Long-Term Safety and Efficacy of rFIXFc in the Prevention... | NCT01425723 | Trialant
NCT01425723
Sponsor
Bioverativ Therapeutics Inc.
Status
Completed
Last Update Posted
Dec 19, 2020Actual
Enrollment
120Actual
Phase
Phase 3
Conditions
Severe Hemophilia B
Interventions
rFIXFc
Countries
United States
Australia
Belgium
Brazil
Canada
China
France
Germany
Hong Kong
India
Ireland
Italy
Japan
Netherlands
Poland
South Africa
Sweden
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT01425723
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
9HB01EXT
Secondary IDs
ID
Type
Description
Link
2011-003075-11
Brief Title
Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B
Official Title
An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B
Acronym
B-YOND
Organization
SanofiINDUSTRY
Status Module
Record Verification Date
Nov 2018
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Dec 8, 2011Actual
Primary Completion Date
Oct 2017Actual
Completion Date
Oct 2017Actual
First Submitted Date
Aug 19, 2011
First Submission Date that Met QC Criteria
Aug 29, 2011
First Posted Date
Aug 30, 2011Estimated
Results Waived
Not provided
Results First Submitted Date
Oct 5, 2018
Results First Submitted that Met QC Criteria
Nov 21, 2018
Results First Posted Date
Nov 23, 2018Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Dec 16, 2020
Last Update Posted Date
Dec 19, 2020Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Bioverativ Therapeutics Inc.INDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
No
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophilia B.
The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.
Detailed Description
Participants will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the patient in the preceding studies, B-LONG 998HB102 (NCT01027364) and Kids B-LONG study 9HB02PED (NCT01440946)
Conditions Module
Conditions
Severe Hemophilia B
Keywords
B-LONG
B-YOND
B-LONG Extension
rFIXFc
Severe Hemophilia B
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
120Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
On-Demand
Experimental
The individual dose of rFIXFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor IX peak (recovery) levels.
Biological: rFIXFc
Prophylaxis
Experimental
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biological: rFIXFc
Interventions
Name
Type
Description
Arm Group Labels
Other Names
rFIXFc
Biological
Administered as specified in the treatment arm.
On-Demand
Prophylaxis
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Number of Participants With Any Positive Inhibitor Development
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.
Approximately 5 years
Secondary Outcomes
Measure
Description
Time Frame
Annualized Bleeding Rate (ABR)
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.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Key Inclusion Criteria:
Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
Ability to understand the purposes & risks of the study and provide signed and dated informed consent.
Key Exclusion Criteria:
High-titer inhibitor (>/=5.00 BU/mL)
NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.
Astermark J, Hermans C, Ezzalfani M, Sidhom A, Barbier S, Kragh N, Falk A, Eriksson D. Recombinant factor IX Fc prophylaxis reduces pain and increases levels of physical activity, with sustained, long-term improvements in patients with hemophilia B: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2023 May 29;14:20406207231170701. doi: 10.1177/20406207231170701. eCollection 2023.
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.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
rFIXFc (Participants From 9HB02PED)
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.
Periods
Title
Milestones
Reasons Not Completed
Overall Study
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Nov 13, 2013
Oct 5, 2018
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Panama
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Non-Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
coagulation factor IX (recombinant) Fc fusion protein
Alprolix
BIIB029
Approximately 5 years
Annualized Spontaneous Joint Bleeding Episodes
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.
Approximately 5 years
Total Number of Exposure Days (EDs)
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.
Approximately 5 years
Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
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.
Approximately 5 years
Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
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.
Approximately 5 years
Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
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.
