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Trial record 18 of 82 for:    bleeding episodes | ( Map: France )

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

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ClinicalTrials.gov Identifier: NCT01425723
Recruitment Status : Completed
First Posted : August 30, 2011
Results First Posted : November 22, 2018
Last Update Posted : November 22, 2018
Sponsor:
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Severe Hemophilia B
Intervention Biological: rFIXFc
Enrollment 120
Recruitment Details  
Pre-assignment Details Participants who completed either of the Phase 3 studies (Study 998HB102 [NCT01027364], Study 9HB02PED [NCT01440946]) were expected to be eligible to enroll in this study.
Arm/Group Title rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102)
Hide Arm/Group Description Participants received Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) intravenously (IV) according to their assigned treatment regimen as follows: Weekly prophylaxis (P): Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of greater than (>)5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg twice weekly versus 50 IU/kg once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event. Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 international units per kilogram (IU/kg) to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5 percent (%), if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Period Title: Overall Study
Started 27 [1] 93
Weekly Prophylaxis 23 51
Individualized Prophylaxis 5 31
Personalized Prophylaxis 2 17
Episodic 0 15
Surgery Subgroup [2] 1 15
Completed 23 75
Not Completed 4 18
Reason Not Completed
Lack of Efficacy             0             1
Withdrawal by Subject             1             4
Lost to Follow-up             0             3
Physician Decision             2             0
Other             1             10
[1]
13 participants in <6 Years Old Age Cohort and 14 participants in 6 to <12 Years Old Age Cohort.
[2]
Participants who have undergone major surgery or have major surgical/rehabilitation period in study.
Arm/Group Title rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102) Total
Hide Arm/Group Description Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of >5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg twice weekly versus 50 IU/kg once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event. Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event. Total of all reporting groups
Overall Number of Baseline Participants 27 93 120
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants 93 participants 120 participants
<18 years
27
 100.0%
8
   8.6%
35
  29.2%
Between 18 and 65 years
0
   0.0%
85
  91.4%
85
  70.8%
> 65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants 93 participants 120 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
27
 100.0%
93
 100.0%
120
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants 93 participants 120 participants
Hispanic or Latino
0
   0.0%
11
  11.8%
11
   9.2%
Not Hispanic or Latino
27
 100.0%
78
  83.9%
105
  87.5%
Unknown or Not Reported
0
   0.0%
4
   4.3%
4
   3.3%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants 93 participants 120 participants
White
19
  70.4%
47
  50.5%
66
  55.0%
Black or African American
2
   7.4%
9
   9.7%
11
   9.2%
Asian
5
  18.5%
27
  29.0%
32
  26.7%
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Other
1
   3.7%
10
  10.8%
11
   9.2%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Number of Participants With Any Positive Inhibitor Development
Hide Description An inhibitor test result greater than or equal to (>=)0.6 Bethesda units per milliliter (BU/mL), confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Data was summarized by treatment regimen for participants from Study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from Study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame Approximately 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set included participants who received at least 1 dose of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in study 9HB01EXT.
Arm/Group Title rFIXFc (9HB02PED [<6 Years Old]) rFIXFc (9HB02PED [6 to <12 Years]) rFIXFc (Participants From 998HB102)
Hide Arm/Group Description:
Participants with less than (<) 6 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants with 6 to <12 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 13 14 93
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
2.Secondary Outcome
Title Annualized Bleeding Rate (ABR)
Hide Description ABR is annualized number of bleeding episodes per participant per year. Bleeding episodes were classified as spontaneous if participant records bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity and classified as traumatic if participant records bleeding event when there is known reason for bleed. ABR=(Number of bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. ABR was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame Approximately 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) included all participants who received at least 1 dose of rFIXFc. Here 'n' (number analyzed) signifies number of participants who were analyzed in each treatment regimen, for each arm, respectively.
Arm/Group Title rFIXFc (9HB02PED [<6 Years Old Age Cohort]) rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort]) rFIXFc (Participants From Study 998HB102)
Hide Arm/Group Description:
Participants with < 6 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants with 6 to <12 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 13 14 93
Median (Inter-Quartile Range)
Unit of Measure: episodes per participant per year
Weekly Prophylaxis Number Analyzed 13 participants 10 participants 51 participants
1.04
(0.00 to 2.28)
1.14
(0.54 to 2.34)
2.26
(0.40 to 5.16)
Individualized Prophylaxis Number Analyzed 0 participants 5 participants 31 participants
3.69
(3.54 to 5.21)
1.85
(0.76 to 4.00)
Personalized Prophylaxis Number Analyzed 1 participants 1 participants 16 participants
0.54
(0.54 to 0.54)
3.13
(3.13 to 3.13)
2.91
(1.14 to 5.36)
Episodic Number Analyzed 0 participants 0 participants 15 participants
11.64
(5.12 to 18.54)
3.Secondary Outcome
Title Annualized Spontaneous Joint Bleeding Episodes
Hide Description Bleeding episodes were classified as spontaneous if participant records a bleeding event when there is no known contributing factor such as definite trauma/antecedent strenuous activity. In addition, location of bleed (joint, internal, skin/mucosa or muscle) were also collected. Annualized spontaneous joint bleeding episodes=(Number of spontaneous joint bleeding episodes during efficacy period/number of days during efficacy period)*365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Bleeding episodes were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame Approximately 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all participants who received at least 1 dose of rFIXFc. Here 'n' (number analyzed) signifies number of participants who were analyzed in each treatment regimen, for each arm, respectively.
Arm/Group Title rFIXFc (9HB02PED [<6 Years Old Age Cohort]) rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort]) rFIXFc (Participants From Study 998HB102)
Hide Arm/Group Description:
Participants with < 6 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants with 6 to <12 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 13 14 93
Median (Inter-Quartile Range)
Unit of Measure: episodes per participant per year
Weekly Prophylaxis Number Analyzed 13 participants 10 participants 51 participants
0.00
(0.00 to 1.06)
0.00
(0.00 to 1.40)
0.38
(0.00 to 2.25)
Individualized Prophylaxis Number Analyzed 0 participants 5 participants 31 participants
0.00
(0.00 to 0.29)
0.38
(0.00 to 1.43)
Personalized Prophylaxis Number Analyzed 1 participants 1 participants 16 participants
0.00
(0.00 to 0.00)
0.00
(0.00 to 0.00)
0.30
(0.00 to 1.37)
Episodic Number Analyzed 0 participants 0 participants 15 participants
2.15
(0.58 to 11.68)
4.Secondary Outcome
Title Total Number of Exposure Days (EDs)
Hide Description An exposure day is a 24-hour period in which one or more rFIXFc injections are given. The total number of days of exposure to rFIXFc were summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame Approximately 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set included participants who received at least 1 dose of rFIXFc in study 9HB01EXT. Here 'n' (number analyzed) signifies number of participants who were analyzed in each treatment regimen, for each arm, respectively.
Arm/Group Title rFIXFc (9HB02PED [<6 Years Old Age Cohort]) rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort]) rFIXFc (Participants From Study 998HB102)
Hide Arm/Group Description:
Participants with < 6 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants with 6 to <12 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 13 14 93
Median (Full Range)
Unit of Measure: days
Weekly Prophylaxis Number Analyzed 13 participants 10 participants 51 participants
55.00
(8.0 to 176.0)
165.00
(3.0 to 202.0)
169.00
(4.0 to 327.0)
Individualized Prophylaxis Number Analyzed 0 participants 5 participants 31 participants
54.00
(4.0 to 141.0)
110.00
(9.0 to 369.0)
Personalized Prophylaxis Number Analyzed 1 participants 1 participants 17 participants
157.00
(157.00 to 157.00)
90.00
(90.00 to 90.00)
146.00
(7.0 to 431.0)
Episodic Number Analyzed 0 participants 0 participants 15 participants
52.00
(0.0 to 164.0)
5.Secondary Outcome
Title Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
Hide Description Annualized consumption = (total international unit per kilogram [IU/kg] of study treatment received during the efficacy period / total number of days during the efficacy period) multiplied by 365.25. Efficacy period reflects sum of all intervals of time during which participants were treated with rFIXFc per treatment regimen excluding major and minor surgical/rehabilitation periods and large injection intervals. Annualized consumption was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED as per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Time Frame Approximately 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all participants who received at least 1 dose of rFIXFc. Here 'n' (number analyzed) signifies number of participants who were analyzed in each treatment regimen, for each arm, respectively.
