Trial Outcomes & Findings for BAX 855 Continuation (NCT NCT01945593)
NCT ID: NCT01945593
Last Updated: 2021-05-24
Results Overview
Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
218 participants
Primary outcome timeframe
Baseline through end of study (53 months)
Results posted on
2021-05-24
Participant Flow
The study was conducted at 86 centers in 23 countries between 15 October 2013 (first participant first visit) and 02 March 2018 (last participant last visit).
A total of 218 subjects were enrolled, of them 216 subjects received treatment.
Participant milestones
| Measure |
BAX 855: Age < 2 Years
Participants of age less than (\<) 2 years received an infusion of 50 +/- 10 International Units (IU)/kilogram (kg) of BAX 855 twice weekly; could be increased to 80 IU/kg or a pharmacokinetically tailored (PK-tailored) prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain Factor VIII (FVIII) trough levels of greater than or equal to (\>=) 3% until at least 100 exposure days (EDs) were reached.
|
BAX 855: Age >= 2 to <12 Years
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
64
|
26
|
125
|
|
Overall Study
COMPLETED
|
3
|
56
|
23
|
105
|
|
Overall Study
NOT COMPLETED
|
0
|
8
|
3
|
20
|
Reasons for withdrawal
| Measure |
BAX 855: Age < 2 Years
Participants of age less than (\<) 2 years received an infusion of 50 +/- 10 International Units (IU)/kilogram (kg) of BAX 855 twice weekly; could be increased to 80 IU/kg or a pharmacokinetically tailored (PK-tailored) prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain Factor VIII (FVIII) trough levels of greater than or equal to (\>=) 3% until at least 100 exposure days (EDs) were reached.
|
BAX 855: Age >= 2 to <12 Years
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
0
|
0
|
5
|
|
Overall Study
Physician Decision
|
0
|
0
|
1
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
2
|
0
|
4
|
|
Overall Study
Protocol Violation
|
0
|
2
|
1
|
3
|
|
Overall Study
Death
|
0
|
0
|
1
|
0
|
|
Overall Study
Other: Terminated
|
0
|
0
|
0
|
1
|
|
Overall Study
Other: Surgical Procedure
|
0
|
2
|
0
|
6
|
|
Overall Study
Other: Screen Failure
|
0
|
2
|
0
|
0
|
Baseline Characteristics
BAX 855 Continuation
Baseline characteristics by cohort
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
Total
n=216 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
4 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
42 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
88 Participants
n=4 Participants
|
152 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
37 Participants
n=4 Participants
|
58 Participants
n=21 Participants
|
|
Age, Continuous
|
1.0 Years
STANDARD_DEVIATION 0.00 • n=5 Participants
|
6.0 Years
STANDARD_DEVIATION 2.47 • n=7 Participants
|
13.8 Years
STANDARD_DEVIATION 1.30 • n=5 Participants
|
33.5 Years
STANDARD_DEVIATION 11.68 • n=4 Participants
|
22.8 Years
STANDARD_DEVIATION 15.67 • n=21 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
61 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
125 Participants
n=4 Participants
|
215 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
10 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
59 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
118 Participants
n=4 Participants
|
206 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Baseline through end of study (53 months)Population: Safety analysis set (SAS) included all participants with at least 1 BAX 855 infusion. The analysis included participants that developed inhibitory antibodies (IA) to FVIII and participants that did not develop IA to FVIII and had 100 or more exposure days (ED) to BAX 855 across all studies and a FVIII inhibitory test result after the 100th ED.
Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=57 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=118 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline through end of study (53 months)Population: Full analysis set (FAS) included all participants with at least 1 BAX 855 infusion.
The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
FDR: Twice Weekly
|
0.614 Bleeds per year
Interval 0.154 to 2.454
|
0.792 Bleeds per year
Interval 0.518 to 1.21
|
1.439 Bleeds per year
Interval 0.942 to 2.2
|
1.293 Bleeds per year
Interval 0.913 to 1.832
|
|
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
Fixed-dose regimen (FDR): Every 5 Days
|
—
|
—
|
1.381 Bleeds per year
Interval 0.728 to 2.62
|
1.160 Bleeds per year
Interval 0.727 to 1.852
|
|
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
FDR: Every 7 Days
|
—
|
—
|
0.669 Bleeds per year
Interval 0.061 to 7.377
|
1.992 Bleeds per year
Interval 0.822 to 4.827
|
|
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
PK-tailored regimen (PK-t R)
|
—
|
1.078 Bleeds per year
Interval 0.45 to 2.581
|
1.602 Bleeds per year
Interval 0.27 to 9.486
|
0.868 Bleeds per year
Interval 0.365 to 2.067
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: FAS included all participants with at least 1 BAX 855 infusion.
