PRevention Of BLeeding in hEmatological Malignancies With Antifibrinolytic (Epsilon Aminocaproic Acid) (PROBLEMA)
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|ClinicalTrials.gov Identifier: NCT02074436|
Recruitment Status : Recruiting
First Posted : February 28, 2014
Last Update Posted : May 12, 2021
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.
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.
|Condition or disease||Intervention/treatment||Phase|
|Hematological Malignancies Thrombocytopenia||Drug: EACA Other: Platelet transfusion||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Randomized Trial of Epsilon Aminocaproic Acid Versus Platelet Transfusions for the Prevention of Bleeding in Thrombocytopenic Patients With Hematological Malignancies|
|Actual Study Start Date :||May 23, 2014|
|Estimated Primary Completion Date :||September 8, 2022|
|Estimated Study Completion Date :||December 1, 2022|
Experimental: Prophylactic EACA
Prophylactic EACA 1000 mg PO twice daily if platelets < 20 x 10⁹/L
EACA 1000 mg twice a day. Patients will receive platelet transfusion in case of grade ≥ 2 bleeding.
Active Comparator: Platelet transfusion
Platelet transfusion if platelet count is < 20 x 10⁹/L in the outpatient or < 10 x 10⁹/L in the inpatient setting
Other: Platelet transfusion
Prophylactic platelet transfusion. Additional platelet transfusions will be administered in case of grade ≥ 2 bleeding.
- Proportion of patients who develop major bleeding episodes (WHO grades 3-4) [ Time Frame: 6 months ]The primary objective of this study is to compare the proportion of patients who develop major bleeding episodes (WHO grades 3-4) in the group randomized to receive prophylactic EACA versus standard of care prophylactic platelet transfusions.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02074436
|Contact: Ana G. Antun, MD, MScfirstname.lastname@example.org|
|Contact: Shannon Gleasonemail@example.com|
|United States, Georgia|
|Emory University Hospital||Completed|
|Atlanta, Georgia, United States, 30322|
|Atlanta VA Medical Center||Recruiting|
|Decatur, Georgia, United States, 30033|
|Contact: Ana Antun, MD, MSc 404-593-6732 firstname.lastname@example.org|
|Principal Investigator: Wayne B. Harris, MD|
|Principal Investigator:||Ana G. Antun, MD, MSc||Emory University|