A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia

• Sponsor: Meghan McCormick (Other)
• Overall Status: Terminated
• CT.gov ID: NCT03806556
• Collaborator: (none)
• Enrollment: 11
• Location: 1
• Arms: 2
• Actual Duration (Months): 16.1
• Patients Per Site Per Month: 0.7

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Pediatric Cancer; Thrombocytopenia; Hemostatic Disorder; Coagulation Defect; Acquired	Drug: Tranexamic Acid; Drug: Normal saline	Phase 1/Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 11 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Intervention Model Description: This trial is designed as a prospective, randomized, controlled, blinded (patient, caregiver, physician, assessor) trial with two parallel groups and a primary endpoint of feasibility and safety. Randomization will be performed as block randomization with a 1:1 allocation within blocks of size four.This trial is designed as a prospective, randomized, controlled, blinded (patient, caregiver, physician, assessor) trial with two parallel groups and a primary endpoint of feasibility and safety. Randomization will be performed as block randomization with a 1:1 allocation within blocks of size four.
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Masking Description: 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.
• Primary Purpose: Prevention
• Official Title: A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia
• Actual Study Start Date: Apr 22, 2019
• Actual Primary Completion Date: Aug 25, 2020
• Actual Study Completion Date: Aug 25, 2020

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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).	Drug: Tranexamic Acid; IV medication administered after patient meets inclusion/exclusion criteria; Other Names: TXA
Placebo Comparator: 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.	Drug: Normal saline; IV medication administered after patient meets inclusion/exclusion criteria; Other Names: NS

Outcome Measures

Primary Outcome Measures

1. 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 [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.

2. Feasibility of Tranexamic Acid as an Adjunct to Standard Therapy: Number of Participants Eligible and Recruited [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.

Secondary Outcome Measures

1. World Health Organization (WHO) Bleeding Scale Grade 2 or Higher Bleeding [30 days after activation of study drug]

   Proportion of patients with bleeding of WHO grade 2 or above, after activation of study drug. Bleeding graded on a scale ranging from 1 (minor bleeding) through 4 (bleeding that is fatal or life-threatening).

2. Number of Platelet and Red Blood Cell Transfusions [30 days after activation of study drug]

   Number of platelet and red blood cell transfusions per patient during the first 30 days post prescription activation of study drug

3. Number of Days Alive and Without WHO Grade 2 Bleeding or Greater [30 days after activation of study drug]

   Number of days alive and without WHO grade 2 bleeding or greater during the first 30 days post activation of study drug. Bleeding graded on a scale ranging from 1 (minor bleeding) through 4 (bleeding that is fatal or life-threatening).

4. 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]

   Any venous or arterial thrombosis on standard diagnostic imaging post-randomization

5. Bleeding of Any Grade [From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug]

   Proportion of patients with bleeding of any grade as assessed by WHO bleeding score, after activation of study drug

6. 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]

   Highest grade of bleeding (as measured on WHO bleeding scale) during study period in each enrolled patient. Bleeding graded on a scale ranging from 1 (minor bleeding) through 4 (bleeding that is fatal or life-threatening).

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 21 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UPMC Childrens Hospital of Pittsburgh	Pittsburgh	Pennsylvania	United States	15224

Sponsors and Collaborators

• Meghan McCormick

Investigators

• Principal Investigator: Meghan McCormick, MD, UPMC Childrens Hospital of Pittsburgh

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Jan 12, 2020
• Informed Consent Form - Jan 2, 2020

More Information

Publications

None provided.

• Responsible Party: Meghan McCormick, Principal Investigator, Fellow, University of Pittsburgh
• ClinicalTrials.gov Identifier: NCT03806556
• Other Study ID Numbers: PRO18100519
• First Posted: Jan 16, 2019
• Last Update Posted: Sep 20, 2021
• Last Verified: Aug 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Meghan McCormick, Principal Investigator, Fellow, University of Pittsburgh: tranexamic acid; anti-fibrinolytic agents; chemotherapy-induced thrombocytopenia
• Additional relevant MeSH terms: Hemostatic Disorders; Thrombocytopenia; Blood Coagulation Disorders; Tranexamic Acid

Study Results

Participant Flow

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Period Title: Overall Study
STARTED	6	5
COMPLETED	6	5
NOT COMPLETED	0	0

Baseline Characteristics

Arm/Group Title	Tranexamic Acid	Placebo	Total
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria	Total of all reporting groups
Overall Participants	6	5	11
Age (years) [Median (Full Range) ]
Median (Full Range) [years]	9.5	11	11
Sex: Female, Male (Count of Participants)
Female	2 33.3%	1 20%	3 27.3%
Male	4 66.7%	4 80%	8 72.7%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%	0 0%	0 0%
Not Hispanic or Latino	6 100%	5 100%	11 100%
Unknown or Not Reported	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	1 16.7%	0 0%	1 9.1%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	0 0%
White	5 83.3%	4 80%	9 81.8%
More than one race	0 0%	1 20%	1 9.1%
Unknown or Not Reported	0 0%	0 0%	0 0%
Diagnosis (Count of Participants)
Lymphoid Leukemia	4 66.7%	0 0%	4 36.4%
Myeloid Leukemia	0 0%	2 40%	2 18.2%
Lymphoma	1 16.7%	1 20%	2 18.2%
Solid Tumor	1 16.7%	2 40%	3 27.3%
Type of Therapy (Count of Participants)
Chemotherapy	4 66.7%	2 40%	6 54.5%
Autologous Hematopoietic Stem Cell Transplant	1 16.7%	2 40%	3 27.3%
Allogeneic Hematopoietic Stem Cell Transplant	1 16.7%	1 20%	2 18.2%

Outcome Measures

1. Primary Outcome

Title	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
Description	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.
Time Frame	From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Grade 3 Adverse Events : Anemia	4 66.7%	4 80%
Grade 3 Adverse Events : Anorexia	0 0%	2 40%
Grade 3 Adverse Events : Bone Pain	1 16.7%	0 0%
Grade 3 Adverse Events : Catheter Related Infection	3 50%	1 20%
Grade 3 Adverse Events : Vomiting	0 0%	2 40%
Grade 3 Adverse Events : Febrile Neutropenia	2 33.3%	3 60%
Grade 3 Adverse Events : Fever	0 0%	1 20%
Grade 3 Adverse Events : Hypoalbuminemia	1 16.7%	0 0%
Grade 3 Adverse Events : Hypotension	0 0%	1 20%
Grade 3 Adverse Events : Hypoxia	0 0%	1 20%
Grade 3 Adverse Events : Hypophosphatemia	0 0%	1 20%
Grade 3 Adverse Events : Rash Maculo-Papular	0 0%	2 40%
Grade 3 Adverse Events : Nausea	0 0%	1 20%
Grade 3 Adverse Events : Mucositis Oral	0 0%	1 20%
Grade 3 Adverse Events : Sinusitis	0 0%	1 20%
Grade 3 Adverse Events : Skin Infection	1 16.7%	0 0%
Grade 3 Adverse Events : White Blood Cell Decreases	0 0%	0 0%
Grade 3 Adverse Events : Platelet Count Decreased	0 0%	0 0%
Grade 3 Adverse Events : Blood Bilirubin Increased	0 0%	0 0%
Grade 4 Adverse Events : Anemia	0 0%	0 0%
Grade 4 Adverse Events : Anorexia	0 0%	0 0%
Grade 4 Adverse Events : Bone Pain	0 0%	0 0%
Grade 4 Adverse Events : Catheter Related Infection	0 0%	0 0%
Grade 4 Adverse Events : Vomiting	0 0%	0 0%
Grade 4 Adverse Events : Febrile Neutropenia	0 0%	1 20%
Grade 4 Adverse Events : Fever	0 0%	0 0%
Grade 4 Adverse Events : Hypoalbuminemia	0 0%	0 0%
Grade 4 Adverse Events : Hypotension	0 0%	0 0%
Grade 4 Adverse Events : Hypoxia	0 0%	0 0%
Grade 4 Adverse Events : Hypophosphatemia	0 0%	0 0%
Grade 4 Adverse Events : Rash Maculo-Papular	0 0%	0 0%
Grade 4 Adverse Events : Nausea	0 0%	0 0%
Grade 4 Adverse Events : Mucositis Oral	0 0%	0 0%
Grade 4 Adverse Events : Sinusitis	0 0%	0 0%
Grade 4 Adverse Events : Skin Infection	0 0%	0 0%
Grade 4 Adverse Events : White Blood Cell Decreases	6 100%	5 100%
Grade 4 Adverse Events : Platelet Count Decreased	6 100%	5 100%
Grade 4 Adverse Events : Blood Bilirubin Increased	1 16.7%	0 0%

2. Primary Outcome

Title	Feasibility of Tranexamic Acid as an Adjunct to Standard Therapy: Number of Participants Eligible and Recruited
Description	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.
Time Frame	From time of recruitment of the first patient until the last patient is enrolled, up to 16 months in duration

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Overall Study
Arm/Group Description	All patients assessed for eligibility as defined by diagnosis of hematologic or solid tumor malignancy and anticipated platelet counts ≤20,000/uL for ≥5 days
Measure Participants	693
Assessed for Eligibility	693 11550%
Screened for Complete Inclusion/Exclusion Criteria	148 2466.7%
Eligible for Enrollment	31 516.7%
Consented to Participate	11 183.3%

3. Secondary Outcome

Title	World Health Organization (WHO) Bleeding Scale Grade 2 or Higher Bleeding
Description	Proportion of patients with bleeding of WHO grade 2 or above, after activation of study drug. Bleeding graded on a scale ranging from 1 (minor bleeding) through 4 (bleeding that is fatal or life-threatening).
Time Frame	30 days after activation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Count of Participants [Participants]	0 0%	3 60%

