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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06079879
Registration number
NCT06079879
Ethics application status
Date submitted
6/10/2023
Date registered
12/10/2023
Titles & IDs
Public title
A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)
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Scientific title
A Phase 3, Randomized, Open-label, Active-Comparator-Controlled Clinical Study to Evaluate the Safety and Efficacy of Bomedemstat (MK-3543/IMG-7289) Versus Best Available Therapy (BAT) in Participants With Essential Thrombocythemia Who Have an Inadequate Response to or Are Intolerant of Hydroxyurea
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Secondary ID [1]
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2023-504865-21
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Secondary ID [2]
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3543-006
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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 - Anagrelide
Treatment: Drugs - Busulfan
Treatment: Drugs - Interferon alfa/pegylated interferon alfa
Treatment: Drugs - Ruxolitinib
Experimental: Bomedemstat - Participants will begin treatment at a dose of 50 mg of bomedemstat daily. 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. All participants will be treated daily for up to 52 weeks, and are eligible for an extended treatment phase up to 152 weeks.
Active comparator: Best Available Therapy - Each participant will receive either anagrelide, busulfan, interferon alfa/pegylated interferon alfa, or ruxolitinib as determined by investigator. All participants will be treated per respective approved product labels for up to 52 weeks. Participants receiving BAT for 52 weeks who stop responding to BAT are eligible to switch to bomedemstat and receive this for up to 152 weeks at the investigators discretion.
Treatment: Drugs: Bomedemstat
Oral Capsule
Treatment: Drugs: Anagrelide
Oral Capsule
Treatment: Drugs: Busulfan
Oral Tablet
Treatment: Drugs: Interferon alfa/pegylated interferon alfa
Subcutaneous Solution
Treatment: Drugs: Ruxolitinib
Oral Tablet
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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, 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 approximately 52 weeks
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Secondary outcome [1]
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Change From Baseline in Myelofibrosis Symptom Assessment Form (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. For this outcome measure, the change from baseline through Week 156 in scores for the individual item of fatigue severity will be presented.
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Timepoint [1]
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Baseline and pre-specified timepoints through Week 156
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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. Change from baseline in PROMIS Fatigue SF-7a through Week 156 will be presented.
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Timepoint [2]
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Baseline and pre-specified timepoints through Week 156
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Secondary outcome [3]
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Change From Baseline in Total Symptom Score as Measured on the MFSAF v4.0
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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. MFSAF total score for all symptoms at baseline through Week 156 will be presented.
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Timepoint [3]
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Baseline and pre-specified timepoints through Week 156
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Secondary outcome [4]
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Duration of Clinicohematologic Response (DOCHR)
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Assessment method [4]
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For participants who demonstrate DCHR, 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, thrombotic or major hemorrhagic events or disease progression to MF, MDS or AML.
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Timepoint [4]
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Up to approximately 52 weeks
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Secondary outcome [5]
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Duration of Hematologic Remission (DOHR)
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Assessment method [5]
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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 [5]
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Up to approximately 52 weeks
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Secondary outcome [6]
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Percentage of Participants with Thrombotic Events
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Assessment method [6]
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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 [6]
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Up to 156 weeks
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Secondary outcome [7]
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Percentage of Participants with Major Hemorrhagic Events
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Assessment method [7]
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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 [7]
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Up to 156 weeks
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Secondary outcome [8]
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Disease Progression Rate
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Assessment method [8]
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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 acute myeloid leukemia as assessed by the adjudication committee. The disease progression rate of participants in each arm will be presented.
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Timepoint [8]
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Up to approximately 52 weeks
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Secondary outcome [9]
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Event Free Survival (EFS)
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Assessment method [9]
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Event free survival (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 [9]
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Up to approximately 52 weeks
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Secondary outcome [10]
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Number of Participants with An Adverse Event (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 180 weeks
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Secondary outcome [11]
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Number of Participants Discontinuing From Study Therapy Due to an AE
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Assessment method [11]
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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. The number of participants who discontinue study treatment due to an adverse event will be presented.
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Timepoint [11]
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Up to 152 weeks
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Eligibility
Key inclusion criteria
* Has a diagnosis of ET per WHO 2016 diagnostic criteria for myeloproliferative neoplasms
* 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 a history of inadequate response to or intolerance of hydroxyurea based on modified European LeukemiaNet (ELN) criteria for hydroxyurea resistance or intolerance: hydroxyurea resistance (or inadequate response) or hydroxyurea Intolerance
* Has an inadequate or loss of response to their most recent prior ET therapy, requiring a change of cytoreductive therapy
* Has a platelet count > 450 × 10^9/L (450k /µL) assessed up to 72 hours before first dose of study intervention
* Has an absolute neutrophil count (ANC) =0.75 × 10^9/L assessed up to 72 hours before first dose of study intervention
* Participants may have received up to 3 prior lines of therapy including hydroxyurea
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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
* Known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to bomedemstat or lysine demethylase or monoamine oxidase inhibitor (LSDi or MAOi) or the chosen best available therapy (including anagrelide, interferon alfa/pegylated interferon, ruxolitinib, or busulfan) that contraindicates participation
* History of any illness/impairment of GI function that might interfere with drug absorption (eg, chronic diarrhea or history of gastric bypass surgical procedure), confound the study results or pose an additional risk to the individual by participation in the study
* Evidence at the time of Screening of increased risk of bleeding
* History of a malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years. Note: The time requirement does not apply to participants who underwent successful definitive resection of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ, excluding carcinoma in situ of the bladder
* Human immunodeficiency virus (HIV)-infected participants with a history of Kaposi's sarcoma and/or Multicentric Castleman's Disease
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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 assessing the outcomes
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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
31/12/2023
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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
18/08/2028
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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)
NSW,SA,VIC,WA
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Recruitment hospital [1]
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Royal Prince Alfred Hospital ( Site 1100) - Camperdown
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Royal North Shore Hospital ( Site 0003) - St Leonards
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Recruitment hospital [3]
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Calvary Mater Newcastle ( Site 0505) - Waratah
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Recruitment hospital [4]
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Royal Adelaide Hospital-Haematology Clinical Trials Unit ( Site 0001) - Adelaide
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Monash Health-Haematology Research ( Site 0006) - Clayton
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Recruitment hospital [6]
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Western Health-Sunshine & Footscray Hospitals-Cancer Services-Cancer Research ( Site 0502) - Melbourne
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Recruitment hospital [7]
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Royal Perth Hospital-Haematology ( Site 0504) - Perth
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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2065 - St Leonards
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Recruitment postcode(s) [3]
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2298 - Waratah
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Recruitment postcode(s) [4]
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5000 - Adelaide
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Recruitment postcode(s) [5]
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3168 - Clayton
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Recruitment postcode(s) [6]
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3021 - Melbourne
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Recruitment postcode(s) [7]
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6000 - Perth
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Recruitment outside Australia
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Michigan
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North Carolina
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France
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France
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Yamanashi
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Antalya
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Edirne
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Istanbul
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Kocaeli
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Samsun
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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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Ethics approval
Ethics application status
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Summary
Brief summary
This is a study evaluating the safety and efficacy of bomedemstat (MK-3543) compared with the best available therapy (BAT) in participants with essential thrombocythemia (ET) who have an inadequate response to or are intolerant of hydroxyurea. The primary study hypothesis is that bomedemstat is superior to the best available therapy with respect to durable clinicohematologic response (DCHR).
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Trial website
https://clinicaltrials.gov/study/NCT06079879
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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 Sharpe & Dohme LLC
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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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0
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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/NCT06079879