Evaluation of Tranexamic Acid in Myelodysplastic Syndromes and Acute Myeloid Leukemia

NCT ID: NCT06599762

Last Updated: 2025-09-17

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 75 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-09-10
• Study Completion Date: 2027-04-01

Brief Summary

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.

Detailed Description

RATIONALE: Myelodysplastic syndromes (MDS) 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.

Conditions

Myelodysplastic Syndromes; Acute Myeloid Leukemia

Study Design

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

Study Groups

Tranexamic Acid

Group Type: EXPERIMENTAL

Tranexamic acid

Intervention Type: DRUG

Tranexamic acid 1000mg orally two or three times daily

Matching Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo orally two or three times daily

Interventions

Tranexamic acid

Tranexamic acid 1000mg orally two or three times daily

Intervention Type: DRUG

Placebo

Placebo orally two or three times daily

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Age ≥ 18 years

2. Diagnosis of myelodysplastic syndromes or acute myeloid leukemia

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

1. Participant is deemed unlikely to survive >30 days (as determined by clinical team)

2. Participant unable to provide informed consent

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Manitoba

OTHER

Sponsor Role: lead

Responsible Party

Brett Houston

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

CancerCare Manitoba

Winnipeg, Manitoba, Canada

Site Status: RECRUITING

Countries

Canada

Central Contacts

Brett Houston, MD, PhD

Role: CONTACT

bhouston@cancercare.mb.ca

204-787-8552

Nora Choi, MSc

Role: CONTACT

nchoi@hsc.mb.ca

204-787-8552

Facility Contacts

Brett Houston, MD

Role: primary

bhouston@cancercare.mb.ca

204-787-1103

Other Identifiers

CHI-001-1

Identifier Type: -

Identifier Source: org_study_id