Evaluation of Tranexamic Acid in Myelodysplastic Syndromes and Acute Myeloid Leukemia (MYELO-CAN:TXA)

Evaluation of Tranexamic Acid Among Outpatients With Myelodysplastic Syndromes and Acute Myeloid Leukemia: a Multicenter Pilot Trial

Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are serious, life changing blood cancers. Patients with MDS and AML commonly experience complications related to bleeding, which affect patient quality-of-life and can sometimes lead to hospitalization or death. The investigators will conduct a randomized controlled trial to evaluate the effectiveness and safety of tranexamic acid (TXA; a medication that prevents clots from dissolving) to prevent bleeding. In this study, 50% of patients will be randomized (like the flip of a coin) to receive TXA; the other 50% of patients will receive placebo. The investigators will monitor both groups of patients to see if the medication improves the risk and/or severity of bleeding. If tranexamic acid were to safely reduced the frequency of bleeding, this would broadly influence how doctors provide care for patients with MDS and AML around the world.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Neoplasm
• Intervention / Treatment: Drug: Tranexamic acid; Drug: Placebo

Detailed Description

RATIONALE: Myelodysplastic syndromes (MDS), myelodysplastic/myeloproliferative neoplasm (MDS/MPN) and acute myeloid leukemia (AML) are serious, life-changing blood cancers. Despite the best efforts of their care team, patients with MDS and AML commonly experience complications related to bleeding. These complications affect patient quality-of-life and can sometimes lead to hospitalization or death. Evaluation of affordable and widely available treatments to minimize bleeding complications among patients with MDS and AML is needed.

STUDY OBJECTIVES: To evaluate the feasibility of tranexamic acid (TXA) that will evaluate the efficacy and safety of treatments to minimize bleeding in patients with MDS and AML treated in the outpatient setting.

METHODOLOGY: The investigators will conduct a multicenter pilot randomized control trial (RCT) for outpatients ≥18 years of age with MDS and AML. Patients with MDS and AML with low platelet counts will receive TXA (a medication that prevents clots from dissolving). TXA is commonly used in other clinical settings but have not been studied in patients with MDS or AML receiving outpatient chemotherapy (ie, chemotherapy that can be given from clinic, rather than a hospital). In this study, 50% of patients will be randomized (like the flip of a coin) to receive the medication the investigators are studying. The other 50% of patients will receive a matching placebo.

OUTCOMES: The primary feasibility outcome is the ability to enroll a mean of 1 patient per site per month.

SITES AND DURATION: The investigators will initially enroll patients from 10-15 sites across Canada. The expected duration of enrollment is 2 years.

SIGNIFICANCE: With a broad range of stakeholders, including patient partners, the trial will address a broadly applicable patient-prioritized question. Tranexamic acid is readily available, inexpensive, and has an established side effect profile. Results of this trial are highly generalizable and will broadly impact the care of patients with MDS and AML.

• Study Type: Interventional
• Enrollment (Estimated): 75
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Brett Houston, MD, PhD; Phone Number: 204-787-8552; Email: bhouston@cancercare.mb.ca
• Study Contact Backup: Name: Nora Choi, MSc; Phone Number: 204-787-8552; Email: nchoi@hsc.mb.ca

Study Locations

• Canada
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3E 0V9
      • Recruiting
      • CancerCare Manitoba
      • Contact: Brett Houston, MD; Phone Number: 204-787-1103; Email: bhouston@cancercare.mb.ca

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Master platform inclusion criteria:

1. Age ≥ 18 years
2. Diagnosis of myelodysplastic syndromes, myelodysplastic/myeloproliferative neoplasm or acute myeloid leukemia

MYELO-CAN TXA inclusion criteria:

1. Receipt of less-intensive chemotherapy (includes both frontline and relapsed/refractory setting)
2. Severe thrombocytopenia (platelets ≤ 30x10^9/L or platelets ≤ 50x10^9/L prior to chemotherapy initiation)

