BAY81-8973 Pediatric Safety and Efficacy Trial | NCT01311648 | Trialant
NCT01311648
Sponsor
Bayer
Status
Completed
Last Update Posted
Dec 5, 2023Actual
Enrollment
94Actual
Phase
Phase 3
Conditions
Haemophilia A
Interventions
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Countries
United States
Argentina
Bulgaria
Canada
Denmark
Hungary
Ireland
Israel
Italy
Latvia
Lithuania
Mexico
Norway
Poland
Romania
Russia
Spain
Protocol Section
Identification Module
NCT ID
NCT01311648
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
13400
Secondary IDs
ID
Type
Description
Link
2010-021781-29
EudraCT Number
Brief Title
BAY81-8973 Pediatric Safety and Efficacy Trial
Official Title
A Multicenter Phase III Uncontrolled Open-label Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy
Acronym
Not provided
Organization
BayerINDUSTRY
Status Module
Record Verification Date
Nov 2023
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
Jun 9, 2011Actual
Primary Completion Date
Sep 9, 2019Actual
Completion Date
Oct 27, 2020Actual
First Submitted Date
Feb 18, 2011
First Submission Date that Met QC Criteria
Mar 8, 2011
First Posted Date
Mar 9, 2011Estimated
Results Waived
Not provided
Results First Submitted Date
Sep 1, 2020
Results First Submitted that Met QC Criteria
Oct 9, 2020
Results First Posted Date
Nov 3, 2020Actual
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
Nov 16, 2023
Last Update Posted Date
Dec 5, 2023Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
BayerINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
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 was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
The secondary objectives were
To assess the safety and efficacy of BAY81-8973 during surgeries.
To assess incremental recovery of BAY81-8973.
To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated patients [PTPs] and previously untreated patients [PUPs] / minimally treated patients [MTPs] - participation in PK sampling was voluntary and required consent).
Detailed Description
Not provided
Conditions Module
Conditions
Haemophilia A
Keywords
Recombinant factor VIII
Pediatric use
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
94Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
PTPs 0-12 years
Experimental
Previously treated patients (PTPs) aged below 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (EDs) in main study - Part A. Participants having reached at least 50 EDs in main study - Part A were offered participation in an open label extension study (optional). Participants who transitioned from main study - Part A to the extension study received BAY81-8973, 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study).
Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
PUPs/MTPs 0-<6 years
Experimental
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more than 3 exposure days (EDs) with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development in main study - Part B. Participants having reached at least 50 EDs in main study - Part B were offered participation in an open label extension study and received BAY81-8973 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study); participants who developed an inhibitor in main study - Part B were offered participation in open label extension study and received Immune Tolerance Induction (ITI) treatment with BAY81-8973 until successful eradication of the inhibitor, or until failure, for approximately 18 months.
Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Biological
Main study: 25-50 IU/kg at least 2x/week for 6 months and at least 50 EDs, IV infusion; Extension study: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study), IV infusion. Exposure day (ED): An ED is a unit of time (1 day) in which replacement treatment of Hemophilia is given to a patient.
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Annualized Number of Total Bleeds Within 48 h
Annualized number (mean +/- standard deviation) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days [EDs]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Within 48 hours post infusion
Annualized Number of Total Bleeds Within 48 h
Annualized number (median [inter-quartile range (Q1-Q3)]) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days [EDs]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Within 48 hours post infusion
Secondary Outcomes
Measure
Description
Time Frame
Annualized Number of Total Bleeds During Prophylaxis Treatment
Annualized number (mean +/- standard deviation) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Male
PTPs (previously treated patients): aged <= 12 years
Severe hemophilia A defined as < 1% FVIII concentration (FVIII:C)
PTPs: >= 50 exposure days (EDs) with any FVIII concentrate, no current evidence of inhibitory antibody, and no history of FVIII inhibitor formation
PUPs: no prior exposure to any FVIII product
MTPs: having no more than 3 EDs with any FVIII product, no current evidence of inhibitory antibody and no history of FVIII inhibitor formation
Exclusion Criteria:
With another bleeding disorder that is different from Hemophilia A
With thrombocytopenia (platelet count < 100 000/mm^3)
Creatinine > 2x upper limit of normal or Aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) > 5x upper limit of normal
Without a negative inhibitor testing at screening (except for PUPs)
Receiving chemotherapy, immune modulatory drugs, has received another investigational FVIII product within the last month, or received another experimental drug within the last 3 months
Requires any pre-medication to tolerate FVIII treatment
Known hypersensitivity to active substance, mouse, or hamster protein
Oldenburg J, Windyga J, Hampton K, Lalezari S, Tseneklidou-Stoeter D, Beckmann H, Maas Enriquez M. Safety and efficacy of BAY 81-8973 for surgery in previously treated patients with haemophilia A: results of the LEOPOLD clinical trial programme. Haemophilia. 2016 May;22(3):349-53. doi: 10.1111/hae.12839. Epub 2016 Mar 1.
Overall, 58 participants were screened in Part A, of which 7 participants were screening failures and 51 participants received the study drug; 52 participants were screened in Part B, of which 9 participants were screening failures and 43 participants received the study drug. 46 participants from Part A and 36 from Part B entered the optional extension study.
Recruitment Details
The study was conducted at multiple centers in 18 countries and consisted of: Part A - between 09-JUN-2011 (FPFV) and 02-JAN-2013 (LPLV); Part B - between 19-SEP-2012 (FPFV) and 09-SEP-2019 (LPLV); Extension study - between 21-DEC-2011 (FPFV) and 27-OCT-2020 (LPLV).
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
PTPs 0-12 Years
Previously treated patients (PTPs) aged below 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (EDs) in main study - Part A. Participants having reached at least 50 EDs in main study - Part A were offered participation in an open label extension study (optional). Participants who transitioned from main study - Part A to the extension study received BAY81-8973, 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study).
Periods
Title
Milestones
Reasons Not Completed
Main Study
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
3
More Info Module
Limitations and Caveats
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
Feb 1, 2019
Sep 1, 2020
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Austria
Greece
Serbia
Sweden
United Kingdom
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
N/A
Intervention Model
Single Group 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
PTPs 0-12 years
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Biological
Main study: 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development, IV infusion; Extension study: For participants having reached at least 50 EDs in main study - Part B: 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study), IV infusion. For participants who developed an inhibitor in main study - Part B: up to 200 IU/kg per day or 100 IU/kg twice a day at the discretion of the investigator and coordinating investigator until successful eradication of the inhibitor, or until failure, for up to18 months (treatment beyond 18 months required an agreement with the sponsor and coordinating investigator), IV infusion
PUPs/MTPs 0-<6 years
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Annualized Number of Total Bleeds During Prophylaxis Treatment
Annualized number (median [inter-quartile range (Q1-Q3)]) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Hemostatic Control During Major and Minor Surgeries
For participants who underwent major or minor surgeries during the study, hemostasis during the surgeries was assessed as excellent, good, moderate or poor. Number of surgeries per assessment was summarized and reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Number of Participants With Inhibitor Development in Main Study
Number of participants with confirmed positive FVIII inhibitor titer (≥ 0.6 Bethesda unit [BU/mL]) during the main study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. > 5.0 BU/mL).
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Number of Participants With New Inhibitor Development in Extension Study
Number of participants who had not developed an inhibitor during the main study but developed an inhibitor (confirmed positive FVIII inhibitor titer [≥ 0.6 BU/mL]) during the extension study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. > 5.0 BU/mL).
From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years)
Factor VIII Recovery Values
Incremental recovery of Factor VIII (FVIII) at 20-30 min after end of infusions was determined and mean recovery values were reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Consumption of Factor VIII in All Infusions
Factor VIII (FVIII) usage/consumption was summarized for all infusions. Consumption per participant's body weight per year was calculated and reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Consumption of FVIII in Infusions for Prophylaxis
Factor VIII (FVIII) usage/consumption was summarized for prophylaxis infusions. Consumption per participant's body weight per year was calculated and reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Consumption of FVIII in Infusions for the Treatment of Bleeds
Factor VIII (FVIII) usage/consumption was summarized for infusions used to treat breakthrough bleeds. Consumption per participant's body weight per year was calculated and reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Number of Infusions Per Bleed
The number of infusions used to treat a bleed was defined as the first infusion to treat the bleed plus all follow-up infusions to treat the same bleed, if any. The mean value of number of infusions for each bleed was calculated and reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Response to Treatment of Bleeds
Participants or caregivers were asked to assess the response to treatment of bleeds as excellent, good, moderate or poor. Percentage of bleeds per assessment was summarized and reported.
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Half-life (t1/2) of BAY81-8973 in Plasma
Half-life (t1/2) of BAY81-8973 in plasma was measured. Geometric mean and percentage geometric coefficient of variation (%CV) were reported.
Pre-infusion and until 24 hours post infusion
Cincinnati
Ohio
45229
United States
Cleveland
Ohio
44106
United States
Columbus
Ohio
43205-2696
United States
Bahía Blanca
Buenos Aires
B8001HXM
Argentina
Plovdiv
4002
Bulgaria
Varna
9010
Bulgaria
Edmonton
Alberta
T6G 2C8
Canada
Hamilton
Ontario
L8N 3Z5
Canada
Toronto
Ontario
M5G 1X8
Canada
Århus N
8200
Denmark
Budapest
1089
Hungary
Debrecen
4032
Hungary
Mohács
7700
Hungary
Crumlin
Dublin
12
Ireland
Ramat Gan
5262000
Israel
Bari
Apulia
70126
Italy
Rome
Lazio
00165
Italy
Milan
Lombardy
20122
Italy
Catania
Sicily
95123
Italy
Padova
Veneto
35128
Italy
Riga
LV-1004
Latvia
Vilnius
08406
Lithuania
Guadalajara
Jalisco
44280
Mexico
San Juan del Río
Querétaro
76800
Mexico
Oaxaca City
68000
Mexico
Oslo
Norway
Lodz
91-738
Poland
Warsaw
00-576
Poland
Wroclaw
50-368
Poland
Bucharest
011026
Romania
Bucharest
022328
Romania
Cluj-Napoca
400177
Romania
Timișoara
300011
Romania
Kazan'
139445
Russia
Kirov
610027
Russia
Volgograd
400138
Russia
Esplugues de Llobregat
Barcelona
8950
Spain
A Coruña
15006
Spain
Alicante
03010
Spain
Cáceres
10003
Spain
Madrid
28046
Spain
Valencia
46026
Spain
Result
Ljung R, Kenet G, Mancuso ME, Kaleva V, Rusen L, Tseneklidou-Stoeter D, Michaels LA, Shah A, Hong W, Maas Enriquez M; investigators of the LEOPOLD Kids Trial. BAY 81-8973 safety and efficacy for prophylaxis and treatment of bleeds in previously treated children with severe haemophilia A: results of the LEOPOLD Kids Trial. Haemophilia. 2016 May;22(3):354-60. doi: 10.1111/hae.12866. Epub 2015 Dec 9.
Shah A, Delesen H, Garger S, Lalezari S. Pharmacokinetic properties of BAY 81-8973, a full-length recombinant factor VIII. Haemophilia. 2015 Nov;21(6):766-71. doi: 10.1111/hae.12691. Epub 2015 May 8.
Maas Enriquez M, Thrift J, Garger S, Katterle Y. BAY 81-8973, a full-length recombinant factor VIII: Human heat shock protein 70 improves the manufacturing process without affecting clinical safety. Protein Expr Purif. 2016 Nov;127:111-115. doi: 10.1016/j.pep.2016.07.009. Epub 2016 Jul 18.
Garmann D, McLeay S, Shah A, Vis P, Maas Enriquez M, Ploeger BA. Population pharmacokinetic characterization of BAY 81-8973, a full-length recombinant factor VIII: lessons learned - importance of including samples with factor VIII levels below the quantitation limit. Haemophilia. 2017 Jul;23(4):528-537. doi: 10.1111/hae.13192. Epub 2017 Feb 20.
Mahlangu JN, Ahuja SP, Windyga J, Church N, Shah A, Schwartz L. BAY 81-8973, a full-length recombinant factor VIII for the treatment of hemophilia A: product review. Ther Adv Hematol. 2018 Jul;9(7):191-205. doi: 10.1177/2040620718777903. Epub 2018 Jun 12.
Kenet G, Ljung R, Rusen L, Kerlin BA, Blanchette V, Saulyte Trakymiene S, Uscatescu V, Beckmann H, Tseneklidou-Stoeter D, Church N. Continued benefit demonstrated with BAY 81-8973 prophylaxis in previously treated children with severe haemophilia A: Interim analysis from the LEOPOLD Kids extension study. Thromb Res. 2020 May;189:96-101. doi: 10.1016/j.thromres.2020.03.005. Epub 2020 Mar 9.
Kenet G, Moulton T, Wicklund BM, Ahuja SP, Escobar M, Mahlangu J. Switching from Sucrose-Formulated rFVIII to Octocog Alfa (BAY 81-8973) Prophylaxis Improves Bleed Outcomes in the LEOPOLD Clinical Trials. J Blood Med. 2023 Jun 7;14:379-388. doi: 10.2147/JBM.S405624. eCollection 2023.
Ljung R, Chan AKC, Glosli H, Afonja O, Becker B, Tseneklidou-Stoeter D, Mancuso ME, Saulyte-Trakymiene S, Kenet G. BAY 81-8973 Efficacy and Safety in Previously Untreated and Minimally Treated Children with Severe Hemophilia A: The LEOPOLD Kids Trial. Thromb Haemost. 2023 Jan;123(1):27-39. doi: 10.1055/s-0042-1757876. Epub 2023 Jan 10.
Ljung R, Chan AKC, Ahuja SP, Mancuso ME, Marquez JFC, Volk F, Blanchette V, Kerlin BA, Trakymiene SS, Glosli H, Kenet G. BAY 81-8973 Demonstrates Long-Term Safety and Efficacy in Children With Severe Haemophilia A: Results From the LEOPOLD Kids Extension Study. Eur J Haematol. 2025 Mar;114(3):556-565. doi: 10.1111/ejh.14362. Epub 2024 Dec 12.
FG001
PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more than 3 exposure days [EDs] with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for at least 50 EDs or until inhibitor development in main study - Part B. Participants having reached at least 50 EDs in main study - Part B were offered participation in an open label extension study and received BAY81-8973 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study); participants who developed an inhibitor in main study - Part B were offered participation in open label extension study and received Immune Tolerance Induction (ITI) treatment with BAY81-8973 until successful eradication of the inhibitor, or until failure, for approximately 18 months.
FG00051 subjects
FG00143 subjects
Started 0-<6 Years
FG00025 subjects
FG00143 subjects
Started 6-12 Years
FG00026 subjects
FG0010 subjects
COMPLETED
FG00051 subjects
FG00122 subjects
NOT COMPLETED
FG0000 subjects
FG00121 subjects
Type
Comment
Reasons
Protocol Violation
FG0000 subjects
FG0011 subjects
Adverse Event
FG0000 subjects
FG0011 subjects
Inhibitor management
FG0000 subjects
FG00117 subjects
Withdrawal by Subject
FG0000 subjects
FG0012 subjects
Extension Study
Type
Comment
Milestone Data
STARTED
FG00046 subjects
FG00136 subjects
COMPLETED
FG00045 subjects
FG00125 subjects
NOT COMPLETED
FG0001 subjects
FG00111 subjects
Type
Comment
Reasons
Physician Decision
FG0000 subjects
FG0011 subjects
ITI therapy with marketed product
FG0000 subjects
FG001
Safety analysis set (SAF): all participants who entered Study Part A or Part B and received at least one infusion of study medication.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
BG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
BG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 exposure days (EDs) with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 EDs or until inhibitor development in main study - Part B.
