Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

BAX 855 Continuation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01945593
Recruitment Status : Completed
First Posted : September 18, 2013
Results First Posted : May 1, 2019
Last Update Posted : May 1, 2019
Sponsor:
Collaborator:
Baxalta Innovations GmbH, now part of Shire
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Condition Hemophilia A
Intervention Biological: BAX855
Enrollment 218
Recruitment Details 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).
Pre-assignment Details A total of 218 subjects were enrolled, of them 216 subjects received treatment.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description 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. 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. 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. 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.
Period Title: Overall Study
Started 3 64 26 125
Completed 3 56 23 105
Not Completed 0 8 3 20
Reason Not Completed
Adverse Event             0             0             0             5
Physician Decision             0             0             1             1
Withdrawal by Subject             0             2             0             4
Protocol Violation             0             2             1             3
Death             0             0             1             0
Other: Terminated             0             0             0             1
Other: Surgical Procedure             0             2             0             6
Other: Screen Failure             0             2             0             0
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years Total
Hide Arm/Group Description 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. 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. 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. 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 of all reporting groups
Overall Number of Baseline Participants 3 62 26 125 216
Hide Baseline Analysis Population Description
Safety analysis set included all participants with at least 1 BAX855 infusion.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants 62 participants 26 participants 125 participants 216 participants
1.0  (0.00) 6.0  (2.47) 13.8  (1.30) 33.5  (11.68) 22.8  (15.67)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 62 participants 26 participants 125 participants 216 participants
Female
0
   0.0%
1
   1.6%
0
   0.0%
0
   0.0%
1
   0.5%
Male
3
 100.0%
61
  98.4%
26
 100.0%
125
 100.0%
215
  99.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 62 participants 26 participants 125 participants 216 participants
Hispanic or Latino
0
   0.0%
3
   4.8%
0
   0.0%
7
   5.6%
10
   4.6%
Not Hispanic or Latino
3
 100.0%
59
  95.2%
26
 100.0%
118
  94.4%
206
  95.4%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 62 participants 26 participants 125 participants 216 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  33.3%
15
  24.2%
5
  19.2%
37
  29.6%
58
  26.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
3
   4.8%
1
   3.8%
0
   0.0%
4
   1.9%
White
2
  66.7%
42
  67.7%
20
  76.9%
88
  70.4%
152
  70.4%
More than one race
0
   0.0%
1
   1.6%
0
   0.0%
0
   0.0%
1
   0.5%
Unknown or Not Reported
0
   0.0%
1
   1.6%
0
   0.0%
0
   0.0%
1
   0.5%
1.Primary Outcome
Title Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 57 26 118
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
2.Primary Outcome
Title Annualized Bleed Rate (ABR) - Spontaneous Bleeds
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set (FAS) included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Median (95% Confidence Interval)
Unit of Measure: Bleeds per year
Fixed-dose regimen (FDR): Every 5 Days Number Analyzed 0 participants 0 participants 7 participants 49 participants
1.381
(0.728 to 2.620)
1.160
(0.727 to 1.852)
FDR: Every 7 Days Number Analyzed 0 participants 0 participants 1 participants 14 participants
0.669
(0.061 to 7.377)
1.992
(0.822 to 4.827)
FDR: Twice Weekly Number Analyzed 3 participants 59 participants 20 participants 104 participants
0.614
(0.154 to 2.454)
0.792
(0.518 to 1.210)
1.439
(0.942 to 2.200)
1.293
(0.913 to 1.832)
PK-tailored regimen (PK-t R) Number Analyzed 0 participants 10 participants 4 participants 11 participants
1.078
(0.450 to 2.581)
1.602
(0.270 to 9.486)
0.868
(0.365 to 2.067)
3.Secondary Outcome
Title Total Annualized Bleed Rate (ABR)
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Mean (Standard Deviation)
Unit of Measure: Bleeds per year
1.855  (1.000) 2.370  (3.027) 3.175  (2.797) 2.426  (3.258)
4.Secondary Outcome