Exclusion Criteria:

Master platform exclusion criteria:

1. Participant is deemed unlikely to survive >30 days (as determined by clinical team)
2. Participant unable to provide informed consent

MYELO-CAN TXA exclusion criteria:

1. Known allergy to tranexamic acid
2. Active thromboembolic disease
3. Active ischemic heart disease
4. Gross hematuria
5. Stage V chronic kidney disease
6. Clinically suspected disseminated intravascular coagulation (DIC)
7. Pregnancy and/or breastfeeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Matching Placebo	Drug: Placebo; Placebo orally two or three times daily
Experimental: Tranexamic Acid	Drug: Tranexamic acid; Tranexamic acid 1000mg orally two or three times daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient enrollment feasibility	The ability to enroll a median of 1 patient per site per month (10 patients / month when all sites are active)	2 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Venous or arterial thromboembolism incidence	The incidence of venous or arterial thromboembolism will be measured as a safety outcome.	2 months
Catheter-associated thrombosis incidence	The incidence of catheter-associated thrombosis will be measured as a safety outcome.	2 months
Study drug discontinuation	Study drug discontinuation due to adverse events will be measured as a safety outcome.	2 months
Ability to consent 30% of eligible patients	The ability to consent 30% of eligible patients will be measured as a feasibility outcome.	2 months
Grade 3 and 4 nausea/vomiting	The incidence of grade 3 and 4 nausea/vomiting (CTCAE) will be measured as a safety outcome.	2 months
Visual disturbance incidence	The incidence of new visual disturbances will be measured as a safety outcome.	2 months
Medication adherence	Protocol adherence of 80% of all intended medication doses per patient will be measured as a feasibility outcome.	2 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Quality of life evaluation using PROMIS questionnaire	Quality of life evaluation at baseline and monthly using the Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue SF7a patient questionnaire.	2 months
Quality of life evaluation using EORTC questionnaire	Quality of life evaluation at baseline and monthly using the Core Quality of Life Questionnaire of the European Organisation For Research And Treatment Of Cancer (EORTC) C30 questionnaire.	2 months
Grade 2 to 5 bleeding incidence	The incidence of World Health Organization (WHO) defined grade 2 to 5 bleeding will be measured as a tertiary clinical outcome.	2 months
WHO bleeding incidence	The incidence of World Health Organization (WHO) bleeding stratified by grade will be measured as a clinical outcome.	2 months
Red blood cell transfusion exposure	The proportion of patients transfused red blood cells (RBC) will be measured as a clinical outcome.	2 months
Red blood cell transfusion volume	The mean/median number of red blood cell units transfused per patient will be measured as a clinical outcome.	2 months
Platelet transfusion volume	The mean/median number of platelet doses administered per patient will be measured as a clinical outcome.	2 months
Platelet transfusion exposure	The proportion of patients transfused with platelets will be measured as a clinical outcome.	2 months

Collaborators and Investigators

• Sponsor: University of Manitoba

Study record dates

Study Major Dates

• Study Start (Actual): September 10, 2025
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): April 1, 2027

Study Registration Dates

• First Submitted: September 3, 2024
• First Submitted That Met QC Criteria: September 12, 2024
• First Posted (Actual): September 19, 2024

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 20, 2026
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; Randomized Controlled Trial; Myelodysplastic Syndrome; Tranexamic Acid; Thrombocytopenia; myeloproliferative neoplasm; myelodysplastic/myeloproliferative neoplasm
• Additional Relevant MeSH Terms: Cytopenia; Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Leukemia; Blood Platelet Disorders; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Myeloproliferative Disorders; Myelodysplastic-Myeloproliferative Diseases; Thrombocytopenia; Organic Chemicals; Carboxylic Acids; Acids, Carbocyclic; Cyclohexanecarboxylic Acids; Tranexamic Acid
• Other Study ID Numbers: CHI-001-1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No