Approximately 5 years
Sacramento
California
95817
United States
Research Site
Aurora
Colorado
80045
United States
Research Site
Atlanta
Georgia
30322
United States
Research Site
Honolulu
Hawaii
96826
United States
Research Site
Indianapolis
Indiana
46260
United States
Research Site
New Orleans
Louisiana
70112
United States
Research Site
East Lansing
Michigan
48823
United States
Research Site
Pittsburgh
Pennsylvania
15213
United States
Research Site
Seattle
Washington
98104
United States
Research Site
Adelaide
South Australia
5000
Australia
Research site
Parkville
Victoria
3052
Australia
Research site
Murdoch
Western Australia
6150
Australia
Research Site
Perth
Western Australia
6008
Australia
Research Site
Brussels
1200
Belgium
Research Site
Leuven
3000
Belgium
Research Site
Campinas
São Paulo
13083-878
Brazil
Research Site
Toronto
Ontario
M5B 1W8
Canada
Research Site
Montreal
Quebec
H3T 1C5
Canada
Research Site
Beijing
Beijingshì
100005
China
Research Site
Guangzhou
Guangdongsheng
510515
China
Research Site
Shanghai
Shà nghaishì
200025
China
Research Site
Tianjing
Tianjinshì
300020
China
Research Site
Marseille
Bouches-Du-Rhône
13385
France
Research Site
Bonn
North Rhine-Westphalia
53127
Germany
Research Site
Hong Kong
New Territories
Hong Kong
Research site
Hong Kong
Hong Kong
Research Site
Bangalore
Karnataka
560034
India
Research Site
Pune
Maharashtra
411004
India
Research Site
Vellore
Tamil Nadu
632004
India
Research Site
Dublin
D12 N512
Ireland
Research Site
Florence
50134
Italy
Research Site
Milan
20122
Italy
Research Site
Nagoya
Aichi-ken
466-8550
Japan
Research Site
Kitakyushu
Fukuoka
807-8555
Japan
Research Site
Kawasaki
Kanagawa
216-8511
Japan
Research Site
Kashihara-shi
Nara
634-8522
Japan
Research Site
Shinjuku-ku
Tokyo-To
160-0023
Japan
Research Site
Tokyo
Tokyo-To
167-8515
Japan
Research Site
Utrecht
3584 CX
Netherlands
Research Site
Lodz
93-510
Poland
Research Site
Johannesburg
Gauteng
2193
South Africa
Research Site
Cape Town
Western Cape
7925
South Africa
Research Site
Malmö
20502
Sweden
Research Site
Stockholm
17176
Sweden
Research Site
Cambridge
Cambridgeshire
CB2 0QQ
United Kingdom
Research Site
London
Greater London
E1 1BB
United Kingdom
Research site
London
Greater London
SE1 7EH
United Kingdom
Research Site
Basingstoke
Hampshire
RG24 9NA
United Kingdom
Derived
Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230.
Shapiro AD, Pasi KJ, Ozelo MC, Kulkarni R, Barnowski C, Winding B, Szamosi J, Lethagen S. Extending recombinant factor IX Fc fusion protein dosing interval to 14 or more days in patients with hemophilia B. Res Pract Thromb Haemost. 2018 Nov 29;3(1):109-113. doi: 10.1002/rth2.12163. eCollection 2019 Jan.
FG001
rFIXFc (Participants From 998HB102)
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.
FG00027 subjects13 participants in \<6 Years Old Age Cohort and 14 participants in 6 to \<12 Years Old Age Cohort.
FG00193 subjects
Weekly Prophylaxis
FG00023 subjects
FG00151 subjects
Individualized Prophylaxis
FG0005 subjects
FG00131 subjects
Personalized Prophylaxis
FG0002 subjects
FG00117 subjects
Episodic
FG0000 subjects
FG00115 subjects
Surgery Subgroup
Participants who have undergone major surgery or have major surgical/rehabilitation period in study.
FG0001 subjects
FG00115 subjects
COMPLETED
FG00023 subjects
FG00175 subjects
NOT COMPLETED
FG0004 subjects
FG00118 subjects
Type
Comment
Reasons
Lack of Efficacy
FG0000 subjects
FG0011 subjects
Withdrawal by Subject
FG0001 subjects
FG0014 subjects
Lost to Follow-up
FG0000 subjects
FG0013 subjects
Physician Decision
FG0002 subjects
FG0010 subjects
Other
FG0001 subjects
FG00110 subjects
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
rFIXFc (Participants From 9HB02PED)
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.
BG001
rFIXFc (Participants From 998HB102)
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.
BG002
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00027
BG00193
BG002120
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Customized
Count of Participants
Participants
Title
Denominators
Categories
<18 years
Title
Measurements
BG00027
BG0018
BG00235
Between 18 and 65 years
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0000
BG0010
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0000
BG00111
BG002
Race/Ethnicity, Customized
Count of Participants
Participants
Title
Denominators
Categories
White
Title
Measurements
BG00019
BG00147
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Number of Participants With Any Positive Inhibitor Development
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.
