Clinical trial NCT06599762: Evaluation of Tranexamic Acid in Myelodysplastic Syndromes and Acute Myeloid Leukemia
Official title: Evaluation of Tranexamic Acid Among Outpatients With Myelodysplastic Syndromes and Acute Myeloid Leukemia: a Multicenter Pilot Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Myelodysplastic Syndromes
Acute Myeloid Leukemia
Keywords: Tranexamic Acid
Randomized Controlled Trial
Myelodysplastic Syndrome
Acute Myeloid Leukemia
Thrombocytopenia
Lead sponsor: University of Manitoba
Sponsor class: OTHER
First posted: 2024-09-19
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Randomized controlled pilot trial
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Patient enrollment feasibility
timeFrame: 2 months
Secondary outcomes:
measure: Venous or arterial thromboembolism incidence
timeFrame: 2 months
measure: Catheter-associated thrombosis incidence
timeFrame: 2 months
measure: Study drug discontinuation
timeFrame: 2 months
measure: Ability to consent 30% of eligible patients
timeFrame: 2 months
measure: Grade 3 and 4 nausea/vomiting
timeFrame: 2 months
measure: Visual disturbance incidence
timeFrame: 2 months
measure: Medication adherence
timeFrame: 2 months
Other outcomes:
measure: Quality of life evaluation using PROMIS questionnaire
timeFrame: 2 months
measure: Quality of life evaluation using EORTC questionnaire
timeFrame: 2 months
measure: Grade 2 to 5 bleeding incidence
timeFrame: 2 months
measure: WHO bleeding incidence
timeFrame: 2 months
measure: Red blood cell transfusion exposure
timeFrame: 2 months
measure: Red blood cell transfusion volume
timeFrame: 2 months
measure: Platelet transfusion volume
timeFrame: 2 months
measure: Platelet transfusion exposure
timeFrame: 2 months
Locations:
city: Winnipeg
country: Canada
facility: CancerCare Manitoba
state: Manitoba
Study officials:
References: