A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia

Meghan McCormick

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Thrombocytopenia; Hemostatic Disorder; Pediatric Cancer; Coagulation Defect; Acquired

Treatments: Drug: Tranexamic Acid; Drug: Normal saline

Study type: Interventional

Funder types: Other

Identifiers

NCT03806556

PRO18100519

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 11 patients

Sex: All

Ages: 2 to 21 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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11 participants in 2 patient groups, including a placebo group

Tranexamic Acid

Experimental group

Description:

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).

Treatment:

Drug: Tranexamic Acid

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Normal saline