BG003
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00025
BG00126
BG00243
BG00394
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
Years
Title
Denominators
Categories
Title
Measurements
BG0003.8± 1.3
BG0018.8± 1.8
BG0021.1± 0.8
BG003
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
BG0001
BG0010
BG002
Race/Ethnicity, Customized
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
White
BG00024
BG00124
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
Annualized Number of Total Bleeds Within 48 h
Annualized number (mean +/- standard deviation) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days [EDs]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD)
Posted
Mean
Standard Deviation
Bleeds
Within 48 hours post infusion
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 exposure days (EDs) with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 EDs or until inhibitor development in main study - Part B.
Units
Counts
Participants
OG00025
OG00126
OG00243
Title
Denominators
Categories
Title
Measurements
OG0002.23± 2.77(2.77 to )
OG0011.86± 3.08(3.08 to )
OG0021.9± 3.3(3.3 to )
Primary
Annualized Number of Total Bleeds Within 48 h
Annualized number (median [inter-quartile range (Q1-Q3)]) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days [EDs]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD)
Posted
Median
Inter-Quartile Range
Bleeds
Within 48 hours post infusion
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Secondary
Annualized Number of Total Bleeds During Prophylaxis Treatment
Annualized number (mean +/- standard deviation) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD)
Posted
Mean
Standard Deviation
Bleeds
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Secondary
Annualized Number of Total Bleeds During Prophylaxis Treatment
Annualized number (median [inter-quartile range (Q1-Q3)]) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD)
Posted
Median
Inter-Quartile Range
Bleeds
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Secondary
Hemostatic Control During Major and Minor Surgeries
For participants who underwent major or minor surgeries during the study, hemostasis during the surgeries was assessed as excellent, good, moderate or poor. Number of surgeries per assessment was summarized and reported.
Participants in SAF in main study who underwent major or minor surgeries during the study
Posted
Count of Units
Surgeries
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Surgeries
Surgeries
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Number of Participants With Inhibitor Development in Main Study
Number of participants with confirmed positive FVIII inhibitor titer (≥ 0.6 Bethesda unit [BU/mL]) during the main study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. > 5.0 BU/mL).
Participants in main study - safety analysis set (SAF, all participants who entered Study Part A or Part B and received at least one infusion of study medication) with inhibitor measurements done
Posted
Count of Participants
Participants
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Secondary
Number of Participants With New Inhibitor Development in Extension Study
Number of participants who had not developed an inhibitor during the main study but developed an inhibitor (confirmed positive FVIII inhibitor titer [≥ 0.6 BU/mL]) during the extension study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. > 5.0 BU/mL).
All participants who entered the extension study and had not developed inhibitors during the main study Part A or Part B
Posted
Count of Participants
Participants
From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years)
ID
Title
Description
OG000
Extension Study - Former Part A Participants
Participants having reached at least 50 exposure days (EDs) in main study - Part A were offered participation in an open label extension study (optional). Participants who transitioned from main study - Part A to the extension study received BAY81-8973, 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study).
OG001
Extension Study - Former Part B Participants
Participants having reached at least 50 exposure days (EDs) in main study - Part B were offered participation in an open label extension study and received BAY81-8973 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study); participants who developed an inhibitor in main study - Part B were offered participation in open label extension study and received Immune Tolerance Induction (ITI) treatment with BAY81-8973 until successful eradication of the inhibitor, or until failure, for approximately 18 months.
Secondary
Factor VIII Recovery Values
Incremental recovery of Factor VIII (FVIII) at 20-30 min after end of infusions was determined and mean recovery values were reported.
Participants in intent-to-treat (ITT) analysis set in main study with valid FVIII recovery values
Posted
Mean
Standard Deviation
International unit (IU)/dL per IU/kg
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Consumption of Factor VIII in All Infusions
Factor VIII (FVIII) usage/consumption was summarized for all infusions. Consumption per participant's body weight per year was calculated and reported.
Intent-to-treat (ITT) analysis set - main study: all participants of the SAF in main study who had infusion/bleeding data from the electronic patient diary (EPD)
Posted
Mean
Standard Deviation
international units/kilogram/year
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Consumption of FVIII in Infusions for Prophylaxis
Factor VIII (FVIII) usage/consumption was summarized for prophylaxis infusions. Consumption per participant's body weight per year was calculated and reported.
Participants in intent-to-treat (ITT) analysis set in main study with at least one dose of prophylaxis treatment with study drug
Posted
Mean
Standard Deviation
international units/kilogram/year
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Consumption of FVIII in Infusions for the Treatment of Bleeds
Factor VIII (FVIII) usage/consumption was summarized for infusions used to treat breakthrough bleeds. Consumption per participant's body weight per year was calculated and reported.
Participants in intent-to-treat (ITT) analysis set in main study with at least one bleed treated with study drug
Posted
Mean
Standard Deviation
international units/kilogram/year
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Number of Infusions Per Bleed
The number of infusions used to treat a bleed was defined as the first infusion to treat the bleed plus all follow-up infusions to treat the same bleed, if any. The mean value of number of infusions for each bleed was calculated and reported.
Participants in intent-to-treat (ITT) analysis set in main study with at least one bleed
Posted
Mean
Standard Deviation
Infusions
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Response to Treatment of Bleeds
Participants or caregivers were asked to assess the response to treatment of bleeds as excellent, good, moderate or poor. Percentage of bleeds per assessment was summarized and reported.
Participants in intent-to-treat (ITT) analysis set in main study with at least one bleed
Posted
Count of Units
Bleeds assessed for the response
Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
Bleeds assessed for the response
Bleeds assessed for the response
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Secondary
Half-life (t1/2) of BAY81-8973 in Plasma
Half-life (t1/2) of BAY81-8973 in plasma was measured. Geometric mean and percentage geometric coefficient of variation (%CV) were reported.
Participants in PK analysis set (PKS) - A with evaluable data for this endpoint (PKS - A: all participants who entered main study - Part A and received at least one infusion of study medication with evaluable pharmacokinetic data)
Posted
Geometric Mean
Geometric Coefficient of Variation
Hours
Pre-infusion and until 24 hours post infusion
ID
Title
Description
OG000
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
OG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
Units
Counts
Participants
Time Frame
Main study - Part A: from the first BAY81-8973 infusion until 3 days after the last infusion (median 6 months); Main study - Part B: from the first BAY81-8973 infusion until 7 days after the last infusion (median 8 months); Extension: from start of extension study until 3 days after the last infusion in extension study (median 3.1 years)
Description
Not provided
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
Main Study - Part A: PTPs 0-<6 Years
Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
0
25
0
25
16
25
EG001
Main Study - Part A: PTPs 6-12 Years
Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED) in main study - Part A.
0
26
5
26
19
26
EG002
Main Study - Part B: PUPs/MTPs 0-<6 Years
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 exposure days (EDs) with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 EDs or until inhibitor development in main study - Part B.
0
43
26
43
28
43
EG003
Extension Study - Former Part A Participants
Participants having reached at least 50 exposure days (EDs) in main study - Part A were offered participation in an open label extension study (optional). Participants who transitioned from main study - Part A to the extension study received BAY81-8973, 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part A and extension study).
