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Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor

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: NCT00851721
Recruitment Status : Completed
First Posted : February 26, 2009
Results First Posted : March 14, 2014
Last Update Posted : November 22, 2017
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Hemophilia A or B With Inhibitors
Hemophilia A
Hemophilia B
Intervention Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
Enrollment 52
Recruitment Details Enrollment was conducted in Europe, North America, Asia-Pacific, and South America at 17 clinical sites beginning in March 2009.
Pre-assignment Details 52 participants were enrolled. Sixteen participants discontinued, (ten were screen failures and six were withdrawn before randomization (2 sponsor's decision- inhibitors, 3 withdrew consent, and 1 due to investigator decision to have participant on prophylaxis). Therefore 36 participants were randomized.
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Period Title: Overall Study
Started 19 17
Completed 17 16
Not Completed 2 1
Reason Not Completed
planned surgery             1             0
Death             1             0
Adverse Event             0             1
Arm/Group Title On-demand Arm Prophylaxis Arm Total
Hide Arm/Group Description Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period Total of all reporting groups
Overall Number of Baseline Participants 19 17 36
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 19 participants 17 participants 36 participants
29.1  (15.2) 25.6  (15.4) 27.4  (15.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 17 participants 36 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
19
 100.0%
17
 100.0%
36
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants 17 participants 36 participants
United States 1 1 2
Poland 1 1 2
Brazil 3 2 5
Ukraine 4 4 8
Romania 0 1 1
Croatia 0 1 1
Russian Federation 8 3 11
Bulgaria 0 2 2
Japan 1 1 2
New Zealand 1 1 2
1.Primary Outcome
Title Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand
Hide Description

Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens:

  1. On-Demand: FEIBA NF dose & dosing interval as prescribed by treating physician
  2. Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period

Annualized rate of bleeding episodes was calculated as:

(Number of bleeding episodes/observed treatment period in days) * 365.25

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Efficacy Intent to Treat Analysis Dataset:

Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Median (Inter-Quartile Range)
Unit of Measure: bleeds/year
28.7
(17.7 to 50.0)
7.9
(2.9 to 11.0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-demand Arm, Prophylaxis Arm
Comments H0: μ(on-demand) = μ(prophylaxis) Versus H1: μ(on-demand) ≠ μ(prophylaxis) (Where H0 implies no difference in mean bleeding episode rate between prophylaxis and on-demand treatment arms and H1 implies otherwise. This test was performed at a significance level of 5%, two-sided, two sample)
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0003
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
2.Secondary Outcome
Title Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Hide Description Spontaneous includes unknown/undermined etiology
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Efficacy Intent to Treat Analysis Dataset:

Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Median (Inter-Quartile Range)
Unit of Measure: Bleeds per year
Spontaneous
18.9
(10.9 to 43.5)
5.6
(2.9 to 8.0)
Traumatic
4.7
(1.9 to 10.7)
2.5
(0.0 to 3.1)
Joint
22.9
(14.1 to 46.9)
6.0
(2.9 to 10.0)
Non-Joint
2.9
(1.0 to 4.9)
0.5
(0.0 to 2.0)
Spontaneous Joint
16.6
(9.9 to 40.8)
4.5
(2.9 to 8.0)
Spontaneous Non-Joint
1.0
(1.0 to 2.9)
0.0
(0.0 to 1.0)
Traumatic Joint
4.0
(1.0 to 7.1)
1.0
(0.0 to 3.1)
Traumatic Non-Joint
0.0
(0.0 to 1.9)
0.0
(0.0 to 1.0)
All Bleeding Etiologies, and Bleed Types
28.7
(17.7 to 50.0)
7.9
(2.9 to 11.0)
3.Secondary Outcome
Title Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Intent to Treat Analysis Dataset
Arm/Group Title On-demand Arm Versus Prophylaxis Arm
Hide Arm/Group Description:

On-demand arm: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician

Prophylaxis arm: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period

Overall Number of Participants Analyzed 36
Mean (Standard Deviation)
Unit of Measure: (bleeds/year)^(1/2)
Spontaneous Bleeds 2.2  (1.8)
Traumatic Bleeds 1.0  (1.2)
Joint Bleeds 2.4  (1.9)
Non-Joint Bleeds 0.8  (0.9)
Spontaneous Joint Bleeds 2.1  (1.8)
Spontaneous Non-Joint Bleeds 0.8  (0.8)
Traumatic Joint Bleeds 0.9  (1.2)
Traumatic Non-Joint Bleeds -0.0  (0.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Spontaneous Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0008
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Traumatic Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0199
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Joint Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0006
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Non-Joint Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0227
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Spontaneous Joint Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0013
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 6 Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Spontaneous Non-Joint Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0030
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 7 Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Traumatic Joint Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0254
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
Show Statistical Analysis 8 Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments Traumatic Non-Joint Bleeds
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9322
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
4.Secondary Outcome
Title Annualized Bleeding Rate for New Target Joints
Hide Description Target joints are ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows, and hips; a target joint bleeding episode refers to an individual anatomical location.
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Efficacy Intent to Treat Analysis Dataset:

Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Median (Inter-Quartile Range)
Unit of Measure: Bleeds per year
5.9
(0.0 to 12.9)
0.0
(0.0 to 4.1)
5.Secondary Outcome
Title Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens: New Target Joints
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Efficacy Intent to Treat Analysis Dataset:

Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.

Arm/Group Title On-demand Arm Versus Prophylaxis Arm
Hide Arm/Group Description:

On-demand arm: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician

Prophylaxis arm: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period

Overall Number of Participants Analyzed 36
Mean (Standard Deviation)
Unit of Measure: (bleeds/year)^(1/2)
1.6  (2.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-demand Arm Versus Prophylaxis Arm
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0271
Comments [Not Specified]
Method Two-sample, two-sided t-test
Comments [Not Specified]
6.Secondary Outcome
Title Number of New Target Joints
Hide Description Target Joints are defined as ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows and hips
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Efficacy Intent to Treat Analysis Dataset:

Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Measure Type: Number
Unit of Measure: new target joints
23 7
7.Secondary Outcome
Title Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
Hide Description

Pain caused by a bleeding episode in adolescents and adults (≥12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 hours (h) and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) on the VAS pain scale in millimeters from 0 (no pain) to 100 (worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (100). Pain assessment occurred after each infusion related to single bleeding episodes. In case participants required an additional infusion within 24h, pain was assessed 6 ± 0.5 h and 24 ±1 h following the subsequent infusion.

Change in VAS scores at 6 ± 0.5 h and 24 ±1 h post-infusion were also compared relative to pre-infusion VAS scores (ie, (pre-infusion VAS score) - (post-infusion VAS score)).

Time Frame Throughout the study period, 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Additional evaluations for bleeding episodes in the intent-to-treat analysis dataset: consists of all participants with at least 1 bleeding episode treated with investigational product.
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 17 12
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes
522 158
Median (Inter-Quartile Range)
Unit of Measure: Scores on a scale
Pre-Infusion (N= 513, 130)
29.0
(12.0 to 55.0)
49.5
(34.7 to 81.6)
6 ± 0.5 hours post-infusion (N= 522, 158)
10.0
(4.0 to 37.0)
33.8
(8.0 to 52.6)
24 ± 1 hours post-infusion (N= 512,131)
3.0
(1.0 to 14.0)
6.2
(1.0 to 20.0)
Change (Pre-Inf to 6h post-inf) (N= 509, 129)
10.0
(4.0 to 21.8)
19.4
(5.0 to 41.6)
Change (Pre-Inf to 24h post-inf) (N= 489, 102)
18.0
(8.0 to 39.6)
37.8
(17.0 to 75.5)
8.Secondary Outcome
Title Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Hide Description

Pain caused by a bleeding episode (BE) in pediatric participants (<12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 h and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) using the children’s VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (worst possible pain).

