A Phase 2 Multi-Center, Open Label Study to Assess the Safety, Efficacy and Pharmacodynamics of IMG-7289 in Patients With Essential Thrombocythemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 73
- 试验地点
- 31
- 主要终点
- Number of Participants Who Experienced an Adverse Event (AE)
研究概览
简要总结
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
详细描述
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.
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
- •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.
排除标准
- •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
研究组 & 干预措施
Bomedemstat
Bomedemstat administered daily for 169 consecutive days
干预措施: Bomedemstat (Drug)
结局指标
主要结局
Number of Participants Who Experienced an Adverse Event (AE)
时间窗: 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
时间窗: 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
时间窗: 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.
次要结局
未报告次要终点