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

Phase 2

Completed

• Conditions: Essential Thrombocythemia
• Interventions: Drug: Bomedemstat

• Registration Number: NCT04254978
• Lead Sponsor: Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA)

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.

Study Timeline

• Study First Submitted: 2020-01-15
• Study First Submitted QC: 2020-02-02
• Study First Posted: 2020-02-05
• Study Start: 2020-09-08
• Primary Completion: 2023-03-23
• Study Completion: 2023-03-23
• Status Verified: 2024-03
• Results First Submitted: 2024-03-06
• Results First Submitted QC: 2024-03-06
• Last Update Submitted: 2024-03-06
• Results First Posted: 2024-04-02
• Last Update Posted: 2024-04-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 73

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Bomedemstat	Bomedemstat	Bomedemstat administered daily for 169 consecutive days

Primary Outcome Measures

Name	Time	Method
Number of Participants Who Experienced an Adverse Event (AE)	Up to approximately 30 months	An AE is any undesirable physical, psychological or behavioral effect experienced by a participant during 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 participant from the time of first dose with study treatment until completion of the study. The number of participants who experienced an AE is reported.
Number of Participants Who Discontinued Study Treatment Due to an AE	Up to approximately 28 months	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 discontinued study treatment due to an AE is reported.
Percentage of Participants With Platelet Count ≤400 k/μL at Day 169	Up to day 169	Blood samples were collected at pre-specified timepoints to determine platelet counts. The percentage of participants who achieved reduction in platelet count to ≤400k/μL in the absence of new thrombolytic events is reported.

Trial Locations

Locations (7)

Middlemore Hospital; 🇳🇿 Auckland, New Zealand

Department of Hematology and Stem Cell Transplantation, West German Cancer Center (WTZ); 🇩🇪 Essen, Germany

Guys and St Thomas Hospital; 🇬🇧 London, United Kingdom

Local Institution; 🇬🇧 Oxford, United Kingdom

CRIMM; Centro Ricerca e Innovazione delle Malattia Mieloproliferative, Azienda ospedaliera Universitaria Careggi; 🇮🇹 Florence, Italy

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

Royal Adelaide Hospital; 🇦🇺 Adelaide, South Australia, Australia