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MYELO-CAN: A Randomized Control Trial Platform

Overview

MYELO-CAN is a trial platform designed by patients and clinicians whose priority is to improve patient-prioritized outcomes for those affected by myelodysplastic syndrome and acute leukemia.  

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Study overview

Recruitment

Closed to recruitment March 5, 2024.

Number of patients enrolled: 8428

Number of Sites: 10

Developing enduring infrastructure to efficiently evaluate treatments for patients with Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

TitleEvaluation of supportive care treatments among outpatients with myelodysplastic syndrome and acute myeloid leukemia: a multicenter pilot trial (MYELO-CAN)
DescriptionMyelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) are serious, life changing blood cancers associated with significant morbidity and mortality. Morbidity is to a large extent due to anemia, neutropenia and thrombocytopenia (i.e. bone marrow failure). Despite the need for supportive care interventions such as transfusions and antimicrobials to manage these patients, evidence-informed outpatient supportive care strategies are lacking to prevent the most common adverse events (eg: bleeding and infection).
AimThe primary objective is to build clinical trial platform infrastructure to create a patient-centred learning health system to advance knowledge and improve patient outcomes, quality-of-life and survival in patients with MDS and AML.
EndpointsThis platform is intended to host trials focused on various aspects of MDS and AML care at different phases of investigation. Therefore, we cannot specify a single primary endpoint for the entire platform.

Two ongoing trials

1. Tranexamic Acid (TXA) Trial

Enrolling as of Sept 2025

Patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) commonly experience complications related to bleeding, despite the best efforts of their care team. 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.

Tranexamic acid (TXA) reduces clinically relevant bleeding events in a variety of settings. However, the efficacy and safety of TXA in outpatients with MDS and AML is unknown; clinical trials are needed to inform best practice.

2. Antibiotic Prophylaxis (ABX) Trial

Anticipated start Mar 2026

Patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) commonly experience complications related to infection, despite the best efforts of their care team. These complications affect patient quality-of-life and can sometimes lead to hospitalization or death. Evaluation of affordable and widely available treatments to minimize infection complications among patients with MDS and AML is needed. 

Levofloxacin (antibiotic) prophylaxis is shown to minimize febrile neutropenia and infectious complications in a variety of settings. However, the efficacy and safety of levofloxacin in outpatients with MDS and AML is unknown; clinical trials are needed to inform best practice. 

Details of our ongoing trials

1. Tranexamic Acid (TXA) Trial2. Antibiotic Prophylaxis (ABX) Trial
Protocol numberCHI-001-1CHI-001-2
TitleEvaluation of tranexamic acid among outpatients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML): a multicenter pilot trial (MYELO-CAN TXA)Evaluation of antibiotic prophylaxis in myelodysplastic syndrome and acute myeloid leukemia: a multicenter pilot trial (MYELO-CAN ABX)
AimTo evaluate trial feasibility to inform our larger phase III trial of TXA among at-risk patients. Ultimately, we will evaluate the efficacy and safety of TXA to minimize bleeding in patients with MDS and AML treated in the outpatient setting.To evaluate trial feasibility to inform our larger phase III trial of levofloxacin among at-risk patients. Ultimately, we will evaluate the efficacy and safety of levofloxacin prophylaxis to minimize infection in patients with MDS and AML treated in the outpatient setting.
Patient populationPatients with MDS and AML receiving less-intensive chemotherapy with severe thrombocytopenia (platelet count <30×109/L).Patients with MDS and AML receiving less-intensive chemotherapy who are at-risk for infection.
InterventionTranexamic acid 1 gram orally 2-3 times per day for 60 days.Levofloxacin 500mg orally once daily for 90 days.
ComparatorPlacebo orally 2-3 times per day for 60 days.Standard of care (no antibiotic prophylaxis).
Primary outcomeAbility to enrol a median of 1 patient / site / month. Ability to enroll a median of 1 patient / site / month.

FUNDERS AND COLLABORATORS

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Participating Sites

MYELO-CAN TEAM

Principal Investigators

UM Trial Operations Team