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A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors (STASEY)

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ClinicalTrials.gov Identifier: NCT03191799
Recruitment Status : Completed
First Posted : June 19, 2017
Results First Posted : June 11, 2021
Last Update Posted : June 11, 2021
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia A
Intervention Drug: Emicizumab
Enrollment 195
Recruitment Details  
Pre-assignment Details A total of 195 patients were enrolled in the clinical trial.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Period Title: Overall Study
Started [1] 195
Received at Least One Dose of Emicizumab [2] 193
Dose Up-Titrated to 3 mg/kg QW 2
Completed 186
Not Completed 9
Reason Not Completed
Withdrawal by Subject             4
Physician Decision             2
Lost to Follow-up             1
Death             2
[1]
Intent-to-Treat (ITT) Population
[2]
Safety Population
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Baseline Participants 195
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 195 participants
32.2  (16.6)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 195 participants
≥12 to <18 Years Old
40
  20.5%
≥18 to <65 Years Old
146
  74.9%
≥65 Years Old
9
   4.6%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 195 participants
Female
0
   0.0%
Male
195
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 195 participants
Hispanic or Latino
41
  21.0%
Not Hispanic or Latino
143
  73.3%
Unknown or Not Reported
11
   5.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 195 participants
American Indian or Alaska Native
19
   9.7%
Asian
38
  19.5%
Native Hawaiian or Other Pacific Islander
1
   0.5%
Black or African American
7
   3.6%
White
121
  62.1%
More than one race
0
   0.0%
Unknown or Not Reported
9
   4.6%
1.Primary Outcome
Title Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale
Hide Description Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. aPCC = activated prothrombin complex concentrate
Time Frame From Baseline until study completion (up to 2 years)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Measure Type: Count of Participants
Unit of Measure: Participants
Any Adverse Event (AE)
163
  84.5%
Fatal AE
2
   1.0%
Serious AE
31
  16.1%
AE Leading to Withdrawal from Treatment
1
   0.5%
AE Leading to Dose Modification/Interruption
4
   2.1%
AE Leading to Study Discontinuation
1
   0.5%
Grade 3-5 AE
39
  20.2%
Related AE
35
  18.1%
Local Injection Site Reaction
22
  11.4%
Systemic Hypersensitivity/Anaphylac(tic/toid) Reaction
0
   0.0%
Thromboembolic Event (TE)
2
   1.0%
TE Related to aPCC and Emicizumab
0
   0.0%
Thrombotic Microangiopathy (TMA)
0
   0.0%
TMA Related to aPCC and Emicizumab
0
   0.0%
Cases of Potential Drug-Induced Liver Injury
0
   0.0%
Suspected Transmission of Infectious Agent by the Study Drug
0
   0.0%
2.Primary Outcome
Title Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade ≥3 AEs
Hide Description Investigators sought information on adverse events (AEs) at each contact with participants. The WHO toxicity grading scale was used for assessing AE severity (i.e., intensity of an AE); any AEs not specifically listed in the WHO toxicity grading scale were assessed for severity according to the following grades: Grade 1 is mild; Grade 2 is moderate, Grade 3 is severe; Grade 4 is life-threatening; and Grade 5 is death. Regardless of severity, some AEs may have also met seriousness criteria. The terms "severe" and "serious" are not synonymous; severity and seriousness were independently assessed for each AE. The AE rate per 100 patient-years was computed as follows: AE Rate = (Number of AEs observed/ Total patient-years at risk)*100. Total patient-years at risk is the sum over all patients of the time intervals (in years) between start of study therapy (study day 1) and the end of follow up.
Time Frame From Baseline until study completion (up to 2 years)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Overall Number of Units Analyzed
Type of Units Analyzed: Adverse Events (AEs)
 800
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: AEs per 100 patient-years
All-Grade AEs Number Analyzed 800 Adverse Events (AEs)
211.92
(197.49 to 227.13)
Serious AEs Number Analyzed 50 Adverse Events (AEs)
13.25
(9.83 to 17.46)
Grade ≥3 AEs Number Analyzed 65 Adverse Events (AEs)
17.22
(13.29 to 21.95)
3.Primary Outcome
Title Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline
Hide Description The World Health Organization (WHO) toxicity grading scale was used for determining the severity of laboratory abnormalities (i.e., test results outside of the reference range) for hematology and biochemistry parameters; Grade 0 is normal and Grades 1 to 4 represent worsening levels of the parameter outside of the normal range in the specified direction of the abnormality (high and low are above and below the range, respectively). Not every laboratory abnormality qualified as an adverse event (AE). A laboratory test result was reported as an AE if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment; resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment. Baseline was defined as the last available assessment prior to first receipt of study drug. Abs = absolute count; SGOT/AST = aspartate aminotransferase; SGPT/ALT = alanine aminotransferase
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Measure Type: Count of Participants
Unit of Measure: Participants
Hemoglobin, Low - Grade 0 to 0
173
  89.6%
Hemoglobin, Low - Grade 0 to 1
8
   4.1%
Hemoglobin, Low - Grade 0 to 2
4
   2.1%
Hemoglobin, Low - Grade 0 to 4
1
   0.5%
Hemoglobin, Low - Grade 1 to 0
1
   0.5%
Hemoglobin, Low - Grade 1 to 1
2
   1.0%
Hemoglobin, Low - Grade 1 to 2
1
   0.5%
Hemoglobin, Low - Grade 1 to 3
1
   0.5%
Hemoglobin, Low - Grade 2 to 1
1
   0.5%
Hemoglobin, Low - Grade 2 to 3
1
   0.5%
Neutrophils, Total (Abs), Low - Grade 0 to 0
173
  89.6%
Neutrophils, Total (Abs), Low - Grade 0 to 1
13
   6.7%
Neutrophils, Total (Abs), Low - Grade 0 to 2
1
   0.5%
Neutrophils, Total (Abs), Low - Grade 0 to 3
2
   1.0%
Neutrophils, Total (Abs), Low - Grade 0 to 4
2
   1.0%
Neutrophils, Total (Abs), Low - Grade 1 to 1
2
   1.0%
Platelets, Low - Grade 0 to 0
190
  98.4%
Platelets, Low - Grade 0 to 1
2
   1.0%
Platelets, Low - Grade 1 to 0
1
   0.5%
Alkaline Phosphatase, High - Grade 0 to 0
187
  96.9%
Alkaline Phosphatase, High - Grade 0 to 1
3
   1.6%
Alkaline Phosphatase, High - Grade 0 to 2
1
   0.5%
Alkaline Phosphatase, High - Grade 1 to 1
2
   1.0%
Bilirubin, High - Grade 0 to 0
169
  87.6%
Bilirubin, High - Grade 0 to 1
14
   7.3%
Bilirubin, High - Grade 1 to 0
3
   1.6%
Bilirubin, High - Grade 1 to 1
2
   1.0%
Bilirubin, High - Grade 1 to 2
3
   1.6%
Bilirubin, High - Grade 2 to 2
2
   1.0%
Blood Urea Nitrogen, High - Grade 0 to 0
187
  96.9%
Blood Urea Nitrogen, High - Grade 0 to 1
5
   2.6%
Blood Urea Nitrogen, High - Grade 1 to 1
1
   0.5%
Calcium (Corrected), High - Grade 0 to 0
192
  99.5%
Calcium (Corrected), High - Grade 1 to 2
1
   0.5%
Calcium (Corrected), Low - Grade 0 to 0
173
  89.6%
Calcium (Corrected), Low - Grade 0 to 1
15
   7.8%
Calcium (Corrected), Low - Grade 0 to 2
1
   0.5%
Calcium (Corrected), Low - Grade 1 to 0
3
   1.6%
Calcium (Corrected), Low - Grade 1 to 1
1
   0.5%
Creatinine, High - Grade 0 to 0
185
  95.9%
Creatinine, High - Grade 0 to 1
5
   2.6%
Creatinine, High - Grade 0 to 2
1
   0.5%
Creatinine, High - Grade 1 to 1
1
   0.5%
Creatinine, High - Grade 1 to 2
1
   0.5%
Glucose, High - Grade 0 to 0
148
  76.7%
Glucose, High - Grade 0 to 1
28
  14.5%
Glucose, High - Grade 0 to 2
6
   3.1%
Glucose, High - Grade 1 to 1
2
   1.0%
Glucose, High - Grade 1 to 2
3
   1.6%
Glucose, High - Grade 2 to 2
1
   0.5%
Glucose, High - Grade 2 to 3
1
   0.5%
Glucose, High - Grade 3 to 2
1
   0.5%
Glucose, High - Grade 3 to 3
2
   1.0%
Glucose, High - Grade 4 to 3
1
   0.5%
Glucose, Low - Grade 0 to 0
180
  93.3%
Glucose, Low - Grade 0 to 1
7
   3.6%
Glucose, Low - Grade 0 to 2
4
   2.1%
Glucose, Low - Grade 1 to 0
2
   1.0%
Magnesium, Low - Grade 0 to 0
188
  97.4%
Magnesium, Low - Grade 0 to 1
4
   2.1%
Magnesium, Low - Grade 1 to 1
1
   0.5%
Phosphorus, Low - Grade 0 to 0
160
  82.9%
Phosphorus, Low - Grade 0 to 1
21
  10.9%
Phosphorus, Low - Grade 1 to 0
2
   1.0%
Phosphorus, Low - Grade 1 to 1
9
   4.7%
Phosphorus, Low - Grade 1 to 2
1
   0.5%
Potassium, High - Grade 0 to 0
189
  97.9%
Potassium, High - Grade 0 to 1
2
   1.0%
Potassium, High - Grade 0 to 2
1
   0.5%
Potassium, High - Grade 0 to 4
1
   0.5%
Potassium, Low - Grade 0 to 0
184
  95.3%
Potassium, Low - Grade 0 to 1
6
   3.1%
Potassium, Low - Grade 1 to 0
3
   1.6%
SGOT/AST, High - Grade 0 to 0
164
  85.0%
SGOT/AST, High - Grade 0 to 1
13
   6.7%
SGOT/AST, High - Grade 0 to 2
8
   4.1%
SGOT/AST, High - Grade 1 to 0
1
   0.5%
SGOT/AST, High - Grade 1 to 1
5
   2.6%
SGOT/AST, High - Grade 1 to 2
1
   0.5%
SGOT/AST, High - Grade 1 to 3
1
   0.5%
SGPT/ALT, High - Grade 0 to 0
158
  81.9%
SGPT/ALT, High - Grade 0 to 1
22
  11.4%
SGPT/ALT, High - Grade 0 to 2
5
   2.6%
SGPT/ALT, High - Grade 1 to 0
1
   0.5%
SGPT/ALT, High - Grade 1 to 1
4
   2.1%
SGPT/ALT, High - Grade 1 to 2
2
   1.0%
SGPT/ALT, High - Grade 1 to 4
1
   0.5%
Sodium, High - Grade 0 to 0
178
  92.2%
Sodium, High - Grade 0 to 1
7
   3.6%
Sodium, High - Grade 0 to 2
2
   1.0%
Sodium, High - Grade 1 to 0
4
   2.1%
Sodium, High - Grade 1 to 1
2
   1.0%
Sodium, Low - Grade 0 to 0
166
  86.0%
Sodium, Low - Grade 0 to 1
19
   9.8%
Sodium, Low - Grade 0 to 2
2
   1.0%
Sodium, Low - Grade 1 to 0
3
   1.6%
Sodium, Low - Grade 1 to 1
2
   1.0%
Sodium, Low - Grade 1 to 2
1
   0.5%
4.Primary Outcome
Title Change From Baseline in Body Temperature at Specified Timepoints