The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. Bleeding occurring at multiple locations related to the same injury (e.g., knee and ankle bleed following a fall) was counted as a single bleeding episode. Total annualized bleed rate (spontaneous and traumatic bleeding episodes) was reported.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Total Annualized Bleed Rate (ABR)
|
1.855 Bleeds per year
Standard Deviation 1.000
|
2.370 Bleeds per year
Standard Deviation 3.027
|
3.175 Bleeds per year
Standard Deviation 2.797
|
2.426 Bleeds per year
Standard Deviation 3.258
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: FAS included all participants with at least 1 BAX 855 infusion. Here 'N' refers to the number of participants evaluable for this outcome.
The participant or caregiver rated the overall treatment response at 24 (+/- 2) hours after the initiation of treatment using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and possibly requires more than 1 infusion for complete resolution; Fair: Slight relief of pain and slight improvement in signs of bleeding after a single infusion and required more than 1 infusion for complete resolution and None: No improvement or condition worsens.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=43 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=24 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=95 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Excellent
|
3 Treated bleeds
|
108 Treated bleeds
|
98 Treated bleeds
|
229 Treated bleeds
|
|
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Good
|
3 Treated bleeds
|
66 Treated bleeds
|
49 Treated bleeds
|
250 Treated bleeds
|
|
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Fair
|
0 Treated bleeds
|
5 Treated bleeds
|
7 Treated bleeds
|
36 Treated bleeds
|
|
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
None
|
0 Treated bleeds
|
0 Treated bleeds
|
1 Treated bleeds
|
3 Treated bleeds
|
|
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Not Reported
|
0 Treated bleeds
|
13 Treated bleeds
|
13 Treated bleeds
|
26 Treated bleeds
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: FAS included all participants with at least 1 BAX 855 infusion.
The BAX 855 infusions to treat each bleeding episode was determined by the participant, the participant's caregiver, and/or investigator, and was based upon the participant's response to treatment. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
BAX 855 Infusions Needed to Treat Bleeding Episodes
|
1.3 Infusions
Standard Deviation 0.52
|
1.4 Infusions
Standard Deviation 1.14
|
1.4 Infusions
Standard Deviation 1.81
|
1.4 Infusions
Standard Deviation 1.10
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: FAS included all participants with at least 1 BAX 855 infusion. Here number of participants analyzed refers to the number of participants evaluable for this outcome.
The time interval between bleeding episodes was calculated based upon the date and time reported for each bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=2 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=36 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=21 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=84 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Total Time Intervals Between Bleeding Episodes
|
5.700 Months
Interval 4.074 to 7.327
|
5.051 Months
Interval 0.756 to 14.867
|
5.232 Months
Interval 1.228 to 14.571
|
5.818 Months
Interval 0.617 to 22.686
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion.
The average dose of BAX 855 per prophylactic infusion was reported.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Average Dose of BAX 855 Per Prophylactic Infusion
|
50.748 International units per kilogram (IU/kg)
Standard Deviation 11.312
|
53.855 International units per kilogram (IU/kg)
Standard Deviation 7.754
|
53.356 International units per kilogram (IU/kg)
Standard Deviation 10.344
|
49.727 International units per kilogram (IU/kg)
Standard Deviation 8.677
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion.
An AE was any unfavorable and unintended sign (eg, an abnormal laboratory finding), symptom (eg, rash, pain, discomfort, fever, dizziness, etc.), disease (eg, peritonitis, bacteremia, etc.), or outcome of death temporally associated with the use of an investigational product (IP), whether or not considered causally related to the IP. A serious adverse event (SAE) was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/resulted in death; was life-threatening; required inpatient hospitalization or resulted in prolongation of an existing hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect; was a medically important event.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
SAE
|
0 Participants
|
7 Participants
|
4 Participants
|
22 Participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
AE
|
3 Participants
|
52 Participants
|
18 Participants
|
101 Participants
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
Change in body temperature at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=61 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Body Temperature
FDR: Pre-Infusion
|
-0.23 Degree celsius
Standard Deviation 0.208
|
-0.05 Degree celsius
Standard Deviation 0.386
|
-0.08 Degree celsius
Standard Deviation 0.409
|
-0.01 Degree celsius
Standard Deviation 0.376
|
|
Change From Baseline in Body Temperature
FDR: Post-Infusion
|
-0.07 Degree celsius
Standard Deviation 0.306
|
-0.03 Degree celsius
Standard Deviation 0.394
|
0.03 Degree celsius
Standard Deviation 0.375
|
-0.02 Degree celsius
Standard Deviation 0.395
|
|
Change From Baseline in Body Temperature
PK-tR: Pre-Infusion
|
—
|
0.28 Degree celsius
Standard Deviation 0.255
|
-0.08 Degree celsius
Standard Deviation 0.189
|
-0.10 Degree celsius
Standard Deviation 0.595
|
|
Change From Baseline in Body Temperature
PK-tR: Post-Infusion
|
—
|
0.13 Degree celsius
Standard Deviation 0.362
|
0.03 Degree celsius
Standard Deviation 0.263
|
0.01 Degree celsius
Standard Deviation 0.593
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
Change in pulse rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=61 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Pulse Rate
FDR: Pre-Infusion
|
-20.3 Beats per minute (beats/min)
Standard Deviation 21.22
|
-0.1 Beats per minute (beats/min)
Standard Deviation 14.31
|
-7.7 Beats per minute (beats/min)
Standard Deviation 9.07
|
-0.4 Beats per minute (beats/min)
Standard Deviation 11.67
|
|
Change From Baseline in Pulse Rate
FDR: Post-Infusion
|
-13 Beats per minute (beats/min)
Standard Deviation 19.47
|
2.4 Beats per minute (beats/min)
Standard Deviation 16.72
|
-6.2 Beats per minute (beats/min)
Standard Deviation 8.55
|
-1 Beats per minute (beats/min)
Standard Deviation 10.07
|
|
Change From Baseline in Pulse Rate
PK-tR: Pre-Infusion
|
—
|
0.1 Beats per minute (beats/min)
Standard Deviation 9.13
|
-1.8 Beats per minute (beats/min)
Standard Deviation 13.91
|
4.0 Beats per minute (beats/min)
Standard Deviation 9.15
|
|
Change From Baseline in Pulse Rate
PK-tR: Post-Infusion
|
—
|
-8.1 Beats per minute (beats/min)
Standard Deviation 14.94
|
-4.5 Beats per minute (beats/min)
Standard Deviation 12.40
|
-0.2 Beats per minute (beats/min)
Standard Deviation 7.31
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
Change in respiratory rate at pre-infusion and post-infusion at end of the study was reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=61 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Respiratory Rate
FDR: Post-Infusion
|
-6.3 Breaths per minute (breaths/min)
Standard Deviation 4.73
|
-1.3 Breaths per minute (breaths/min)
Standard Deviation 3.55
|
-1.5 Breaths per minute (breaths/min)
Standard Deviation 1.85
|
0.2 Breaths per minute (breaths/min)
Standard Deviation 2.88
|
|
Change From Baseline in Respiratory Rate
PK-tR: Post-Infusion
|
—
|
0.4 Breaths per minute (breaths/min)
Standard Deviation 5.73
|
-3.3 Breaths per minute (breaths/min)
Standard Deviation 5.85
|
0.3 Breaths per minute (breaths/min)
Standard Deviation 2.55
|
|
Change From Baseline in Respiratory Rate
FDR: Pre-Infusion
|
-6.3 Breaths per minute (breaths/min)
Standard Deviation 4.73
|
-0.8 Breaths per minute (breaths/min)
Standard Deviation 3.97
|
-1.3 Breaths per minute (breaths/min)
Standard Deviation 1.67
|
0.4 Breaths per minute (breaths/min)
Standard Deviation 2.85
|
|
Change From Baseline in Respiratory Rate
PK-tR: Pre-Infusion
|
—
|
1.5 Breaths per minute (breaths/min)
Standard Deviation 7.01
|
-3.5 Breaths per minute (breaths/min)
Standard Deviation 5.74
|
0.2 Breaths per minute (breaths/min)
Standard Deviation 2.79
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: SAS with evaluable participants for this endpoint were analyzed. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
Change in systolic and diastolic blood pressure at pre-infusion and post-infusion at end of the study were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, SBP refers to systolic blood pressure, DBP refers to diastolic blood pressure.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=61 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Blood Pressure
SBP - PK-tR: Post-Infusion
|
—
|
-1.8 Millimeter of mercury (mmHg)
Standard Deviation 8.61
|
3.8 Millimeter of mercury (mmHg)
Standard Deviation 6.4
|
4.9 Millimeter of mercury (mmHg)
Standard Deviation 15.14
|
|
Change From Baseline in Blood Pressure
DBP - PK-tR: Post-Infusion
|
—
|
-2.4 Millimeter of mercury (mmHg)
Standard Deviation 6.67
|
-2.3 Millimeter of mercury (mmHg)
Standard Deviation 7.68
|
-0.9 Millimeter of mercury (mmHg)
Standard Deviation 7.93
|
|
Change From Baseline in Blood Pressure
SBP - FDR: Pre-Infusion
|
-2.7 Millimeter of mercury (mmHg)
Standard Deviation 1.53
|
4.5 Millimeter of mercury (mmHg)
Standard Deviation 11.82
|
4.0 Millimeter of mercury (mmHg)
Standard Deviation 9.25
|
1.8 Millimeter of mercury (mmHg)
Standard Deviation 12.87
|
|
Change From Baseline in Blood Pressure
SBP - FDR: Post-Infusion
|
-8.7 Millimeter of mercury (mmHg)
Standard Deviation 8.50
|
3 Millimeter of mercury (mmHg)
Standard Deviation 11.49
|
2.7 Millimeter of mercury (mmHg)
Standard Deviation 8.65
|
-1.4 Millimeter of mercury (mmHg)
Standard Deviation 12.08
|
|
Change From Baseline in Blood Pressure
SBP - PK-tR: Pre-Infusion
|
—
|
0.9 Millimeter of mercury (mmHg)
Standard Deviation 8.04
|
3.5 Millimeter of mercury (mmHg)
Standard Deviation 9.15
|
1.0 Millimeter of mercury (mmHg)
Standard Deviation 14.54
|
|
Change From Baseline in Blood Pressure
DBP - FDR: Pre-Infusion
|
0.7 Millimeter of mercury (mmHg)
Standard Deviation 8.02
|
1.8 Millimeter of mercury (mmHg)
Standard Deviation 9.73
|
1.4 Millimeter of mercury (mmHg)
Standard Deviation 9.69
|
1.8 Millimeter of mercury (mmHg)
Standard Deviation 8.66
|
|
Change From Baseline in Blood Pressure
DBP - FDR: Post-Infusion
|
5.3 Millimeter of mercury (mmHg)
Standard Deviation 8.74
|
3 Millimeter of mercury (mmHg)
Standard Deviation 9.72
|
0.3 Millimeter of mercury (mmHg)
Standard Deviation 6.43
|
0.4 Millimeter of mercury (mmHg)
Standard Deviation 9.21
|
|
Change From Baseline in Blood Pressure
DBP - PK-tR: Pre-Infusion
|
—
|
2.3 Millimeter of mercury (mmHg)
Standard Deviation 9.65
|
0.5 Millimeter of mercury (mmHg)
Standard Deviation 10.28
|
1.5 Millimeter of mercury (mmHg)
Standard Deviation 7.90
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen at the time of sampling, AlA refers to alanine aminotransferase, AP refers to alkaline phosphatase, AsA refers to aspartate aminotransferase.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=60 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Normal to Normal
|
3 Participants
|
43 Participants
|
20 Participants
|
55 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Normal to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
10 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
1 Participants
|
7 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
6 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Normal to Normal
|
3 Participants
|
8 Participants
|
11 Participants
|
100 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Normal to Abnormal NCS
|
0 Participants
|
4 Participants
|
4 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Normal to Normal
|
3 Participants
|
7 Participants
|
15 Participants
|
53 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Normal to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
12 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
5 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Normal to Normal
|
2 Participants
|
4 Participants
|
13 Participants
|
82 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Abnormal NCS to Normal
|
0 Participants
|
1 Participants
|
1 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
4 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Normal to Normal
|
2 Participants
|
20 Participants
|
11 Participants
|
61 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Normal to Abnormal NCS
|
0 Participants
|
9 Participants
|
4 Participants
|
13 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Abnormal NCS to Abnormal NCS
|
1 Participants
|
3 Participants
|
1 Participants
|
8 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Abnormal CS to Normal
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
4 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AlA- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
14 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
4 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AP- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
5 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
AsA- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
7 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Normal to Abnormal NCS
|
1 Participants
|
1 Participants
|
0 Participants
|
6 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Bilirubin- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Glucose- FDR: Abnormal NCS to Normal
|
0 Participants
|
11 Participants
|
5 Participants
|
20 Participants
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion. Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported. In the below table, FDR refers to fixed dose regimen, PK-tR refers to PK tailored regimen at the time of sampling, Leu refers to leukocytes, MCV refers to mean corpuscular volume, Lym/Leu refers to lymphocytes/leukocytes.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=60 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Normal to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
4 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Normal to Normal
|
2 Participants
|
28 Participants
|
11 Participants
|
97 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Normal to Abnormal NCS
|
0 Participants
|
5 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Abnormal NCS to Normal
|
0 Participants
|
5 Participants
|
2 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Abnormal NCS to Abnormal NCS
|
1 Participants
|
5 Participants
|
6 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Eosinophils/Leu- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Normal to Normal
|
3 Participants
|
34 Participants
|
18 Participants
|
70 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Normal to Abnormal NCS
|
0 Participants
|
3 Participants
|
3 Participants
|
8 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Abnormal NCS toAbnormal NCS
|
0 Participants
|
4 Participants
|
0 Participants
|
7 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Erythrocytes MCV- FDR:Abnormal CS to Abnormal CS
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Normal to Normal
|
2 Participants
|
34 Participants
|
15 Participants
|
84 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Normal to Abnormal NCS
|
0 Participants
|
0 Participants
|
2 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Abnormal NCS to Normal
|
0 Participants
|
6 Participants
|
4 Participants
|
6 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Abnormal CS to Normal
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Abnormal CS to Abnormal NCS
|
1 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Normal to Normal
|
2 Participants
|
37 Participants
|
20 Participants
|
89 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Abnormal NCS to Normal
|
0 Participants
|
3 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
1 Participants
|
1 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Abnormal CS to Normal
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Normal to Normal
|
1 Participants
|
37 Participants
|
13 Participants
|
87 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Normal to Abnormal NCS
|
1 Participants
|
4 Participants
|
4 Participants
|
5 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Abnormal NCS to Normal
|
1 Participants
|
2 Participants
|
2 Participants
|
10 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Leukocytes- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Normal to Normal
|
0 Participants
|
39 Participants
|
19 Participants
|
98 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Normal to Abnormal NCS
|
2 Participants
|
0 Participants
|
1 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Abnormal NCS to Normal
|
0 Participants
|
3 Participants
|
1 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Abnormal NCS to Abnormal NCS
|
1 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Lym/Leu- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Normal to Normal
|
2 Participants
|
40 Participants
|
21 Participants
|
88 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Normal to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