4. Secondary Outcome

Title	Number of Platelet and Red Blood Cell Transfusions
Description	Number of platelet and red blood cell transfusions per patient during the first 30 days post prescription activation of study drug
Time Frame	30 days after activation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Number of platelet transfusions per patient	1.5	4
Number of pRBC transfusions per patient	0.5	1

5. Secondary Outcome

Title	Number of Days Alive and Without WHO Grade 2 Bleeding or Greater
Description	Number of days alive and without WHO grade 2 bleeding or greater during the first 30 days post activation of study drug. Bleeding graded on a scale ranging from 1 (minor bleeding) through 4 (bleeding that is fatal or life-threatening).
Time Frame	30 days after activation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Mean (Full Range) [days]	5.5	10.4

6. Secondary Outcome

Title	The Occurrence of Thromboembolic Adverse Events and Serious Adverse Events
Description	Any venous or arterial thrombosis on standard diagnostic imaging post-randomization
Time Frame	From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Number [events]	0	0

7. Secondary Outcome

Title	Bleeding of Any Grade
Description	Proportion of patients with bleeding of any grade as assessed by WHO bleeding score, after activation of study drug
Time Frame	From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Count of Participants [Participants]	4 66.7%	5 100%

8. Secondary Outcome

Title	Highest Observed Grade of Bleeding (as Measured on WHO Bleeding Scale) During the Study Period
Description	Highest grade of bleeding (as measured on WHO bleeding scale) during study period in each enrolled patient. Bleeding graded on a scale ranging from 1 (minor bleeding) through 4 (bleeding that is fatal or life-threatening).
Time Frame	From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Tranexamic Acid	Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria	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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
Measure Participants	6	5
Grade 1 Bleeding	4 66.7%	2 40%
Grade 2 Bleeding	0 0%	3 60%
Grade 3 Bleeding	0 0%	0 0%
Grade 4 Bleeding	0 0%	0 0%
Grade 5 Bleeding	0 0%	0 0%

Adverse Events

Time Frame	From the time of activation of the study drug up to and including 30 days after the last dose of the study drug, an average of 41 days
Arm/Group Title	Tranexamic Acid		Placebo
Arm/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). Tranexamic Acid: IV medication administered after patient meets inclusion/exclusion criteria		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. Normal saline: IV medication administered after patient meets inclusion/exclusion criteria
All Cause Mortality
	Tranexamic Acid		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/6 (0%)		0/5 (0%)
Serious Adverse Events
	Tranexamic Acid		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/6 (100%)		5/5 (100%)
Blood and lymphatic system disorders
White Blood Cell Decreased	6/6 (100%)		5/5 (100%)
Platelet Count Decreased	6/6 (100%)		5/5 (100%)
Febrile Neutropenia	0/6 (0%)		1/5 (20%)
Hepatobiliary disorders
Blood Bilirubin Increased	1/6 (16.7%)		0/5 (0%)
Other (Not Including Serious) Adverse Events
	Tranexamic Acid		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/6 (100%)		5/5 (100%)
Blood and lymphatic system disorders
Anemia	4/6 (66.7%)		4/5 (80%)
Febrile Neutropenia	2/6 (33.3%)		3/5 (60%)
Gastrointestinal disorders
Vomiting	0/6 (0%)		2/5 (40%)
Nausea	0/6 (0%)		1/5 (20%)
Mucositis Oral	0/6 (0%)		1/5 (20%)
General disorders
Fever	0/6 (0%)		1/5 (20%)
Infections and infestations
Catheter Related Infection	3/6 (50%)		1/5 (20%)
Sinusitis	0/6 (0%)		1/5 (20%)
Skin Infection	1/6 (16.7%)		0/5 (0%)
Metabolism and nutrition disorders
Anorexia	0/6 (0%)		2/5 (40%)
Hypoalbuminemia	1/6 (16.7%)		0/5 (0%)
Hypophosphatemia	0/6 (0%)		1/5 (20%)
Musculoskeletal and connective tissue disorders
Bone Pain	1/6 (16.7%)		0/5 (0%)
Respiratory, thoracic and mediastinal disorders
Hypoxia	0/6 (0%)		1/5 (20%)
Skin and subcutaneous tissue disorders
Rash Maculo-papular	0/6 (0%)		2/5 (40%)
Vascular disorders
Hypotension	0/6 (0%)		1/5 (20%)

Limitations/Caveats

The pre-specified analysis described in the study protocol stated the following: "For secondary endpoints, events of WHO Grade 2 or higher bleeding will be compared through the use of odds ratio or T-test statistic of means. The mean number of platelet transfusions will be analyzed using a T-test statistic of means." This analysis was not completed as it would be scientifically inappropriate due to insufficient enrollment. Instead a descriptive analysis was completed.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.