Trial Outcomes & Findings for Minimize Menorrhagia in Women With Von Willebrand Disease (NCT NCT02606045)
NCT ID: NCT02606045
Last Updated: 2024-07-01
Results Overview
As measured by Pictorial Blood Assessment Chart (PBAC), 0- no theoretical limit, higher score means greater severity
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
39 participants
Primary outcome timeframe
4 weeks.
Results posted on
2024-07-01
Participant Flow
Participant milestones
| Measure |
Recombinant Von Willebrand Factor, Then Tranexamic Acid
Group I will receive Arm A recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5 of each of two menstrual cycles, Cycles 3 and 4.
|
Tranexamic Acid Then Recombinant Von Willebrand Factor
Group II will receive Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm A, recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 on each of two menstrual cycles, Cycles 3 and 4.
|
|---|---|---|
|
Overall Study
STARTED
|
18
|
21
|
|
Overall Study
COMPLETED
|
17
|
19
|
|
Overall Study
NOT COMPLETED
|
1
|
2
|
Reasons for withdrawal
| Measure |
Recombinant Von Willebrand Factor, Then Tranexamic Acid
Group I will receive Arm A recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5 of each of two menstrual cycles, Cycles 3 and 4.
|
Tranexamic Acid Then Recombinant Von Willebrand Factor
Group II will receive Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm A, recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 on each of two menstrual cycles, Cycles 3 and 4.
|
|---|---|---|
|
Overall Study
Study closed before treament could begin
|
1
|
2
|
Baseline Characteristics
Minimize Menorrhagia in Women With Von Willebrand Disease
Baseline characteristics by cohort
| Measure |
Group I
n=17 Participants
Group I will receive Arm A recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5 of each of two menstrual cycles, Cycles 3 and 4.
|
Group II
n=19 Participants
Group II will receive Arm B, tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, Cycles 1 and 2. They will then be crossed over to Arm A, recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 on each of two menstrual cycles, Cycles 3 and 4.
|
Total
n=36 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
17 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
31.9 years
STANDARD_DEVIATION 6.3 • n=5 Participants
|
26.7 years
STANDARD_DEVIATION 10.3 • n=7 Participants
|
29.2 years
STANDARD_DEVIATION 8.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 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
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 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
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
25 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
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
17 participants
n=5 Participants
|
19 participants
n=7 Participants
|
36 participants
n=5 Participants
|
|
Menstrual Bleeding Severity
|
597 score on a scale
STANDARD_DEVIATION 389 • n=5 Participants
|
483 score on a scale
STANDARD_DEVIATION 270 • n=7 Participants
|
537 score on a scale
STANDARD_DEVIATION 331 • n=5 Participants
|
PRIMARY outcome
Timeframe: 4 weeks.As measured by Pictorial Blood Assessment Chart (PBAC), 0- no theoretical limit, higher score means greater severity
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Menstrual Bleeding Severity
|
225 score on a scale
Standard Deviation 207
|
272 score on a scale
Standard Deviation 223
|
SECONDARY outcome
Timeframe: 4 weeks.Menorrhagia cycle length by days of bleeding (score 0 - theoretically any length)
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Cycle Duration
|
7.1 days
Standard Deviation 4.5
|
7.0 days
Standard Deviation 3.9
|
SECONDARY outcome
Timeframe: 4 weeks.Unit measure derived from quality of life questions reported as a single score, Minimum 0 to 100 maximum, with a higher score indicating worse outcome
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Ruta Menorrhagia Severity Scale
|
32.4 units on a scale
Standard Deviation 13.5
|
34.8 units on a scale
Standard Deviation 13.0
|
SECONDARY outcome
Timeframe: 4 weeks.Reported by questions on depression, Minimum 0 to 60 Maximum, with higher scores indicating greater depression.