Arm/Group Title rFIXFc (9HB02PED [<6 Years Old Age Cohort]) rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort]) rFIXFc (Participants From Study 998HB102)
Hide Arm/Group Description:
Participants with < 6 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants with 6 to <12 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 13 14 93
Median (Inter-Quartile Range)
Unit of Measure: IU per kilogram per participant per year
Weekly Prophylaxis Number Analyzed 13 participants 10 participants 51 participants
3382.5
(2930.9 to 3667.4)
3212.0
(2838.5 to 3344.4)
2598.0
(2129.8 to 3370.9)
Individualized Prophylaxis Number Analyzed 0 participants 5 participants 31 participants
3700.7
(3689.2 to 3716.5)
2894.8
(2520.0 to 4023.5)
Personalized Prophylaxis Number Analyzed 1 participants 1 participants 16 participants
3331.7
(3331.7 to 3331.7)
8931.2
(8931.2 to 8931.2)
3671.2
(2394.8 to 5563.3)
Episodic Number Analyzed 0 participants 0 participants 15 participants
595.6
(363.8 to 1024.5)
6.Secondary Outcome
Title Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
Hide Description Participants were assessed for response to their rFIXFc regimen using following 4-point scale: 1=Excellent: bleeding episodes responded to less than or equal to (<=)usual number of injections or dose of rFIXFc or rate of breakthrough bleeding during prophylaxis was <= that usually observed; 2=Effective: most bleeding episodes responded to same number of injections and dose, but some required more injections or higher doses, or there was minor increase in rate of breakthrough; 3=Partially Effective: bleeding episodes most often required more injections and/or higher doses than expected or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses and 4=Ineffective: routine failure to control hemostasis/hemostatic control require additional agents. Total number of scale responses =total count of scale responses for all participants; multiple responses per participant including those at scheduled and unscheduled visits are counted.
Time Frame Approximately 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
FAS included all participants who received at least 1 dose of rFIXFc. Data was summarized by treatment regimen for participants from Study 998HB102 and Study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Arm/Group Title rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102)
Hide Arm/Group Description:
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of >5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg twice weekly versus 50 IU/kg once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 27 93
Overall Number of Units Analyzed
Type of Units Analyzed: Responses
151 815
Count of Units
Unit of Measure: Responses
Excellent
131
  86.8%
622
  76.3%
Effective
19
  12.6%
184
  22.6%
Partially Effective
1
   0.7%
9
   1.1%
Ineffective
0
   0.0%
0
   0.0%
7.Secondary Outcome
Title Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
Hide Description Using eDiary, participant received rating for treatment response to any bleeding episode (BE) using 4-point scale- 1=Excellent: Abrupt pain relief and/or improvement in signs of bleeding within approximately (approx.) 8 hours (h) after initial injection (inj.); 2=Good: Definite pain relief and/or improvement in signs of bleeding within approx. 8h after an injection, but possibly requiring more than 1 injection after 24–48h for complete resolution; 3=Moderate: Probable/slight beneficial effect within 8h after initial injection and requires more than 1 injection and 4=None: No improvement, or condition worsens within approx. 8h after initial injection. This assessment was to be made approx. 8 to 12h from time the injection was given to treat BE and prior to any additional doses of rFIXFc given for same bleeding episode. Percentages are based on the number of bleeding episodes for which a response (excellent or good) was provided for the first injection during the efficacy period.
Time Frame Approximately 5 years
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Hide Analysis Population Description
FAS was analyzed. Data was summarized by treatment regimen for participants from study 998HB102 and by age cohort (<6 years and 6 to <12 years old) and treatment regimen for participants from study 9HB02PED per planned analysis. Participants were included in summary of more than 1 treatment regimen if their regimen changed during study.
Arm/Group Title rFIXFc (9HB02PED [<6 Years Old Age Cohort]) rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort]) rFIXFc (Participants From 998HB102)
Hide Arm/Group Description:
Participants with < 6 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants with 6 to <12 years old age received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of > 5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event.
Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event.
Overall Number of Participants Analyzed 13 14 93
Overall Number of Units Analyzed
Type of Units Analyzed: Injections
75 90 1646
Count of Units
Unit of Measure: Injections
Weekly Prophylaxis Number Analyzed 74 Injections 57 Injections 461 Injections
60
  81.1%
47
  82.5%
342
  74.2%
Individualized Prophylaxis Number Analyzed 0 Injections 31 Injections 386 Injections
25
  80.6%
336
  87.0%
Personalized Prophylaxis Number Analyzed 1 Injections 2 Injections 178 Injections
0
   0.0%
1
  50.0%
135
  75.8%
Episodic Number Analyzed 0 Injections 0 Injections 621 Injections
603
  97.1%
Time Frame From signing of Informed Consent Form (ICF) through follow-up (approximately 5 years)
Adverse Event Reporting Description Adverse events (AEs) data was planned to be reported for each group and for the overall participants. AEs emergent during major surgical/rehabilitation periods are analyzed separately as per planned analysis and are presented as separate groups.
 
Arm/Group Title rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102) Overall rFIXFc (Participants From 9HB02PED and 998HB102) rFIXFc (Participants From 9HB02PED in Surgery Subgroup) rFIXFc (Participants From 998HB102 in Surgery Subgroup)
Hide Arm/Group Description Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis (P): Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile in parent study and individual pharmacokinetic profile, trough and/or peak (recovery) values; Individualized P: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized P: included addition of prevention doses prior to strenuous activity; targeting FIX trough level of >5%, if warranted by bleeding history and/or activity level or dosing twice a week (25 IU/kg twice weekly versus 50 IU/kg once weekly) for participants who may have better control with such regimen. Participants who reached age of 12 during study could also choose to switch to episodic treatment group, with dosing based on participants clinical condition and type and severity of bleeding event. Participants received rFIXFc IV according to their assigned treatment regimen as follows: Weekly prophylaxis: Participants received 20 IU/kg to 100 IU/kg rFIXFc once weekly. Dose was based on participants clinical profile observed in parent study and individual pharmacokinetic profile, trough, and/or peak (recovery) values. Individualized prophylaxis: 100 IU/kg rFIXFc every 8 to 16 days or twice a month; Personalized Prophylaxis: included addition of prevention doses prior to strenuous activity; targeting a FIX trough level of > 5%, if warranted by the bleeding history and/or activity level or dosing twice a week (25 IU/kg, twice weekly, versus 50 IU/kg, once weekly) for participants who may have better control with such a regimen. Episodic (On Demand): The individual dose of rFIXFc to treat bleeding episodes was based on participants clinical condition, type and severity of the bleeding event. All participants who received rFIXFc drug in study 9HB01EXT, from studies 9HB02PED and 998HB102 (combined). AEs emergent during major surgical/rehabilitation periods are excluded and are presented as separate groups. Participants who required emergent or elective surgery while participating in this study and treated with the dose and regimen of rFIXFc as appropriate for the type of surgery. Participants returned to a regular rFIXFc regimen once all dosing for the postoperative rehabilitation period had been completed. Participants who required emergent or elective surgery while participating in this study and treated with the dose and regimen of rFIXFc as appropriate for the type of surgery. Participants returned to a regular rFIXFc regimen once all dosing for the postoperative rehabilitation period had been completed.
All-Cause Mortality
rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102) Overall rFIXFc (Participants From 9HB02PED and 998HB102) rFIXFc (Participants From 9HB02PED in Surgery Subgroup) rFIXFc (Participants From 998HB102 in Surgery Subgroup)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/27 (0.00%)   0/93 (0.00%)   0/120 (0.00%)   0/1 (0.00%)   0/15 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102) Overall rFIXFc (Participants From 9HB02PED and 998HB102) rFIXFc (Participants From 9HB02PED in Surgery Subgroup) rFIXFc (Participants From 998HB102 in Surgery Subgroup)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   5/27 (18.52%)   31/93 (33.33%)   36/120 (30.00%)   0/1 (0.00%)   1/15 (6.67%) 
Blood and lymphatic system disorders           
Anaemia   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Eye disorders           
Necrotising retinitis   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Gastrointestinal disorders           
Enterocolitis   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Gastrointestinal haemorrhage   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Anal Sphincter Atony   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
General disorders           
Pain   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Infections and infestations           
Anal abscess   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Cellulitis   0/27 (0.00%)  3/93 (3.23%)  3/120 (2.50%)  0/1 (0.00%)  0/15 (0.00%) 
Hepatitis C   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Orchitis   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Pilonidal cyst   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Pneumonia   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Staphylococcal bacteraemia   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Tonsillitis   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Tooth abscess   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Wound infection   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Injury, poisoning and procedural complications           
Concussion   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Extradural haematoma   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Fall   3/27 (11.11%)  4/93 (4.30%)  7/120 (5.83%)  0/1 (0.00%)  0/15 (0.00%) 
Femoral neck fracture   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Foreign body   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Head injury   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Laceration   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Post procedural haematoma   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Post procedural haemorrhage   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Rib fracture   0/27 (0.00%)  2/93 (2.15%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Road traffic accident   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Traumatic haematoma   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Traumatic intracranial haemorrhage   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Upper limb fracture   1/27 (3.70%)  0/93 (0.00%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Musculoskeletal and connective tissue disorders           
Arthropathy   0/27 (0.00%)  2/93 (2.15%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Haemarthrosis   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Haemophilic arthropathy   0/27 (0.00%)  4/93 (4.30%)  4/120 (3.33%)  0/1 (0.00%)  0/15 (0.00%) 
Intervertebral disc protrusion   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Slipping rib syndrome   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Spinal column stenosis   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)           
Hepatic neoplasm malignant   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Nervous system disorders           
Epilepsy   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Haemorrhage intracranial   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Posterior interosseous syndrome   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Renal and urinary disorders           
Calculus ureteric   0/27 (0.00%)  2/93 (2.15%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Haematuria   0/27 (0.00%)  2/93 (2.15%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Renal colic   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Renal failure acute   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Reproductive system and breast disorders           
Epididymitis   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
Respiratory, thoracic and mediastinal disorders           
Epistaxis   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Tonsillar haemorrhage   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Social circumstances           
Victim of crime   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Surgical and medical procedures           
Knee arthroplasty   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Tonsillectomy   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Vascular disorders           
Haematoma   0/27 (0.00%)  1/93 (1.08%)  1/120 (0.83%)  0/1 (0.00%)  0/15 (0.00%) 