0
46
23
46
41
46
EG004
Extension Study - Former Part B Participants
Participants having reached at least 50 exposure days (EDs) in main study - Part B were offered participation in an open label extension study and received BAY81-8973 25-50 IU/kg at least 2x/week for at least 100 cumulative EDs (main study - Part B and extension study); participants who developed an inhibitor in main study - Part B were offered participation in open label extension study and received Immune Tolerance Induction (ITI) treatment with BAY81-8973 until successful eradication of the inhibitor, or until failure, for approximately 18 months.
0
36
14
36
24
36
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Blood loss anaemia
Blood and lymphatic system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG0030 events0 affected46 at risk
EG004
Factor VIII inhibition
Blood and lymphatic system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Gastritis
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Gastritis haemorrhagic
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Gastrointestinal haemorrhage
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Mouth haemorrhage
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Catheter site granuloma
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Catheter site haematoma
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Catheter site haemorrhage
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Catheter site related reaction
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Decreased activity
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Extravasation
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Facial pain
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Pyrexia
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Appendicitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Bacteraemia
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Bacterial infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Bronchitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Cellulitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Cellulitis orbital
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Enterococcal sepsis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Epidemic pleurodynia
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Escherichia urinary tract infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Gastroenteritis rotavirus
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Gastroenteritis staphylococcal
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Gastroenteritis viral
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Otitis media acute
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Peritonsillar abscess
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pulpitis dental
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Respiratory tract infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Sepsis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tonsillitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tooth abscess
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tracheitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Vascular device infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Viral infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Craniocerebral injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Mouth injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Post procedural haemorrhage
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Radius fracture
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Subcutaneous haematoma
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Subdural haematoma
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Traumatic haemorrhage
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Traumatic haemothorax
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Ulna fracture
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Anti factor VIII antibody positive
Investigations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG00224 events22 affected43 at risk
EG003
Catheterisation cardiac
Investigations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Electroencephalogram abnormal
Investigations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Metabolic syndrome
Metabolism and nutrition disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Arthritis reactive
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Haemarthrosis
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0025 events3 affected43 at risk
EG003
Haematoma muscle
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Soft tissue haemorrhage
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0022 events2 affected43 at risk
EG003
Cerebral haemorrhage
Nervous system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Encephalomalacia
Nervous system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Multiple sclerosis
Nervous system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Subarachnoid haemorrhage
Nervous system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Device failure
Product Issues
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Device occlusion
Product Issues
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Internal device exposed
Product Issues
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Haematuria
Renal and urinary disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Laryngeal haematoma
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Nasal polyps
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Drug eruption
Skin and subcutaneous tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Adenotonsillectomy
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Central venous catheter removal
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Central venous catheterisation
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Dental cleaning
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Immune tolerance induction
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tooth extraction
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Haematoma
Vascular disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0013 events1 affected26 at risk
EG0023 events3 affected43 at risk
EG0030 events0 affected46 at risk
EG00410 events3 affected36 at risk
Blood loss anaemia
Blood and lymphatic system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0022 events2 affected43 at risk
EG003
Iron deficiency anaemia
Blood and lymphatic system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Phimosis
Congenital, familial and genetic disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Ear pain
Ear and labyrinth disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Conjunctivitis allergic
Eye disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Photophobia
Eye disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Strabismus
Eye disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0013 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Dental caries
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0003 events2 affected25 at risk
EG0012 events1 affected26 at risk
EG0028 events6 affected43 at risk
EG003
Functional gastrointestinal disorder
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0012 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Glossodynia
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Haematochezia
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Stomatitis
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Teething
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0023 events2 affected43 at risk
EG003
Toothache
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0011 events1 affected26 at risk
EG0028 events4 affected43 at risk
EG003
Catheter site haematoma
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Fatigue
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Hyperthermia
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Infusion site swelling