Scores on the children's VAS scale are presented as:

  • No Pain
  • Mild Pain
  • Moderate pain
  • Severe pain
  • Very severe pain
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Additional evaluations for bleeding episodes in the intent-to-treat analysis dataset: consists of all participants with at least 1 bleeding episode treated with investigational product.
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 2 2
Overall Number of Units Analyzed
Type of Units Analyzed: Bleeding episodes (BEs)
77 9
Measure Type: Number
Unit of Measure: Bleeding episodes
Pre-Infusion (N= 54, 8) - No Pain 27 3
Pre-Infusion (N= 54, 8) - Mild Pain 3 1
Pre-Infusion (N= 54, 8) - Moderate Pain 12 3
Pre-Infusion (N= 54, 8) - Severe Pain 10 1
Pre-Infusion (N= 54, 8) - Very Severe Pain 2 0
6 ± 0.5 h post-infusion (N= 74, 8) - No Pain 47 3
± 0.5 h post-infusion (N= 74, 8) - Mild Pain 9 3
± 0.5 h post-infusion (N= 74, 8) - Moderate Pain 15 2
± 0.5 h post-infusion (N= 74, 8) - Severe Pain 3 0
± 0.5 h post-infusion (N= 74, 8)- Very Severe Pain 0 0
24 ± 1 h post-infusion (N= 77, 9) - No Pain 60 7
24 ± 1 h post-infusion (N= 77, 9) - Mild Pain 11 1
24 ± 1 h post-infusion (N= 77, 9) - Moderate Pain 6 1
24 ± 1 h post-infusion (N= 77, 9) - Severe Pain 0 0
24± 1h post-infusion (N = 77, 9) -Very Severe Pain 0 0
9.Secondary Outcome
Title Assessment of Clinical Symptoms - Range of Motion (ROM)
Hide Description ROM was measured using a goniometer for 3 key joints (ie, ankles, knees, and elbows) at screening, month 6, and termination (end of study visit)
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Median (Inter-Quartile Range)
Unit of Measure: degrees
Left Elbow - Extension: Screening
15.0
(0.0 to 45.0)
10.0
(0.0 to 25.0)
Left Elbow - Extension: Month 6 (n = 18,16)
10.0
(0.0 to 41.0)
8.0
(0.0 to 42.5)
Left Elbow - Extension: Termination (n = 17,16)
20.0
(0.0 to 41.0)
15.5
(0.0 to 40.0)
Left Elbow - Flexion: Screening (n = 19,17)
125.0
(90.0 to 140.0)
115.0
(80.0 to 140.0)
Left Elbow - Flexion: Month 6 (n = 18,16)
130.0
(90.0 to 140.0)
125.0
(72.5 to 143.0)
Left Elbow - Flexion: Termination (n = 17,16)
110.0
(90.0 to 140.0)
125.0
(75.0 to 145.0)
Left Elbow - Pronation: Screening (n = 18,16)
64.0
(55.0 to 90.0)
55.0
(22.5 to 77.5)
Left Elbow - Pronation: Month 6 (n = 17,14)
80.0
(60.0 to 90.0)
67.5
(35.0 to 80.0)
Left Elbow - Pronation: Termination (n = 17,15)
68.0
(60.0 to 90.0)
65.0
(30.0 to 80.0)
Left Elbow - Supination: Screening (n = 18,16)
75.0
(50.0 to 90.0)
42.5
(17.5 to 77.5)
Left Elbow - Supination: Month 6 (n = 17,14)
80.0
(50.0 to 90.0)
60.0
(35.0 to 80.0)
Left Elbow - Supination: Termination (n = 17,15)
70.0
(50.0 to 90.0)
70.0
(35.0 to 80.0)
Right Elbow - Extension: Screening (n = 19,17)
20.0
(0.0 to 50.0)
10.0
(0.0 to 50.0)
Right Elbow - Extension: Month 6 (n = 18,16)
17.5
(0.0 to 50.0)
10.0
(0.0 to 42.5)
Right Elbow - Extension: Termination (n = 18,16)
22.5
(0.0 to 45.0)
20.0
(0.0 to 62.5)
Right Elbow - Flexion: Screening (n = 19,17)
120.0
(105.0 to 140.0)
100.0
(80.0 to 130.0)
Right Elbow - Flexion: Month 6 (n = 18,16)
122.5
(110.0 to 140.0)
100.0
(85.0 to 136.0)
Right Elbow - Flexion: Termination (n = 18,16)
121.0
(110.0 to 140.0)
99.0
(70.0 to 127.5)
Right Elbow - Pronation: Screening (n = 18,16)
75.0
(55.0 to 90.0)
57.5
(27.5 to 80.0)
Right Elbow - Pronation: Month 6 (n = 17,14)
80.0
(55.0 to 90.0)
72.5
(40.0 to 80.0)
Right Elbow - Pronation: Termination (n = 18,15)
67.5
(55.0 to 90.0)
70.0
(40.0 to 80.0)
Right Elbow - Supination: Screening (n = 18,16)
75.0
(45.0 to 90.0)
52.5
(20.0 to 80.0)
Right Elbow - Supination: Month 6 (n = 17,14)
70.0
(45.0 to 90.0)
72.5
(30.0 to 80.0)
Right Elbow - Supination: Termination (n = 18,15)
75.0
(45.0 to 90.0)
70.0
(30.0 to 80.0)
Left Knee - Extension: Screening (n = 18,16)
15.0
(5.0 to 90.0)
1.5
(0.0 to 55.5)
Left Knee - Extension: Month 6 (n = 17,14)
15.0
(5.0 to 70.0)
0.0
(0.0 to 20.0)
Left Knee - Extension: Termination (n = 18,15)
15.0
(5.0 to 90.0)
0.0
(0.0 to 70.0)
Left Knee - Flexion: Screening (n = 19,17)
100.0
(80.0 to 130.0)
90.0
(65.0 to 115.0)
Left Knee - Flexion: Month 6 (n = 18,16)
112.5
(80.0 to 130.0)
97.5
(65.0 to 125.0)
Left Knee - Flexion: Termination (n = 18,16)
105.0
(60.0 to 130.0)
92.5
(70.0 to 125.0)
Right Knee - Extension: Screening (n = 18,16)
35.0
(10.0 to 90.0)
5.0
(0.0 to 30.0)
Right Knee - Extension: Month 6 (n = 17,14)
20.0
(10.0 to 90.0)
10.0
(2.0 to 30.0)
Right Knee - Extension: Termination (n = 18,15)
17.5
(10.0 to 90.0)
5.0
(0.0 to 30.0)
Right Knee - Flexion: Screening (n = 19,17)
90.0
(20.0 to 122.0)
80.0
(60.0 to 125.0)
Right Knee - Flexion: Month 6 (n = 18,16)
90.0
(20.0 to 130.0)
82.5
(56.0 to 127.5)
Right Knee - Flexion: Termination (n = 18,16)
85.0
(20.0 to 120.0)
87.5
(56.0 to 127.5)
Left Ankle - Dorsiflexion: Screening (n = 18,16)
20.0
(15.0 to 30.0)
17.5
(9.0 to 37.5)
Left Ankle - Dorsiflexion: Month 6 (n = 17,14)
20.0
(11.0 to 35.0)
17.5
(8.0 to 50.0)
Left Ankle - Dorsiflexion: Termination (n = 17,15)
15.0
(11.0 to 25.0)
20.0
(5.0 to 50.0)
Left Ankle - Plantarflexion: Screening (n = 18,16)
34.5
(30.0 to 40.0)
30.0
(10.0 to 35.0)
Left Ankle - Plantarflexion: Month 6 (n = 17,14)
34.0
(25.0 to 35.0)
30.0
(15.0 to 40.0)
Left Ankle - Plantarflexion: Termination (n=17,15)
35.0
(30.0 to 40.0)
30.0
(15.0 to 35.0)
Left Ankle - Pronation: Screening (n = 19,17)
14.0
(10.0 to 20.0)
15.0
(10.0 to 15.0)
Left Ankle - Pronation: Month 6 (n = 18,15)
15.0
(10.0 to 20.0)
15.0
(10.0 to 16.0)
Left Ankle - Pronation: Termination (n = 18,16)
15.0
(10.0 to 20.0)
12.5
(10.0 to 15.5)
Left Ankle - Supination: Screening (n = 19,17)
25.0
(10.0 to 30.0)
15.0
(10.0 to 27.0)
Left Ankle - Supination: Month 6 (n = 18,15)
25.0
(20.0 to 30.0)
25.0
(10.0 to 30.0)
Left Ankle - Supination: Termination (n = 18,16)
25.0
(16.0 to 30.0)
20.0
(10.0 to 27.5)
Right Ankle - Dorsiflexion: Screening (n = 18,16)
15.0
(10.0 to 20.0)
20.0
(7.5 to 35.0)
Right Ankle - Dorsiflexion: Month 6 (n = 17,14)
15.0
(15.0 to 20.0)
25.0
(6.0 to 40.0)
Right Ankle - Dorsiflexion: Termination (n= 17,15)
15.0
(14.0 to 20.0)
20.0
(10.0 to 40.0)
Right Ankle - Plantarflexion: Screening (n= 18,16)
35.0
(30.0 to 40.0)
20.0
(9.0 to 39.0)
Right Ankle - Plantarflexion: Month 6 (n= 17,14)
30.0
(25.0 to 40.0)
23.0
(10.0 to 40.0)
Right Ankle - Plantarflexion: Termination(n=17,15)
30.0
(25.0 to 40.0)
21.0
(10.0 to 40.0)
Right Ankle - Pronation: Screening (n = 19,17)
10.0
(10.0 to 15.0)
10.0
(10.0 to 20.0)
Right Ankle - Pronation: Month 6 (n= 18,15)
10.0
(10.0 to 20.0)
10.0
(10.0 to 20.0)
Right Ankle - Pronation: Termination (n= 18,16)
10.0
(10.0 to 17.0)
11.0
(10.0 to 17.5)
Right Ankle - Supination: Screening (n = 19,17)
17.0
(10.0 to 30.0)
20.0
(10.0 to 30.0)
Right Ankle - Supination: Month 6 (n= 18,15)
20.0
(10.0 to 30.0)
20.0
(10.0 to 30.0)
Right Ankle - Supination: Termination (n= 18,16)
20.0
(10.0 to 30.0)
20.0
(10.0 to 30.0)
10.Secondary Outcome
Title Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Hide Description

Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale):

Excellent: Full pain relief & bleeding cessation within ~6 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis;

Good: Definite pain relief and/or improvement in bleeding within ~6 hours after infusion. Possibly requires >1 infusion for complete resolution;

Fair: Probable or slight relief of pain & slight improvement in bleeding within

~6 hours after infusion. Requires >1 infusion for complete resolution;

None: No improvement or condition worsens

Time Frame 6 h ± 30 min post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding Episodes Analysis Set Consists of all participants who experienced a bleeding episode (BE)
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 14
Measure Type: Number
Unit of Measure: bleeding episodes
Excellent (n = 16, 5) 109 16
Good (n = 19, 14) 355 106
Fair (n = 15, 6) 142 42
None (n = 6, 2) 14 5
Rating Not Done (n = 3, 2) 3 2
Not Available (n = 0, 1) 0 2
11.Secondary Outcome
Title Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Hide Description

Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale):

Excellent: Full pain relief & bleeding cessation within ~24 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis;

Good: Definite pain relief and/or improvement in bleeding within ~24 hours after infusion. Possibly requires >1 infusion for complete resolution;

Fair: Probable or slight relief of pain & slight improvement in bleeding within

~24 hours after infusion. Requires >1 infusion for complete resolution;

None: No improvement or condition worsens

Time Frame 24 ± 1 h post-infusion
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding Episodes Analysis Set Consists of all participants who experienced a bleeding episode (BE)
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 14
Measure Type: Number
Unit of Measure: bleeding episodes
Excellent (n = 16, 6) 282 42
Good (n = 19, 14) 280 89
Fair (n = 9, 3) 36 12
None (n = 1,0) 1 0
Rating Not Done (n = 8, 3) 24 28
Not Available (n = 0, 1) 0 2
12.Secondary Outcome
Title Total Weight Adjusted Dose to Control a Bleeding Episode
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Additional Evaluations for Bleeding Episodes Analysis Set

- Consists of all participants with at least 1 bleeding episode treated with investigational product, FEIBA NF.

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 14
Median (Inter-Quartile Range)
Unit of Measure: Units/kg
4049.7
(2324.5 to 7408.4)
1524.9
(293.2 to 2883.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection On-demand Arm, Prophylaxis Arm
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0067
Comments [Not Specified]
Method Mann-Whitney tests (Wilcoxon-Rank Sum)
Comments [Not Specified]
13.Secondary Outcome
Title The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Additional Evaluations for Bleeding Episodes Analysis Set

- Consists of all participants with at least 1 bleeding episode treated with investigational product, FEIBA NF.

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 14
Measure Type: Number
Unit of Measure: Bleeding Episodes (BEs)
1 infusion 352 98
2 infusions 134 41
3 infusions 62 13
≥4 infusions 75 21
14.Secondary Outcome
Title Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
Hide Description The normal reference range of values for aPTT is 22.8 – 31 seconds.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 16
Median (Inter-Quartile Range)
Unit of Measure: seconds
Screening visit (n= 19, 16)
67.3
(63.1 to 75.1)
68.5
(61.0 to 73.1)
Month 3 (n= 14, 15)
68.0
(62.7 to 76.2)
62.7
(57.5 to 67.2)
Month 6 (n= 17, 15)
68.2
(64.6 to 72.1)
65.6
(62.6 to 69.5)
Month 9 (n= 17, 15)
70.0
(64.6 to 77.9)
68.1
(58.1 to 73.9)
Termination visit (n= 18, 16)
69.8
(63.4 to 75.6)
69.4
(60.5 to 77.1)
15.Secondary Outcome
Title Abnormal D-Dimer Assay Results
Hide Description The normal reference range of values for D-dimers is <500 ng/mL.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 9 9
Median (Inter-Quartile Range)
Unit of Measure: ng/mL
Screening visit (n= 7, 6)
862.0
(647.0 to 1035.0)
833.5
(644.0 to 1164.0)
Month 3 (n= 7, 8)
690.0
(561.0 to 830.0)
1021.5
(730.0 to 1633.5)
Month 6 (n= 9, 8)
969.0
(753.0 to 1103.0)
1032.0
(652.0 to 1442.5)
Month 9 (n= 6, 8)
731.5
(592.0 to 1398.0)
1121.0
(942.5 to 1448.5)
Termination visit (n= 5, 9)
813.0
(695.0 to 1386.0)
1031.0
(872.0 to 1258.0)
16.Secondary Outcome
Title Abnormal Fibrinogen Assay Results
Hide Description The normal reference range of values for fibrinogen is 200-400 mg/dL.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 4 6
Median (Inter-Quartile Range)
Unit of Measure: mg/dL
Screening visit (n= 4, 6)
428.0
(414.0 to 478.5)
422.0
(411.0 to 428.0)
Month 3 (n= 0, 2)
NA [1] 
(NA to NA)
402.0
(199.0 to 605.0)
Month 6 (n= 3, 1)
415.0
(412.0 to 418.0)
759.0
(759.0 to 759.0)
Month 9 (n= 1, 4)
508.0
(508.0 to 508.0)
326.5
(183.5 to 635.5)
Termination visit (n= 2, 2)
456.5
(425.0 to 488.0)
522.5
(414.0 to 631.0)
[1]
There were no abnormal fibrinogen assay results for this arm/group at this time point
17.Secondary Outcome
Title Abnormal Fibrin Degradation Products (FDP) Assay Results
Hide Description The normal reference range of values for FDP is 0-5 ug/mL.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 3 2
Median (Inter-Quartile Range)
Unit of Measure: ug/mL
Screening visit (n= 1, 2)
16.0
(16.0 to 16.0)
12.0
(8.0 to 16.0)
Month 3 (n= 0, 2)
NA [1] 
(NA to NA)
8.0
(8.0 to 8.0)
Month 6 (n= 1, 1)
8.0
(8.0 to 8.0)
8.0
(8.0 to 8.0)
Month 9 (n= 1, 2)
8.0
(8.0 to 8.0)
8.0
(8.0 to 8.0)
Termination visit (n= 3, 1)
8.0
(8.0 to 8.0)
8.0
(8.0 to 8.0)
[1]
There were no abnormal fibrin degradation products assay results for this arm/group at this time point
18.Secondary Outcome
Title Abnormal Prothrombin Fragment F 1.2 Assay Results
Hide Description The normal reference range of values for prothrombin fragment F 1.2 is 69-229 pmol/L.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 6 9
Median (Inter-Quartile Range)
Unit of Measure: pmol/L
Screening visit (n= 2, 1)
284.0
(278.0 to 290.0)
308.0
(308.0 to 308.0)
Month 3 (n= 6, 7)
336.5
(267.0 to 414.0)
579.0
(354.0 to 972.0)
Month 6 (n= 3, 9)
448.0
(272.0 to 714.0)
430.0
(366.0 to 504.0)
Month 9 (n= 4, 9)
382.0
(313.0 to 405.5)
415.0
(272.0 to 472.0)
Termination visit (n= 4, 7)
252.5
(250.0 to 265.0)
643.0
(376.0 to 1679.0)
19.Secondary Outcome
Title Abnormal Prothrombin Time Assay Results
Hide Description The normal reference range of values for PT is 9.7-12.3 sec.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 1 1
Median (Inter-Quartile Range)
Unit of Measure: seconds
Screening visit (n= 1, 1)
9.4
(9.4 to 9.4)
9.6
(9.6 to 9.6)
Month 3 (n= 1, 0)
9.5
(9.5 to 9.5)
NA [1] 
(NA to NA)
Month 6 (n= 0, 1)
NA [1] 
(NA to NA)
12.5
(12.5 to 12.5)
Month 9 (n= 1, 1)
12.7
(12.7 to 12.7)
13.6
(13.6 to 13.6)
Termination visit (n= 0, 1)
NA [1] 
(NA to NA)
13.4
(13.4 to 13.4)
[1]
There were no abnormal prothrombin time assay results for this arm/group at this time point
20.Secondary Outcome
Title Abnormal Thrombin-Antithrombin III (TAT) Assay Results
Hide Description The normal reference range of values for TAT is 1-4.1 ug/L.
Time Frame Screening visit, Month 3, Month 6, Month 9, and Termination visit
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set Participants with Clinically Significant Laboratory Results
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 4 7
Median (Inter-Quartile Range)
Unit of Measure: ug/L
Screening visit (n= 4, 1)
5.1
(4.4 to 5.9)
5.6
(5.6 to 5.6)
Month 3 (n= 3, 7)
4.7
(4.5 to 5.9)
12.3
(4.4 to 40.8)
Month 6 (n= 3, 6)
7.9
(6.3 to 10.5)
5.6
(4.4 to 6.1)
Month 9 (n= 0, 3)
NA [1] 
(NA to NA)
6.0
(5.6 to 52.9)
Termination visit (n= 2, 5)
10.3
(8.0 to 12.5)
5.6
(5.1 to 7.3)
[1]
There were no abnormal Thrombin-Antithrombin III assay results for this arm/group at this time point
21.Secondary Outcome
Title Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
Hide Description
  • Hepatitis A Virus Antibody (HAV Ab)
  • Hepatitis B Virus Core Antibody (HBcAb)
  • Hepatitis B Virus Surface Antibody (HBsAb)
  • Hepatitis B Virus Surface Antigen (HBsAg)
  • Hepatitis C Virus (HCV)
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Measure Type: Number
Unit of Measure: participants
HAV Ab: Screening Negative; Termination Positive 1 0
HAV Ab: Screening Negative; Termination Negative 8 11
HAV Ab: Screening Positive; Termination Positive 8 4
HAV Ab: Screening Positive; Termination Negative 1 1
HAV Ab: Serology not available 1 1
HBcAb: Screening Negative; Termination Positive 0 1
HBcAb: Screening Negative; Termination Negative 8 11
HBcAb: Screening Positive; Termination Positive 9 4
HBcAb: Screening Positive; Termination Negative 1 0
HBcAb: Serology not available 1 1
HBsAb: Screening Negative; Termination Positive 2 5
HBsAb: Screening Negative; Termination Negative 2 2
HBsAb: Screening Positive; Termination Positive 13 9
HBsAb: Screening Positive; Termination Negative 1 0
HBsAb: Serology not available 1 1
HBsAg: Screening Negative; Termination Positive 0 0
HBsAg: Screening Negative; Termination Negative 18 16
HBsAg: Serology not available 1 1
HCV: Screening Negative; Termination Positive 0 0
HCV: Screening Negative; Termination Negative 6 10
HCV: Screening Positive; Termination Positive 12 6
HCV: Screening Positive; Termination Negative 0 0
HCV: Serology not available 1 1
22.Secondary Outcome
Title Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab)
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Prophylaxis
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Measure Type: Number
Unit of Measure: participants
HIV 1/2 Ab: Screen Negative; Termination Positive 0 0
HIV 1/2 Ab: Screen Negative; Termination Negative 18 16
HIV 1/2 Ab: : Serology not available 1 1
23.Secondary Outcome
Title Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Hide Description

Normal range (0 - 0.89 IV); High (> 0.89 IV)

- Parvovirus B19 IgG Antibody [IV] (Parvo IgG Ab)

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Prophylaxis
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Measure Type: Number
Unit of Measure: participants
Parvo IgG Ab: Screening High ; Termination Normal 3 1
Parvo IgG Ab: Screening High ; Termination High 14 13
Parvo IgG Ab: Screening Normal; Termination Normal 0 2
Parvo IgG Ab: Screening Normal ; Termination High 1 0
Parvo IgG Ab: Serology not available 1 1
24.Secondary Outcome
Title Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Hide Description

Normal range (0 - 0.89 IV); High (> 0.89 IV)

- Parvovirus B19 IgM Antibody [IV] (Parvo IgM Ab)

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Prophylaxis
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Measure Type: Number
Unit of Measure: participants
Parvo IgM Ab: Screening High ; Termination Normal 0 0
Parvo IgM Ab: Screening High ; Termination High 1 0
Parvo IgM Ab: Screening Normal; Termination Normal 17 16
Parvo IgM Ab: Screening Normal ; Termination High 0 0
Parvo IgM Ab: Serology not available 1 1
25.Secondary Outcome
Title Rate of Related Adverse Events (AEs) Per Year
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Median (Inter-Quartile Range)
Unit of Measure: Related AEs per year
0.000
(0.000 to 0.000)
0.000
(0.000 to 0.979)
26.Secondary Outcome
Title Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Median (Inter-Quartile Range)
Unit of Measure: Related AEs within/during 1hr per year
0.000
(0.000 to 0.000)
0.000
(0.000 to 0.000)
27.Secondary Outcome
Title Number of Related Thromboembolic Adverse Events (AEs)
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 19 17
Measure Type: Number
Unit of Measure: Related thromboembolic AEs
0 0
28.Secondary Outcome
Title Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Hide Description

Absolute Changes in Inhibitor Titer (or no change in low or high titer status):

  • Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU)
  • Inhibitor Titer went from Low (≤5 BU) to High (>5 BU)
  • Inhibitor Titer went from High (>5 BU) to Low (≤5 BU)
  • Inhibitor Titer went from High (>5 BU) to High (>5 BU)
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Safety Analysis Set

- Hemophilia A study participants

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 17 16
Median (Inter-Quartile Range)
Unit of Measure: Bethesda Units (BU)
Screening to 3 Month- Low to High (N=2, 3)
12.1
(12.1 to 12.1)
4.0
(0.7 to 16.8)
Screening to 3 Month- High to Low (N=1, 0)
2.0
(2 to 2)
NA [1] 
(NA to NA)
Screening to 6 Month- Low to High (N=2, 1)
12.9
(4.9 to 20.8)
5.3
(5.3 to 5.3)
Screening to 6 Month- High to Low (N=1, 0)
3.4
(3.4 to 3.4)
NA [1] 
(NA to NA)
Screening to 9 Month- Low to High (N=2, 1)
5.0
(4.3 to 5.6)
3.7
(3.7 to 3.7)
Screening to 9 Month- High to Low (N=0, 2)
NA [1] 
(NA to NA)
2.1
(0.9 to 3.2)
Screening to Termination- Low to High (N=2, 1)
39.2
(3.8 to 74.7)
5.8
(5.8 to 5.8)
Screening to Termination- High to Low (N=1, 2)
14.6
(14.6 to 14.6)
3.3
(3.3 to 3.3)
[1]
There were no inhibitor titer shifts for this arm/group at this time point
29.Secondary Outcome
Title Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Hide Description

Absolute Changes in Inhibitor Titer (or no change in low or high titer status):

  • Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU)
  • Inhibitor Titer went from Low (≤5 BU) to High (>5 BU)
  • Inhibitor Titer went from High (>5 BU) to Low (≤5 BU)
  • Inhibitor Titer went from High (>5 BU) to High (>5 BU)
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description

Safety Analysis Set

- Hemophilia B study participants

Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 1 1
Median (Inter-Quartile Range)
Unit of Measure: Bethesda Units (BU)
Screening to 3 Month- Low to Low (N=1, 0)
3.2
(3.2 to 3.2)
NA [1] 
(NA to NA)
Screening to 3 Month- Low to High (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to 3 Month- High to Low (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to 3 Month- High to High (N=1, 1)
0.3
(0.3 to 0.3)
10.7
(10.7 to 10.7)
Screening to 6 Month- Low to Low (N=1, 0)
1.5
(1.5 to 1.5)
NA [1] 
(NA to NA)
Screening to 6 Month- Low to High (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to 6 Month- High to Low (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to 6 Month- High to High (N=1, 1)
1.7
(1.7 to 1.7)
23.2
(23.2 to 23.2)
Screening to 9 Month- Low to Low (N=1, 0)
3.2
(3.2 to 3.2)
NA [1] 
(NA to NA)
Screening to 9 Month- Low to High (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to 9 Month- High to Low (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to 9 Month- High to High (N=0, 1)
NA [1] 
(NA to NA)
37.0
(37.0 to 37.0)
Screening to Termination - Low to Low (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to Termination- Low to High (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to Termination- High to Low (N=0, 0)
NA [1] 
(NA to NA)
NA [1] 
(NA to NA)
Screening to Termination - High to High (N=1, 1)
11.3
(11.3 to 11.3)
21.4
(21.4 to 21.4)
[1]
There were no inhibitor titer shifts for this arm/group at this time point
30.Secondary Outcome
Title Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School)
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 17 16
Mean (Standard Deviation)
Unit of Measure: days
16.4  (25.76) 8.8  (14.42)
31.Secondary Outcome
Title Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 18 16
Mean (Standard Deviation)
Unit of Measure: hospitalizations
0.6  (1.15) 0.6  (2.03)
32.Secondary Outcome
Title Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 18 16
Mean (Standard Deviation)
Unit of Measure: hospitalizations
0.1  (0.32) 0.1  (0.25)
33.Secondary Outcome
Title Pharmacoeconomics: Annual Number of Emergency Room Visits
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 18 16
Mean (Standard Deviation)
Unit of Measure: Emergency room visits
0.4  (0.98) 0.4  (1.09)
34.Secondary Outcome
Title Pharmacoeconomics: Annual Number of Physician’s Office Visits
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 18 16
Mean (Standard Deviation)
Unit of Measure: Physician's office visits
2.2  (4.15) 2.6  (4.60)
35.Secondary Outcome
Title Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 18 16
Mean (Standard Deviation)
Unit of Measure: Days
5.0  (9.48) 5.3  (17.00)
36.Secondary Outcome
Title Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 18 16
Mean (Standard Deviation)
Unit of Measure: Days
0.7  (2.61) 0.7  (2.75)
37.Secondary Outcome
Title Pharmacoeconomics: Annual Total Number of Days Lost (Work or School)
Hide Description [Not Specified]
Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacoeconomic Analysis Set
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 17 16
Mean (Standard Deviation)
Unit of Measure: Days
17.4  (25.42) 15.4  (24.28)
38.Secondary Outcome
Title Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
Hide Description

EQ-5D is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The EQ-5D total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome.

EQ-5D Index scores based on EQ-5D questionnaire were calculated for participants ≥14 years of age, at screening, 6 months, and at termination visit. Changes in scores at 6 months and termination were also calculated.

A relatively higher score represents better quality of life.

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
HRQoL Intent-to-Treat Analysis Dataset - comprised of all participants ≥14 years of age who were randomized, had any available assessments at any available study visits (baseline, 6- month, and 12-month) as defined in the protocol
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 16 12
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Screening (N= 16, 12) 0.627  (0.2067) 0.620  (0.1841)
6 Months (N= 15, 11) 0.621  (0.1880) 0.729  (0.1392)
Termination (N= 15, 10) 0.605  (0.2146) 0.700  (0.1233)
Change (Screening - Month 6) (N= 15, 11) -0.006  (0.2088) -0.096  (0.2403)
Change (Screening - Termination) (N= 15, 10) 0.010  (0.2470) -0.075  (0.2594)
Change (Month 6 - Termination) (N= 15, 10) 0.016  (0.1765) 0.001  (0.1493)
39.Secondary Outcome
Title Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Hide Description

The Haem-A-QoL instrument has been developed and used in Hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & Leisure (S&L), School & Work (W&S), Dealing with Hemophilia (Dealing), Family Planning (FP), Feeling, Relationships (R'ships), Treatment, View, and Outlook for the Future (Future). A Haem-A-QoL Total Score (Total) was also calculated. For the Haem-A-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.

Haem-A-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset for all participants ≥ 16 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 15 12
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Dealing- Screening (N=15, 12) 35.0  (19.72) 26.0  (15.84)
Dealing- Month 6 (N=14, 11) 25.0  (16.67) 18.9  (21.11)
Dealing- Termination (N=14, 11) 25.6  (25.21) 23.7  (22.15)
Dealing- Change (Screening - Month 6) (N=14, 11) 10.7  (18.61) 3.4  (22.62)
Dealing- Change(Screening - Termination)(N=14, 11) 10.1  (27.19) -1.4  (21.37)
Dealing- Change (Month 6 - Termination)(N=14, 11) -0.6  (16.81) -4.8  (21.26)
FP- Screening (N=9, 8) 16.7  (20.49) 34.9  (31.31)
FP- Month 6 (N=12, 7) 20.5  (26.71) 14.3  (11.25)
FP- Termination (N=13, 8) 19.1  (16.25) 21.1  (22.14)
FP- Change (Screening - Month 6) (N=9, 7) -3.7  (26.94) 14.9  (28.73)
FP- Change(Screening - Termination)(N=9, 7) -2.5  (25.57) 13.1  (20.26)
FP- Change (Month 6 - Termination)(N=11, 6) -0.8  (32.68) -8.3  (28.41)
Feeling- Screening (N=15, 12) 39.6  (28.22) 48.2  (29.02)
Feeling- Month 6 (N=14, 11) 33.9  (28.77) 29.0  (17.29)
Feeling- Termination (N=14, 11) 30.8  (28.53) 26.1  (20.69)
Feeling- Change (Screening - Month 6) (N=14, 11) 4.9  (27.75) 17.3  (34.72)
Feeling- Change(Screening - Termination)(N=14, 11) 8.0  (31.91) 20.2  (24.58)
Feeling- Change (Month 6 - Termination)(N=14, 11) 3.1  (16.40) 2.8  (23.94)
Future- Screening (N=14, 12) 46.8  (28.46) 48.6  (23.32)
Future- Month 6 (N=14, 11) 40.2  (26.14) 46.0  (18.03)
Future- Termination (N=14, 11) 47.9  (25.47) 46.8  (18.74)
Future- Change (Screening - Month 6) (N=13, 11) 4.0  (26.84) 1.1  (24.34)
Future- Change(Screening - Termination)(N=13, 11) -3.8  (20.53) 0.3  (16.58)
Future- Change (Month 6 - Termination)(N=14, 11) -7.7  (20.25) -0.8  (16.30)
PH- Screening (N=15, 12) 64.0  (22.46) 66.4  (24.21)
PH- Month 6 (N=14, 11) 50.8  (27.55) 43.1  (25.24)
PH- Termination (N=14, 11) 56.1  (24.98) 42.3  (18.86)
PH- Change (Screening - Month 6) (N=14, 11) 14.6  (21.70) 21.1  (28.77)
PH- Change(Screening - Termination)(N=14, 11) 9.3  (23.93) 21.9  (24.79)
PH- Change (Month 6 - Termination)(N=14, 11) -5.3  (10.65) 0.8  (20.20)
R'ships- Screening (N=15, 11) 28.3  (36.57) 26.9  (28.83)
R'ships- Month 6 (N=14, 11) 19.0  (28.20) 15.2  (13.85)
R'ships- Termination (N=14, 10) 17.9  (22.13) 15.0  (17.48)
R'ships- Change (Screening - Month 6) (N=14, 10) 7.7  (34.04) 10.4  (27.45)
R'ships- Change(Screening - Termination)(N=14, 10) 8.9  (30.57) 12.1  (25.49)
R'ships- Change (Month 6 - Termination)(N=14, 10) 1.2  (21.40) 1.7  (16.57)
S&L- Screening (N=12, 8) 69.3  (17.11) 83.1  (14.13)
S&L- Month 6 (N=12, 8) 63.1  (23.09) 69.7  (19.75)
S&L- Termination (N=12, 8) 72.3  (17.30) 78.1  (12.52)
S&L- Change (Screening - Month 6) (N=9, 5) 9.9  (19.75) 20.5  (27.97)
S&L- Change(Screening - Termination)(N=9, 5) 1.2  (15.91) 7.0  (21.68)
S&L- Change (Month 6 - Termination)(N=11, 7) -10.2  (13.44) -2.5  (15.61)
Treatment- Screening (N=15, 12) 36.9  (13.32) 39.6  (20.70)
Treatment- Month 6 (N=14, 11) 30.4  (19.82) 40.9  (16.96)
Treatment- Termination (N=14, 11) 34.8  (14.87) 42.6  (17.80)
Treatment- Change (Screening - Month 6) (N=14, 11) 6.5  (23.72) 0.0  (16.74)
Treatment-Change(Screening -Termination)(N=14, 11) 2.1  (17.81) -1.7  (23.86)
Treatment- Change (Month 6- Termination)(N=14, 11) -4.4  (15.07) -1.7  (18.70)
View- Screening (N=15, 12) 52.3  (24.49) 55.6  (11.97)
View- Month 6 (N=14, 11) 44.3  (27.66) 40.0  (13.23)
View- Termination (N=14, 11) 47.9  (22.59) 37.7  (21.26)
View- Change (Screening - Month 6) (N=14, 11) 7.5  (23.68) 14.3  (17.25)
View- Change (Screening -Termination)(N=14, 11) 3.9  (23.79) 16.6  (15.01)
View- Change (Month 6- Termination)(N=14, 11) -3.6  (17.03) 2.3  (20.78)
W&S-Screening (N=13, 10) 43.8  (25.90) 57.7  (17.87)
W&S- Month 6 (N=13, 9) 31.7  (26.82) 37.5  (20.73)
W&S- Termination (N=12, 9) 28.6  (22.53) 36.8  (19.63)
W&S- Change (Screening - Month 6) (N=12, 8) 10.9  (30.98) 25.3  (25.60)
W&S- Change (Screening -Termination)(N=12, 8) 16.1  (33.86) 25.3  (19.01)
W&S- Change (Month 6- Termination)(N=12, 9) 5.2  (15.95) 0.7  (21.75)
Total- Screening (N=14, 12) 44.0  (15.50) 49.2  (15.43)
Total- Month 6 (N=14, 11) 37.4  (19.37) 38.1  (11.34)
Total- Termination (N=14, 11) 40.2  (17.55) 38.7  (14.21)
Total- Change (Screening - Month 6) (N=13, 11) 9.2  (18.44) 10.2  (17.84)
Total- Change (Screening -Termination)(N=13, 11) 6.1  (15.41) 9.5  (12.77)
Total- Change (Month 6- Termination)(N=14, 11) -2.7  (9.21) -0.7  (14.40)
40.Secondary Outcome
Title Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Hide Description

The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & School (S&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.

Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - for all participants <16 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 4 5
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Dealing- Screening (N=4, 4) 25.9  (11.80) 39.3  (17.00)
Dealing- Month 6 (N=4, 4) 25.6  (19.31) 36.6  (16.07)
Dealing- Termination (N=4, 5) 21.4  (16.24) 28.6  (13.36)
Dealing- Change (Screening - Month 6) (N=4, 4) 0.3  (8.61) 2.7  (7.92)
Dealing- Change(Screening - Termination)(N=4, 4) 4.5  (7.92) 13.4  (5.36)
Dealing- Change (Month 6 - Termination)(N=4, 4) 4.2  (11.85) 10.7  (7.72)
Family- Screening (N=4, 5) 55.3  (19.05) 50.9  (8.72)
Family- Month 6 (N=4, 5) 50.3  (27.65) 49.1  (10.27)
Family- Termination (N=4, 5) 45.8  (31.03) 39.8  (13.77)
Family- Change (Screening - Month 6) (N=4, 5) 5.0  (9.26) 1.7  (15.65)
Family- Change(Screening - Termination)(N=4, 5) 9.5  (15.52) 11.1  (19.22)
Family- Change (Month 6 - Termination)(N=4, 5) 4.5  (12.16) 9.3  (5.24)
Feeling- Screening (N=4, 5) 44.1  (14.25) 38.2  (2.77)
Feeling- Month 6 (N=4, 5) 50.3  (27.02) 37.5  (22.21)
Feeling- Termination (N=4, 5) 33.6  (36.07) 27.8  (16.97)
Feeling- Change (Screening - Month 6) (N=4, 5) -6.3  (24.27) 0.7  (24.02)
Feeling- Change(Screening - Termination)(N=4, 5) 10.5  (27.92) 10.5  (19.45)
Feeling- Change (Month 6 - Termination)(N=4, 5) 16.7  (13.85) 9.7  (20.64)
Friends- Screening (N=4, 5) 35.9  (20.65) 52.1  (17.18)
Friends- Month 6 (N=4, 5) 26.6  (17.95) 36.3  (29.78)
Friends- Termination (N=4, 5) 34.4  (14.88) 66.3  (20.06)
Friends- Change (Screening - Month 6) (N=4, 5) 9.4  (27.72) 15.8  (21.88)
Friends- Change(Screening - Termination)(N=4, 5) 1.6  (17.95) -14.2  (28.31)
Friends- Change (Month 6 - Termination)(N=4, 5) -7.8  (22.46) -30.0  (31.68)
Future- Screening (N=2, 2) 53.1  (13.26) 43.8  (17.68)
Future- Month 6 (N=2, 2) 40.6  (13.26) 37.5  (8.84)
Future- Termination (N=2, 2) 31.3  (0.00) 43.8  (8.84)
Future- Change (Screening - Month 6) (N=2, 2) 12.5  (0.00) 6.3  (8.84)
Future- Change(Screening - Termination)(N=2, 2) 21.9  (13.26) 0.0  (8.84)
Future- Change (Month 6 - Termination)(N=2, 2) 9.4  (13.26) -6.3  (0.00)
Others- Screening (N=4, 5) 27.1  (34.44) 29.2  (16.67)
Others- Month 6 (N=4, 5) 42.7  (39.14) 24.2  (15.70)
Others- Termination (N=4, 5) 28.1  (32.87) 25.8  (13.63)
Others- Change (Screening - Month 6) (N=4, 5) -15.6  (16.80) 5.0  (20.28)
Others- Change(Screening - Termination)(N=4, 5) -1.0  (15.36) 3.3  (15.98)
Others- Change (Month 6 - Termination)(N=4, 5) 14.6  (19.98) -1.7  (10.87)
PH- Screening (N=4, 5) 58.0  (11.06) 52.1  (23.23)
PH- Month 6 (N=4, 5) 53.6  (13.98) 48.0  (17.23)
PH- Termination (N=4, 5) 41.1  (11.85) 40.4  (17.02)
PH- Change (Screening - Month 6) (N=4, 5) 4.5  (12.16) 4.1  (32.71)
PH- Change(Screening - Termination)(N=4, 5) 17.0  (17.10) 11.8  (29.60)
PH- Change (Month 6 - Termination)(N=4, 5) 12.5  (22.87) 7.7  (22.25)
R'ships- Screening (N=2, 2) 12.5  (17.68) 43.8  (8.84)
R'ships- Month 6 (N=2, 2) 25.0  (35.36) 43.8  (8.84)
R'ships- Termination (N=2, 2) 0.0  (0.00) 37.5  (17.68)
R'ships- Change (Screening - Month 6) (N=2, 2) -12.5  (17.68) 0.0  (0.00)
R'ships-Change(Screening -Termination)(N=2, 2) 12.5  (17.68) 6.3  (8.84)
R'ships- Change (Month 6- Termination)(N=2, 2) 25.0  (35.36) 6.3  (8.84)
S&S- Screening (N=4, 5) 67.1  (10.68) 59.1  (23.73)
S&S- Month 6 (N=4, 5) 62.5  (7.74) 56.5  (9.07)
S&S- Termination (N=4, 5) 55.6  (17.16) 57.2  (17.29)
S&S- Change (Screening - Month 6) (N=4, 5) 4.6  (6.89) 2.6  (16.32)
S&S- Change (Screening -Termination)(N=4, 5) 11.5  (12.02) 1.9  (10.22)
S&S- Change (Month 6- Termination)(N=4, 5) 6.9  (18.07) -0.8  (8.94)
Support-Screening (N=4, 4) 28.1  (25.77) 51.6  (9.38)
Support-Month 6 (N=4, 4) 18.8  (29.32) 42.2  (17.95)
Support- Termination (N=4, 5) 32.8  (32.02) 48.8  (20.44)
Support- Change (Screening - Month 6) (N=4, 4) 9.4  (24.21) 9.4  (10.83)
Support- Change (Screening -Termination)(N=4, 4 -4.7  (13.86) 3.1  (15.73)
Support- Change (Month 6- Termination)(N=4, 4) -14.1  (21.27) -6.3  (8.84)
Treatment- Screening (N=4, 5) 29.0  (13.23) 64.2  (22.60)
Treatment- Month 6 (N=4, 5) 21.9  (6.90) 54.8  (14.53)
Treatment- Termination (N=4, 5) 17.1  (22.12) 41.0  (13.34)
Treatment- Change (Screening - Month 6) (N=4, 5) 7.1  (10.10) 9.4  (13.06)
Treatment- Change(Screening -Termination)(N=4, 5) 11.9  (17.16) 23.2  (16.94)
Treatment- Change (Month 6- Termination)(N=4, 5) 4.8  (22.47) 13.8  (11.14)
View-Screening (N=4, 5) 47.4  (18.25) 36.7  (16.94)
View-Month 6 (N=4, 5) 46.3  (24.45) 36.4  (22.04)
View- Termination (N=4, 5) 32.8  (35.68) 30.8  (12.85)
View- Change (Screening - Month 6) (N=4, 5) 1.1  (6.38) 0.3  (7.63)
View- Change (Screening -Termination)(N=4, 5) 14.7  (18.62) 5.9  (10.08)
View- Change (Month 6- Termination)(N=4, 5) 13.5  (13.02) 5.6  (16.70)
Total-Screening (N=4, 5) 43.7  (13.67) 48.1  (8.84)
Total-Month 6 (N=4, 5) 42.0  (13.84) 42.3  (11.08)
Total- Termination (N=4, 5) 34.5  (21.83) 39.5  (8.52)
Total- Change (Screening - Month 6) (N=4, 5) 1.7  (1.19) 5.7  (14.97)
Total- Change (Screening -Termination)(N=4, 5) 9.1  (8.57) 8.6  (8.72)
Total- Change (Month 6- Termination)(N=4, 5) 7.4  (8.91) 2.8  (7.06)
41.Secondary Outcome
Title Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Hide Description