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
rFIXFc (Participants From 9HB02PED) rFIXFc (Participants From 998HB102) Overall rFIXFc (Participants From 9HB02PED and 998HB102) rFIXFc (Participants From 9HB02PED in Surgery Subgroup) rFIXFc (Participants From 998HB102 in Surgery Subgroup)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   23/27 (85.19%)   66/93 (70.97%)   89/120 (74.17%)   0/1 (0.00%)   4/15 (26.67%) 
Gastrointestinal disorders           
Diarrhoea   2/27 (7.41%)  4/93 (4.30%)  6/120 (5.00%)  0/1 (0.00%)  0/15 (0.00%) 
Gingival bleeding   2/27 (7.41%)  0/93 (0.00%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Gastrooesophageal reflux disease   1/27 (3.70%)  7/93 (7.53%)  8/120 (6.67%)  0/1 (0.00%)  0/15 (0.00%) 
Nausea   0/27 (0.00%)  5/93 (5.38%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Toothache   0/27 (0.00%)  5/93 (5.38%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Vomiting   5/27 (18.52%)  7/93 (7.53%)  12/120 (10.00%)  0/1 (0.00%)  0/15 (0.00%) 
Food poisoning   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
General disorders           
Pyrexia   6/27 (22.22%)  5/93 (5.38%)  11/120 (9.17%)  0/1 (0.00%)  0/15 (0.00%) 
Immune system disorders           
Seasonal allergy   4/27 (14.81%)  3/93 (3.23%)  7/120 (5.83%)  0/1 (0.00%)  0/15 (0.00%) 
Infections and infestations           
Ear infection   3/27 (11.11%)  1/93 (1.08%)  4/120 (3.33%)  0/1 (0.00%)  0/15 (0.00%) 
Influenza   1/27 (3.70%)  8/93 (8.60%)  9/120 (7.50%)  0/1 (0.00%)  1/15 (6.67%) 
Hepatitis C   0/27 (0.00%)  5/93 (5.38%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Lower respiratory tract infection   3/27 (11.11%)  1/93 (1.08%)  4/120 (3.33%)  0/1 (0.00%)  0/15 (0.00%) 
Nasopharyngitis   3/27 (11.11%)  14/93 (15.05%)  17/120 (14.17%)  0/1 (0.00%)  0/15 (0.00%) 
Paronychia   2/27 (7.41%)  0/93 (0.00%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Tonsillitis   2/27 (7.41%)  0/93 (0.00%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Sinusitis   1/27 (3.70%)  5/93 (5.38%)  6/120 (5.00%)  0/1 (0.00%)  0/15 (0.00%) 
Upper respiratory tract infection   2/27 (7.41%)  6/93 (6.45%)  8/120 (6.67%)  0/1 (0.00%)  0/15 (0.00%) 
Varicella   2/27 (7.41%)  1/93 (1.08%)  3/120 (2.50%)  0/1 (0.00%)  0/15 (0.00%) 
Viral infection   2/27 (7.41%)  3/93 (3.23%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Injury, poisoning and procedural complications           
Excoriation   2/27 (7.41%)  1/93 (1.08%)  3/120 (2.50%)  0/1 (0.00%)  0/15 (0.00%) 
Fall   7/27 (25.93%)  4/93 (4.30%)  11/120 (9.17%)  0/1 (0.00%)  0/15 (0.00%) 
Head injury   2/27 (7.41%)  2/93 (2.15%)  4/120 (3.33%)  0/1 (0.00%)  0/15 (0.00%) 
Joint dislocation   2/27 (7.41%)  0/93 (0.00%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Joint injury   2/27 (7.41%)  2/93 (2.15%)  4/120 (3.33%)  0/1 (0.00%)  0/15 (0.00%) 
Ligament sprain   2/27 (7.41%)  4/93 (4.30%)  6/120 (5.00%)  0/1 (0.00%)  0/15 (0.00%) 
Laceration   3/27 (11.11%)  5/93 (5.38%)  8/120 (6.67%)  0/1 (0.00%)  0/15 (0.00%) 
Post Procedural Complication   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
Procedural Pain   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  2/15 (13.33%) 
Investigations           
Serum ferritin decreased   2/27 (7.41%)  0/93 (0.00%)  2/120 (1.67%)  0/1 (0.00%)  0/15 (0.00%) 
Musculoskeletal and connective tissue disorders           
Arthralgia   4/27 (14.81%)  10/93 (10.75%)  14/120 (11.67%)  0/1 (0.00%)  0/15 (0.00%) 
Pain in extremity   2/27 (7.41%)  1/93 (1.08%)  3/120 (2.50%)  0/1 (0.00%)  0/15 (0.00%) 
Nervous system disorders           
Headache   3/27 (11.11%)  15/93 (16.13%)  18/120 (15.00%)  0/1 (0.00%)  0/15 (0.00%) 
Convulsion   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
Psychiatric disorders           
Insomnia   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
Stress   0/27 (0.00%)  0/93 (0.00%)  0/120 (0.00%)  0/1 (0.00%)  1/15 (6.67%) 
Renal and urinary disorders           
Haematuria   0/27 (0.00%)  5/93 (5.38%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Respiratory, thoracic and mediastinal disorders           
Cough   3/27 (11.11%)  5/93 (5.38%)  8/120 (6.67%)  0/1 (0.00%)  0/15 (0.00%) 
Nasal congestion   5/27 (18.52%)  0/93 (0.00%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Epistaxis   5/27 (18.52%)  0/93 (0.00%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Skin and subcutaneous tissue disorders           
Acne   0/27 (0.00%)  5/93 (5.38%)  5/120 (4.17%)  0/1 (0.00%)  0/15 (0.00%) 
Vascular disorders           
Hypertension   0/27 (0.00%)  7/93 (7.53%)  7/120 (5.83%)  0/1 (0.00%)  0/15 (0.00%) 
Indicates events were collected by 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.
Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Bioverativ Study Medical Director
Organization: Bioverativ Therapeutics Inc.
Phone: 781-6631801
EMail: clinicaltrials@bioverativ.com
Layout table for additonal information
Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01425723     History of Changes
Other Study ID Numbers: 9HB01EXT
2011-003075-11
First Submitted: August 19, 2011
First Posted: August 30, 2011
Results First Submitted: October 5, 2018
Results First Posted: November 22, 2018
Last Update Posted: November 22, 2018