Safety Analysis Set included participants who received at least 1 dose of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in study 9HB01EXT.
Posted
Count of Participants
Participants
Approximately 5 years
ID
Title
Description
OG000
rFIXFc (9HB02PED [<6 Years Old])
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.
OG001
rFIXFc (9HB02PED [6 to <12 Years])
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.
OG002
rFIXFc (Participants From 998HB102)
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.
Units
Counts
Participants
OG00013
OG00114
OG00293
Title
Denominators
Categories
Title
Measurements
OG0000
OG0010
OG0020
Secondary
Annualized Bleeding Rate (ABR)
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.
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.
Posted
Median
Inter-Quartile Range
episodes per participant per year
Approximately 5 years
ID
Title
Description
OG000
rFIXFc (9HB02PED [<6 Years Old Age Cohort])
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.
Secondary
Annualized Spontaneous Joint Bleeding Episodes
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.
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.
Posted
Median
Inter-Quartile Range
episodes per participant per year
Approximately 5 years
ID
Title
Description
OG000
rFIXFc (9HB02PED [<6 Years Old Age Cohort])
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.
Secondary
Total Number of Exposure Days (EDs)
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.
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.
Posted
Median
Full Range
days
Approximately 5 years
ID
Title
Description
OG000
rFIXFc (9HB02PED [<6 Years Old Age Cohort])
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.
Secondary
Annualized rFIXFc Consumption (International Units Per Kilogram [IU/kg])
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.
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.
Posted
Median
Inter-Quartile Range
IU per kilogram per participant per year
Approximately 5 years
ID
Title
Description
OG000
rFIXFc (9HB02PED [<6 Years Old Age Cohort])
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.
Secondary
Physicians' Global Assessment of Participant's Response to rFIXFc Regimen Using a 4-Point Scale
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.
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.
Posted
Count of Units
Responses
Approximately 5 years
Responses
Responses
ID
Title
Description
OG000
rFIXFc (Participants From 9HB02PED)
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.
Secondary
Participant's Assessment of Response (Excellent or Good Response) to rFIXFc Injections for the Treatment of Bleeding Episodes Using a 4-Point Scale
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.
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.
Posted
Count of Units
Injections
Approximately 5 years
Injections
Injections
ID
Title
Description
OG000
rFIXFc (9HB02PED [<6 Years Old Age Cohort])
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.
Time Frame
From signing of Informed Consent Form (ICF) through follow-up (approximately 5 years)
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.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
rFIXFc (Participants From 9HB02PED)
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.
0
27
5
27
23
27
EG001
rFIXFc (Participants From 998HB102)
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.
0
93
31
93
66
93
EG002
Overall rFIXFc (Participants From 9HB02PED and 998HB102)
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.
0
120
36
120
89
120
EG003
rFIXFc (Participants From 9HB02PED in Surgery Subgroup)
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.
0
1
0
1
0
1
EG004
rFIXFc (Participants From 998HB102 in Surgery Subgroup)
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.
