A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Feasibility of Tranexamic Acid as an Adjunct to Standard Therapy: Number of Participants Eligible and Recruited
研究概览
简要总结
This study evaluates the use of tranexamic acid (TXA) in addition to standard therapy in children receiving chemotherapy or blood and/or marrow transplantation to decrease the risk of bleeding. Half of participants will receive tranexamic acid and half of participants will receive placebo.
详细描述
The purpose of this study is to conduct a prospective, randomized, blinded, placebo controlled trial to evaluate the safety and feasibility of the addition of antifibrinolytic therapy with tranexamic acid to the standard care in patients who are thrombocytopenic due to primary bone marrow disorders or chemotherapy, immunotherapy and/or radiation therapy in order to prevent bleeding. The results of this study will change practice by providing evidence as to whether or not TXA is effective and safe treatment when used as an adjunct to platelet transfusion therapy in the thrombocytopenic patient.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
The investigational pharmacy will be notified when patients enroll on trial. When participants meet criteria for randomization, this will be completed by the Investigational Pharmacy. The pharmacy will prepare and distribute to floor nursing staff all doses of tranexamic acid (available in IV form as a colorless liquid) or placebo (as an equal volume of normal saline). Labels will not carry identifiers of which study arm the enrolled patient is on.
入排标准
- 年龄范围
- 2 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have a confirmed diagnosis of hematologic malignancy or solid tumor malignancy
- •Patients must be undergoing or planned chemotherapy or BMT
- •Patients will only be eligible to receive study drug or placebo during inpatient periods
- •Patients must be predicted to have thrombocytopenia ≤20,000/microliter (uL) for ≥5 days
- •Patient must have a platelet transfusion threshold of ≤30,000/uL
- •Patients must be >14 days beyond their last dose of Pegylated(PEG)-Asparaginase or >72 hours beyond their last dose of Erwinia Asparaginase
- •Patients must be able to comply with treatment and monitoring
排除标准
- •Diagnosis of acute promyelocytic leukemia (APL)
- •History of Immune Thrombocytopenic Purpura (ITP), Thrombotic Thrombocytopenic Purpura (TTP) or Hemolytic Uremic Syndrome (HUS)
- •Diagnosis of Disseminated Intravascular Coagulopathy (DIC)
- •History of inherited or acquired bleeding disorder AND/OR inherited or acquired prothrombotic disorder
- •Patient must not have WHO Grade 2 bleeding or greater within 48 hours prior to enrollment or study drug activation
- •Patient must not have received PEG-Asparaginase within the 7 day period prior to enrollment. If given within the 8-14 day period prior to enrollment patients are eligible if prothrombin time (PT), partial thromboplastin time (PTT), international normalized ratio (INR) and fibrinogen are obtained and are within 1.5 times the upper limits of normal.
- •Patient must not be receiving tranexamic acid or other anti-fibrinolytic agent or any other agent to promote hemostasis (which includes DDAVP, recombinant Factor VII, Prothrombin Complex Concentrate, Estrogen Derivatives and Progestins)
- •Patient must not be receiving therapy with anticoagulation or antiplatelet therapy (which includes heparin infusion, enoxaparin, aspirin. If anticoagulant/antiplatelet therapy is discontinued when platelet count is <50,000/uL patient will be eligible for enrollment)
- •Patient must not be receiving platelet growth factors
- •Current thromboembolic event
- •History of thromboembolic event <6 months prior to enrollment
- •Current/prior history of sinusoidal obstruction disease
- •Visible hematuria
- •Renal dysfunction (as defined by age-specific creatinine values calculated by Schwartz equation) or hemodialysis or anuria (defined as <10 mL urine/hour over 24 hours)
- •History of seizures
- •Allergy to tranexamic acid
- •Pregnancy
- •Unwilling to accept blood product transfusions
研究组 & 干预措施
Tranexamic Acid
Doses will be given intravenous (IV). Doses are administered every 8 hours or three times daily (TID) per the discretion of the treating investigator. TXA dose will be 10mg/kg, diluted in normal saline to a total volume of 15 milliliters (mL).
干预措施: Tranexamic Acid (Drug)
Placebo
Doses will be given intravenous (IV). Doses are administered every 8 hours or TID per the discretion of the treating investigator. Normal saline will be administered at a total volume of 15mL.
干预措施: Normal saline (Drug)
结局指标
主要结局
Feasibility of Tranexamic Acid as an Adjunct to Standard Therapy: Number of Participants Eligible and Recruited
时间窗: From time of recruitment of the first patient until the last patient is enrolled, up to 16 months in duration
Number of participants eligible for study enrollment and recruited. The hypothesis is that tranexamic acid can be safely added to standard care regimens in patients with hematologic malignancies or solid tumors during periods of severe thrombocytopenia. A descriptive assessment of feasibility of recruitment by monitoring number of patients screened, number of patients eligible for enrollment and rate of recruitment (both start-up and ongoing) during study period to be completed by collecting data on the number of patients screened, the number of patients eligible for study inclusion, the number of eligible patients who consent to study inclusion and the number of consented patients activated to the study drug, organized in visual form by CONSORT diagram.
Safety and Tolerability of Tranexamic Acid in Participants as the Number of Patients With Any Adverse Events and Serious Adverse Events (SAE) as Assessed by CTCAE v4.03
时间窗: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug
Adverse Events and Serious Adverse Events (SAE) will be collected on subjects throughout their participation in the study and up to 30 days following discontinuation of the study drug, regardless of attribution. Adverse events and serious adverse events will be tabulated by type and grade according to the NCI CTCAE v 4.03. The hypothesis is that tranexamic acid can be safely added to standard care regimens in patients with hematologic malignancies or solid tumors during periods of severe thrombocytopenia. Analysis limited to a descriptive assessment of the safety of tranexamic acid in patients with hematologic malignancies or solid tumors by reported adverse events, serious adverse events and death.
次要结局
- The Occurrence of Thromboembolic Adverse Events and Serious Adverse Events(From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug)
- Number of Platelet and Red Blood Cell Transfusions(30 days after activation of study drug)
- Number of Days Alive and Without WHO Grade 2 Bleeding or Greater(30 days after activation of study drug)
- Highest Observed Grade of Bleeding (as Measured on WHO Bleeding Scale) During the Study Period(From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug)
- World Health Organization (WHO) Bleeding Scale Grade 2 or Higher Bleeding(30 days after activation of study drug)
- Bleeding of Any Grade(From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug)
研究者
Meghan McCormick
Principal Investigator, Fellow
University of Pittsburgh