4 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Abnormal NCS to Normal
|
0 Participants
|
2 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Abnormal NCS to Abnormal NCS
|
1 Participants
|
0 Participants
|
0 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Platelets- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Normal to Normal
|
0 Participants
|
4 Participants
|
3 Participants
|
10 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Normal to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Abnormal NCS to Normal
|
0 Participants
|
2 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Abnormal NCS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hematocrit- PK-t R: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Normal to Normal
|
0 Participants
|
7 Participants
|
4 Participants
|
11 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Normal to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Abnormal NCS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Abnormal CS to Normal
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Hematology Laboratory Assessments
Hemoglobin- PK-t R: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion.Here, number of participants analyzed refer to the number of participants evaluable for this outcome at specified time point.
The number of participants with clinically significant shifts from "normal" "abnormal clinically significant (CS)" and "abnormal not clinically significant (abnormal NCS)" at baseline to "normal" "abnormal clinically significant (CS) and abnormal clinically significant (NCS)" at completion were reported.. In the below table, HDL refers to high density lipoprotein, LDL refers to low density lipoprotein, VLDL refers to very low density lipoprotein.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=60 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Normal to Normal
|
3 Participants
|
8 Participants
|
12 Participants
|
65 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Normal to Abnormal NCS
|
0 Participants
|
5 Participants
|
0 Participants
|
4 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
2 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
3 Participants
|
8 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
19 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Cholesterol- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Normal to Normal
|
0 Participants
|
5 Participants
|
10 Participants
|
68 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Normal to Abnormal NCS
|
0 Participants
|
2 Participants
|
3 Participants
|
13 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
2 Participants
|
7 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
0 Participants
|
5 Participants
|
20 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
2 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- PK-tR: Normal to Normal
|
0 Participants
|
6 Participants
|
3 Participants
|
7 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- PK-tR: Normal to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- PK-tR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- PK-tR: Abnormal NCS to Normal
|
0 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDLCholesterol-PK-tR:Abnormal NCS to Abnormal NCS
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol-PK-tR:Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol-PK-tR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Normal to Normal
|
3 Participants
|
30 Participants
|
14 Participants
|
67 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Normal to Abnormal NCS
|
0 Participants
|
4 Participants
|
2 Participants
|
8 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Abnormal NCS to Normal
|
0 Participants
|
7 Participants
|
4 Participants
|
10 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- FDR: Abnormal NCS toAbnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
14 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol- PK-tR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
VLDL Cholesterol-PK-tR:Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
2 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
HDL Cholesterol- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Normal to Normal
|
3 Participants
|
7 Participants
|
7 Participants
|
56 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Normal to Abnormal NCS
|
0 Participants
|
2 Participants
|
0 Participants
|
9 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Abnormal NCS to Normal
|
0 Participants
|
0 Participants
|
2 Participants
|
7 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Abnormal NCS to Abnormal NCS
|
0 Participants
|
0 Participants
|
5 Participants
|
20 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Abnormal CS to Abnormal NCS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
LDL Cholesterol- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Normal to Normal
|
1 Participants
|
3 Participants
|
9 Participants
|
84 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Normal to Abnormal NCS
|
0 Participants
|
11 Participants
|
5 Participants
|
8 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Normal to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Abnormal NCS to Normal
|
1 Participants
|
4 Participants
|
2 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Abnormal NCS to Abnormal NCS
|
1 Participants
|
7 Participants
|
2 Participants
|
3 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Abnormal NCS to Abnormal CS
|
0 Participants
|
0 Participants
|
2 Participants
|
2 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Abnormal CS to Normal
|
0 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Number of Participants With Shifts in Lipid Panel Assessments
Triglycerides- FDR: Abnormal CS to Abnormal CS
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion.
