Clinical trial NCT05558696: A Study of Bomedemstat (MK-3543) in Participants With Polycythemia Vera (MK-3543-004)
Official 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Polycythemia Vera
Keywords: polycythemia vera
bomedemstat
Lead sponsor: Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)
Sponsor class: INDUSTRY
First posted: 2022-09-28
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics of the orally administered lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat, in participants with polycythemia vera (PV). At Week 36 of dosing, participants will be assessed for eligibility to receive additional treatment through Week 52. Participants deriving clinical benefit and safely tolerating bomedemstat will qualify for continued treatment at the Investigator's discretion.

Detailed description: With Amendment 3, after all ongoing participants have reached 52 weeks of treatment, eligible participants may transition to a bomedemstat extension study if available. With Amendment 4, all secondary PK and patient reported outcome measures were designated as exploratory.

Eligibility criteria: 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

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

Primary outcomes:
measure: Number of participants with adverse events (AEs)
timeFrame: Up to ~56 weeks
measure: Number of participants who discontinued study intervention due to AEs
timeFrame: Up to ~52 weeks
measure: Number of participants with sustained change from baseline of hematocrit to <45% without concomitant phlebotomy at Week 36
timeFrame: Baseline through Week 36
Secondary outcomes:
measure: Duration of reduction of hematocrit to <45% without phlebotomy
timeFrame: Baseline through Week 36
measure: Number of participants with platelet count ≤ 450 x 10^9/L at Week 36
timeFrame: Baseline through Week 36
measure: Duration of platelet count ≤ 450 x 10^9/L in participants at Week 36
timeFrame: Baseline through Week 36
measure: Number of participants with white blood cell (WBC) count of <10 x 10^9/L at Week 36
timeFrame: Baseline through Week 36
measure: Duration of white blood cell (WBC) count <10 x 10^9/L in participants at Week 36
timeFrame: Baseline through Week 36
measure: Number of participants with thrombotic events
timeFrame: Baseline through Week 36
measure: Number of participants with major hemorrhagic events
timeFrame: Baseline through Week 36
measure: Number of participants with a reduction in splenic volume in patients with an enlarged spleen at baseline
timeFrame: Baseline through Week 36
measure: Number of participants with progressive disease (PD)
timeFrame: Baseline through Week 36
Other outcomes:
Locations:
city: Plantation
country: United States
facility: BRCR Global ( Site 0120)
state: Florida
city: Skokie
country: United States
facility: Hematology Oncology of the North Shore ( Site 0104)
state: Illinois
city: Ann Arbor
country: United States
facility: University of Michigan Comprehensive Cancer Center ( Site 0008)
state: Michigan
city: Las Vegas
country: United States
facility: Comprehensive Cancer Centers of Nevada - Peak ( Site 0118)
state: Nevada
city: Durham
country: United States
facility: Duke University Medical Center ( Site 0016)
state: North Carolina
city: Columbus
country: United States
facility: Ohio State University Comprehensive Cancer Center ( Site 0103)
state: Ohio
city: Portland
country: United States
facility: OHSU Knight Cardiovascular Institute Cardiology Clinic - South Waterfront ( Site 0102)
state: Oregon
city: Salt Lake City
country: United States
facility: Huntsman Cancer Hospital at the University of Utah ( Site 0119)
state: Utah
city: Sunshine Coast
country: Australia
facility: Sunshine Coast Hematology and Oncology Clinic (Site 0506)
state: Queensland
city: Clayton
country: Australia
facility: Monash Medical Centre ( Site 0006)
state: Victoria
city: Perth
country: Australia
facility: Royal Perth Hospital ( Site 0504)
state: Western Australia
city: Gloucester
country: United Kingdom
facility: Gloucestershire Royal Hospital ( Site 0205)
state: England
city: Lincoln
country: United Kingdom
facility: United Lincolnshire Hospitals NHS Trust ( Site 0204)
state: Great Britain
city: London
country: United Kingdom
facility: Imperial College London ( Site 0025)
state: Great Britain
city: Boston
country: United Kingdom
facility: Boston Pilgrim Hospital ( Site 0207)
state: Lincolnshire
city: London
country: United Kingdom
facility: Guys and St Thomas NHS Foundation Trust - Guys Hospital ( Site 0020)
state: London, City Of
city: Newport
country: United Kingdom
facility: Royal Gwent Hospital ( Site 0201)
state: Wales
Study officials:
affiliation: Merck Sharp & Dohme LLC
name: Medical Director
role: STUDY_DIRECTOR
References: