A Phase 2 Multi-Center, Open Label Study to Assess the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Bomedemstat in Patients With Polycythemia Vera (PV)

3543-004

This is a Phase 2 open label study of an orally administered lysine-specific demethylase 1 (LSD1) inhibitor, bomedemstat, in participants with polycythemia vera. The primary hypothesis is that bomedemstat is a safe and tolerable orally available agent when administered to participants with PV; and inhibition of LSD1 by bomedemstat will induce hematologic response in this population by 36 weeks, improve symptom burden and reduce spleen size in participants with enlarged spleen at baseline. With Amendment 3, after all ongoing patients have reached 52 weeks of treatment, eligible patients may transition to a bomedemstat extension study if available.

2023-09-07

2025-03-24

2025-03-24

Active (not recruiting)

Early phase I

20

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 20, 'type': 'ESTIMATED'}}

2024-04-22