PRevention Of BLeeding in hEmatological Malignancies With Antifibrinolytic (Epsilon Aminocaproic Acid)

• Status: Recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

Summary

STUDY BACKGROUND AND PURPOSE:

Patients with hematological malignancies (blood-related cancers) often develop thrombocytopenia (low platelet count), which can be made worse by cancer treatment.

Preventive (prophylactic) platelet transfusion remains the standard of care for thrombocytopenic patients. However, bleeding remains a significant problem in these patients, affecting approximately 20% of patients with acute myeloid leukemia and 34-58% of hematopoietic stem cell transplant recipients. Platelet transfusion refractoriness, the repeated failure to obtain satisfactory response to platelet transfusions, is a common problem. Alternatives to platelet transfusions are desperately needed for these patients.

Epsilon aminocaproic acid (EACA) blocks a process called fibrinolysis that is an essential step in the bleeding process. EACA is approved by the FDA for the treatment of severe bleeding-related diseases and complications. A small study has shown EACA to be well tolerated and associated with low risk of bleeding in patients with hematological malignancies.

This study will compare EACA versus standard prophylactic platelet transfusion for the prevention of bleeding in thrombocytopenic patients with hematological malignancies.

STUDY DESCRIPTION:

This is Phase II study to compare EACA versus standard prophylactic platelet transfusion to prevent bleeding in thrombocytopenic patients with hematological malignancies. Patients who are eligible to take part must give their written agreement before they can be enrolled.

The study will enroll 100 patients who will be assigned randomly to take EACA twice daily or to undergo standard prophylactic platelet transfusion. Patients will be followed for any bleeding events, need for platelet transfusion, and any side effects experienced. Patients will complete questionnaires to assess their quality of life while on the study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Emory University
• Collaborator: Akorn, Inc.
• Treatments: Aminocaproic Acid

Criteria

Inclusion Criteria:

• Age > 18 with a hematological malignancy

• Informed consent

• Thrombocytopenia with untransfused platelet counts < 20 x 10⁹/L in the out-patient or in the in-patient setting and one of the following criteria:

• Acute thrombocytopenia in patients with hematological malignancies who are in remission and are receiving myelosuppressive consolidation chemotherapy that is expected to induce marrow aplasia for at least 2 weeks; or

• Acute or chronic thrombocytopenia in patients with acute leukemia (myeloblastic or lymphoblastic) receiving induction or re-induction chemotherapy that is expected to induce marrow aplasia for at least 2 weeks; or

• Expected chronic thrombocytopenia in patients with newly diagnosed marrow failure syndromes, myelodysplastic syndromes, aplastic anemia, chronic myelomonocytic leukemia or myelofibrosis; or

• Expected chronic thrombocytopenia in patients with relapsed or refractory hematological malignancies; or

• Hematopoietic stem cell transplant recipients with chronic thrombocytopenia due to chronic graft-versus-host disease (GVHD) or other causes

Exclusion Criteria:

• Acute promyelocytic leukemia

• Patient receiving anticoagulation

• Patient receiving antiplatelet agents

• Patient treated with antifibrinolytic agents (including EACA) within 14 days prior to screening

• Subjects receiving procoagulant agent including DDAVP, recombinant factor VII or prothrombin complex concentrate within 24 hours of enrollment

• Subject with known congenital bleeding disorders or platelet dysfunction

• Disseminated intravascular coagulation

• Fibrinogen level < 150 mg/dl

• Patients with known lupus anticoagulant or positive antiphospholipid antibody

• History of arterial or venous thromboembolic disease 6 months prior to screening

• Patient requiring platelet transfusion threshold of > 20 x 10⁹/L

• Active grade ≥ 2 bleeding at the time of randomization, including hematuria

• History of grade 4 bleeding

• Hematopoietic stem cell transplant recipient within 100 days post-transplant

• Pregnancy

• Known allergy to EACA

• History of veno-occlusive disease of the liver

• Myocardial infarction 6 months prior to screening

• Unstable angina

• Grade 2 renal dysfunction: creatinine > 2-3 times the upper limit of normal