Study of Bomedemstat in Participants With Essential Thrombocythemia (IMG-7289-CTP-201/MK-3543-003)

Study Overview
=================
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.

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

Conditions
-----------------
Essential Thrombocythemia

Intervention / Treatment
-----------------
* Drug: Bomedemstat


Participation Criteria
=================
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Multi-center, open-label
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Bomedemstat<br>Bomedemstat administered daily for 169 consecutive days | Drug: Bomedemstat<br>* Oral administration<br>* Other names: MK-3543;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Who Experience an Adverse Event (AE) | An AE is any undesirable physical, psychological or behavioral effect experienced by a patient during participation in the study, in conjunction with the use of the drug or biologic, whether or not product related. This includes any untoward signs or symptoms experienced by the patient from the time of first dose with study treatment until completion of the study. The number of participants who experience an AE will be reported. | Up to approximately 30 months | 
| Number of Participants Who Discontinue Study Treatment Due to an AE | An AE is any undesirable physical, psychological or behavioral effect experienced by a patient during participation in the study, in conjunction with the use of the drug or biologic, whether or not product-related. This includes any untoward signs or symptoms experienced by the patient from the time of first dose with study treatment until completion of the study. The number of participants who discontinue study treatment due to an AE will be reported. | Up to approximately 30 months | 
| Change From Baseline in Percentage of Participants with Platelet Count ≤400k/µL | Blood samples will be collected at pre-specified timepoints to determine platelet counts. Change from baseline in percentage of participants who achieve reduction in platelet count to ≤400k/µL in the absence of new thrombolytic events will be reported. | Baseline and up to 30 months |