Hide Description Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: degrees Celsius
Baseline (BL) - Value at Visit Number Analyzed 192 participants
36.33  (0.52)
Change from BL at Week 2 Number Analyzed 190 participants
0.04  (0.41)
Change from BL at Week 3 Number Analyzed 191 participants
0.03  (0.40)
Change from BL at Week 5 Number Analyzed 191 participants
0.03  (0.45)
Change from BL at Month 3 Number Analyzed 187 participants
0.04  (0.45)
Change from BL at Month 6 Number Analyzed 183 participants
0.07  (0.50)
Change from BL at Month 9 Number Analyzed 184 participants
0.06  (0.48)
Change from BL at Month 12 Number Analyzed 182 participants
0.07  (0.48)
Change from BL at Month 18 Number Analyzed 176 participants
0.10  (0.53)
Change from BL at Early Termination/Study Completion Number Analyzed 163 participants
0.08  (0.48)
5.Primary Outcome
Title Change From Baseline in Systolic Blood Pressure at Specified Timepoints
Hide Description Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: millimetres of mercury (mmHg)
Baseline (BL) - Value at Visit Number Analyzed 193 participants
121.1  (14.3)
Change from BL at Week 2 Number Analyzed 193 participants
-0.9  (11.1)
Change from BL at Week 3 Number Analyzed 192 participants
-1.2  (10.6)
Change from BL at Week 5 Number Analyzed 192 participants
-1.3  (12.3)
Change from BL at Month 3 Number Analyzed 188 participants
0.1  (11.5)
Change from BL at Month 6 Number Analyzed 185 participants
-0.2  (13.1)
Change from BL at Month 9 Number Analyzed 186 participants
0.1  (13.1)
Change from BL at Month 12 Number Analyzed 184 participants
0.1  (13.9)
Change from BL at Month 18 Number Analyzed 177 participants
-0.1  (13.8)
Change from BL at Early Termination/Study Completion Number Analyzed 166 participants
1.9  (14.1)
6.Primary Outcome
Title Change From Baseline in Diastolic Blood Pressure at Specified Timepoints
Hide Description Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: millimetres of mercury (mmHg)
Baseline (BL) - Value at Visit Number Analyzed 193 participants
75.5  (10.4)
Change from BL at Week 2 Number Analyzed 193 participants
-1.2  (8.5)
Change from BL at Week 3 Number Analyzed 192 participants
-1.0  (8.9)
Change from BL at Week 5 Number Analyzed 192 participants
-1.2  (9.2)
Change from BL at Month 3 Number Analyzed 188 participants
-0.3  (9.8)
Change from BL at Month 6 Number Analyzed 185 participants
-0.7  (10.1)
Change from BL at Month 9 Number Analyzed 186 participants
-1.3  (9.4)
Change from BL at Month 12 Number Analyzed 184 participants
-1.2  (10.4)
Change from BL at Month 18 Number Analyzed 177 participants
-0.9  (9.6)
Change from BL at Early Termination/Study Completion Number Analyzed 166 participants
0.0  (11.0)
7.Primary Outcome
Title Change From Baseline in Pulse Rate at Specified Timepoints
Hide Description Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: beats per minute
Baseline (BL) - Value at Visit Number Analyzed 193 participants
76.6  (11.6)
Change from BL at Week 2 Number Analyzed 193 participants
1.0  (9.9)
Change from BL at Week 3 Number Analyzed 192 participants
1.6  (11.6)
Change from BL at Week 5 Number Analyzed 192 participants
-0.5  (11.3)
Change from BL at Month 3 Number Analyzed 188 participants
-0.8  (10.4)
Change from BL at Month 6 Number Analyzed 185 participants
-0.7  (11.6)
Change from BL at Month 9 Number Analyzed 186 participants
-1.0  (11.3)
Change from BL at Month 12 Number Analyzed 184 participants
-0.5  (12.2)
Change from BL at Month 18 Number Analyzed 177 participants
-2.1  (12.7)
Change from BL at Early Termination/Study Completion Number Analyzed 166 participants
-1.2  (11.9)
8.Primary Outcome
Title Change From Baseline in Respiratory Rate at Specified Timepoints
Hide Description Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: breaths per minute
Baseline (BL) - Value at Visit Number Analyzed 192 participants
17.0  (3.1)
Change from BL at Week 2 Number Analyzed 192 participants
0.2  (2.2)
Change from BL at Week 3 Number Analyzed 191 participants
0.0  (2.3)
Change from BL at Week 5 Number Analyzed 189 participants
-0.1  (2.5)
Change from BL at Month 3 Number Analyzed 187 participants
0.3  (4.7)
Change from BL at Month 6 Number Analyzed 180 participants
-0.1  (2.5)
Change from BL at Month 9 Number Analyzed 184 participants
0.0  (2.7)
Change from BL at Month 12 Number Analyzed 180 participants
0.3  (2.9)
Change from BL at Month 18 Number Analyzed 173 participants
-0.4  (2.7)
Change from BL at Early Termination/Study Completion Number Analyzed 160 participants
-0.7  (3.1)
9.Primary Outcome
Title Change From Baseline in Body Weight at Specified Timepoints
Hide Description Vital signs that were measured included body temperature, pulse and respiratory rates, systolic and diastolic blood pressure, and weight. On treatment days, measurement occurred prior to emicizumab administration.
Time Frame Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: kilograms (kg)
Baseline (BL) - Value at Visit Number Analyzed 193 participants
69.55  (16.60)
Change from BL at Week 2 Number Analyzed 193 participants
0.14  (1.06)
Change from BL at Week 3 Number Analyzed 192 participants
0.34  (1.18)
Change from BL at Week 5 Number Analyzed 192 participants
0.52  (1.56)
Change from BL at Month 3 Number Analyzed 188 participants
0.98  (2.44)
Change from BL at Month 6 Number Analyzed 186 participants
1.11  (3.20)
Change from BL at Month 9 Number Analyzed 186 participants
1.61  (4.08)
Change from BL at Month 12 Number Analyzed 184 participants
1.85  (4.88)
Change from BL at Month 18 Number Analyzed 177 participants
2.58  (5.56)
Change from BL at Early Termination/Study Completion Number Analyzed 165 participants
3.15  (6.78)
10.Secondary Outcome
Title Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Hide Description The number of bleeds over the efficacy period was analyzed as an ABR using a negative binomial regression model. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all bleed types, bleeds due to surgery/procedure were excluded. Bleeds occurring after dose up-titration have been excluded.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: bleeds per year
Treated Bleeds
0.5
(0.27 to 0.89)
All Bleeds
1.1
(0.80 to 1.47)
Treated Joint Bleeds
0.4
(0.15 to 0.86)
Treated Target Joint Bleeds
0.2
(0.07 to 0.68)
Treated Spontaneous Bleeds
0.3
(0.15 to 0.73)
11.Secondary Outcome
Title Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Hide Description The number of bleeds over the efficacy period was calculated as: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all types, bleeds due to surgery/procedure and bleeds occurring after dose up-titration were excluded.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Mean (95% Confidence Interval)
Unit of Measure: bleeds per year
Treated Bleeds
0.6
(0.00 to 4.85)
All Bleeds
1.3
(0.06 to 6.02)
Treated Joint Bleeds
0.4
(0.00 to 4.55)
Treated Target Joint Bleeds
0.3
(0.00 to 4.28)
Treated Spontaneous Bleeds
0.4
(0.00 to 4.49)
12.Secondary Outcome
Title Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Hide Description The number of bleeds over the efficacy period was calculated as: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". All bleeds: included both treated and non-treated bleeds. Treated joint bleeds: treated bleeds where bleed type was "joint bleed" accompanied by at least one of following symptoms: "increased swelling or warmth of the skin over the joint", "increasing pain" or "decreased range of motion or difficulty using the joint compared with baseline". Treated target joint bleeds: treated joint bleeds that occurred in a target joint, defined as a joint in which ≥3 treated joint bleeds occurred during the 24 weeks prior to study entry. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. For all types, bleeds due to surgery/procedure and bleeds occurring after dose up-titration were excluded.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Median (Inter-Quartile Range)
Unit of Measure: bleeds per year
Treated Bleeds
0.0
(0.00 to 0.00)
All Bleeds
0.0
(0.00 to 1.01)
Treated Joint Bleeds
0.0
(0.00 to 0.00)
Treated Target Joint Bleeds
0.0
(0.00 to 0.00)
Treated Spontaneous Bleeds
0.0
(0.00 to 0.00)
13.Secondary Outcome
Title Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds
Hide Description Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
0 Bleeds
82.6
(76.5 to 87.6)
1-3 Bleeds
12.3
(8.0 to 17.8)
4-10 Bleeds
4.1
(1.8 to 7.9)
>10 Bleeds
1.0
(0.1 to 3.7)
14.Secondary Outcome
Title Percentage of Participants by the Categorized Number of Bleeds for All Bleeds
Hide Description All bleeds: included both treated and non-treated bleeds. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
0 Bleeds
54.9
(47.6 to 62.0)
1-3 Bleeds
30.3
(23.9 to 37.2)
4-10 Bleeds
12.3
(8.0 to 17.8)
>10 Bleeds
2.6
(0.8 to 5.9)
15.Secondary Outcome
Title Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds
Hide Description Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
0 Bleeds
89.2
(84.0 to 93.2)
1-3 Bleeds
8.7
(5.2 to 13.6)
4-10 Bleeds
1.5
(0.3 to 4.4)
>10 Bleeds
0.5
(0.0 to 2.8)
16.Secondary Outcome
Title Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds
Hide Description The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated bleeds: bleeds followed by a hemophilia medication reported to be a "Treatment for bleed". Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
ABR <1
90.3
(85.2 to 94.0)
ABR 1-3.4
6.2
(3.2 to 10.5)
ABR 3.5-10
2.6
(0.8 to 5.9)
ABR >10
1.0
(0.1 to 3.7)
17.Secondary Outcome
Title Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds
Hide Description The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. All bleeds: included both treated and non-treated bleeds. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
ABR <1
72.3
(65.5 to 78.5)
ABR 1-3.4
20.5
(15.1 to 26.9)
ABR 3.5-10
4.6
(2.1 to 8.6)
ABR >10
2.6
(0.8 to 5.9)
18.Secondary Outcome
Title Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds
Hide Description The number of bleeds over the efficacy period was calculated as an ABR for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25. Treated spontaneous bleeds: treated bleeds with no other known contributing factor such as trauma or procedure/surgery. Bleeds due to surgery/procedure were excluded. The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location are counted as one bleed if the second bleed occurs within 72 hours from the last treatment for the first bleed. Included data before up-titration only, for participants whose dose was up-titrated.
Time Frame From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) Population: all enrolled participants
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
ABR <1
95.4
(91.4 to 97.9)
ABR 1-3.4
2.6
(0.8 to 5.9)
ABR 3.5-10
1.0
(0.1 to 3.7)
ABR >10
1.0
(0.1 to 3.7)
19.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. The Total Score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
41.09  (16.15)
Change from BL at Month 3 Number Analyzed 144 participants
-17.39  (12.86)
Change from BL at Month 6 Number Analyzed 141 participants
-16.40  (14.58)
Change from BL at Month 12 Number Analyzed 140 participants
-17.44  (14.16)
Change from BL at Month 18 Number Analyzed 132 participants
-16.27  (15.39)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-14.13  (13.70)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 3 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 6 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 12 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 18 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Early Terminatinon/Study Completion vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
20.Secondary Outcome
Title Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. The Total Score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflective of better quality of life. An improvement larger than the responder threshold was defined as a decrease from baseline of at least 7 points in the Haem-A-QoL Total score.