Binding antibodies (IgG and IgM) against FVIII, polyethylene glycol (PEG) and PEGylated FVIII (PEG-FVIII) were analyzed using enzyme-linked immunosorbent assay (ELISA).
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Binding Antibodies
IgG to FVIII
|
0 Participants
|
2 Participants
|
1 Participants
|
2 Participants
|
|
Number of Participants With Binding Antibodies
IgM to FVIII
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Binding Antibodies
IgG to PEG-FVIII
|
1 Participants
|
3 Participants
|
0 Participants
|
4 Participants
|
|
Number of Participants With Binding Antibodies
IgM to PEG-FVIII
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Binding Antibodies
IgG to PEG
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants With Binding Antibodies
IgM to PEG
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline through end of study (53 months)Population: SAS included all participants with at least 1 BAX 855 infusion.
Testing for binding of anti-CHO protein antibodies was performed on citrate-anti-coagulated plasma using an ELISA employing polyclonal antihuman IgG antibodies.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=3 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 Participants
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 Participants
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: FAS with population of age greater than or equal to 18 years were analyzed.
Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the bleed severity for participants \>=18 years of age as: severity of spontaneous bleeding in my joints (unrelated to injury or activity), spontaneous bleeding in my muscles (unrelated to injury or activity), prolonged bleeding after injury in spite of treatment, intense pain because of bleeding event, joint pain due to active bleed and bleeding during personal hygiene routine, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)\*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=125 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Bleed Severity
Fixed dose regimen
|
-7.824 Score on a scale
Standard Deviation 18.514
|
—
|
—
|
—
|
|
Change From Baseline in Bleed Severity
PK-tailored regimen
|
-16.667 Score on a scale
Standard Deviation 14.337
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: FAS with population of age greater than or equal to 18 years were analyzed.
Hemophilia symptom (haemo-SYM) questionnaire has two subscales: pain and bleed. It was used to asses the pain severity for participants \>=18 years of age as: pain because of swelling in my joints, climbing stairs, upon waking in the morning, active arthritis; constant pain, in my muscles, that needs medication; joint sensitivity to weather conditions; reduced range of joint movement, joint deformity, sleep disturbance because of pain or bleeds, blood in my urine, nose bleeds and assigned a score of 0=Absent, 1=very mild, 2=mild, 3=moderate, 4=severe and 5=very severe. The score was determined as (mean score/5)\*100 where mean score is the mean of the available results in the particular subscale. Higher scores on the Haemo-SYM indicate more severe symptoms. Therefore, negative change scores indicate that symptoms have improved. Here 'n' refers to the number of participants evaluable for this endpoint.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=125 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Pain Severity
Fixed dose regimen
|
-1.341 Score on a scale
Standard Deviation 11.531
|
—
|
—
|
—
|
|
Change From Baseline in Pain Severity
PK-tailored regimen
|
-8.889 Score on a scale
Standard Deviation 20.659
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: FAS with population of age greater than or equal to 14 years were analyzed.
HRQoL in participants aged \>=14 years was measured using the SF-36 questionnaire. The questionnaire was divided into 8 domains and scored as: physical functioning (1=yes, limited a lot to 3=no, not limited at all), role-physical (1=all of the time to 5=none of the time), bodily pain (1=very severe to 6=none), general health (1=poor to 5=excellent), vitality (1=none of the time to 5=all of the time), social functioning (1=all of the time: to 5=none of the time), role emotional (1=all of the time to 5=none of the time) and mental health (1=all of the time to 5=none of the time). The score for each domain is then to be transformed to a 0-100 range as \[(actual raw score-lowest possible raw score)/possible raw score range\]\*100. Positive change scores indicate improved HRQoL. in the below table 'FDR' indicates fixed dose regimen, 'PK-tr' indicates pharmacokinetically tailored regimen and 'n' refers to the number of participants evaluable for this endpoint.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=26 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=125 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Physical functioning
|
3.875 Score on a scale
Standard Deviation 4.912
|
-0.108 Score on a scale
Standard Deviation 3.021
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Physical functioning
|
1.000 Score on a scale
Standard Deviation 1.414
|
-0.364 Score on a scale
Standard Deviation 4.456
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Role-physical
|
0.8 Score on a scale
Standard Deviation 4.13
|
0.4 Score on a scale
Standard Deviation 3.90
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Bodily pain
|
1.30 Score on a scale
Standard Deviation 2.530
|
0.81 Score on a scale
Standard Deviation 2.051
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Bodily pain
|
-0.30 Score on a scale
Standard Deviation 2.687
|
0.39 Score on a scale
Standard Deviation 3.250
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: General health
|
2.33 Score on a scale
Standard Deviation 3.533
|
0.48 Score on a scale
Standard Deviation 3.837
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Role-physical
|
1.5 Score on a scale
Standard Deviation 3.54
|
1.2 Score on a scale
Standard Deviation 2.86
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Social functioning
|
1.0 Score on a scale
Standard Deviation 1.41
|
-1.2 Score on a scale
Standard Deviation 2.18
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Role emotional
|
-0.6 Score on a scale
Standard Deviation 2.26
|
-0.4 Score on a scale
Standard Deviation 2.44
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Role emotional
|
0.5 Score on a scale
Standard Deviation 0.71
|
-1.1 Score on a scale
Standard Deviation 2.47
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Mental health
|
-1.0 Score on a scale
Standard Deviation 1.93