The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports & School (S&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100.

Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.

Time Frame 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants <16 years old, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 4 5
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Dealing- Screening (N=4, 4) 33.0  (17.10) 44.6  (20.72)
Dealing- Month 6 (N=4, 4) 21.4  (2.92) 37.5  (13.20)
Dealing- Termination (N=4, 5) 25.0  (9.22) 32.9  (12.73)
Dealing- Change (Screening - Month 6) (N=4, 4) 11.6  (15.26) 7.1  (8.75)
Dealing- Change(Screening - Termination)(N=4, 4) 8.0  (15.26) 14.3  (12.37)
Dealing- Change (Month 6 - Termination)(N=4, 4) -3.6  (6.52) 7.1  (6.52)
Family- Screening (N=4, 5) 50.5  (23.94) 35.6  (22.05)
Family- Month 6 (N=4, 5) 43.4  (28.78) 36.4  (23.06)
Family- Termination (N=4, 5) 38.3  (21.25) 32.5  (21.09)
Family- Change (Screening - Month 6) (N=4, 5) 7.0  (20.31) -0.7  (16.38)
Family- Change(Screening - Termination)(N=4, 5) 12.2  (25.72) 3.1  (15.51)
Family- Change (Month 6 - Termination)(N=4, 5) 5.2  (13.66) 3.9  (12.20)
Feeling- Screening (N=4, 5) 54.8  (31.91) 34.9  (12.89)
Feeling- Month 6 (N=4, 5) 41.4  (43.03) 22.0  (18.68)
Feeling- Termination (N=4, 5) 34.4  (39.48) 19.2  (15.39)
Feeling- Change (Screening - Month 6) (N=4, 5) 13.4  (16.91) 12.9  (27.77)
Feeling- Change(Screening - Termination)(N=4, 5) 20.4  (11.77) 15.7  (23.20)
Feeling- Change (Month 6 - Termination)(N=4, 5) 7.0  (7.79) 2.8  (15.26)
Friends- Screening (N=4, 5) 34.4  (25.26) 58.8  (20.54)
Friends- Month 6 (N=4, 5) 28.1  (15.73) 42.5  (29.78)
Friends- Termination (N=4, 5) 32.8  (12.88) 56.3  (27.24)
Friends- Change (Screening - Month 6) (N=4, 5) 6.3  (25.52) 16.3  (24.84)
Friends- Change(Screening - Termination)(N=4, 5) 1.6  (28.58) 2.5  (22.79)
Friends- Change (Month 6 - Termination)(N=4, 5) -4.7  (17.95) -13.8  (20.44)
Future- Screening (N=2, 2) 40.6  (13.26) 43.8  (17.68)
Future- Month 6 (N=2, 2) 43.8  (17.68) 43.8  (17.68)
Future- Termination (N=2, 2) 46.9  (22.10) 28.1  (22.10)
Future- Change (Screening - Month 6) (N=2, 2) -3.1  (4.42) 0.0  (0.00)
Future- Change(Screening - Termination)(N=2, 2) -6.3  (8.84) 15.6  (4.42)
Future- Change (Month 6 - Termination)(N=2, 2) -3.1  (4.42) 15.6  (4.42)
Others- Screening (N=4, 5) 42.7  (33.74) 26.7  (20.11)
Others- Month 6 (N=4, 5) 33.3  (40.11) 16.7  (15.02)
Others- Termination (N=4, 5) 18.8  (21.11) 13.3  (12.64)
Others- Change (Screening - Month 6) (N=4, 5) 9.4  (8.59) 10.0  (25.79)
Others- Change(Screening - Termination)(N=4, 5) 24.0  (14.18) 13.3  (23.09)
Others- Change (Month 6 - Termination)(N=4, 5) 14.6  (19.39) 3.3  (16.24)
PH- Screening (N=4, 5) 67.0  (24.98) 42.1  (32.28)
PH- Month 6 (N=4, 5) 49.1  (14.69) 33.9  (16.66)
PH- Termination (N=4, 5) 41.1  (23.05) 25.7  (10.83)
PH- Change (Screening - Month 6) (N=4, 5) 17.9  (19.56) 8.2  (34.43)
PH- Change(Screening - Termination)(N=4, 5) 25.9  (14.40) 16.4  (29.73)
PH- Change (Month 6 - Termination)(N=4, 5) 8.0  (12.16) 8.2  (20.66)
R'ships- Screening (N=2, 2) 6.3  (8.84) 43.8  (8.84)
R'ships- Month 6 (N=2, 2) 18.8  (26.52) 43.8  (8.84)
R'ships- Termination (N=2, 2) 6.3  (8.84) 18.8  (26.52)
R'ships- Change (Screening - Month 6) (N=2, 2) -12.5  (17.68) 0.0  (0.00)
R'ships-Change(Screening -Termination)(N=2, 2) 0.0  (0.00) 25.0  (17.68)
R'ships- Change (Month 6- Termination)(N=2, 2) 12.5  (17.68) 25.0  (17.68)
S&S- Screening (N=4, 5) 53.3  (14.74) 51.3  (19.82)
S&S- Month 6 (N=4, 5) 46.8  (13.42) 54.2  (5.53)
S&S- Termination (N=4, 5) 33.2  (5.64) 49.8  (18.49)
S&S- Change (Screening - Month 6) (N=4, 5) 6.5  (11.42) -2.8  (18.53)
S&S- Change (Screening -Termination)(N=4, 5) 20.1  (14.74) 1.5  (18.74)
S&S- Change (Month 6- Termination)(N=4, 5) 13.5  (9.10) 4.4  (15.19)
Support-Screening (N=4, 4) 32.8  (3.13) 62.5  (31.04)
Support-Month 6 (N=4, 4) 28.1  (10.83) 56.3  (31.87)
Support- Termination (N=4, 5) 17.2  (17.21) 65.0  (15.69)
Support- Change (Screening - Month 6) (N=4, 4) 4.7  (12.88) 6.3  (31.46)
Support- Change (Screening -Termination)(N=4, 4 15.6  (18.75) -3.1  (18.75)
Support- Change (Month 6- Termination)(N=4, 4) 10.9  (10.67) -9.4  (25.77)
Treatment- Screening (N=4, 5) 27.3  (15.38) 69.6  (25.63)
Treatment- Month 6 (N=4, 5) 30.6  (16.50) 56.8  (15.89)
Treatment- Termination (N=4, 5) 35.9  (26.10) 44.2  (27.10)
Treatment- Change (Screening - Month 6) (N=4, 5) -3.2  (4.43) 12.9  (21.22)
Treatment- Change(Screening -Termination)(N=4, 5) -8.6  (13.86) 25.4  (28.00)
Treatment- Change (Month 6- Termination)(N=4, 5) -5.3  (11.25) 12.6  (15.53)
View-Screening (N=4, 5) 46.0  (19.33) 31.9  (14.36)
View-Month 6 (N=4, 5) 39.3  (33.02) 32.4  (19.41)
View- Termination (N=4, 5) 32.5  (32.75) 32.2  (8.60)
View- Change (Screening - Month 6) (N=4, 5) 6.7  (14.96) -0.5  (18.94)
View- Change (Screening -Termination)(N=4, 5) 13.5  (16.56) -0.3  (10.77)
View- Change (Month 6- Termination)(N=4, 5) 6.8  (8.67) 0.2  (13.48)
Total-Screening (N=4, 5) 44.9  (12.28) 45.1  (9.16)
Total-Month 6 (N=4, 5) 37.4  (17.91) 37.7  (9.87)
Total- Termination (N=4, 5) 32.2  (17.96) 35.3  (9.06)
Total- Change (Screening - Month 6) (N=4, 5) 7.5  (7.21) 7.4  (16.57)
Total- Change (Screening -Termination)(N=4, 5) 12.7  (8.69) 9.8  (12.35)
Total- Change (Month 6- Termination)(N=4, 5) 5.2  (3.55) 2.5  (9.46)
42.Secondary Outcome
Title Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Hide Description