Time Frame At 3, 6, 12, and 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Measure Type: Number
Unit of Measure: Percentage of participants
Month 3 Number Analyzed 147 participants
78.2
Month 6 Number Analyzed 145 participants
72.4
Month 12 Number Analyzed 144 participants
70.8
Month 18 Number Analyzed 136 participants
69.9
21.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
51.13  (23.02)
Change from BL at Month 3 Number Analyzed 144 participants
-27.36  (20.70)
Change from BL at Month 6 Number Analyzed 141 participants
-26.10  (23.12)
Change from BL at Month 12 Number Analyzed 140 participants
-25.21  (23.31)
Change from BL at Month 18 Number Analyzed 132 participants
-23.14  (24.51)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-23.29  (25.16)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 3 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 6 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 12 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 18 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Early Terminatinon/Study Completion vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
22.Secondary Outcome
Title Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life. An improvement larger than the responder threshold was defined as a decrease from baseline of at least 10 points in the Haem-A-QoL Physical Health domain score.
Time Frame At 3, 6, 12, and 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Measure Type: Number
Unit of Measure: Percentage of participants
Month 3 Number Analyzed 147 participants
79.6
Month 6 Number Analyzed 145 participants
74.5
Month 12 Number Analyzed 144 participants
72.9
Month 18 Number Analyzed 136 participants
71.3
23.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
39.40  (19.89)
Change from BL at Month 3 Number Analyzed 144 participants
-23.44  (22.07)
Change from BL at Month 6 Number Analyzed 141 participants
-21.01  (23.74)
Change from BL at Month 12 Number Analyzed 140 participants
-22.21  (22.57)
Change from BL at Month 18 Number Analyzed 132 participants
-20.34  (25.37)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-21.16  (22.31)
24.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 125 participants
39.23  (25.31)
Change from BL at Month 3 Number Analyzed 114 participants
-21.40  (26.51)
Change from BL at Month 6 Number Analyzed 106 participants
-21.91  (27.17)
Change from BL at Month 12 Number Analyzed 106 participants
-22.43  (25.55)
Change from BL at Month 18 Number Analyzed 101 participants
-21.43  (26.65)
Change from BL at Early Termination/Study Completion Number Analyzed 51 participants
-18.83  (29.54)
25.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
26.61  (19.76)
Change from BL at Month 3 Number Analyzed 144 participants
5.32  (30.06)
Change from BL at Month 6 Number Analyzed 141 participants
5.73  (28.14)
Change from BL at Month 12 Number Analyzed 140 participants
5.24  (29.18)
Change from BL at Month 18 Number Analyzed 132 participants
7.70  (29.75)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
10.60  (33.42)
26.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
36.25  (24.53)
Change from BL at Month 3 Number Analyzed 144 participants
-21.22  (22.69)
Change from BL at Month 6 Number Analyzed 141 participants
-20.88  (24.11)
Change from BL at Month 12 Number Analyzed 140 participants
-22.37  (23.78)
Change from BL at Month 18 Number Analyzed 132 participants
-19.55  (25.43)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-17.95  (23.62)
27.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 91 participants
25.64  (28.49)
Change from BL at Month 3 Number Analyzed 67 participants
-8.64  (20.95)
Change from BL at Month 6 Number Analyzed 68 participants
-9.07  (20.78)
Change from BL at Month 12 Number Analyzed 65 participants
-10.83  (22.51)
Change from BL at Month 18 Number Analyzed 58 participants
-12.39  (25.20)
Change from BL at Early Termination/Study Completion Number Analyzed 26 participants
-9.38  (24.81)
28.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
42.70  (21.95)
Change from BL at Month 3 Number Analyzed 144 participants
-15.56  (17.63)
Change from BL at Month 6 Number Analyzed 141 participants
-15.46  (17.83)
Change from BL at Month 12 Number Analyzed 140 participants
-14.46  (20.10)
Change from BL at Month 18 Number Analyzed 132 participants
-14.43  (19.83)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-13.29  (19.56)
29.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
23.17  (28.65)
Change from BL at Month 3 Number Analyzed 144 participants
-7.35  (25.25)
Change from BL at Month 6 Number Analyzed 141 participants
-6.03  (22.76)
Change from BL at Month 12 Number Analyzed 140 participants
-7.74  (27.60)
Change from BL at Month 18 Number Analyzed 132 participants
-9.72  (28.16)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-4.05  (21.32)
30.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 114 participants
68.86  (21.24)
Change from BL at Month 3 Number Analyzed 89 participants
-19.65  (23.67)
Change from BL at Month 6 Number Analyzed 83 participants
-17.63  (25.86)
Change from BL at Month 12 Number Analyzed 87 participants
-22.56  (22.17)
Change from BL at Month 18 Number Analyzed 76 participants
-20.20  (25.86)
Change from BL at Early Termination/Study Completion Number Analyzed 42 participants
-20.65  (28.65)
31.Secondary Outcome
Title Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants
Hide Description The Haem-A-QoL questionnaire was designed for adult participants (greater than or equal to 18 years old) with hemophilia. It consists of 46 items comprising 10 domains: physical health, treatment, work and school, dealing with hemophilia, feelings, familly planning, future, partnerships and sexuality, sports and leisure, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always; although, for some items there is also a 'not applicable' option. Scale scores for each domain range from 0 to 100, with lower scores reflective of better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adult participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 155
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 150 participants
43.57  (22.05)
Change from BL at Month 3 Number Analyzed 144 participants
-13.51  (20.63)
Change from BL at Month 6 Number Analyzed 141 participants
-15.04  (21.19)
Change from BL at Month 12 Number Analyzed 140 participants
-15.71  (19.88)
Change from BL at Month 18 Number Analyzed 132 participants
-15.19  (21.88)
Change from BL at Early Termination/Study Completion Number Analyzed 70 participants
-9.50  (20.70)
32.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. The Total Score is derived from the scores for all domains and scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
35.10  (12.42)
Change from BL at Month 3 Number Analyzed 32 participants
-9.60  (12.51)
Change from BL at Month 6 Number Analyzed 34 participants
-12.65  (12.72)
Change from BL at Month 12 Number Analyzed 32 participants
-13.57  (13.20)
Change from BL at Month 18 Number Analyzed 35 participants
-14.02  (11.83)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-18.37  (17.53)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 3 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 6 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 12 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 18 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Early Terminatinon/Study Completion vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0004
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
33.Secondary Outcome
Title Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. The Total Score is derived from the scores for all domains and scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life. An improvement larger than the responder threshold was defined as a decrease of at least 5 points in the Haemo-QoL-SF Total score.
