Pediatric CancerPhase 1/2NCT03806556

A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia

A Phase 1/2 study testing Tranexamic Acid and Normal saline in people with Pediatric Cancer and Thrombocytopenia, run by Meghan McCormick at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 2 to 21 years
Conditions the study is for: Pediatric Cancer, Thrombocytopenia, Hemostatic Disorder
What is being tested?: Tranexamic Acid and Normal saline
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 11 participants
Where: 1 siteUnited States
Study dates: 22 Apr 2019to 25 Aug 2020
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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

Who cannot join

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.

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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 locations and contacts

1 site in 1 country.

United States1 site

UPMC Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224

Other trials in United States

About this study

Read the 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
Being tested: Tranexamic Acid, Normal saline
Keywords: tranexamic acid, anti-fibrinolytic agents, chemotherapy-induced thrombocytopenia
Medical subject headings: Neoplasms, Thrombocytopenia, Hemostatic Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Tranexamic AcidDrug
Also called TXA
IV medication administered after patient meets inclusion/exclusion criteria
Normal salineDrug
Also called NS
IV medication administered after patient meets inclusion/exclusion criteria

Study groups in NCT03806556
Study group	Role	What they receive
Tranexamic Acid	Experimental	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). Receives: Drug: Tranexamic Acid
Placebo	Placebo comparator	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. Receives: Drug: Normal saline

What is being measured

The primary outcome is the main question the study is designed to answer.

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
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.
Measured: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug
Feasibility of Tranexamic Acid as an Adjunct to Standard Therapy: Number of Participants Eligible and Recruited
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.
Measured: From time of recruitment of the first patient until the last patient is enrolled, up to 16 months in duration

Secondary outcomes (6)

World Health Organization (WHO) Bleeding Scale Grade 2 or Higher Bleeding
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).
Measured: 30 days after activation of study drug
Number of Platelet and Red Blood Cell Transfusions
Number of platelet and red blood cell transfusions per patient during the first 30 days post prescription activation of study drug
Measured: 30 days after activation of study drug
Number of Days Alive and Without WHO Grade 2 Bleeding or Greater
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).
Measured: 30 days after activation of study drug
The Occurrence of Thromboembolic Adverse Events and Serious Adverse Events
Any venous or arterial thrombosis on standard diagnostic imaging post-randomization
Measured: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug
Bleeding of Any Grade
Proportion of patients with bleeding of any grade as assessed by WHO bleeding score, after activation of study drug
Measured: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug
Highest Observed Grade of Bleeding (as Measured on WHO Bleeding Scale) During the Study Period
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).
Measured: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Apr 201925 Aug 2020

First posted16 Jan 2019
Study start22 Apr 2019
Primary completion25 Aug 2020
Study completion25 Aug 2020

Why it stopped: Low accrual rate, strategic reasons

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Pediatric CancerStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
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.
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 11 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03806556
Sponsor's protocol ID: PRO18100519
Status last verified: Aug 2021
First submitted: 4 Jan 2019
Last updated: 20 Sep 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Anonymized public data set will be available after publication of primary results

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 20 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Pediatric Trial Using Tranexamic Acid in Thrombocytopenia
ClinicalTrials.gov ID: NCT03806556
Acronym / sponsor ID: PRO18100519
Conditions: Pediatric Cancer, Thrombocytopenia, Hemostatic Disorder, Coagulation Defect; Acquired
Keywords: tranexamic acid, anti-fibrinolytic agents, chemotherapy-induced thrombocytopenia
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Low accrual rate, strategic reasons
Enrolment: 11 participants (actual)
Lead sponsor: Meghan McCormickUniversity, hospital or other
Study start: 22 Apr 2019 (actual)
Primary completion: 25 Aug 2020 (actual)
Study completion: 25 Aug 2020 (actual)
First posted: 16 Jan 2019 (actual)
Results: Posted 20 Sep 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2
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.

Arm	Type	Receives
Tranexamic Acid	Experimental	Drug: Tranexamic AcidDoses 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).
Placebo	Placebo comparator	Drug: Normal salineDoses 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.

Who may qualify

Ages: 2 to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (7 listed)

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

Key exclusion criteria (18 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Tranexamic AcidAlso: TXAIV medication administered after patient meets inclusion/exclusion criteria	Drug	Tranexamic Acid
Normal salineAlso: NSIV medication administered after patient meets inclusion/exclusion criteria	Drug	Placebo

Primary outcome

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

6 secondary outcomes

World Health Organization (WHO) Bleeding Scale Grade 2 or Higher Bleeding
Time frame: 30 days after activation of study drug
Number of Platelet and Red Blood Cell Transfusions
Time frame: 30 days after activation of study drug
Number of Days Alive and Without WHO Grade 2 Bleeding or Greater
Time frame: 30 days after activation of study drug
The Occurrence of Thromboembolic Adverse Events and Serious Adverse Events
Time frame: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug
Bleeding of Any Grade
Time frame: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug
Highest Observed Grade of Bleeding (as Measured on WHO Bleeding Scale) During the Study Period
Time frame: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug

Results and safety

Results posted to the registry on 20 Sep 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Tranexamic Acid	6	6
Placebo	5	5

Primary outcome: 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

Time frame: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug. Count of participants. Unit: Participants

Measure	Tranexamic Acid n = 6	Placebo n = 5
Grade 3 Adverse Events : Anemia	4	4
Grade 3 Adverse Events : Anorexia	0	2
Grade 3 Adverse Events : Bone Pain	1	0
Grade 3 Adverse Events : Catheter Related Infection	3	1
Grade 3 Adverse Events : Vomiting	0	2
Grade 3 Adverse Events : Febrile Neutropenia	2	3
Grade 3 Adverse Events : Fever	0	1
Grade 3 Adverse Events : Hypoalbuminemia	1	0

Primary outcome: Feasibility of Tranexamic Acid as an Adjunct to Standard Therapy: Number of Participants Eligible and Recruited

Time frame: From time of recruitment of the first patient until the last patient is enrolled, up to 16 months in duration. Count of participants. Unit: Participants

Measure	Overall Study n = 693
Assessed for Eligibility	693
Screened for Complete Inclusion/Exclusion Criteria	148
Eligible for Enrollment	31
Consented to Participate	11

6 secondary outcomes are reported in the full results.

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. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Tranexamic Acid	6 of 6 (100%)	6 of 6 (100%)	0 of 6
Placebo	5 of 5 (100%)	5 of 5 (100%)	0 of 5

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. Anonymized public data set will be available after publication of primary results

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Meghan McCormick, MD principal investigator

UPMC Childrens Hospital of Pittsburgh

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Tranexamic Acid and Normal saline in people with Pediatric Cancer and Thrombocytopenia, run by Meghan McCormick at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 20 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Sep 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 (time frame: From activation of the study drug (maximum 30 days) through 30 days from discontinuation of study drug). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03806556.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Sep 2021.