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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05558696
Registration number
NCT05558696
Ethics application status
Date submitted
20/09/2022
Date registered
28/09/2022
Date last updated
11/06/2024
Titles & IDs
Public title
A Study of Bomedemstat (MK-3543) in Participants With Polycythemia Vera (MK-3543-004)
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Scientific title
A Phase 2 Multi-Center, Open Label Study to Assess the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Bomedemstat in Patients With Polycythemia Vera (PV)
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Secondary ID [1]
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MK-3543-004
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Secondary ID [2]
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3543-004
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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:
Polycythemia Vera
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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
Experimental: bomedemstat - Participants will receive bomedemstat daily for 36 weeks and may qualify for additional treatment thereafter if deriving clinical benefit.
Treatment: Drugs: bomedemstat
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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Number of participants with adverse events (AEs)
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Assessment method [1]
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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 presented.
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Timepoint [1]
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Up to ~40 weeks
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Primary outcome [2]
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Number of participants who discontinued study intervention due to AEs
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Assessment method [2]
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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 presented.
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Timepoint [2]
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Up to ~36 weeks
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Primary outcome [3]
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Number of participants with change from baseline of hematocrit to <45% without phlebotomy at Week 36
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Assessment method [3]
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Hematocrit will be analyzed by taking blood samples from participants at designated time points during the study. The number of participants who have a change from baseline of hematocrit to \<45% without phlebotomy at Week 36 will be presented.
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Timepoint [3]
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Baseline through Week 36
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Secondary outcome [1]
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Duration of reduction of hematocrit to <45% without phlebotomy
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Assessment method [1]
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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 presented.
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Timepoint [1]
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Baseline through Week 36
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Secondary outcome [2]
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Number of participants with platelet count = 450 x 10^9/L at Week 36
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Assessment method [2]
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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 presented.
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Timepoint [2]
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Baseline through Week 36
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Secondary outcome [3]
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Duration of platelet count = 450 x 10^9/L in participants at Week 36
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Assessment method [3]
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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 presented.
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Timepoint [3]
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Baseline through Week 36
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Secondary outcome [4]
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Number of participants with white blood cell (WBC) count of <10 x 10^9/L at Week 36
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Assessment method [4]
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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 presented.
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Timepoint [4]
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Baseline through Week 36
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Secondary outcome [5]
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Duration of white blood cell (WBC) count <10 x 10^9/L in participants at Week 36
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Assessment method [5]
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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 presented.
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Timepoint [5]
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Baseline through Week 36
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Secondary outcome [6]
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Number of participants with thrombotic events
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Assessment method [6]
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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 presented.
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Timepoint [6]
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Baseline through Week 36
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Secondary outcome [7]
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Number of participants with major hemorrhagic events
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Assessment method [7]
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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 presented.
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Timepoint [7]
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Baseline through Week 36
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Secondary outcome [8]
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Number of participants with a reduction in splenic volume in patients with an enlarged spleen at baseline
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Assessment method [8]
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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 presented.
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Timepoint [8]
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Baseline through Week 36
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Secondary outcome [9]
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Number of participants with progressive disease (PD)
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Assessment method [9]
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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 presented.
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Timepoint [9]
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Baseline through Week 36
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Secondary outcome [10]
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Maximum plasma concentration (Cmax) of bomedemstat
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Assessment method [10]
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Cmax is defined as the peak concentration over the dosing interval. Blood samples collected pre-dose and at multiple timepoints post-dose will be used to determine Cmax of bomedemstat.
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Timepoint [10]
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At designated time points on Day 1, Day 15 and at investigators choice of one of any regularly scheduled study visit from Week 4 (Day 29) to Week 8 (Day 57)
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Secondary outcome [11]
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Area under the curve 0-24 (AUC 0-24) of bomedemstat
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Assessment method [11]
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AUC is defined as area under curve exposure. Blood samples collected pre-dose and at multiple timepoints post-dose will be used to determine AUC of bomedemstat.
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Timepoint [11]
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At designated time points on Day 1, Day 15 and at investigators choice of one of any regularly scheduled study visit from Week 4 (Day 29) to Week 8 (Day 57)
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Secondary outcome [12]
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Half life (t½) of bomedemstat
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Assessment method [12]
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t½ is the time required for 50% of drug to be cleared from serum. Blood samples will be collected pre-dose and post-dose at designated timepoints to determine t½ of bomedemstat.
