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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06456346
Registration number
NCT06456346
Ethics application status
Date submitted
7/06/2024
Date registered
13/06/2024
Titles & IDs
Public title
Bomedemstat vs Hydroxyurea for Essential Thrombocythemia (MK-3543-007)
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Scientific title
A Phase 3, Randomized, Double-blind, Active-Comparator-Controlled Clinical Study to Evaluate the Efficacy and Safety of Bomedemstat (MK-3543) Versus Hydroxyurea in Cytoreductive Therapy Naïve Essential Thrombocythemia Participants
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Secondary ID [1]
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2023-505232-36
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Secondary ID [2]
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3543-007
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Essential Thrombocythemia
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Bomedemstat
Treatment: Drugs - Hydroxyurea
Treatment: Drugs - Bomedemstat placebo
Treatment: Drugs - Hydroxyurea placebo
Experimental: Bomedemstat - Participants will receive active bomedemstat and hydroxyurea placebo daily for up to approximately 52 weeks. Dosage will be adjusted either up or down within specified time parameters for each participant to the dose that provides sufficient exposure to safely inhibit thrombopoiesis to decrease platelet counts to the target range. Participants who complete treatment at Week 52 will be eligible to continue treatment in the extended treatment phase. Unblinding will occur and placebo discontinued once all participants have completed at least 52 weeks of therapy or otherwise discontinued.
Active comparator: Hydroxyurea - Participants will receive active hydroxyurea and bomedemstat placebo daily for up to 52 weeks. Dosage will be adjusted either up or down within specified time parameters for each participant to the dose that provides sufficient exposure to safely inhibit thrombopoiesis to decrease platelet counts to the target range. Participants who complete treatment at Week 52 will be eligible to continue treatment in the extended treatment phase. Unblinding will occur and placebo discontinued once all participants have completed at least 52 weeks of therapy or otherwise discontinued.
Treatment: Drugs: Bomedemstat
Oral capsule
Treatment: Drugs: Hydroxyurea
Oral capsule
Treatment: Drugs: Bomedemstat placebo
Oral capsule placebo
Treatment: Drugs: Hydroxyurea placebo
Oral capsule placebo
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Durable Clinicohematologic Response (DCHR) Rate
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Assessment method [1]
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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.
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Timepoint [1]
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Up to Week 52
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Secondary outcome [1]
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Change From Baseline in Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) Individual Fatigue Symptom Item Score
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline and pre-specified timepoints up to Week 52
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Secondary outcome [2]
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Change From Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue SF-7a Total Fatigue Score
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline and pre-specified timepoints up to Week 52
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Secondary outcome [3]
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Change From Baseline in MFSAF v4.0 Total Symptom Score
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline and Week 52
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Secondary outcome [4]
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Duration of Hematologic Remission (DOHR)
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Assessment method [4]
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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.
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Timepoint [4]
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Up to Week 52
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Secondary outcome [5]
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Number of Participants Who Experience Thrombotic Events
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Assessment method [5]
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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.
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Timepoint [5]
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Up to approximately 52 weeks
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Secondary outcome [6]
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Number of Participants Who Experience Major Hemorrhagic Events
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Assessment method [6]
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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.
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Timepoint [6]
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Up to approximately 52 weeks
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Secondary outcome [7]
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Disease Progression Rate
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Assessment method [7]
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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.
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Timepoint [7]
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Up to Week 52
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Secondary outcome [8]
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Event Free Survival (EFS)
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Assessment method [8]
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EFS is defined as the time from randomization to the first documented thrombotic or major hemorrhagic event or disease progression as assessed by the adjudication committee, or death due to any cause, whichever occurs first.
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Timepoint [8]
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Up to Week 52
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Secondary outcome [9]
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Number of Participants Who Experience One or More Adverse Events (AEs)
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Assessment method [9]
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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.
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Timepoint [9]
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Up to approximately 52 weeks
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Secondary outcome [10]
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Number of Participants Who Discontinue Study Intervention Due to an AE
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Assessment method [10]
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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.
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Timepoint [10]
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Up to approximately 52 weeks
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Eligibility
Key inclusion criteria
* Diagnosis of Essential Thrombocythemia (ET) based on World Health Organization Criteria for myeloproliferative neoplasms, and in indication for cytoreductive therapy
* Has a bone marrow fibrosis score of Grade 0 or Grade 1, as per a modified version of the European Consensus Criteria for Grading Myelofibrosis
* Has received no prior cytoreductive treatment for their ET
* Human Immunodeficiency Virus (HIV)-infected participants have well controlled HIV on antiretroviral therapy
* Participants who are Hepatitis B surface antigen (HBsAg) positive are eligible if they have received Hepatitis B Virus (HBV) antiviral therapy for at least 4 weeks and have undetectable HBV viral load
* Participants with history of Hepatitis C Virus (HCV) infection are eligible if HCV viral load is undetectable
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* History of any illness/impairment of gastrointestinal function that might interfere with drug absorption
* History of a malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years
* HIV-infected participants with a history of Kaposi's sarcoma and/or Multicentric Castleman's Disease
* Has an active infection requiring systemic therapy
* Has had a major surgery <4 weeks prior to first dose of study intervention or has not recovered from side effects of major surgery >4 weeks prior to first dose
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/07/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
14/05/2029
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Actual
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Sample size
Target
300
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA
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Recruitment hospital [1]
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Royal Adelaide Hospital-Haematology Clinical Trials Unit ( Site 0203) - Adelaide
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment outside Australia
Country [1]
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Israel
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State/province [1]
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Jerusalem
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Country [2]
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Israel
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State/province [2]
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Ramat Gan
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Country [3]
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Japan
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State/province [3]
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Hokkaido
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Country [4]
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Japan
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State/province [4]
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Hyogo
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Country [5]
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Japan
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State/province [5]
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Ishikawa
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Country [6]
0
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Japan
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State/province [6]
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Mie
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Country [7]
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Japan
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State/province [7]
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Osaka
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Country [8]
0
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Japan
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State/province [8]
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Tokyo
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Country [9]
0
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Japan
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State/province [9]
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Yamanashi
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Country [10]
0
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Japan
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State/province [10]
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Fukushima
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Country [11]
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Japan
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State/province [11]
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Miyazaki
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Country [12]
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Japan
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State/province [12]
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Okayama
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Country [13]
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Taiwan
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State/province [13]
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Tainan
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Merck Sharp & Dohme LLC
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT06456346
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Director
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Address
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Merck Sharp & Dohme LLC
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Toll Free Number
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Address
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Country
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Phone
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1-888-577-8839
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: http://engagezone.msd.com/ds_documentation.php
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06456346