Time Frame At 3, 6, 12, and 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Measure Type: Number
Unit of Measure: Percentage of participants
Month 3 Number Analyzed 36 participants
63.9
Month 6 Number Analyzed 37 participants
75.7
Month 12 Number Analyzed 36 participants
75.0
Month 18 Number Analyzed 39 participants
69.2
34.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
42.14  (18.40)
Change from BL at Month 3 Number Analyzed 32 participants
-29.10  (20.12)
Change from BL at Month 6 Number Analyzed 34 participants
-29.78  (17.55)
Change from BL at Month 12 Number Analyzed 32 participants
-30.47  (20.74)
Change from BL at Month 18 Number Analyzed 35 participants
-30.71  (18.83)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-34.03  (25.66)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 3 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 6 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 12 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 18 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Early Terminatinon/Study Completion vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
35.Secondary Outcome
Title Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life. An improvement larger than the responder threshold was defined as a decrease of at least 10 points in the Haemo-QoL-SF Physical Health domain score.
Time Frame At 3, 6, 12, and 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Measure Type: Number
Unit of Measure: Percentage of participants
Month 3 Number Analyzed 36 participants
77.8
Month 6 Number Analyzed 37 participants
78.4
Month 12 Number Analyzed 36 participants
75.0
Month 18 Number Analyzed 39 participants
79.5
36.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
31.61  (18.12)
Change from BL at Month 3 Number Analyzed 32 participants
-9.38  (20.94)
Change from BL at Month 6 Number Analyzed 34 participants
-8.64  (23.23)
Change from BL at Month 12 Number Analyzed 32 participants
-10.74  (21.31)
Change from BL at Month 18 Number Analyzed 35 participants
-12.68  (21.68)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-20.83  (23.87)
37.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
62.32  (21.30)
Change from BL at Month 3 Number Analyzed 32 participants
-15.82  (25.55)
Change from BL at Month 6 Number Analyzed 34 participants
-20.04  (29.27)
Change from BL at Month 12 Number Analyzed 32 participants
-22.07  (26.71)
Change from BL at Month 18 Number Analyzed 35 participants
-28.21  (27.80)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-23.61  (28.16)
38.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
24.11  (14.79)
Change from BL at Month 3 Number Analyzed 32 participants
-7.23  (18.25)
Change from BL at Month 6 Number Analyzed 34 participants
-7.72  (17.75)
Change from BL at Month 12 Number Analyzed 32 participants
-10.35  (20.24)
Change from BL at Month 18 Number Analyzed 35 participants
-3.39  (25.70)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-12.50  (23.48)
39.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
26.43  (25.10)
Change from BL at Month 3 Number Analyzed 32 participants
-9.57  (20.08)
Change from BL at Month 6 Number Analyzed 34 participants
-16.36  (21.54)
Change from BL at Month 12 Number Analyzed 32 participants
-13.09  (22.80)
Change from BL at Month 18 Number Analyzed 35 participants
-15.18  (25.51)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-23.96  (32.67)
40.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
46.07  (20.79)
Change from BL at Month 3 Number Analyzed 32 participants
-6.84  (15.66)
Change from BL at Month 6 Number Analyzed 34 participants
-11.95  (20.84)
Change from BL at Month 12 Number Analyzed 32 participants
-14.84  (23.32)
Change from BL at Month 18 Number Analyzed 35 participants
-15.36  (21.67)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-15.28  (19.08)
41.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
35.95  (26.02)
Change from BL at Month 3 Number Analyzed 32 participants
-0.52  (24.95)
Change from BL at Month 6 Number Analyzed 34 participants
-3.19  (28.13)
Change from BL at Month 12 Number Analyzed 32 participants
-5.47  (27.57)
Change from BL at Month 18 Number Analyzed 35 participants
-5.95  (36.41)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-7.41  (31.56)
42.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
21.43  (17.50)
Change from BL at Month 3 Number Analyzed 32 participants
-2.93  (18.72)
Change from BL at Month 6 Number Analyzed 34 participants
-5.51  (19.08)
Change from BL at Month 12 Number Analyzed 32 participants
-8.01  (18.53)
Change from BL at Month 18 Number Analyzed 35 participants
-6.79  (17.51)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-11.81  (18.55)
43.Secondary Outcome
Title Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants
Hide Description The Haemo-QoL-SF was designed as a series of age-related questionnaires to measure health-related quality of life in children and adolescents (12-17 year olds) with hemophilia. This version contains 35 items and covers nine domains considered relevant for children's health-related quality of life: physical health, treatment, sports and school, dealing with hemophilia, feelings, family, friends, other people, and view of yourself. Items are rated by participants with one of five response options: never, seldom, sometimes, often, and always. Scale scores range from 0 to 100, with a lower score reflective of better health-related quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of adolescent participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 35 participants
26.07  (22.04)
Change from BL at Month 3 Number Analyzed 32 participants
-2.73  (26.03)
Change from BL at Month 6 Number Analyzed 34 participants
-8.27  (23.27)
Change from BL at Month 12 Number Analyzed 32 participants
-5.08  (24.82)
Change from BL at Month 18 Number Analyzed 35 participants
-5.89  (21.54)
Change from BL at Early Termination/Study Completion Number Analyzed 18 participants
-13.19  (26.59)
44.Secondary Outcome
Title Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints
Hide Description The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations. The index utility score is based on the participant's assessment of their health on five scales: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. It ranges from -0.224 to 1, with 0 corresponding to death and 1 corresponding to full health; negative values correspond to health states worse than death. Higher scores indicate better quality of life.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 188 participants
0.67  (0.23)
Change from BL at Month 3 Number Analyzed 182 participants
0.08  (0.18)
Change from BL at Month 6 Number Analyzed 178 participants
0.11  (0.18)
Change from BL at Month 12 Number Analyzed 176 participants
0.09  (0.19)
Change from BL at Month 18 Number Analyzed 170 participants
0.07  (0.21)
Change from BL at Early Termination/Study Completion Number Analyzed 89 participants
0.10  (0.22)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 3 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 6 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 12 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 18 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Early Terminatinon/Study Completion vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
45.Secondary Outcome
Title Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints
Hide Description The EQ-5D-5L is a generic, self-reported, preference-based health utility measure that consists of six questions and is used to assess health status and inform pharmacoeconomic evaluations. The visual analogue scale (VAS) ranges from 0 to 100 points, on which the participant self-assesses their current health status; 0 being the worst health you can imagine and 100 being the best health you can imagine. Higher scores are reflective of better health.
