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 approximately 52 weeks
Clinicohematologic Response (CHR) Rate
CHR rate is the percentage of participants with CHR, 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 12 and maintained for at least 12 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 approximately 52 weeks
Hematologic Response (HR) Rate
HR rate is the percentage of participants with HR, HR is defined as the time from the first confirmed hematologic response to first documented evidence of confirmed platelet increase or confirmed WBC increase after latest confirmed hematologic response date.
Time frame: Up to approximately 52 weeks
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. The total score is used in the analysis and is obtained by summing keyed scores of all items. Scores can range from 7 to 35, with higher scores indicating greater fatigue.
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Oral capsule placebo
Palo Verde Cancer Specialists ( Site 0052)
Glendale, Arizona, United States
Los Angeles Cancer Network ( Site 0025)
Glendale, California, United States
Stanford Cancer Center ( Site 0024)
Palo Alto, California, United States
Exempla Lutheran Medical Center ( Site 0014)
Golden, Colorado, United States
Yale University School of Medicine ( Site 0051)
New Haven, Connecticut, United States
Parkview Research Center at Parkview Regional Medical Center ( Site 0006)
Fort Wayne, Indiana, United States
University of Michigan ( Site 0003)
Ann Arbor, Michigan, United States
Optum Care Cancer Center ( Site 0053)
Las Vegas, Nevada, United States
Levine Cancer Institute ( Site 0009)
Charlotte, North Carolina, United States
Duke University Health System (DUHS) ( Site 0012)
Durham, North Carolina, United States
...and 161 more locations
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 pre-specified timepoints up to Week 52
Duration of Durable Clinicohematologic Response (DODCHR)
For participants who demonstrate DCHR, DODCHR is defined as the time from the first documented evidence of confirmed reduction of platelet and WBC count until confirmed increase of platelet and WBC counts to above acceptable threshold, locally assessed to be due to ET after latest confirmed hematologic response date or thrombotic or major hemorrhagic events or disease progression to MF, MDS or AML.
Time frame: Up to approximately 52 weeks
Duration of Clinicohematologic Response (DOCHR)
For participants who demonstrate CHR, duration of clinicohematologic response is defined as the time from the first documented evidence of confirmed reduction of platelet and WBC count until confirmed increase of platelet and WBC counts to above acceptable threshold, locally assessed to be due to ET after latest confirmed hematologic response date or thrombotic or major hemorrhagic events or disease progression to MF, MDS or AML.
Time frame: Up to approximately 52 weeks
Duration of Hematologic Response (DOHR)
For participants who show confirmed hematologic response, DOHR is defined as the time from the first confirmed hematologic response to first documented evidence of confirmed platelet increase or confirmed WBC increase after latest confirmed hematologic response date.
Time frame: Up to approximately 52 weeks
Percentage 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
Percentage 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
Percentage of Participants with Disease Progression to post-essential thrombocythemia myelofibrosis or myelodysplastic syndrome (MDS) /acute myeloid leukemia (AML)
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 approximately 52 weeks
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