in splenic volume in patients with an enlarged spleen at baseline
Spleen volume will be measured by magnetic resonance imaging (MRI) (or computerized tomography \[CT\] if participant is not a candidate for MRI) of the abdomen according to standard procedures. The number of participants with a reduction in spleen volume by Week 36 will be reported.
Time frame: Baseline through Week 36
Number of participants with progressive disease (PD)
PD is defined as the worsening of PV to post-polycythemia vera myelofibrosis, myelodysplastic syndrome or transformation to acute myeloid leukemia. The number of participants with PD will be reported.
Time frame: Baseline through Week 36
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