A Phase II Trial of Avatrombopag for the Treatment of Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplant
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 主要终点
- Failure rate of platelet recovery
研究概览
简要总结
This phase II trial studies the side effects and how well avatrombopag works for the treatment of thrombocytopenia after donor hematopoietic stem cell transplant. Thrombocytopenia is defined as abnormally low level of platelets in the blood. Avatrombopag is a small molecule thrombopoietin receptor agonist which stimulates thrombopoietin receptor leading to increase production of platelets.
详细描述
PRIMARY OBJECTIVE:
I. To determine the safety and efficacy of avatrombopag for the treatment of thrombocytopenia after allogenic hematopoietic stem cell transplantation.
SECONDARY OBJECTIVE:
I. To identify predictors of response to avatrombopag.
OUTLINE:
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
- •Failure to engraft platelets by day 30 (D30) after hematopoietic cell transplantation (HCT) defined as: Platelet count less than 20,000/uL and patient is still dependent on platelet transfusion support
- •Patient must be able to start treatment with avatrombopag within 30-60 days following transplant
- •Able to provide written informed consent from patient or legal representative
排除标准
- •Serious uncontrolled infections
- •Steroid refractory graft versus host disease (GVHD)
- •Patients with thrombotic microangiopathy
- •Pregnant or lactating women
- •Creatinine clearance < 30 ml/min
- •Active thromboembolism requiring anticoagulation
- •Unable to understand the investigational nature of the study or provide informed consent
- •Evidence of disease relapse by flow cytometry of chimerisms
- •Concomitant use of other thrombopoietin receptor agonists (TPO-RA) medication during the treatment phase of the study or two weeks prior to enrollment
研究组 & 干预措施
Treatment (avatrombopag)
Patients receive avatrombopag PO QD for up to 1 year in the absence of disease progression or unacceptable toxicity. Avatrombopag will be titrated weekly until platelet count of greater than or equal to 60,000/uL is achieved and persists for 7 consecutive days, and the patient remains free from platelet transfusion.
干预措施: Avatrombopag (Drug)
结局指标
主要结局
Failure rate of platelet recovery
时间窗: At day 90
The proportion will be provided with 95% exact binomial confidence interval.
Incidence of adverse events of avatrombopag treatment
时间窗: Up to 30 days after the last dose
Toxicities will be evaluated using the Common Terminology Criteria for Adverse Events (CTCAE) version (v).5 standard toxicity grading. Frequency and other descriptive statistics will be used to present the toxicity pattern.
次要结局
- Independence from platelet transfusion(Up to 1 year)
- Duration of exposure to avatrombopag(Up to 1 year)
- Progression-free survival (PFS) of underlying malignant hematologic disorder(From the time of HCT to progression and death, assessed up to 1 year)
- Duration of platelet response(Up to 1 year)
- Overall survival (OS)(From the time of HCT to death from any cause, assessed up to 1 year)
- Incidence of adverse events associated with avatrombopag treatment(Up to 30 days after last dose)
- Platelet count >= 50,000/uL for 7 consecutive days without transfusion support(Up to 1 year)
- Transplant-related mortality(At day 100 and 1 year post-hematopoietic stem cell transplant (HCT))
研究者
Ayman Saad
Principal Investigator
Ohio State University Comprehensive Cancer Center