The purpose of this study is to evaluate the efficacy and safety of bomedemstat compared with hydroxyurea in cytoreductive therapy naïve essential thrombocythemia (ET) participants for whom cytoreductive therapy is indicated. Its primary objective is to compare bomedemstat to hydroxyurea with respect to durable clinicohematologic response (DCHR). The primary hypothesis is that bomedemstat is superior to hydroxyurea with respect to DCHR.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
300
Durable Clinicohematologic Response (DCHR) Rate
DCHR rate is the percentage of participants with DCHR, defined as a confirmed reduction of platelet count to ≤400 × 10\^9/L, absence of white blood cell (WBC) count elevation to \>10 × 10\^9/L locally assessed to be due to ET, starting by Week 24 and maintained for at least 24 weeks, and the absence of any thrombotic or major hemorrhagic events or disease progression to myelofibrosis (MF), myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) by Week 52.
Time frame: Up to Week 52
Change From Baseline in Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) Individual Fatigue Symptom Item Score
The MFSAF v4.0 is a 7-item participant-reported myelofibrosis symptom assessment which asks respondents to report symptom severity at its worst for each of the 7 items on a 0 (Absent) to 10 (Worst Imaginable) numeric rating scale.
Time frame: Baseline and pre-specified timepoints up to Week 52
Change From Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a Total Fatigue Score
The PROMIS F SF-7a is a 7-item participant-reported assessment that measures both the experience of fatigue and the interference of fatigue on daily activities over the past week. Response options are on a 5-point Likert scale, ranging from 1 = never to 5 = always.
Time frame: Baseline and pre-specified timepoints up to Week 52
Change From Baseline in MFSAF v4.0 Total Symptom Score
The MFSAF v4.0 is a 7-item participant-reported myelofibrosis symptom assessment which asks respondents to report symptom severity at its worst for each of the 7 items on a 0 (Absent) to 10 (Worst Imaginable) numeric rating scale. The change from baseline in MFSAF total score for all symptoms will be presented.
Time frame: Baseline and Week 52
Duration of Hematologic Remission (DOHR)
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Oral capsule placebo
Palo Verde Cancer Specialists ( Site 0052)
Glendale, Arizona, United States
RECRUITINGLos Angeles Cancer Network ( Site 0025)
Glendale, California, United States
RECRUITINGStanford Cancer Center ( Site 0024)
Palo Alto, California, United States
RECRUITINGExempla Lutheran Medical Center ( Site 0014)
Golden, Colorado, United States
RECRUITINGParkview Research Center at Parkview Regional Medical Center ( Site 0006)
Fort Wayne, Indiana, United States
COMPLETEDUniversity of Michigan ( Site 0003)
Ann Arbor, Michigan, United States
RECRUITINGLevine Cancer Institute ( Site 0009)
Charlotte, North Carolina, United States
RECRUITINGDuke University Health System (DUHS) ( Site 0012)
Durham, North Carolina, United States
RECRUITINGWake Forest Baptist Health-Internal Medicine, Section on Hematology & Oncology ( Site 0013)
Winston-Salem, North Carolina, United States
RECRUITINGThe Ohio State University Wexner Medical Center ( Site 0028)
Columbus, Ohio, United States
RECRUITING...and 153 more locations
For participants who demonstrate hematologic remission, DOHR is defined as the time from the first documented evidence of platelet and WBC counts reduction until platelet or WBC counts increase to above acceptable threshold.
Time frame: Up to Week 52
Number of Participants Who Experience Thrombotic Events
Thrombotic events include but are not limited to new or recurrent acute myocardial infarction, unstable angina, stroke, transient ischemic attack, deep venous thrombosis, pulmonary embolism, thrombotic digital ischemia, or other thrombotic events.
Time frame: Up to approximately 52 weeks
Number of Participants Who Experience Major Hemorrhagic Events
Major hemorrhagic events include but are not limited to 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 decrease in hemoglobin level of 2 g/dL or more, or leading to transfusion of 2 or more units of whole blood or red cells.
Time frame: Up to approximately 52 weeks
Disease Progression Rate
Disease progression rate is the percentage of participants with disease progression, defined as the transformation to post-essential thrombocythemia myelofibrosis, myelodysplastic syndrome, or AML as assessed by the adjudication committee.
Time frame: Up to Week 52
Number of Participants Who Experience One or More Adverse Events (AEs)
An AE is 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.
Time frame: Up to approximately 52 weeks
Number of Participants Who Discontinue Study Intervention Due to an AE
An AE is 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.
Time frame: Up to approximately 52 weeks