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Center for Epidemiology Studies Depression Scale (CES-D)
|
13.5 score on a scale
Standard Deviation 10.6
|
11.0 score on a scale
Standard Deviation 10.2
|
SECONDARY outcome
Timeframe: 4 weeksSF-36 reported by answer to questions, Minimum 0 to Maximum 100 for each of 8 domains (1-physical functioning, 2-role limitations due to physical health, 3-role limitations due to emotional problems, 4-energy and fatigue, 5-emotional wellbeing, 6-social functioning, 7-pain, and 8-general health), with higher scores for each domain meaning greater severity of physical and mental health.
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Rand Short Form 36-Question Health Survey (SF-36)
General health
|
58.8 units on a scale
Standard Deviation 19.9
|
59.3 units on a scale
Standard Deviation 21.2
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Physical functioning
|
84.4 units on a scale
Standard Deviation 19.6
|
83.6 units on a scale
Standard Deviation 20.0
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Role limitations of physical health
|
70.1 units on a scale
Standard Deviation 39.1
|
67.4 units on a scale
Standard Deviation 37.7
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Role limitations of emotional health
|
63.9 units on a scale
Standard Deviation 41.7
|
68.5 units on a scale
Standard Deviation 39.0
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Energy/fatigue
|
50.3 units on a scale
Standard Deviation 20.6
|
47.9 units on a scale
Standard Deviation 22.8
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Emotional well-being
|
69.2 units on a scale
Standard Deviation 17.7
|
72.9 units on a scale
Standard Deviation 20.0
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Social functioning
|
76.4 units on a scale
Standard Deviation 21.9
|
80.9 units on a scale
Standard Deviation 20.2
|
|
Rand Short Form 36-Question Health Survey (SF-36)
Pain
|
71.2 units on a scale
Standard Deviation 23.5
|
68.8 units on a scale
Standard Deviation 34.2
|
SECONDARY outcome
Timeframe: 4 weeks.Reported by answer to questions, over the past two weeks with minimum 0 to maximum 14, with higher scores indicating greater severity of physical and mental health.
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Center for Disease Control Health-Related Quality of Life 14 Questions (CDC-HRQoL-14)
Physically Unhealthy Days
|
3.1 units on a scale
Standard Deviation 4.0
|
1.9 units on a scale
Standard Deviation 3.2
|
|
Center for Disease Control Health-Related Quality of Life 14 Questions (CDC-HRQoL-14)
Mentally Unhealthy Days
|
4.7 units on a scale
Standard Deviation 5.1
|
5.7 units on a scale
Standard Deviation 7.0
|
SECONDARY outcome
Timeframe: 4 weeksMenorrhagia cycle severity by Likert scale, with minimum 0 to maximum 3, with higher score indicating worse severity.
Outcome measures
| Measure |
Tranexamic Acid
n=36 Participants
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 Participants
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Cycle Severity
|
0.96 units on a scale
Standard Deviation 0.879
|
1.07 units on a scale
Standard Deviation 0.738
|
Adverse Events
Tranexamic Acid
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Recombinant Von Willebrand Factor
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Tranexamic Acid
n=36 participants at risk
Participants who received tranexamic acid 650 mg 2 tablets orally (po) three times daily on days 1-5, for each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
Recombinant Von Willebrand Factor
n=36 participants at risk
Participants who received recombinant von Willebrand factor 40 IU/kg intravenously (IV) infusion on day 1 of each of two menstrual cycles, in either first 2 cycle weeks of the study or the last 2 cycle weeks of the study
|
|---|---|---|
|
Blood and lymphatic system disorders
Mucosal Bleeding
|
5.6%
2/36 • Number of events 4 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
0.00%
0/36 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
|
Blood and lymphatic system disorders
Other Bleeding
|
8.3%
3/36 • Number of events 4 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
5.6%
2/36 • Number of events 2 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
|
Blood and lymphatic system disorders
Hematoma
|
0.00%
0/36 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
2.8%
1/36 • Number of events 1 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
|
Blood and lymphatic system disorders
Injury/ Trauma Bleeding
|
0.00%
0/36 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
2.8%
1/36 • Number of events 1 • Adverse Events were collected over collected a 24 week period
Includes all participants who received study intervention
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60