Time Frame Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
The number analyzed represents the number of participants responding to the questionnaire who had a baseline value and a post-baseline value at a given timepoint.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 195
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline (BL) - Value at Visit Number Analyzed 188 participants
71.01  (19.41)
Change from BL at Month 3 Number Analyzed 182 participants
8.02  (18.70)
Change from BL at Month 6 Number Analyzed 177 participants
10.27  (17.24)
Change from BL at Month 12 Number Analyzed 176 participants
10.69  (18.18)
Change from BL at Month 18 Number Analyzed 170 participants
9.49  (21.08)
Change from BL at Early Termination/Study Completion Number Analyzed 89 participants
14.52  (17.91)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 3 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 6 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 12 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Month 18 vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection 1.5 mg/kg Emicizumab QW
Comments Early Terminatinon/Study Completion vs. Baseline
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Paired t-test
Comments [Not Specified]
46.Secondary Outcome
Title Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire
Hide Description The EmiPref questionnaire asked participants to specify the treatment they would prefer to continue to receive after receiving treatment with their previous episodic or prophylactic regimen and subcutaneous (SC) emicizumab, or if they had no preference. The 95% confidence intervals were calculated for one sample binomial using the Pearson-Clopper method.
Time Frame Month 3
Hide Outcome Measure Data
Hide Analysis Population Description
The number of participants analyzed represents the total number who responded to this questionnaire.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 179
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
Prefer the New Study Drug Treatment (Emicizumab)
96.6
(92.85 to 98.76)
Prefer my Old Hemophilia Treatment
0.6
(0.01 to 3.07)
Have no Preference
2.8
(0.91 to 6.40)
47.Secondary Outcome
Title Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline
Hide Description 'Total ADA Negative' is the sum of all subjects who tested negative for ADA in the 2 following categories: 'ADA Negative', those who are pre-dose ADA negative or are missing pre-dose ADA data and who have all negative post-dose ADA results; and 'ADA Negative (Treatment Unaffected)', a subset who are pre-dose ADA positive but do not have a ≥4-fold increase in post-dose ADA levels compared to baseline measurement. 'Total ADA Positive' is the sum of all subjects who tested positive for ADA in the 2 following categories: 'ADA Positive (Treatment Boosted)', those who are pre-dose ADA positive and have a ≥4-fold increase in post-dose ADA levels compared to baseline measurement; and 'ADA Positive (Treatment Induced)', those who are pre-dose ADA negative or missing data and who have at least one post-dose ADA positive sample.
Time Frame Baseline (Week 1), Week 5, and at 3, 6, 9, 12, and 18 months, and at early termination/study completion (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least one dose of emicizumab. The number analyzed included participants with at least one baseline and one post-baseline assessments.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Measure Type: Count of Participants
Unit of Measure: Participants
Total ADA Negative (Neg + Neg Unaffected)
183
  94.8%
ADA Negative
180
  93.3%
ADA Negative (Treatment Unaffected)
3
   1.6%
Total ADA Positive (Boosted + Induced)
10
   5.2%
ADA Positive (Treatment Boosted)
2
   1.0%
ADA Positive (Treatment Induced)
8
   4.1%
48.Secondary Outcome
Title Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints
Hide Description The trough concentration is a measure of the plasma concentration of a study drug at the end of of the dosage interval.
Time Frame Pre-dose at Weeks 2, 3, and 5, Months 3, 6, 12, and 18, and at treatment discontinuation (up to 2 years)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic-Evaluable Population: all participants who received at least one dose of emicizumab and had at least one post-baseline emicizumab concentration result.
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description:
Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
Overall Number of Participants Analyzed 193
Mean (Standard Deviation)
Unit of Measure: micrograms per millilitre (μg/mL)
Week 2 Number Analyzed 188 participants
16.7  (5.5)
Week 3 Number Analyzed 190 participants
30.7  (9.2)
Week 5 Number Analyzed 187 participants
52.4  (15.5)
Month 3 Number Analyzed 186 participants
52.5  (18.3)
Month 6 Number Analyzed 183 participants
53.4  (19.3)
Month 12 Number Analyzed 183 participants
54.2  (20.6)
Month 18 Number Analyzed 171 participants
53.6  (18.3)
Treatment Discontinuation Number Analyzed 154 participants
54.6  (19.0)
Time Frame From Baseline until study completion (up to 2 years)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 1.5 mg/kg Emicizumab QW
Hide Arm/Group Description Participants were enrolled to receive initial weekly doses of prophylactic 3.0 mg/kg emicizumab subcutaneously (SC) for 4 weeks, followed by prophylactic maintenance doses consisting of 1.5 mg/kg emicizumab once a week (QW), administered SC for the remainder of the 2-year treatment period.