0
15
1
15
4
15
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG0030 affected1 at risk
EG0040 affected15 at risk
Necrotising retinitis
Eye disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Enterocolitis
Gastrointestinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Gastrointestinal haemorrhage
Gastrointestinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Pain
General disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Anal abscess
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Cellulitis
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0013 affected93 at risk
EG0023 affected120 at risk
EG003
Hepatitis C
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Orchitis
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Pilonidal cyst
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Pneumonia
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Staphylococcal bacteraemia
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Tonsillitis
Infections and infestations
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Tooth abscess
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Wound infection
Infections and infestations
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Concussion
Injury, poisoning and procedural complications
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Extradural haematoma
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Fall
Injury, poisoning and procedural complications
Systematic Assessment
EG0003 affected27 at risk
EG0014 affected93 at risk
EG0027 affected120 at risk
EG003
Femoral neck fracture
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Foreign body
Injury, poisoning and procedural complications
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Head injury
Injury, poisoning and procedural complications
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Laceration
Injury, poisoning and procedural complications
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Post procedural haematoma
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Post procedural haemorrhage
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Rib fracture
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0012 affected93 at risk
EG0022 affected120 at risk
EG003
Road traffic accident
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Traumatic haematoma
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Traumatic intracranial haemorrhage
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Upper limb fracture
Injury, poisoning and procedural complications
Systematic Assessment
EG0001 affected27 at risk
EG0010 affected93 at risk
EG0021 affected120 at risk
EG003
Arthropathy
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0012 affected93 at risk
EG0022 affected120 at risk
EG003
Haemarthrosis
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Haemophilic arthropathy
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0014 affected93 at risk
EG0024 affected120 at risk
EG003
Intervertebral disc protrusion
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Slipping rib syndrome
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Spinal column stenosis
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Hepatic neoplasm malignant
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Epilepsy
Nervous system disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Haemorrhage intracranial
Nervous system disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Posterior interosseous syndrome
Nervous system disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Calculus ureteric
Renal and urinary disorders
Systematic Assessment
EG0000 affected27 at risk
EG0012 affected93 at risk
EG0022 affected120 at risk
EG003
Haematuria
Renal and urinary disorders
Systematic Assessment
EG0000 affected27 at risk
EG0012 affected93 at risk
EG0022 affected120 at risk
EG003
Renal colic
Renal and urinary disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Renal failure acute
Renal and urinary disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Tonsillar haemorrhage
Respiratory, thoracic and mediastinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Victim of crime
Social circumstances
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Knee arthroplasty
Surgical and medical procedures
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Tonsillectomy
Surgical and medical procedures
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Haematoma
Vascular disorders
Systematic Assessment
EG0000 affected27 at risk
EG0011 affected93 at risk
EG0021 affected120 at risk
EG003
Anal Sphincter Atony
Gastrointestinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Epididymitis
Reproductive system and breast disorders
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Hypertension
Vascular disorders
Systematic Assessment
EG0000 affected27 at risk
EG0017 affected93 at risk
EG0027 affected120 at risk
EG0030 affected1 at risk
EG0040 affected15 at risk
Excoriation
Injury, poisoning and procedural complications
Systematic Assessment
EG0002 affected27 at risk
EG0011 affected93 at risk
EG0023 affected120 at risk
EG003
Fall
Injury, poisoning and procedural complications
Systematic Assessment
EG0007 affected27 at risk
EG0014 affected93 at risk
EG00211 affected120 at risk
EG003
Head injury
Injury, poisoning and procedural complications
Systematic Assessment
EG0002 affected27 at risk
EG0012 affected93 at risk
EG0024 affected120 at risk
EG003
Joint dislocation