Title Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all participants with at least 1 BAX 855 infusion. Here ‘N’ refers to the number of participants evaluable for this outcome.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 43 24 95
Measure Type: Number
Unit of Measure: Treated bleeds
Excellent 3 108 98 229
Good 3 66 49 250
Fair 0 5 7 36
None 0 0 1 3
Not Reported 0 13 13 26
5.Secondary Outcome
Title BAX 855 Infusions Needed to Treat Bleeding Episodes
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Mean (Standard Deviation)
Unit of Measure: Infusions
1.3  (0.52) 1.4  (1.14) 1.4  (1.81) 1.4  (1.10)
6.Secondary Outcome
Title Total Time Intervals Between Bleeding Episodes
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 2 36 21 84
Median (Full Range)
Unit of Measure: Months
5.700
(4.074 to 7.327)
5.051
(0.756 to 14.867)
5.232
(1.228 to 14.571)
5.818
(0.617 to 22.686)
7.Secondary Outcome
Title Average Dose of BAX 855 Per Prophylactic Infusion
Hide Description The average dose of BAX 855 per prophylactic infusion was reported.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
SAS included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Mean (Standard Deviation)
Unit of Measure: International units per kilogram (IU/kg)
50.748  (11.312) 53.855  (7.754) 53.356  (10.344) 49.727  (8.677)
8.Secondary Outcome
Title Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
SAS included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Measure Type: Count of Participants
Unit of Measure: Participants
AE
3
 100.0%
52
  83.9%
18
  69.2%
101
  80.8%
SAE
0
   0.0%
7
  11.3%
4
  15.4%
22
  17.6%
9.Secondary Outcome
Title Change From Baseline in Body Temperature
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 61 26 125
Mean (Standard Deviation)
Unit of Measure: Degree celsius
FDR: Pre-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-0.23  (0.208) -0.05  (0.386) -0.08  (0.409) -0.01  (0.376)
FDR: Post-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-0.07  (0.306) -0.03  (0.394) 0.03  (0.375) -0.02  (0.395)
PK-tR: Pre-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
0.28  (0.255) -0.08  (0.189) -0.10  (0.595)
PK-tR: Post-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
0.13  (0.362) 0.03  (0.263) 0.01  (0.593)
10.Secondary Outcome
Title Change From Baseline in Pulse Rate
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 61 26 125
Mean (Standard Deviation)
Unit of Measure: Beats per minute (beats/min)
FDR: Pre-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-20.3  (21.22) -0.1  (14.31) -7.7  (9.07) -0.4  (11.67)
FDR: Post-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-13  (19.47) 2.4  (16.72) -6.2  (8.55) -1  (10.07)
PK-tR: Pre-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
0.1  (9.13) -1.8  (13.91) 4.0  (9.15)
PK-tR: Post-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
-8.1  (14.94) -4.5  (12.40) -0.2  (7.31)
11.Secondary Outcome
Title Change From Baseline in Respiratory Rate
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 61 26 125
Mean (Standard Deviation)
Unit of Measure: Breaths per minute (breaths/min)
FDR: Pre-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-6.3  (4.73) -0.8  (3.97) -1.3  (1.67) 0.4  (2.85)
FDR: Post-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-6.3  (4.73) -1.3  (3.55) -1.5  (1.85) 0.2  (2.88)
PK-tR: Pre-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
1.5  (7.01) -3.5  (5.74) 0.2  (2.79)
PK-tR: Post-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
0.4  (5.73) -3.3  (5.85) 0.3  (2.55)
12.Secondary Outcome
Title Change From Baseline in Blood Pressure
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 61 26 125
Mean (Standard Deviation)
Unit of Measure: Millimeter of mercury (mmHg)
SBP - FDR: Pre-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-2.7  (1.53) 4.5  (11.82) 4.0  (9.25) 1.8  (12.87)
SBP - FDR: Post-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
-8.7  (8.50) 3  (11.49) 2.7  (8.65) -1.4  (12.08)
SBP - PK-tR: Pre-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
0.9  (8.04) 3.5  (9.15) 1.0  (14.54)
SBP - PK-tR: Post-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
-1.8  (8.61) 3.8  (6.4) 4.9  (15.14)
DBP - FDR: Pre-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
0.7  (8.02) 1.8  (9.73) 1.4  (9.69) 1.8  (8.66)
DBP - FDR: Post-Infusion Number Analyzed 3 participants 61 participants 26 participants 125 participants
5.3  (8.74) 3  (9.72) 0.3  (6.43) 0.4  (9.21)
DBP - PK-tR: Pre-Infusion Number Analyzed 0 participants 10 participants 4 participants 11 participants
2.3  (9.65) 0.5  (10.28) 1.5  (7.90)
DBP - PK-tR: Post-Infusion Number Analyzed 0 participants 61 participants 26 participants 125 participants
-2.4  (6.67) -2.3  (7.68) -0.9  (7.93)
13.Secondary Outcome
Title Number of Participants With Shifts in Clinical Chemistry Laboratory Assessments.