All-Cause Mortality
1.5 mg/kg Emicizumab QW
Affected / at Risk (%)
Total   2/193 (1.04%)    
Hide Serious Adverse Events
1.5 mg/kg Emicizumab QW
Affected / at Risk (%) # Events
Total   31/193 (16.06%)    
Blood and lymphatic system disorders   
THROMBOCYTOPENIA † 1  1/193 (0.52%)  1
Cardiac disorders   
ACUTE MYOCARDIAL INFARCTION † 1  1/193 (0.52%)  1
Gastrointestinal disorders   
ABDOMINAL COMPARTMENT SYNDROME † 1  1/193 (0.52%)  1
DIVERTICULUM INTESTINAL HAEMORRHAGIC † 1  1/193 (0.52%)  1
GASTROINTESTINAL HAEMORRHAGE † 1  1/193 (0.52%)  5
RETROPERITONEAL HAEMATOMA † 1  1/193 (0.52%)  1
Hepatobiliary disorders   
SUBCAPSULAR HEPATIC HAEMATOMA † 1  1/193 (0.52%)  1
Infections and infestations   
ABSCESS † 1  1/193 (0.52%)  1
APPENDICITIS † 1  1/193 (0.52%)  1
CATHETER SITE ABSCESS † 1  2/193 (1.04%)  2
DEVICE RELATED INFECTION † 1  1/193 (0.52%)  1
INFECTED SKIN ULCER † 1  1/193 (0.52%)  1
PNEUMONIA † 1  1/193 (0.52%)  1
WOUND INFECTION † 1  1/193 (0.52%)  1
Injury, poisoning and procedural complications   
FEMUR FRACTURE † 1  2/193 (1.04%)  2
FIBULA FRACTURE † 1  1/193 (0.52%)  1
FRACTURED SACRUM † 1  1/193 (0.52%)  1
HEAD INJURY † 1  1/193 (0.52%)  1
HIP FRACTURE † 1  1/193 (0.52%)  1
INJURY † 1  1/193 (0.52%)  1
JOINT INJURY † 1  1/193 (0.52%)  1
LOWER LIMB FRACTURE † 1  1/193 (0.52%)  1
MULTIPLE INJURIES † 1  1/193 (0.52%)  1
POST PROCEDURAL HAEMORRHAGE † 1  1/193 (0.52%)  1
TIBIA FRACTURE † 1  1/193 (0.52%)  1
WOUND DEHISCENCE † 1  2/193 (1.04%)  2
WOUND HAEMATOMA † 1  1/193 (0.52%)  1
Musculoskeletal and connective tissue disorders   
HAEMARTHROSIS † 1  1/193 (0.52%)  1
HAEMATOMA MUSCLE † 1  2/193 (1.04%)  2
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
ADENOCARCINOMA OF COLON † 1  1/193 (0.52%)  1
BLADDER NEOPLASM † 1  1/193 (0.52%)  1
Nervous system disorders   
GENERALISED TONIC-CLONIC SEIZURE † 1  1/193 (0.52%)  1
TENSION HEADACHE † 1  1/193 (0.52%)  1
Product Issues   
DEVICE DISLOCATION † 1  1/193 (0.52%)  1
Renal and urinary disorders   
NEPHROTIC SYNDROME † 1  1/193 (0.52%)  1
RENAL PAIN † 1  1/193 (0.52%)  1
URETEROLITHIASIS † 1  1/193 (0.52%)  1
Respiratory, thoracic and mediastinal disorders   
EPISTAXIS † 1  1/193 (0.52%)  1
PNEUMONIA ASPIRATION † 1  1/193 (0.52%)  1
Surgical and medical procedures   
CENTRAL VENOUS CATHETER REMOVAL † 1  1/193 (0.52%)  1
Vascular disorders   
HAEMATOMA † 1  1/193 (0.52%)  1
HAEMORRHAGE † 1  1/193 (0.52%)  1
1
Term from vocabulary, MedDRA v23.1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
1.5 mg/kg Emicizumab QW
Affected / at Risk (%) # Events
Total   104/193 (53.89%)    
General disorders   
INJECTION SITE REACTION † 1  22/193 (11.40%)  29
PYREXIA † 1  21/193 (10.88%)  26
Infections and infestations   
INFLUENZA † 1  16/193 (8.29%)  25
NASOPHARYNGITIS † 1  30/193 (15.54%)  47
Injury, poisoning and procedural complications   
FALL † 1  12/193 (6.22%)  16
LIMB INJURY † 1  10/193 (5.18%)  12
Musculoskeletal and connective tissue disorders   
ARTHRALGIA † 1  33/193 (17.10%)  52
Nervous system disorders   
HEADACHE † 1  29/193 (15.03%)  52
Respiratory, thoracic and mediastinal disorders   
COUGH † 1  12/193 (6.22%)  13
1
Term from vocabulary, MedDRA v23.1
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The study being conducted under this agreement is part of the overall study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the study, but only after the first publication or presentation that involves the overall study. The Sponsor may request that confidential information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann-La Roche
Phone: 800-821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03191799    
Other Study ID Numbers: MO39129
2016-004366-25 ( EudraCT Number )
First Submitted: June 15, 2017
First Posted: June 19, 2017
Results First Submitted: May 17, 2021
Results First Posted: June 11, 2021
Last Update Posted: June 11, 2021