A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia

NCT ID: NCT03806556

Last Updated: 2021-09-20

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-04-22
• Study Completion Date: 2020-08-25

Brief Summary

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.

Detailed 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.

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: PREVENTION
• Blinding Strategy: QUADRUPLE

Study Groups

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).

Group Type: EXPERIMENTAL

Tranexamic Acid

Intervention Type: DRUG

IV medication administered after patient meets inclusion/exclusion criteria

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.

Group Type: PLACEBO_COMPARATOR

Normal saline

Intervention Type: DRUG

IV medication administered after patient meets inclusion/exclusion criteria

Interventions

Tranexamic Acid

IV medication administered after patient meets inclusion/exclusion criteria

Intervention Type: DRUG

Normal saline

IV medication administered after patient meets inclusion/exclusion criteria

Intervention Type: DRUG

Other Intervention Names

TXA; NS

Eligibility Criteria

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

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Meghan McCormick

OTHER

Sponsor Role: lead

Responsible Party

Meghan McCormick

Principal Investigator, Fellow

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Meghan McCormick, MD

Role: PRINCIPAL_INVESTIGATOR

UPMC Childrens Hospital of Pittsburgh

Locations

UPMC Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

PRO18100519

Identifier Type: -

Identifier Source: org_study_id