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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)
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)
Secondary ID [1] 0 0
MK-3543-004
Secondary ID [2] 0 0
3543-004
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polycythemia Vera 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with adverse events (AEs)
Timepoint [1] 0 0
Up to ~40 weeks
Primary outcome [2] 0 0
Number of participants who discontinued study intervention due to AEs
Timepoint [2] 0 0
Up to ~36 weeks
Primary outcome [3] 0 0
Number of participants with change from baseline of hematocrit to <45% without phlebotomy at Week 36
Timepoint [3] 0 0
Baseline through Week 36
Secondary outcome [1] 0 0
Duration of reduction of hematocrit to <45% without phlebotomy
Timepoint [1] 0 0
Baseline through Week 36
Secondary outcome [2] 0 0
Number of participants with platelet count = 450 x 10^9/L at Week 36
Timepoint [2] 0 0
Baseline through Week 36
Secondary outcome [3] 0 0
Duration of platelet count = 450 x 10^9/L in participants at Week 36
Timepoint [3] 0 0
Baseline through Week 36
Secondary outcome [4] 0 0
Number of participants with white blood cell (WBC) count of <10 x 10^9/L at Week 36
Timepoint [4] 0 0
Baseline through Week 36
Secondary outcome [5] 0 0
Duration of white blood cell (WBC) count <10 x 10^9/L in participants at Week 36
Timepoint [5] 0 0
Baseline through Week 36
Secondary outcome [6] 0 0
Number of participants with thrombotic events
Timepoint [6] 0 0
Baseline through Week 36
Secondary outcome [7] 0 0
Number of participants with major hemorrhagic events
Timepoint [7] 0 0
Baseline through Week 36
Secondary outcome [8] 0 0
Number of participants with a reduction in splenic volume in patients with an enlarged spleen at baseline
Timepoint [8] 0 0
Baseline through Week 36
Secondary outcome [9] 0 0
Number of participants with progressive disease (PD)
Timepoint [9] 0 0
Baseline through Week 36
Secondary outcome [10] 0 0
Maximum plasma concentration (Cmax) of bomedemstat
Timepoint [10] 0 0
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)
Secondary outcome [11] 0 0
Area under the curve 0-24 (AUC 0-24) of bomedemstat
Timepoint [11] 0 0
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)
Secondary outcome [12] 0 0
Half life (t½) of bomedemstat
Timepoint [12] 0 0
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)
Secondary outcome [13] 0 0
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
Timepoint [13] 0 0
Baseline through Week 36
Secondary outcome [14] 0 0
Number of participants with change from baseline in Patient Global Impression of Change (PGIC)
Timepoint [14] 0 0
Week 12 through Week 36
Secondary outcome [15] 0 0
Number of participants with a durable change in participant-reported symptom burden on the MFSAF v4.0 7-day recall
Timepoint [15] 0 0
Baseline through Week 36

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/09/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
24/03/2025
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC,WA
Recruitment hospital [1] 0 0
Sunshine Coast Hematology and Oncology Clinic (Site 0506) - Sunshine Coast
Recruitment hospital [2] 0 0
Monash Medical Centre ( Site 0006) - Clayton
Recruitment hospital [3] 0 0
Royal Perth Hospital ( Site 0504) - Perth
Recruitment postcode(s) [1] 0 0
4556 - Sunshine Coast
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
Nevada
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Oregon
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
United Kingdom
State/province [9] 0 0
England
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Great Britain
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Lincolnshire
Country [12] 0 0
United Kingdom
State/province [12] 0 0
London, City Of
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Wales

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT05558696
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Merck Sharp & Dohme LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries