Trial Outcomes & Findings for PRevention Of BLeeding in hEmatological Malignancies With Antifibrinolytic (Epsilon Aminocaproic Acid) (NCT NCT02074436)
NCT ID: NCT02074436
Last Updated: 2022-07-19
Results Overview
The primary objective of this study is to compare the percentage 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. Grade 3 bleeding is defined as a bleed that significantly limits the patient's ability to perform routine activities despite symptomatic therapy and leads to reduction of the dose or discontinuation of the study drug. Grade 4 bleeding is a life-threatening bleed that requires discontinuation of the study drug and the patient is at immediate risk of death
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
29 participants
Primary outcome timeframe
6 months
Results posted on
2022-07-19
Participant Flow
Participant milestones
| Measure |
Prophylactic EACA
Prophylactic EACA 1000 mg PO twice daily if platelets \< 20 x 10⁹/L
EACA: EACA 1000 mg twice a day. Patients will receive platelet transfusion in case of grade ≥ 2 bleeding.
|
Platelet Transfusion
Platelet transfusion if platelet count is \< 20 x 10⁹/L in the outpatient or \< 10 x 10⁹/L in the inpatient setting
Platelet transfusion: Prophylactic platelet transfusion. Additional platelet transfusions will be administered in case of grade ≥ 2 bleeding.
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
14
|
|
Overall Study
COMPLETED
|
15
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
PRevention Of BLeeding in hEmatological Malignancies With Antifibrinolytic (Epsilon Aminocaproic Acid)
Baseline characteristics by cohort
| Measure |
Prophylactic EACA
n=15 Participants
Prophylactic EACA 1000 mg PO twice daily if platelets \< 20 x 10⁹/L
EACA: EACA 1000 mg twice a day. Patients will receive platelet transfusion in case of grade ≥ 2 bleeding.
|
Platelet Transfusion
n=14 Participants
Platelet transfusion if platelet count is \< 20 x 10⁹/L in the outpatient or \< 10 x 10⁹/L in the inpatient setting
Platelet transfusion: Prophylactic platelet transfusion. Additional platelet transfusions will be administered in case of grade ≥ 2 bleeding.
|
Total
n=29 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
14 participants
n=7 Participants
|
29 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: Study was terminated early due to difficulties with enrollment. No outcome measures were assessed.
The primary objective of this study is to compare the percentage 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. Grade 3 bleeding is defined as a bleed that significantly limits the patient's ability to perform routine activities despite symptomatic therapy and leads to reduction of the dose or discontinuation of the study drug. Grade 4 bleeding is a life-threatening bleed that requires discontinuation of the study drug and the patient is at immediate risk of death
Outcome measures
| Measure |
Prophylactic EACA
n=11 Participants
Prophylactic EACA 1000 mg PO twice daily if platelets \< 20 x 10⁹/L
EACA: EACA 1000 mg twice a day. Patients will receive platelet transfusion in case of grade ≥ 2 bleeding.
|
Platelet Transfusion
n=11 Participants
Platelet transfusion if platelet count is \< 20 x 10⁹/L in the outpatient or \< 10 x 10⁹/L in the inpatient setting
Platelet transfusion: Prophylactic platelet transfusion. Additional platelet transfusions will be administered in case of grade ≥ 2 bleeding.
|
|---|---|---|
|
Percentage of Participants Who Develop Major Bleeding Episodes
Grade 3 Bleeding
|
0 Participants
|
11 Participants
|
|
Percentage of Participants Who Develop Major Bleeding Episodes
Grade 4 Bleeding
|
11 Participants
|
0 Participants
|
Adverse Events
Prophylactic EACA
Serious events: 9 serious events
Other events: 15 other events
Deaths: 5 deaths
Platelet Transfusion
Serious events: 8 serious events
Other events: 14 other events
Deaths: 5 deaths
Serious adverse events
| Measure |
Prophylactic EACA
n=15 participants at risk
Prophylactic EACA 1000 mg PO twice daily if platelets \< 20 x 10⁹/L
EACA: EACA 1000 mg twice a day. Patients will receive platelet transfusion in case of grade ≥ 2 bleeding.
|
Platelet Transfusion
n=14 participants at risk
Platelet transfusion if platelet count is \< 20 x 10⁹/L in the outpatient or \< 10 x 10⁹/L in the inpatient setting
Platelet transfusion: Prophylactic platelet transfusion. Additional platelet transfusions will be administered in case of grade ≥ 2 bleeding.
|
|---|---|---|
|
Cardiac disorders
Chest pain
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Psychiatric disorders
Bipolar Disorder
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Infections and infestations
Neutropenic Fever
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Infections and infestations
Sepsis
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Infections and infestations
Pneumonia
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Cardiac disorders
Pulmonary Edema
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Blood and lymphatic system disorders
Epistaxis
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Immune system disorders
Transfusion reaction
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Blood and lymphatic system disorders
Pancytopenia
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Gastrointestinal disorders
Gastrointestinal Bleed
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Nervous system disorders
Confusion
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
General disorders
Failure to Thrive
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
General disorders
Dehydration
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
Other adverse events
| Measure |
Prophylactic EACA
n=15 participants at risk
Prophylactic EACA 1000 mg PO twice daily if platelets \< 20 x 10⁹/L
EACA: EACA 1000 mg twice a day. Patients will receive platelet transfusion in case of grade ≥ 2 bleeding.
|
Platelet Transfusion
n=14 participants at risk
Platelet transfusion if platelet count is \< 20 x 10⁹/L in the outpatient or \< 10 x 10⁹/L in the inpatient setting
Platelet transfusion: Prophylactic platelet transfusion. Additional platelet transfusions will be administered in case of grade ≥ 2 bleeding.
|
|---|---|---|
|
Cardiac disorders
Chest Pain
|
6.7%
1/15 • Number of events 2 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Cardiac disorders
Hypertension
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Cardiac disorders
Hypotension
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Cardiac disorders
Lower extremity Edema
|
33.3%
5/15 • Number of events 5 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Cardiac disorders
Sinus Tachycardia
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Psychiatric disorders
Depression
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Infections and infestations
Neutropenic Fever
|
20.0%
3/15 • Number of events 4 • Through study completion, an average of 6 months.
|
42.9%
6/14 • Number of events 7 • Through study completion, an average of 6 months.
|
|
Infections and infestations
Sepsis
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Infections and infestations
Mucositis
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.3%
2/15 • Number of events 3 • Through study completion, an average of 6 months.
|
28.6%
4/14 • Number of events 4 • Through study completion, an average of 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.0%
3/15 • Number of events 5 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Hemoptysis
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Blood and lymphatic system disorders
Epistaxis
|
26.7%
4/15 • Number of events 17 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Blood and lymphatic system disorders
Anemia
|
13.3%
2/15 • Number of events 3 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Skin and subcutaneous tissue disorders
Bruising
|
40.0%
6/15 • Number of events 7 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Blood and lymphatic system disorders
Oral Bleeding
|
53.3%
8/15 • Number of events 18 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
40.0%
6/15 • Number of events 8 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Eye disorders
Subconjunctival Hemorrhage
|
6.7%
1/15 • Number of events 3 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Gastrointestinal disorders
Abdominal Pain
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Gastrointestinal disorders
Diarrhea
|
13.3%
2/15 • Number of events 3 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Gastrointestinal disorders
Nausea
|
13.3%
2/15 • Number of events 3 • Through study completion, an average of 6 months.
|
42.9%
6/14 • Number of events 8 • Through study completion, an average of 6 months.
|
|
Gastrointestinal disorders
Liver enzymes
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Nervous system disorders
Headache
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Nervous system disorders
Confusion
|
13.3%
2/15 • Number of events 3 • Through study completion, an average of 6 months.
|
0.00%
0/14 • Through study completion, an average of 6 months.
|
|
Endocrine disorders
Hyperglycemia
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 4 • Through study completion, an average of 6 months.
|
|
Renal and urinary disorders
Acute Kidney Injury
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
Endocrine disorders
Hypokalemia
|
33.3%
5/15 • Number of events 5 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Renal and urinary disorders
Hematuria
|
26.7%
4/15 • Number of events 6 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
Reproductive system and breast disorders
Menorrhagia
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
General disorders
chills
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
General disorders
Anorexia
|
20.0%
3/15 • Number of events 3 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
General disorders
Dehydration
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
21.4%
3/14 • Number of events 3 • Through study completion, an average of 6 months.
|
|
General disorders
Fatigue
|
33.3%
5/15 • Number of events 9 • Through study completion, an average of 6 months.
|
50.0%
7/14 • Number of events 8 • Through study completion, an average of 6 months.
|
|
General disorders
Hiccups
|
0.00%
0/15 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
General disorders
Low grade fever
|
13.3%
2/15 • Number of events 2 • Through study completion, an average of 6 months.
|
14.3%
2/14 • Number of events 2 • Through study completion, an average of 6 months.
|
|
General disorders
Generalized weakness
|
20.0%
3/15 • Number of events 3 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
|
General disorders
Weight loss
|
6.7%
1/15 • Number of events 1 • Through study completion, an average of 6 months.
|
7.1%
1/14 • Number of events 1 • Through study completion, an average of 6 months.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place