Clinical trial NCT04254978: Study of Bomedemstat in Participants With Essential Thrombocythemia (IMG-7289-CTP-201/MK-3543-003)
Official title: A Phase 2 Multi-Center, Open Label Study to Assess the Safety, Efficacy and Pharmacodynamics of IMG-7289 in Patients With Essential Thrombocythemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Essential Thrombocythemia
Lead sponsor: Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)
Sponsor class: INDUSTRY
First posted: 2020-02-05
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Multi-center, open-label
Primary purpose: TREATMENT

Brief summary: This is a Phase 2b open label study of an orally administered LSD1 inhibitor, Bomedemstat (MK-3543, formerly called IMG-7289), in patients with essential thrombocythemia.

This study investigates the following:

* The safety and tolerability of Bomedemstat
* The pharmacodynamic effect of Bomedemstat

Detailed description: This is a Phase 2 multi-center, open-label study evaluating the safety, efficacy and pharmacodynamics of Bomedemstat administered orally once daily in patients with essential thrombocythemia (ET). Patients will be dosed with Bomedemstat for 169 consecutive days in the Initial Treatment Period (ITP). Qualifying patients may continue to receive Bomedemstat in the Additional Treatment Period (ATP).

Safety will be evaluated by clinical assessments of safety parameters i.e., safety laboratory testing, adverse event reporting, physical examination and vital sign assessments. Pharmacodynamics will be evaluated by hematology assessment, patient reported symptom burden, change in spleen size by palpation and other measures.

To ensure safety, a Safety Advisory Board will perform periodic reviews of safety parameters and pharmacodynamic markers.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of Essential Thrombocythemia per World Health Organization (WHO) diagnostic criteria for myeloproliferative neoplasms.
* Requires treatment in order to lower platelet count based on patient age over 60 or history of thrombosis.
* Have failed at least one standard therapy
* Must have discontinued ET therapy at least 1 week (4 weeks for interferon) prior to study drug initiation.

Exclusion Criteria:

* Has undergone major surgery ≤4 weeks prior to starting study drug or has not recovered from side effects of such surgery.
* Unresolved treatment related toxicities from prior therapies (unless resolved to ≤ Grade 1).
* Uncontrolled active infection.
* Current use of prohibited medications
* Known HIV infection or active Hepatitis B or Hepatitis C virus infection
* Other hematologic/biochemistry requirements, as per protocol
* Use of investigational agent within last 14 days
* Pregnant or lactating females

Primary outcomes:
measure: Number of Participants Who Experienced an Adverse Event (AE)
timeFrame: Up to approximately 30 months
measure: Number of Participants Who Discontinued Study Treatment Due to an AE
timeFrame: Up to approximately 28 months
measure: Percentage of Participants With Platelet Count ≤400 k/μL at Day 169
timeFrame: Up to day 169
Secondary outcomes:
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: Local Institution
state: Florida
city: Ann Arbor
country: United States
facility: Local Institution
state: Michigan
city: New York
country: United States
facility: Local Institution
state: New York
city: Durham
country: United States
facility: Local Institution
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Pittsburgh
country: United States
facility: Local Institution
state: Pennsylvania
city: Seattle
country: United States
facility: Local Institution
state: Washington
city: Herston
country: Australia
facility: Local Institution
state: Brisbane
city: Camperdown
country: Australia
facility: Local Institution
state: New South Wales
city: St Leonards
country: Australia
facility: Local Institution
state: New South Wales
city: Southport
country: Australia
facility: Local Institution
state: Queensland
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Clayton
country: Australia
facility: Local Institution
state: Victoria
city: Essen
country: Germany
facility: Department of Hematology and Stem Cell Transplantation, West German Cancer Center (WTZ)
city: Jena
country: Germany
facility: Local Institution
city: Hong Kong
country: Hong Kong
facility: Local Institution
city: Alessandria
country: Italy
facility: Local Institution
city: Bologna
country: Italy
facility: Local Institution
city: Florence
country: Italy
facility: CRIMM; Centro Ricerca e Innovazione delle Malattia Mieloproliferative, Azienda ospedaliera Universitaria Careggi
city: Varese
country: Italy
facility: Local Institution
city: Auckland
country: New Zealand
facility: Local Institution
city: Auckland
country: New Zealand
facility: Middlemore Hospital
city: London
country: United Kingdom
facility: Local Institution
city: London
country: United Kingdom
facility: Guys and St Thomas Hospital
city: London
country: United Kingdom
facility: Local Institution
city: Oxford
country: United Kingdom
facility: Local Institution
Study officials:
affiliation: Merck Sharp & Dohme LLC
name: Medical Director
role: STUDY_DIRECTOR
References: