Evaluation of Tranexamic Acid in Myelodysplastic Syndromes and Acute Myeloid Leukemia

Not Applicable

Recruiting

• Conditions: Myelodysplastic Syndromes; Acute Myeloid Leukemia
• Interventions: Drug: Tranexamic acid; Drug: Placebo

• Registration Number: NCT06599762
• Lead Sponsor: University of Manitoba

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.

Study Timeline

• Study First Submitted: 2024-09-03
• Study First Submitted QC: 2024-09-12
• Study First Posted: 2024-09-19
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-05
• Last Update Posted: 2025-05-08
• Study Start: 2025-05-12
• Primary Completion: 2027-04-01
• Study Completion: 2027-04-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

Master platform inclusion criteria:

1. Age ≥ 18 years
2. Diagnosis of myelodysplastic syndromes 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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Tranexamic Acid	Tranexamic acid	-
Matching Placebo	Placebo	-

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

CancerCare Manitoba; 🇨🇦 Winnipeg, Manitoba, Canada