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 60 26 125
Measure Type: Count of Participants
Unit of Measure: Participants
AlA- FDR: Normal to Normal
3
 100.0%
43
  71.7%
20
  76.9%
55
  44.0%
AlA- FDR: Normal to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
10
   8.0%
AlA- FDR: Normal to Abnormal CS
0
   0.0%
0
   0.0%
1
   3.8%
7
   5.6%
AlA- FDR: Abnormal NCS to Normal
0
   0.0%
0
   0.0%
0
   0.0%
6
   4.8%
AlA- FDR: Abnormal NCS to Abnormal NCS
0
   0.0%
1
   1.7%
0
   0.0%
14
  11.2%
AlA- FDR: Abnormal NCS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
AlA- FDR: Abnormal CS to Normal
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
AlA- FDR: Abnormal CS to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
AlA- FDR: Abnormal CS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
3
   2.4%
AP- FDR: Normal to Normal
3
 100.0%
8
  13.3%
11
  42.3%
100
  80.0%
AP- FDR: Normal to Abnormal NCS
0
   0.0%
4
   6.7%
4
  15.4%
3
   2.4%
AP- FDR: Normal to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
AP- FDR: Abnormal NCS to Normal
0
   0.0%
0
   0.0%
1
   3.8%
0
   0.0%
AP- FDR: Abnormal NCS to Abnormal NCS
0
   0.0%
4
   6.7%
0
   0.0%
2
   1.6%
AP- FDR: Abnormal NCS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
AP- FDR: Abnormal CS to Normal
0
   0.0%
0
   0.0%
0
   0.0%
3
   2.4%
AP- FDR: Abnormal CS to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
AP- FDR: Abnormal CS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
AsA- FDR: Normal to Normal
3
 100.0%
7
  11.7%
15
  57.7%
53
  42.4%
AsA- FDR: Normal to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
12
   9.6%
AsA- FDR: Normal to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
5
   4.0%
AsA- FDR: Abnormal NCS to Normal
0
   0.0%
0
   0.0%
0
   0.0%
5
   4.0%
AsA- FDR: Abnormal NCS to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
7
   5.6%
AsA- FDR: Abnormal NCS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
AsA- FDR: Abnormal CS to Normal
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
AsA- FDR: Abnormal CS to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
AsA- FDR: Abnormal CS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Bilirubin- FDR: Normal to Normal
2
  66.7%
4
   6.7%
13
  50.0%
82
  65.6%
Bilirubin- FDR: Normal to Abnormal NCS
1
  33.3%
1
   1.7%
0
   0.0%
6
   4.8%
Bilirubin- FDR: Normal to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Bilirubin- FDR: Abnormal NCS to Normal
0
   0.0%
1
   1.7%
1
   3.8%
2
   1.6%
Bilirubin- FDR: Abnormal NCS to Abnormal NCS
0
   0.0%
1
   1.7%
0
   0.0%
4
   3.2%
Bilirubin- FDR: Abnormal NCS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Bilirubin- FDR: Abnormal CS to Normal
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Bilirubin- FDR: Abnormal CS to Abnormal NCS
0
   0.0%
0
   0.0%
1
   3.8%
0
   0.0%
Bilirubin- FDR: Abnormal CS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Glucose- FDR: Normal to Normal
2
  66.7%
20
  33.3%
11
  42.3%
61
  48.8%
Glucose- FDR: Normal to Abnormal NCS
0
   0.0%
9
  15.0%
4
  15.4%
13
  10.4%
Glucose- FDR: Normal to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Glucose- FDR: Abnormal NCS to Normal
0
   0.0%
11
  18.3%
5
  19.2%
20
  16.0%
Glucose- FDR: Abnormal NCS to Abnormal NCS
1
  33.3%
3
   5.0%
1
   3.8%
8
   6.4%
Glucose- FDR: Abnormal NCS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Glucose- FDR: Abnormal CS to Normal
0
   0.0%
1
   1.7%
0
   0.0%
0
   0.0%
Glucose- FDR: Abnormal CS to Abnormal NCS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Glucose- FDR: Abnormal CS to Abnormal CS
0
   0.0%
0
   0.0%
0
   0.0%
4
   3.2%
14.Secondary Outcome
Title Number of Participants With Shifts in Hematology Laboratory Assessments
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 60 26 125
Measure Type: Count of Participants
Unit of Measure: Participants
Eosinophils/Leu- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
2
  66.7%
28
  46.7%
11
  42.3%
97
  77.6%
Eosinophils/Leu- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
5
   8.3%
1
   3.8%
1
   0.8%
Eosinophils/Leu- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Eosinophils/Leu- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
5
   8.3%
2
   7.7%
2
   1.6%
Eosinophils/Leu- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
5
   8.3%
6
  23.1%
1
   0.8%
Eosinophils/Leu- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Eosinophils/Leu- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Eosinophils/Leu- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Eosinophils/Leu- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
0
   0.0%
Erythrocytes MCV- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
3
 100.0%
34
  56.7%
18
  69.2%
70
  56.0%
Erythrocytes MCV- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
3
   5.0%
3
  11.5%
8
   6.4%
Erythrocytes MCV- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Erythrocytes MCV- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
Erythrocytes MCV- FDR: Abnormal NCS toAbnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
4
   6.7%
0
   0.0%
7
   5.6%
Erythrocytes MCV- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Erythrocytes MCV- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Erythrocytes MCV- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Erythrocytes MCV- FDR:Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
1
   0.8%
Hematocrit- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
2
  66.7%
34
  56.7%
15
  57.7%
84
  67.2%
Hematocrit- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
3
   2.4%
Hematocrit- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
3
   2.4%
Hematocrit- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
6
  10.0%
4
  15.4%
6
   4.8%
Hematocrit- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
3
   2.4%
Hematocrit- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Hematocrit- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
0
   0.0%
Hematocrit- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
0
   0.0%
0
   0.0%
1
   0.8%
Hematocrit- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
2
  66.7%
37
  61.7%
20
  76.9%
89
  71.2%
Hemoglobin- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
4
   3.2%
Hemoglobin- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
Hemoglobin- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
3
   5.0%
0
   0.0%
3
   2.4%
Hemoglobin- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
1
   3.8%
3
   2.4%
Hemoglobin- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Hemoglobin- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Hemoglobin- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
0
   0.0%
Leukocytes- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
37
  61.7%
13
  50.0%
87
  69.6%
Leukocytes- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
4
   6.7%
4
  15.4%
5
   4.0%
Leukocytes- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Leukocytes- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
2
   3.3%
2
   7.7%
10
   8.0%
Leukocytes- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
1
   0.8%
Leukocytes- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Leukocytes- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
0
   0.0%
Leukocytes- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Leukocytes- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Lym/Leu- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
39
  65.0%
19
  73.1%
98
  78.4%
Lym/Leu- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
2
  66.7%
0
   0.0%
1
   3.8%
2
   1.6%
Lym/Leu- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Lym/Leu- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
3
   5.0%
1
   3.8%
2
   1.6%
Lym/Leu- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
1
   1.7%
0
   0.0%
0
   0.0%
Lym/Leu- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Lym/Leu- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Lym/Leu- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Lym/Leu- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Platelets- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
2
  66.7%
40
  66.7%
21
  80.8%
88
  70.4%
Platelets- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
4
   3.2%
Platelets- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Platelets- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
2
   3.3%
0
   0.0%
3
   2.4%
Platelets- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
0
   0.0%
0
   0.0%
3
   2.4%
Platelets- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Platelets- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Platelets- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Platelets- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Hematocrit- PK-t R: Normal to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
4
  40.0%
3
  75.0%
10
  90.9%
Hematocrit- PK-t R: Normal to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
1
  10.0%
0
   0.0%
0
   0.0%
Hematocrit- PK-t R: Normal to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hematocrit- PK-t R: Abnormal NCS to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
2
  20.0%
1
  25.0%
0
   0.0%
Hematocrit- PK-t R: Abnormal NCS to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hematocrit- PK-t R: Abnormal NCS to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hematocrit- PK-t R: Abnormal CS to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
1
   9.1%
Hematocrit- PK-t R: Abnormal CS to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hematocrit- PK-t R: Abnormal CS to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Normal to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
7
  70.0%
4
 100.0%
11
 100.0%
Hemoglobin- PK-t R: Normal to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Normal to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Abnormal NCS to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Abnormal NCS to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Abnormal NCS to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Abnormal CS to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
1
  10.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Abnormal CS to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
Hemoglobin- PK-t R: Abnormal CS to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
15.Secondary Outcome
Title Number of Participants With Shifts in Lipid Panel Assessments
Hide Description 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.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 60 26 125
Measure Type: Count of Participants
Unit of Measure: Participants
Cholesterol- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
3
 100.0%
8
  13.3%
12
  46.2%
65
  52.0%
Cholesterol- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
5
   8.3%
0
   0.0%
4
   3.2%
Cholesterol- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
2
   1.6%
Cholesterol- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
3
  11.5%
8
   6.4%
Cholesterol- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
19
  15.2%
Cholesterol- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Cholesterol- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
Cholesterol- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Cholesterol- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
HDL Cholesterol- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
5
   8.3%
10
  38.5%
68
  54.4%
HDL Cholesterol- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
2
   3.3%
3
  11.5%
13
  10.4%
HDL Cholesterol- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
3
   2.4%
HDL Cholesterol- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
7
   5.6%
HDL Cholesterol- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
5
  19.2%
20
  16.0%
HDL Cholesterol- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
0
   0.0%
HDL Cholesterol- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
HDL Cholesterol- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
HDL Cholesterol- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
LDL Cholesterol- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
3
 100.0%
7
  11.7%
7
  26.9%
56
  44.8%
LDL Cholesterol- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
2
   3.3%
0
   0.0%
9
   7.2%
LDL Cholesterol- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
3
   2.4%
LDL Cholesterol- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
7
   5.6%
LDL Cholesterol- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
5
  19.2%
20
  16.0%
LDL Cholesterol- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
0
   0.0%
LDL Cholesterol- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
LDL Cholesterol- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
LDL Cholesterol- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Triglycerides- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
3
   5.0%
9
  34.6%
84
  67.2%
Triglycerides- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
11
  18.3%
5
  19.2%
8
   6.4%
Triglycerides- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
Triglycerides- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
4
   6.7%
2
   7.7%
3
   2.4%
Triglycerides- FDR: Abnormal NCS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
1
  33.3%
7
  11.7%
2
   7.7%
3
   2.4%
Triglycerides- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
2
   7.7%
2
   1.6%
Triglycerides- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
1
   0.8%
Triglycerides- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
1
   1.7%
0
   0.0%
0
   0.0%
Triglycerides- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
VLDL Cholesterol- FDR: Normal to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
3
 100.0%
30
  50.0%
14
  53.8%
67
  53.6%
VLDL Cholesterol- FDR: Normal to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
4
   6.7%
2
   7.7%
8
   6.4%
VLDL Cholesterol- FDR: Normal to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.6%
VLDL Cholesterol- FDR: Abnormal NCS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
7
  11.7%
4
  15.4%
10
   8.0%
VLDL Cholesterol- FDR: Abnormal NCS toAbnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
14
  11.2%
VLDL Cholesterol- FDR: Abnormal NCS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
1
   0.8%
VLDL Cholesterol- FDR: Abnormal CS to Normal Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
1
   3.8%
1
   0.8%
VLDL Cholesterol- FDR: Abnormal CS to Abnormal NCS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
VLDL Cholesterol- FDR: Abnormal CS to Abnormal CS Number Analyzed 3 participants 60 participants 26 participants 125 participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
VLDL Cholesterol- PK-tR: Normal to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
6
  60.0%
3
  75.0%
7
  63.6%
VLDL Cholesterol- PK-tR: Normal to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
VLDL Cholesterol- PK-tR: Normal to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
VLDL Cholesterol- PK-tR: Abnormal NCS to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
1
  10.0%
1
  25.0%
1
   9.1%
VLDLCholesterol-PK-tR:Abnormal NCS to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
1
  10.0%
0
   0.0%
1
   9.1%
VLDL Cholesterol-PK-tR:Abnormal NCS to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
VLDL Cholesterol- PK-tR: Abnormal CS to Normal Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
2
  18.2%
VLDL Cholesterol-PK-tR:Abnormal CS to Abnormal NCS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
VLDL Cholesterol-PK-tR: Abnormal CS to Abnormal CS Number Analyzed 0 participants 10 participants 4 participants 11 participants
0
0
   0.0%
0
   0.0%
0
   0.0%
16.Secondary Outcome
Title Number of Participants With Binding Antibodies
Hide Description Binding antibodies (IgG and IgM) against FVIII, polyethylene glycol (PEG) and PEGylated FVIII (PEG-FVIII) were analyzed using enzyme-linked immunosorbent assay (ELISA).
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
SAS included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Measure Type: Count of Participants
Unit of Measure: Participants
IgG to FVIII
0
   0.0%
2
   3.2%
1
   3.8%
2
   1.6%
IgM to FVIII
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
IgG to PEG-FVIII
1
  33.3%
3
   4.8%
0
   0.0%
4
   3.2%
IgM to PEG-FVIII
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
IgG to PEG
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
IgM to PEG
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
17.Secondary Outcome
Title Number of Participants With Anti-Chinese Hamster Ovary (CHO) Antibodies
Hide Description Testing for binding of anti-CHO protein antibodies was performed on citrate-anti-coagulated plasma using an ELISA employing polyclonal antihuman IgG antibodies.
Time Frame Baseline through end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
SAS included all participants with at least 1 BAX 855 infusion.
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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.
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.
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 Number of Participants Analyzed 3 62 26 125
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
18.Secondary Outcome
Title Change From Baseline in Bleed Severity
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS with population of age greater than or equal to 18 years were analyzed.
Arm/Group Title BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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 Number of Participants Analyzed 125
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Fixed dose regimen Number Analyzed 99 participants
-7.824  (18.514)
PK-tailored regimen Number Analyzed 9 participants
-16.667  (14.337)
19.Secondary Outcome
Title Change From Baseline in Pain Severity
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS with population of age greater than or equal to 18 years were analyzed.
Arm/Group Title BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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 Number of Participants Analyzed 125
Mean (Standard Deviation)
Unit of Measure: Score on a scale
Fixed dose regimen Number Analyzed 99 participants
-1.341  (11.531)
PK-tailored regimen Number Analyzed 9 participants
-8.889  (20.659)
20.Secondary Outcome
Title Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Short Form-36 (SF-36)
Hide Description

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.

Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS with population of age greater than or equal to 14 years were analyzed.
Arm/Group Title BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 26 125
Mean (Standard Deviation)
Unit of Measure: Score on a scale
FDR: Physical functioning Number Analyzed 8 participants 75 participants
3.875  (4.912) -0.108  (3.021)
PK-t R: Physical functioning Number Analyzed 2 participants 11 participants
1.000  (1.414) -0.364  (4.456)
FDR: Role-physical Number Analyzed 8 participants 75 participants
0.8  (4.13) 0.4  (3.90)
PK-t R: Role-physical Number Analyzed 2 participants 11 participants
1.5  (3.54) 1.2  (2.86)
FDR: Bodily pain Number Analyzed 8 participants 75 participants
1.30  (2.530) 0.81  (2.051)
PK-t R: Bodily pain Number Analyzed 2 participants 11 participants
-0.30  (2.687) 0.39  (3.250)
FDR: General health Number Analyzed 8 participants 74 participants
2.33  (3.533) 0.48  (3.837)
PK-t R: General health Number Analyzed 2 participants 11 participants
2.30  (0.424) 1.56  (4.328)
FDR: Vitality Number Analyzed 8 participants 74 participants
-0.3  (3.20) 0.1  (2.39)
PK-t R: Vitality Number Analyzed 2 participants 11 participants
2.5  (3.54) 0.7  (3.80)
FDR: Social functioning Number Analyzed 8 participants 75 participants
0.4  (1.69) 0.0  (1.39)
PK-t R: Social functioning Number Analyzed 2 participants 11 participants
1.0  (1.41) -1.2  (2.18)
FDR: Role emotional Number Analyzed 8 participants 75 participants
-0.6  (2.26) -0.4  (2.44)
PK-t R: Role emotional Number Analyzed 2 participants 11 participants
0.5  (0.71) -1.1  (2.47)
FDR: Mental health Number Analyzed 8 participants 74 participants
-1.0  (1.93) -0.3  (3.17)
PK-t R: Mental health Number Analyzed 2 participants 11 participants
0.5  (0.71) -0.5  (2.70)
FDR: Physical component score Number Analyzed 8 participants 74 participants
8.280  (12.985) 1.986  (7.059)
PK-t R: Physical component score Number Analyzed 2 participants 11 participants
2.647  (7.990) 3.414  (9.405)
FDR: Mental component score Number Analyzed 8 participants 74 participants
-4.780  (6.680) -1.501  (9.059)
PK-t R: Mental component score Number Analyzed 2 participants 11 participants
3.931  (7.769) -4.185  (8.634)
21.Secondary Outcome
Title Change From Baseline in Patient Reported Outcomes: Health-related Quality of Life (HRQoL): Pediatrics Quality of Life (PedsQL) Questionnaire
Hide Description 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.
Time Frame Baseline, end of study (53 months)
Hide Outcome Measure Data
Hide Analysis Population Description
FAS with population of age less than 14 years were analyzed.
Arm/Group Title BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years
Hide Arm/Group Description:
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.
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.
Overall Number of Participants Analyzed 62 26
Mean (Standard Deviation)
Unit of Measure: Score on a scale
FDR: Physical functioning Number Analyzed 41 participants 3 participants
0.534  (17.896) 3.125  (15.625)
PK-t R: Physical functioning Number Analyzed 7 participants 2 participants
-0.446  (12.425) 7.813  (6.629)
FDR: Emotional functioning Number Analyzed 41 participants 3 participants
-1.0  (16.93) 8.3  (20.21)
PK-t R: Emotional functioning Number Analyzed 7 participants 2 participants
-2.1  (11.85) 10.0  (14.14)
FDR: Social functioning Number Analyzed 41 participants 3 participants
-0.4  (16.22) 8.3  (15.28)
PK-t R: Social functioning Number Analyzed 7 participants 2 participants
-6.4  (19.73) 5.0  (7.07)
FDR: School functioning Number Analyzed 36 participants 3 participants
3.472  (24.107) 15.000  (13.229)
PK-t R: School functioning Number Analyzed 6 participants 2 participants
-9.167  (16.591) -12.500  (17.678)
FDR: Psychosocial functioning Number Analyzed 41 participants 3 participants
0.331  (15.283) 10.556  (15.486)
PK-t R: Psychosocial functioning Number Analyzed 7 participants 2 participants
-6.099  (13.025) 0.833  (3.536)
FDR: Total score Number Analyzed 41 participants 3 participants
0.366  (14.811) 7.971  (15.230)
PK-t R: Total score Number Analyzed 7 participants 2 participants
-3.915  (12.201) 3.261  (0.000)
Time Frame From start of study drug administration up to 53 months
Adverse Event Reporting Description Safety analysis was analyzed for the safety population (216 participants) and not for the enrolled population as in participant flow (218 participants).
 
Arm/Group Title BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Hide Arm/Group Description 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. 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. 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. 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.
All-Cause Mortality
BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)      0/62 (0.00%)      1/26 (3.85%)      0/125 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/3 (0.00%)      7/62 (11.29%)      4/26 (15.38%)      22/125 (17.60%)    
Blood and lymphatic system disorders         
Anaemia * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Splenic haematoma * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 1/26 (3.85%)  1 0/125 (0.00%)  0
Gastrointestinal disorders         
Ileus * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Pancreatitis * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 2/125 (1.60%)  2
General disorders         
Pyrexia * 1  0/3 (0.00%)  0 2/62 (3.23%)  2 0/26 (0.00%)  0 0/125 (0.00%)  0
Hepatobiliary disorders         
Cholecystitis * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Infections and infestations         
Abscess oral * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Appendicitis * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Bacteraemia * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Cystitis * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Device related infection * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  2
Device related sepsis * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Incision site abscess * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Laryngitis * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Plasmodium falciparum infection * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Pneumonia * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 2/125 (1.60%)  2
Skin infection * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Streptococcal bacteraemia * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Tonsillitis * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Upper respiratory tract infection * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Urinary tract infection * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 2/125 (1.60%)  2
Injury, poisoning and procedural complications         
Facial bones fracture * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Fall * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Femoral neck fracture * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Head injury * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 1/125 (0.80%)  1
Nasal injury * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Scapula fracture * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Splenic rupture * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 1/26 (3.85%)  1 0/125 (0.00%)  0
Traumatic fracture * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Wound haemorrhage * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Wrist fracture * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Investigations         
Transaminases increased * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Musculoskeletal and connective tissue disorders         
Haemarthrosis * 1  0/3 (0.00%)  0 2/62 (3.23%)  2 0/26 (0.00%)  0 0/125 (0.00%)  0
Muscle haemorrhage * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 1/26 (3.85%)  1 0/125 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Metastases to lung * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Renal cancer metastatic * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Nervous system disorders         
Cerebral haemorrhage * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 1/26 (3.85%)  1 0/125 (0.00%)  0
Renal and urinary disorders         
Ureterolithiasis * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Respiratory, thoracic and mediastinal disorders         
Dyspnoea * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Epistaxis * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Pleural effusion * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 0/26 (0.00%)  0 1/125 (0.80%)  1
Skin and subcutaneous tissue disorders         
Rash * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Vascular disorders         
Haematoma * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 1/26 (3.85%)  1 2/125 (1.60%)  2
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BAX 855: Age < 2 Years BAX 855: Age >= 2 to <12 Years BAX 855: Age >= 12 to <17 Years BAX 855: Age >= 17 Years
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      40/62 (64.52%)      12/26 (46.15%)      62/125 (49.60%)    
Blood and lymphatic system disorders         
Iron deficiency anaemia * 1  1/3 (33.33%)  1 1/62 (1.61%)  1 0/26 (0.00%)  0 0/125 (0.00%)  0
Gastrointestinal disorders         
Diarrhoea * 1  0/3 (0.00%)  0 6/62 (9.68%)  8 1/26 (3.85%)  1 7/125 (5.60%)  8
Vomiting * 1  0/3 (0.00%)  0 6/62 (9.68%)  8 1/26 (3.85%)  2 1/125 (0.80%)  2
General disorders         
Pyrexia * 1  0/3 (0.00%)  0 11/62 (17.74%)  15 0/26 (0.00%)  0 5/125 (4.00%)  5
Infections and infestations         
Ear infection * 1  1/3 (33.33%)  4 4/62 (6.45%)  6 1/26 (3.85%)  2 0/125 (0.00%)  0
Enterovirus infection * 1  1/3 (33.33%)  1 0/62 (0.00%)  0 0/26 (0.00%)  0 0/125 (0.00%)  0
Gastroenteritis * 1  1/3 (33.33%)  1 1/62 (1.61%)  1 0/26 (0.00%)  0 2/125 (1.60%)  2
Influenza * 1  0/3 (0.00%)  0 5/62 (8.06%)  5 0/26 (0.00%)  0 3/125 (2.40%)  3
Nasopharyngitis * 1  0/3 (0.00%)  0 13/62 (20.97%)  24 2/26 (7.69%)  5 25/125 (20.00%)  39
Otitis media acute * 1  1/3 (33.33%)  1 0/62 (0.00%)  0 0/26 (0.00%)  0 0/125 (0.00%)  0
Pharyngitis * 1  1/3 (33.33%)  1 3/62 (4.84%)  3 0/26 (0.00%)  0 5/125 (4.00%)  9
Pharyngitis streptococcal * 1  0/3 (0.00%)  0 4/62 (6.45%)  5 1/26 (3.85%)  1 1/125 (0.80%)  1
Rhinitis * 1  0/3 (0.00%)  0 4/62 (6.45%)  4 3/26 (11.54%)  5 1/125 (0.80%)  2
Upper respiratory tract infection * 1  0/3 (0.00%)  0 11/62 (17.74%)  22 1/26 (3.85%)  1 12/125 (9.60%)  13
Injury, poisoning and procedural complications         
Ligament sprain * 1  0/3 (0.00%)  0 1/62 (1.61%)  1 2/26 (7.69%)  2 4/125 (3.20%)  6
Limb injury * 1  0/3 (0.00%)  0 2/62 (3.23%)  2 2/26 (7.69%)  2 2/125 (1.60%)  2
Skull fracture * 1  1/3 (33.33%)  1 0/62 (0.00%)  0 0/26 (0.00%)  0 0/125 (0.00%)  0
Musculoskeletal and connective tissue disorders         
Arthralgia * 1  0/3 (0.00%)  0 3/62 (4.84%)  4 2/26 (7.69%)  2 14/125 (11.20%)  20
Back pain * 1  0/3 (0.00%)  0 0/62 (0.00%)  0 1/26 (3.85%)  1 7/125 (5.60%)  7
Nervous system disorders         
Febrile convulsion * 1  1/3 (33.33%)  1 0/62 (0.00%)  0 0/26 (0.00%)  0 0/125 (0.00%)  0
Headache * 1  0/3 (0.00%)  0 6/62 (9.68%)  6 1/26 (3.85%)  1 12/125 (9.60%)  23
Respiratory, thoracic and mediastinal disorders         
Cough * 1  0/3 (0.00%)  0 13/62 (20.97%)  17 0/26 (0.00%)  0 7/125 (5.60%)  8
Pharyngeal ulceration * 1  1/3 (33.33%)  1 0/62 (0.00%)  0 0/26 (0.00%)  0 0/125 (0.00%)  0
Rhinorrhoea * 1  0/3 (0.00%)  0 6/62 (9.68%)  7 0/26 (0.00%)  0 1/125 (0.80%)  1
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: Study Director
Organization: Baxalta, now part of Shire
Phone: +1 866 842 5335
Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT01945593     History of Changes
Other Study ID Numbers: 261302
2013-002236-24 ( EudraCT Number )
First Submitted: September 16, 2013
First Posted: September 18, 2013
Results First Submitted: February 27, 2019
Results First Posted: May 1, 2019
Last Update Posted: May 1, 2019