A Phase 2 Multi-Center, Open Label Study to Assess the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Bomedemstat in Patients With Polycythemia Vera (PV)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 34
- 主要终点
- Number of participants with adverse events (AEs)
研究概览
简要总结
This study will evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics of the orally administered lysine-specific demethylase 1 (LSD1) inhibitor bomedemstat, in participants with polycythemia vera (PV). At Week 36 of dosing, participants will be assessed for eligibility to receive additional treatment through Week 52. Participants deriving clinical benefit and safely tolerating bomedemstat will qualify for continued treatment at the Investigator's discretion.
详细描述
With Amendment 3, after all ongoing participants have reached 52 weeks of treatment, eligible participants may transition to a bomedemstat extension study if available. With Amendment 4, all secondary PK and participant reported outcome measures were designated as exploratory.
Per protocol, participants who derived clinical benefit (no progressive disease) and safely tolerated drug may have qualified for enrollment into the bomedemstat extension study (NCT06351631).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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 A, Hepatitis B or Hepatitis C virus infection
- •Has evidence of increased risk of bleeding, including known bleeding disorders
- •Is pregnant or lactating
研究组 & 干预措施
Bomedemstat
Participants will receive bomedemstat daily for 36 weeks and may qualify for additional treatment through Week 52 if deriving clinical benefit.
干预措施: Bomedemstat (Drug)
结局指标
主要结局
Number of participants with adverse events (AEs)
时间窗: Up to ~56 weeks
An AE is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants with one or more AEs will be reported.
Number of participants who discontinued study intervention due to AEs
时间窗: Up to ~52 weeks
An AE is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants who discontinue study intervention due to AEs will be reported.
Number of participants with sustained change from baseline of hematocrit to <45% without concomitant phlebotomy at Week 36
时间窗: Baseline through Week 36
Hematocrit will be analyzed by taking blood samples from participants at designated time points during the study. The number of participants who have achieved a sustained change from baseline of hematocrit to \<45% without concomitant phlebotomy at Week 36 will be reported.
次要结局
- Number of participants with platelet count ≤ 450 x 10^9/L at Week 36(Baseline through Week 36)
- Duration of reduction of hematocrit to <45% without phlebotomy(Baseline through Week 36)
- Duration of platelet count ≤ 450 x 10^9/L in participants at Week 36(Baseline through Week 36)
- Number of participants with white blood cell (WBC) count of <10 x 10^9/L at Week 36(Baseline through Week 36)
- Duration of white blood cell (WBC) count <10 x 10^9/L in participants at Week 36(Baseline through Week 36)
- Number of participants with thrombotic events(Baseline through Week 36)
- Number of participants with major hemorrhagic events(Baseline through Week 36)
- Number of participants with a reduction in splenic volume in patients with an enlarged spleen at baseline(Baseline through Week 36)
- Number of participants with progressive disease (PD)(Baseline through Week 36)