Injury, poisoning and procedural complications
Systematic Assessment
EG0002 affected27 at risk
EG0010 affected93 at risk
EG0022 affected120 at risk
EG003
Joint injury
Injury, poisoning and procedural complications
Systematic Assessment
EG0002 affected27 at risk
EG0012 affected93 at risk
EG0024 affected120 at risk
EG003
Ligament sprain
Injury, poisoning and procedural complications
Systematic Assessment
EG0002 affected27 at risk
EG0014 affected93 at risk
EG0026 affected120 at risk
EG003
Laceration
Injury, poisoning and procedural complications
Systematic Assessment
EG0003 affected27 at risk
EG0015 affected93 at risk
EG0028 affected120 at risk
EG003
Serum ferritin decreased
Investigations
Systematic Assessment
EG0002 affected27 at risk
EG0010 affected93 at risk
EG0022 affected120 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
Systematic Assessment
EG0003 affected27 at risk
EG0015 affected93 at risk
EG0028 affected120 at risk
EG003
Nasal congestion
Respiratory, thoracic and mediastinal disorders
Systematic Assessment
EG0005 affected27 at risk
EG0010 affected93 at risk
EG0025 affected120 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
Systematic Assessment
EG0005 affected27 at risk
EG0010 affected93 at risk
EG0025 affected120 at risk
EG003
Seasonal allergy
Immune system disorders
Systematic Assessment
EG0004 affected27 at risk
EG0013 affected93 at risk
EG0027 affected120 at risk
EG003
Headache
Nervous system disorders
Systematic Assessment
EG0003 affected27 at risk
EG00115 affected93 at risk
EG00218 affected120 at risk
EG003
Pyrexia
General disorders
Systematic Assessment
EG0006 affected27 at risk
EG0015 affected93 at risk
EG00211 affected120 at risk
EG003
Diarrhoea
Gastrointestinal disorders
Systematic Assessment
EG0002 affected27 at risk
EG0014 affected93 at risk
EG0026 affected120 at risk
EG003
Gingival bleeding
Gastrointestinal disorders
Systematic Assessment
EG0002 affected27 at risk
EG0010 affected93 at risk
EG0022 affected120 at risk
EG003
Gastrooesophageal reflux disease
Gastrointestinal disorders
Systematic Assessment
EG0001 affected27 at risk
EG0017 affected93 at risk
EG0028 affected120 at risk
EG003
Nausea
Gastrointestinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0015 affected93 at risk
EG0025 affected120 at risk
EG003
Toothache
Gastrointestinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0015 affected93 at risk
EG0025 affected120 at risk
EG003
Vomiting
Gastrointestinal disorders
Systematic Assessment
EG0005 affected27 at risk
EG0017 affected93 at risk
EG00212 affected120 at risk
EG003
Haematuria
Renal and urinary disorders
Systematic Assessment
EG0000 affected27 at risk
EG0015 affected93 at risk
EG0025 affected120 at risk
EG003
Acne
Skin and subcutaneous tissue disorders
Systematic Assessment
EG0000 affected27 at risk
EG0015 affected93 at risk
EG0025 affected120 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0004 affected27 at risk
EG00110 affected93 at risk
EG00214 affected120 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
Systematic Assessment
EG0002 affected27 at risk
EG0011 affected93 at risk
EG0023 affected120 at risk
EG003
Ear infection
Infections and infestations
Systematic Assessment
EG0003 affected27 at risk
EG0011 affected93 at risk
EG0024 affected120 at risk
EG003
Influenza
Infections and infestations
Systematic Assessment
EG0001 affected27 at risk
EG0018 affected93 at risk
EG0029 affected120 at risk
EG003
Hepatitis C
Infections and infestations
Systematic Assessment
EG0000 affected27 at risk
EG0015 affected93 at risk
EG0025 affected120 at risk
EG003
Lower respiratory tract infection
Infections and infestations
Systematic Assessment
EG0003 affected27 at risk
EG0011 affected93 at risk
EG0024 affected120 at risk
EG003
Nasopharyngitis
Infections and infestations
Systematic Assessment
EG0003 affected27 at risk
EG00114 affected93 at risk
EG00217 affected120 at risk
EG003
Paronychia
Infections and infestations
Systematic Assessment
EG0002 affected27 at risk
EG0010 affected93 at risk
EG0022 affected120 at risk
EG003
Tonsillitis
Infections and infestations
Systematic Assessment
EG0002 affected27 at risk
EG0010 affected93 at risk
EG0022 affected120 at risk
EG003
Sinusitis
Infections and infestations
Systematic Assessment
EG0001 affected27 at risk
EG0015 affected93 at risk
EG0026 affected120 at risk
EG003
Upper respiratory tract infection
Infections and infestations
Systematic Assessment
EG0002 affected27 at risk
EG0016 affected93 at risk
EG0028 affected120 at risk
EG003
Varicella
Infections and infestations
Systematic Assessment
EG0002 affected27 at risk
EG0011 affected93 at risk
EG0023 affected120 at risk
EG003
Viral infection
Infections and infestations
Systematic Assessment
EG0002 affected27 at risk
EG0013 affected93 at risk
EG0025 affected120 at risk
EG003
Food poisoning
Gastrointestinal disorders
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Post Procedural Complication
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Procedural Pain
Injury, poisoning and procedural complications
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Insomnia
Psychiatric disorders
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Stress
Psychiatric disorders
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Convulsion
Nervous system disorders
Systematic Assessment
EG0000 affected27 at risk
EG0010 affected93 at risk
EG0020 affected120 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
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.
