Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2 |
Blinding Index (BI) score at the end of cycle 4 of treatment period 1 and 2 |
2 years |
|
Primary |
Proportion of participant drop-out at the end of treatment period 1 and 2 |
Proportion of participant drop-out at the end of treatment period 1 and 2 |
2 years |
|
Primary |
Proportion of participants with completed for the candidate primary clinical efficacy outcomes at the end of treatment period 1 and 2 |
Proportion of patients with completed for the candidate primary clinical efficacy outcomes at the end of treatment period 1 and 2 |
2 years |
|
Primary |
Number of participants enrolled in 2 years (i.e. ability to enroll at least 10 participants in 2 years) |
Ability to enroll at least 10 participants in 2 years |
2 years |
|
Primary |
Proportion of participants with carryover effect for the candidate primary clinical efficacy outcomes from period 1 to period 2 |
Proportion of participants with carryover effect for the candidate primary clinical efficacy outcomes from period 1 to period 2 |
2 years |
|
Secondary |
Mean of the 3 highest daily Modified PBAC (mPBAC) scores within each individual participant cycle averaged across 4 individual participant cycles at the end of each treatment period |
Mean of the 3 highest daily Modified PBAC (mPBAC) scores within each individual participant cycle averaged across 4 individual participant cycles at the end of each treatment period |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
The proportion of patients who use of rescue therapy at the end of each treatment period |
The proportion of patients who use of rescue therapy (i.e. more than two days of oral tranexamic acid use, additional treatment with Wilate®, additional hormonal therapy for HMB, urgent/emergent gynecological surgery for HMB, treatment with intravenous iron, red blood cell transfusion, or hospital admission for HMB) at the end of each treatment period |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Mean of the mPBAC score within each individual participant cycle averaged across 4 individual participant cycles |
Mean of the mPBAC score within each individual participant cycle averaged across 4 individual participant cycles |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Median of the mPBAC score within each individual participant cycle used to derive the median across 4 individual participant cycles |
Median of the mPBAC score within each individual participant cycle used to derive the median across 4 individual participant cycles |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Number of days of oral tranexamic acid use |
Number of days of oral tranexamic acid use |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Number of days of Wilate® treatment received |
Number of days of Wilate® treatment received |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Duration of menstruation (measured in days) |
Duration of menstruation (measured in days) |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Major bleed according to the International Society on Thrombosis and Haemostasis (ISTH) definition |
Major bleed according to the International Society on Thrombosis and Haemostasis (ISTH) definition |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Clinically relevant non-major bleed |
Clinically relevant non-major bleed |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Hemoglobin levels (g/L) |
Hemoglobin levels (g/L) |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Ferritin levels (mcg/L) |
Ferritin levels (mcg/L) |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Use of additional hormonal therapy for heavy menstrual bleeding |
Use of additional hormonal therapy for heavy menstrual bleeding |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Requirement of urgent/emergent gynecological surgery for heavy menstrual bleeding |
Requirement of urgent/emergent gynecological surgery for heavy menstrual bleeding |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Fatigue scores (as measured by the FACIT fatigue scale) |
Fatigue scores (as measured by the FACIT fatigue scale) |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Short Form-12 Scores |
Short Form-12 Scores |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Scores on the individual components of the Short Form-12 |
Scores on the individual components of the Short Form-12 |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Requirement of hemostatic care (tranexamic acid, Wilate®, platelet transfusion) |
Requirement of hemostatic care (tranexamic acid, Wilate®, platelet transfusion) |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Requirement of anemia focused care (intravenous iron and/or red blood cell transfusion) |
Requirement of anemia focused care (intravenous iron and/or red blood cell transfusion) |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Number of hypersensitivity infusion reactions |
Number of hypersensitivity infusion reactions |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Number of thromboembolic events (defined as symptomatic or incidental, suspected or confirmed via diagnostic imaging and/or electrocardiogram where appropriate); |
Number of thromboembolic events (defined as symptomatic or incidental, suspected or confirmed via diagnostic imaging and/or electrocardiogram where appropriate); |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Proportion of participants who development of VWF inhibitors |
Proportion of participants who development of VWF inhibitors |
At the end of 8 menstrual cycles (approximately 10 days) |
|
Secondary |
Number of adverse events |
Number of adverse events |
8 cycles |
|