|
-0.3 Score on a scale
Standard Deviation 3.17
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Mental health
|
0.5 Score on a scale
Standard Deviation 0.71
|
-0.5 Score on a scale
Standard Deviation 2.70
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Physical component score
|
8.280 Score on a scale
Standard Deviation 12.985
|
1.986 Score on a scale
Standard Deviation 7.059
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Physical component score
|
2.647 Score on a scale
Standard Deviation 7.990
|
3.414 Score on a scale
Standard Deviation 9.405
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Mental component score
|
-4.780 Score on a scale
Standard Deviation 6.680
|
-1.501 Score on a scale
Standard Deviation 9.059
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Mental component score
|
3.931 Score on a scale
Standard Deviation 7.769
|
-4.185 Score on a scale
Standard Deviation 8.634
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: General health
|
2.30 Score on a scale
Standard Deviation 0.424
|
1.56 Score on a scale
Standard Deviation 4.328
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Vitality
|
-0.3 Score on a scale
Standard Deviation 3.20
|
0.1 Score on a scale
Standard Deviation 2.39
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
PK-t R: Vitality
|
2.5 Score on a scale
Standard Deviation 3.54
|
0.7 Score on a scale
Standard Deviation 3.80
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
FDR: Social functioning
|
0.4 Score on a scale
Standard Deviation 1.69
|
0.0 Score on a scale
Standard Deviation 1.39
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, end of study (53 months)Population: FAS with population of age less than 14 years were analyzed.
HRQoL in participants aged \<14 years was measured using the PedsQL. It capture data for the following domains: physical functioning, emotional functioning, social functioning, school functioning, psychosocial functioning, physical health and a total score. Each question of the PedsQL was scored as Never: 100, almost never: 75, sometimes: 50, often: 25, almost always: 0. The mean of the individual question scores was calculated. Lower scores on the PedsQL indicating worse HRQoL. Here, FDR refers to fixed dose regimen, PK-t R refers to PK-tailored regimen. Here 'n' refers to the number of participants evaluable for this endpoint. Here 'n' refers to the number of participants evaluable for this endpoint.
Outcome measures
| Measure |
BAX 855: Age < 2 Years
n=62 Participants
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=26 Participants
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
PK-t R: Physical functioning
|
-0.446 Score on a scale
Standard Deviation 12.425
|
7.813 Score on a scale
Standard Deviation 6.629
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
FDR: Social functioning
|
-0.4 Score on a scale
Standard Deviation 16.22
|
8.3 Score on a scale
Standard Deviation 15.28
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
FDR: School functioning
|
3.472 Score on a scale
Standard Deviation 24.107
|
15.000 Score on a scale
Standard Deviation 13.229
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
FDR: Physical functioning
|
0.534 Score on a scale
Standard Deviation 17.896
|
3.125 Score on a scale
Standard Deviation 15.625
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
FDR: Emotional functioning
|
-1.0 Score on a scale
Standard Deviation 16.93
|
8.3 Score on a scale
Standard Deviation 20.21
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
PK-t R: Emotional functioning
|
-2.1 Score on a scale
Standard Deviation 11.85
|
10.0 Score on a scale
Standard Deviation 14.14
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
PK-t R: Social functioning
|
-6.4 Score on a scale
Standard Deviation 19.73
|
5.0 Score on a scale
Standard Deviation 7.07
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
PK-t R: School functioning
|
-9.167 Score on a scale
Standard Deviation 16.591
|
-12.500 Score on a scale
Standard Deviation 17.678
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
FDR: Psychosocial functioning
|
0.331 Score on a scale
Standard Deviation 15.283
|
10.556 Score on a scale
Standard Deviation 15.486
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
PK-t R: Psychosocial functioning
|
-6.099 Score on a scale
Standard Deviation 13.025
|
0.833 Score on a scale
Standard Deviation 3.536
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
FDR: Total score
|
0.366 Score on a scale
Standard Deviation 14.811
|
7.971 Score on a scale
Standard Deviation 15.230
|
—
|
—
|
|
Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
PK-t R: Total score
|
-3.915 Score on a scale
Standard Deviation 12.201
|
3.261 Score on a scale
Standard Deviation 0.000
|
—
|
—
|
Adverse Events
BAX 855: Age < 2 Years
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
BAX 855: Age >= 2 to <12 Years
Serious events: 7 serious events
Other events: 40 other events
Deaths: 0 deaths
BAX 855: Age >= 12 to <17 Years
Serious events: 4 serious events
Other events: 12 other events
Deaths: 1 deaths
BAX 855: Age >= 17 Years
Serious events: 22 serious events
Other events: 62 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BAX 855: Age < 2 Years
n=3 participants at risk
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 participants at risk
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 participants at risk
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 participants at risk
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Blood and lymphatic system disorders
Splenic haematoma
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.2%
2/62 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Musculoskeletal and connective tissue disorders
Muscle haemorrhage
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to lung
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Renal cancer metastatic
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Vascular disorders
Haematoma
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
2/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Gastrointestinal disorders
Ileus
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Gastrointestinal disorders
Pancreatitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
2/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
General disorders
Pyrexia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.2%
2/62 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Abscess oral