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D040181
Genetic Diseases, X-Linked
Browse Leaves
Not provided
Browse Branches
Not provided
ID
Term
C000599709
factor IX Fc fusion protein
Ancestor Terms
Not provided
Browse Leaves
Not provided
Browse Branches
Not provided
Title
Measurements
BG0000
BG00185
BG00285
> 65 years
Title
Measurements
BG0000
BG0010
BG0020
0
Male
BG00027
BG00193
BG002120
11
Not Hispanic or Latino
BG00027
BG00178
BG002105
Unknown or Not Reported
BG0000
BG0014
BG0024
66
Black or African American
Title
Measurements
BG0002
BG0019
BG00211
Asian
Title
Measurements
BG0005
BG00127
BG00232
American Indian or Alaska Native
Title
Measurements
BG0000
BG0010
BG0020
Native Hawaiian or other Pacific Islander
Title
Measurements
BG0000
BG0010
BG0020
Other
Title
Measurements
BG0001
BG00110
BG00211
Unknown or Not Reported
Title
Measurements
BG0000
BG0010
BG0020
OG001
rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort])
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.
OG002
rFIXFc (Participants From Study 998HB102)
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.
Units
Counts
Participants
OG00013
OG00114
OG00293
Title
Denominators
Categories
Weekly Prophylaxis
ParticipantsOG00013
ParticipantsOG00110
ParticipantsOG00251
Title
Measurements
OG0001.04(0.00 to 2.28)
OG0011.14(0.54 to 2.34)
OG0022.26(0.40 to 5.16)
Individualized Prophylaxis
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG00231
Title
Measurements
OG001
Personalized Prophylaxis
ParticipantsOG0001
ParticipantsOG0011
ParticipantsOG00216
Title
Measurements
OG000
Episodic
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG00215
Title
Measurements
OG002
OG001
rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort])
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.
OG002
rFIXFc (Participants From Study 998HB102)
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.
Units
Counts
Participants
OG00013
OG00114
OG00293
Title
Denominators
Categories
Weekly Prophylaxis
ParticipantsOG00013
ParticipantsOG00110
ParticipantsOG00251
Title
Measurements
OG0000.00(0.00 to 1.06)
OG0010.00(0.00 to 1.40)
OG0020.38(0.00 to 2.25)
Individualized Prophylaxis
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG00231
Title
Measurements
OG001
Personalized Prophylaxis
ParticipantsOG0001
ParticipantsOG0011
ParticipantsOG00216
Title
Measurements
OG000
Episodic
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG00215
Title
Measurements
OG002
OG001
rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort])
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.
OG002
rFIXFc (Participants From Study 998HB102)
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.
Units
Counts
Participants
OG00013
OG00114
OG00293
Title
Denominators
Categories
Weekly Prophylaxis
ParticipantsOG00013
ParticipantsOG00110
ParticipantsOG00251
Title
Measurements
OG00055.00(8.0 to 176.0)
OG001165.00(3.0 to 202.0)
OG002169.00(4.0 to 327.0)
Individualized Prophylaxis
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG00231
Title
Measurements
OG001
Personalized Prophylaxis
ParticipantsOG0001
ParticipantsOG0011
ParticipantsOG00217
Title
Measurements
OG000
Episodic
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG00215
Title
Measurements
OG002
OG001
rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort])
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.
OG002
rFIXFc (Participants From Study 998HB102)
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.
Units
Counts
Participants
OG00013
OG00114
OG00293
Title
Denominators
Categories
Weekly Prophylaxis
ParticipantsOG00013
ParticipantsOG00110
ParticipantsOG00251
Title
Measurements
OG0003382.5(2930.9 to 3667.4)
OG0013212.0(2838.5 to 3344.4)
OG0022598.0(2129.8 to 3370.9)
Individualized Prophylaxis
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG00231
Title
Measurements
OG001
Personalized Prophylaxis
ParticipantsOG0001
ParticipantsOG0011
ParticipantsOG00216
Title
Measurements
OG000
Episodic
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG00215
Title
Measurements
OG002
OG001
rFIXFc (Participants From 998HB102)
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.
Units
Counts
Participants
OG00027
OG00193
Responses
OG000151
OG001815
Title
Denominators
Categories
Excellent
Title
Measurements
OG000131
OG001622
Effective
Title
Measurements
OG00019
OG001184
Partially Effective
Title
Measurements
OG0001
OG0019
Ineffective
Title
Measurements
OG0000
OG0010
OG001
rFIXFc (9HB02PED [6 to <12 Years Old Age Cohort])
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.
OG002
rFIXFc (Participants From 998HB102)
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.