General pain was assessed using a VAS pain scale at screening, 6 months, and at termination. Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. For the pain scale, a higher number indicates worse pain.

The visual analog scale ranges from 0 to 100 where the endpoints are labeled 'Worst imaginable health state' (=0) and 'Best imaginable health state' (=100). A positive change from baseline indicates improvement.

Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination).

Time Frame Baseline, 6 months and 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants ≥12 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 17 15
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Screening (N= 17, 15) 35.2  (30.15) 55.5  (23.68)
Month 6 (N= 16, 14) 36.6  (25.81) 32.7  (26.24)
Termination (N= 16, 14) 32.4  (21.97) 29.8  (30.48)
Change (Screening - Month 6) (N= 16, 14) 0.8  (31.77) 20.3  (38.91)
Change (Screening - Termination) (N= 16, 14) 5.0  (28.70) 23.2  (46.61)
Change (Month 6 - Termination) (N= 16, 14) 4.2  (21.82) 2.9  (19.62)
43.Secondary Outcome
Title Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Hide Description

General pain was assessed using the children’s VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). Assessments were done at the screening, 6 months, and termination visits.

Scores on the children's VAS scale are presented as:

  • No Pain
  • Mild Pain
  • Moderate pain
  • Severe pain
  • Very severe pain

Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account.

Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination).

Time Frame Baseline, 6 months and 12 months ± 14 days
Hide Outcome Measure Data
Hide Analysis Population Description
Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants <12 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description:
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : FEIBA NF dose and dosing interval as prescribed by the treating physician
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
Overall Number of Participants Analyzed 2 2
Measure Type: Number
Unit of Measure: participants
Screening - No Pain 1 0
Screening - Mild Pain 0 1
Screening - Moderate Pain 1 0
Screening - Severe Pain 0 1
Screening - Very Severe Pain 0 0
Month 6 - No Pain 0 0
Month 6 - Mild Pain 1 2
Month 6 - Moderate Pain 1 0
Month 6 - Severe Pain 0 0
Month 6 - Very Severe Pain 0 0
Termination visit - No Pain 0 1
Termination visit - Mild Pain 0 0
Termination visit - Moderate Pain 1 1
Termination visit - Severe Pain 0 0
Termination visit - Very Severe Pain 1 0
Time Frame Throughout the study period of 3 years and 7 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title On-demand Arm Prophylaxis Arm
Hide Arm/Group Description Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month ± 14 days prophylactic period
All-Cause Mortality
On-demand Arm Prophylaxis Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
On-demand Arm Prophylaxis Arm
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/19 (36.84%)      6/17 (35.29%)    
Gastrointestinal disorders     
Abdominal Wall Haematoma  0/19 (0.00%)  0 1/17 (5.88%)  1
Hepatobiliary disorders     
Cholecystitis Acute  1/19 (5.26%)  1 0/17 (0.00%)  0
Infections and infestations     
Haematoma Infection  1/19 (5.26%)  1 0/17 (0.00%)  0
Injury, poisoning and procedural complications     
Femoral Neck Fracture  1/19 (5.26%)  1 0/17 (0.00%)  0
Investigations     
Hepatitis B Surface Antibody Positive  1/19 (5.26%)  1 2/17 (11.76%)  2
Musculoskeletal and connective tissue disorders     
Arthropathy  1/19 (5.26%)  1 0/17 (0.00%)  0
Haemarthrosis  1/19 (5.26%)  3 2/17 (11.76%)  8
Muscle Haemorrhage  0/19 (0.00%)  0 1/17 (5.88%)  1
Renal and urinary disorders     
Haematuria  1/19 (5.26%)  1 0/17 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Epistaxis  1/19 (5.26%)  1 0/17 (0.00%)  0
Surgical and medical procedures     
Catheter Removal  1/19 (5.26%)  1 0/17 (0.00%)  0
Vascular disorders     
Arteriosclerosis  1/19 (5.26%)  1 0/17 (0.00%)  0
Haematoma  0/19 (0.00%)  0 1/17 (5.88%)  2
Haemorrhage  1/19 (5.26%)  1 0/17 (0.00%)  0
Hypertensive Crisis  1/19 (5.26%)  1 0/17 (0.00%)  0
1
Term from vocabulary, MedDRA (Unspecified)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
On-demand Arm Prophylaxis Arm
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/19 (21.05%)      3/17 (17.65%)    
Blood and lymphatic system disorders     
Anaemia  2/19 (10.53%)  2 0/17 (0.00%)  0
Gastrointestinal disorders     
Diarrhoea  1/19 (5.26%)  2 1/17 (5.88%)  1
Nausea  2/19 (10.53%)  2 0/17 (0.00%)  0
Vomiting  2/19 (10.53%)  2 0/17 (0.00%)  0
General disorders     
Pyrexia  2/19 (10.53%)  4 0/17 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Arthralgia  2/19 (10.53%)  3 1/17 (5.88%)  1
Arthropathy  1/19 (5.26%)  1 0/17 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Cough  1/19 (5.26%)  1 1/17 (5.88%)  1
Oropharyngeal Pain  1/19 (5.26%)  1 1/17 (5.88%)  1
Skin and subcutaneous tissue disorders     
Acne  1/19 (5.26%)  1 1/17 (5.88%)  1
1
Term from vocabulary, MedDRA (Unspecified)
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.
Baxter's agreements with PIs may vary per requirements of individual PI, but contain common elements. For this study: PIs may require prior written approval, or be restricted from independently publishing results until the earlier of the primary multicenter publication or ≤36 months after study completion. Baxter requires a review of results communications (e.g., for confidential information) prior to submission for publication, and shall review and if necessary amend the manuscript in ≤60 days
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Wing-Yen Wong, MD, Global Sr Medical Dir Hemophilia
Organization: Baxter Healthcare Corporation
EMail: wing_yen_wong@baxter.com
Layout table for additonal information
Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT00851721     History of Changes
Other Study ID Numbers: 090701
First Submitted: February 25, 2009
First Posted: February 26, 2009
Results First Submitted: January 30, 2014
Results First Posted: March 14, 2014
Last Update Posted: November 22, 2017