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Appendicitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Cystitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Device related infection
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Device related sepsis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Incision site abscess
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Laryngitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Plasmodium falciparum infection
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Pneumonia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
2/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Skin infection
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Streptococcal bacteraemia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
2/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Femoral neck fracture
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Nasal injury
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Scapula fracture
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Splenic rupture
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Traumatic fracture
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Wound haemorrhage
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Wrist fracture
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Investigations
Transaminases increased
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Renal and urinary disorders
Ureterolithiasis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Nervous system disorders
Cerebral haemorrhage
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
Other adverse events
| Measure |
BAX 855: Age < 2 Years
n=3 participants at risk
Participants of age \< 2 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 2 to <12 Years
n=62 participants at risk
Participants of age \>= 2 to \<12 years received an infusion of 50 +/- 10 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 12 to <17 Years
n=26 participants at risk
Participants of age \>= 12 to \<17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
BAX 855: Age >= 17 Years
n=125 participants at risk
Participants of age \>= 17 years received an infusion of 45 +/- 5 IU/kg of BAX 855 twice weekly; could be increased to 80 IU/kg or a PK-tailored prophylactic dose (should not exceed 80 IU/kg and the FVIII peak level was not to exceed 200%) at least twice weekly based on the participant's individual PK to maintain FVIII trough levels of \>= 3% until at least 100 EDs were reached.
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
4.8%
3/62 • Number of events 4 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
7.7%
2/26 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
11.2%
14/125 • Number of events 20 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
5.6%
7/125 • Number of events 7 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
9.7%
6/62 • Number of events 8 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
5.6%
7/125 • Number of events 8 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
9.7%
6/62 • Number of events 8 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
General disorders
Pyrexia
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
17.7%
11/62 • Number of events 15 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
4.0%
5/125 • Number of events 5 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Ear infection
|
33.3%
1/3 • Number of events 4 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
6.5%
4/62 • Number of events 6 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Enterovirus infection
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Gastroenteritis
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
2/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Influenza
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
8.1%
5/62 • Number of events 5 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
2.4%
3/125 • Number of events 3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
21.0%
13/62 • Number of events 24 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
7.7%
2/26 • Number of events 5 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
20.0%
25/125 • Number of events 39 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Otitis media acute
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Pharyngitis
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
4.8%
3/62 • Number of events 3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
4.0%
5/125 • Number of events 9 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
6.5%
4/62 • Number of events 5 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Rhinitis
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
6.5%
4/62 • Number of events 4 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
11.5%
3/26 • Number of events 5 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
17.7%
11/62 • Number of events 22 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
9.6%
12/125 • Number of events 13 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
1/62 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
7.7%
2/26 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.2%
4/125 • Number of events 6 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.2%
2/62 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
7.7%
2/26 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
1.6%
2/125 • Number of events 2 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Injury, poisoning and procedural complications
Skull fracture
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Nervous system disorders
Febrile convulsion
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
9.7%
6/62 • Number of events 6 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
3.8%
1/26 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
9.6%
12/125 • Number of events 23 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
21.0%
13/62 • Number of events 17 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
5.6%
7/125 • Number of events 8 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal ulceration
|
33.3%
1/3 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/62 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/125 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/3 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
9.7%
6/62 • Number of events 7 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.00%
0/26 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
0.80%
1/125 • Number of events 1 • From start of study drug administration up to 53 months
Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER