A Clinical Trial of Bomedemstat in Participants With Polycythemia Vera (MK-3543-025)

Official title

A Phase 2/3, Randomized, Open-label, Active-comparator-controlled, Parallel-group, Multicenter Study to Evaluate the Safety and Efficacy of Bomedemstat (MK-3543) Versus Best Available Therapy in Participants With Polycythemia Vera Who Have an Inadequate Response to or Are Intolerant to Hydroxyurea

Overview

Researchers are looking for new ways to treat polycythemia vera (PV). People with PV may receive treatment to lower the number of red blood cells in the blood, but the usual treatments may not work for everyone. Researchers want to learn if a trial medicine called bomedemstat, also called MK-3543, can treat PV. In this study, researchers will compare bomedemstat to 2 usual treatments for PV. The goal of this study is to learn if more participants who take bomedemstat reach healthy blood cell counts and avoid major health problems from PV, compared to those who receive a usual treatment.

Interventions

• Drug Bomedemstat
  Oral Capsule
• Drug Ruxolitinib
  Oral Tablet
• Drug Ropeginterferon alfa-2b
  Subcutaneous Solution

Primary outcome measures

• Clinicohematologic Response (CHR) Rate [Time frame: Up to approximately Week 52]

Secondary outcome measures (12)

• Clinicohematologic Response Sustained for a 24-Week Time Period (CHR24) [Time frame: Up to approximately Week 52]
• Number of Participants Who Experience an Adverse Event (AE) [Time frame: Up to approximately Week 52]
• Number of Participants Who Discontinue Study Treatment Due to an AE [Time frame: Up to approximately Week 52]
• Duration of Clinicohematologic Response Sustained for a 24-Week Time Period (DOCHR24) [Time frame: Up to approximately Week 52]
• Duration of Clinicohematologic Response (DOCHR) [Time frame: Up to approximately Week 52]
• Duration of Hematologic Remission (DOHR) [Time frame: Up to approximately Week 52]
• Number of Participants Who Experience Phlebotomies [Time frame: Up to approximately Week 52]
• Disease Progression Rate [Time frame: Up to approximately Week 52]
• Number of Participants Who Experience Thrombotic Events [Time frame: Up to approximately Week 52]
• Number of Participants Who Experience Major Hemorrhagic Events [Time frame: Up to approximately Week 52]
• Change From Baseline in Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4) Total Symptom Score [Time frame: Up to approximately Week 52]
• Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form (SF) 7a Total Fatigue Score [Time frame: Up to approximately Week 52]

Eligibility criteria

Inclusion criteria

The main inclusion criteria include but are not limited to the following:

• Has confirmed local diagnosis of polycythemia vera (PV) per World Health Organization (WHO) diagnostic criteria for PV
• Must have discontinued prior cytoreductive therapy for condition under study for protocol specified duration
• Has failed at least one prior line of cytoreductive therapy to lower hematocrit
• Has a history of inadequate response, resistance to, or intolerant to hydroxyurea (HU) per protocol specified criteria
• Has no evidence of splenomegaly and no symptoms attributable to splenomegaly, including early satiety, left upper quadrant discomfort, or splenic pain
• Has locally assessed bone marrow (BM) fibrosis score of Grade 0 or Grade 1 as per modified version of the European Consensus Criteria for Grading Myelofibrosis
• Human Immunodeficiency Virus (HIV)-infected participants have well controlled HIV on antiretroviral therapy (ART)
• Participants who are Hepatitis B surface antigen (HBsAg) positive are eligible if they have received Hepatitis B Virus (HBV) antiviral therapy for at least 4 weeks and have undetectable HBV viral load
• Participants with history of Hepatitis C Virus (HCV) infection are eligible if HCV viral load is undetectable
• Participants must be able to swallow oral medication and follow instructions for at home dosing of bomedemstat

Exclusion criteria

The main exclusion criteria include but are not limited to the following:

• Has history of any illness/impairment of gastrointestinal (GI) function that might interfere with drug absorption
• Has evidence at the time of screening of increased risk of bleeding
• Has history of malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years
• HIV-infected participants with a history of Kaposi's sarcoma and/or Multicentric Castleman's Disease
• Is currently receiving anticancer therapy
• Has an active infection requiring systemic therapy
• Has had major surgical procedure ≤4 weeks before first dose of study intervention or has not recovered from side effects of major surgical procedure

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07722611 · 3543-025 · MK-3543-025

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