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Timepoint [12]
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At designated time points on Day 1, Day 15 and at investigators choice of one of any regularly scheduled study visit from Week 4 (Day 29) to Week 8 (Day 57)
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Secondary outcome [13]
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Number of participants with change from baseline in The Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) 7-day recall at Week 36
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Assessment method [13]
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The MFSAF v4.0 is a 7-item questionnaire used for the assessment of myelofibrosis symptoms that asks participants to report symptom severity at its worst for each of the seven items on a 0 (Absent) to 10 (Worst Imaginable) numeric rating scale and has a minimum score of 0 and a maximum score of 70. Participants will be evaluated with the MFSAF v4.0 at designated time points during the study. The number of participants with a change from baseline by Week 36 will be reported.
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Timepoint [13]
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Baseline through Week 36
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Secondary outcome [14]
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Number of participants with change from baseline in Patient Global Impression of Change (PGIC)
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Assessment method [14]
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The PGIC is a one-item questionnaire based on the Clinical Global Impressions (CGI) scale and adapted to the participant. It measures change in clinical status from a scale of 1-7 with 1 being very much improved to 7 being very much worse. The number of participants with a change in PGIC score from Week 12 to Week 36 will be presented.
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Timepoint [14]
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Week 12 through Week 36
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Secondary outcome [15]
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Number of participants with a durable change in participant-reported symptom burden on the MFSAF v4.0 7-day recall
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Assessment method [15]
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The MFSAF v4.0 is a 7-item questionnaire used for the assessment of myelofibrosis symptoms that asks participants to report symptom severity at its worst for each of the seven items on a 0 (Absent) to 10 (Worst Imaginable) numeric rating scale and has a minimum score of 0 and a maximum score of 70. Participants will be evaluated with the MFSAF v4.0 at designated time points during the study. The number of participants achieving a durable change of the MFSAF Score will be presented.
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Timepoint [15]
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Baseline through Week 36
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Eligibility
Key inclusion criteria
* Has a diagnosis of Polycythemia Vera per World Health Organization (WHO) diagnostic criteria for myeloproliferative neoplasms
* Has a bone marrow fibrosis score of Grade 0 or Grade 1
* Has failed at least one standard cytoreductive therapy to lower hematocrit
* Has a life expectancy >36 weeks
* Has discontinued prior cytoreductive therapy for 2 weeks (4 weeks for interferon) prior to study drug initiation
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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
* Has an Eastern Cooperative Oncology Group (ECOG) performance status of 3 or greater
* Has unresolved treatment related toxicities from prior therapies (unless resolved to = Grade 1)
* Has an uncontrolled active infection
* Has a known human immunodeficiency virus (HIV) infection or active Hepatitis B or Hepatitis C virus infection
* Has evidence of increased risk of bleeding, including known bleeding disorders
* Is pregnant or lactating
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not 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
7/09/2023
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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
24/03/2025
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Actual
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Sample size
Target
20
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
QLD,VIC,WA
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Recruitment hospital [1]
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Sunshine Coast Hematology and Oncology Clinic (Site 0506) - Sunshine Coast
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Recruitment hospital [2]
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Monash Medical Centre ( Site 0006) - Clayton
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Recruitment hospital [3]
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Royal Perth Hospital ( Site 0504) - Perth
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Recruitment postcode(s) [1]
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4556 - Sunshine Coast
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Recruitment postcode(s) [2]
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3168 - Clayton
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Recruitment postcode(s) [3]
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6000 - Perth
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Florida
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Country [2]
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United States of America
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State/province [2]
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Illinois
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Country [3]
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United States of America
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State/province [3]
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Michigan
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Country [4]
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United States of America
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State/province [4]
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Nevada
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Country [5]
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United States of America
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State/province [5]
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North Carolina
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Country [6]
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United States of America
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State/province [6]
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Ohio
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Country [7]
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United States of America
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State/province [7]
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Oregon
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Country [8]
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United States of America
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State/province [8]
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Utah
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Country [9]
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United Kingdom
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State/province [9]
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England
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Country [10]
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United Kingdom
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State/province [10]
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Great Britain
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Country [11]
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United Kingdom
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State/province [11]
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Lincolnshire
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Country [12]
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United Kingdom
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State/province [12]
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London, City Of
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Country [13]
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United Kingdom
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State/province [13]
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Wales
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a Phase 2 open label study of an orally administered lysine-specific demethylase 1 (LSD1) inhibitor, bomedemstat, in participants with polycythemia vera. The primary hypothesis is that bomedemstat is a safe and tolerable orally available agent when administered to participants with PV; and inhibition of LSD1 by bomedemstat will induce hematologic response in this population by 36 weeks, improve symptom burden and reduce spleen size in participants with enlarged spleen at baseline.
With Amendment 3, after all ongoing patients have reached 52 weeks of treatment, eligible patients may transition to a bomedemstat extension study if available.
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Trial website
https://clinicaltrials.gov/study/NCT05558696
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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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Address
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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 scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/study/NCT05558696
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