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Injection site bruising
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Oedema peripheral
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Peripheral swelling
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pyrexia
General disorders
MedDRA (23.1)
Non-systematic Assessment
EG00010 events5 affected25 at risk
EG0012 events2 affected26 at risk
EG00219 events12 affected43 at risk
EG003
Hypersensitivity
Immune system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Bronchiolitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0024 events2 affected43 at risk
EG003
Bronchitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Catheter site infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Conjunctivitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0002 events2 affected25 at risk
EG0010 events0 affected26 at risk
EG0023 events3 affected43 at risk
EG003
Cystitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Dysentery
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Ear infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0022 events2 affected43 at risk
EG003
Enterovirus infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Gastroenteritis viral
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0023 events3 affected43 at risk
EG003
Gastrointestinal viral infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Hand-foot-and-mouth disease
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Hookworm infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Influenza
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0024 events4 affected43 at risk
EG003
Laryngitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0003 events2 affected25 at risk
EG0012 events2 affected26 at risk
EG00216 events6 affected43 at risk
EG003
Oral herpes
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Otitis media
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0023 events1 affected43 at risk
EG003
Otitis media acute
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Pharyngitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pharyngitis streptococcal
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Respiratory tract infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Rhinitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0011 events1 affected26 at risk
EG0024 events3 affected43 at risk
EG003
Tonsillitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0002 events1 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Tooth abscess
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0012 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tracheitis
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0012 events1 affected26 at risk
EG0023 events3 affected43 at risk
EG003
Varicella
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Vascular device infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Viral infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0003 events3 affected25 at risk
EG0012 events2 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Viral upper respiratory tract infection
Infections and infestations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0003 events2 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Eye contusion
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Face injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0022 events2 affected43 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0022 events2 affected43 at risk
EG003
Genital contusion
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Head injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Joint injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Limb injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Lip injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Procedural pain
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Road traffic accident
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Skin abrasion
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Skin injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Skin laceration
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Subcutaneous haematoma
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tongue injury
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Ulna fracture
Injury, poisoning and procedural complications
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Haemoglobin decreased
Investigations
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Neutrophil count increased
Investigations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
White blood cell count increased
Investigations
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Dehydration
Metabolism and nutrition disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Iron deficiency
Metabolism and nutrition disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0012 events2 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Groin pain
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Joint swelling
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Synovitis
Musculoskeletal and connective tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Skin papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Headache
Nervous system disorders
MedDRA (23.1)
Non-systematic Assessment
EG0003 events2 affected25 at risk
EG0014 events4 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Device failure
Product Issues
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Attention deficit hyperactivity disorder
Psychiatric disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Perineal pain
Reproductive system and breast disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0011 events1 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Adenoidal hypertrophy
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0004 events2 affected25 at risk
EG0014 events4 affected26 at risk
EG0021 events1 affected43 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0013 events2 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Productive cough
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0003 events2 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Rhinitis allergic
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tonsillar hypertrophy
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Dermatitis atopic
Skin and subcutaneous tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Ecchymosis
Skin and subcutaneous tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0003 events3 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0013 events2 affected26 at risk
EG0023 events3 affected43 at risk
EG003
Central venous catheter removal
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Tooth extraction
Surgical and medical procedures
MedDRA (23.1)
Non-systematic Assessment
EG0000 events0 affected25 at risk
EG0013 events2 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Increased upper airway secretion
Respiratory, thoracic and mediastinal disorders
MedDRA (23.1)
Non-systematic Assessment
EG0001 events1 affected25 at risk
EG0010 events0 affected26 at risk
EG0020 events0 affected43 at risk
EG003
Due to the small number of participants per reporting group, all presented summary measures (e.g. mean and proportion) have to be evaluated with caution. If displayed standard deviation should be taken into account. The participant who had a low titer inhibitor detected after 549 EDs during the extension study was considered to have a false positive result due to cross reactivity with IgG anticardiolipin antibodies.
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
The sponsor can review results communications prior to public release and can embargo communications regarding trial results within 60 days. The sponsor can require changes to the communication for any patentable subject matter or termed confidential information. The PI cannot publish the results prior the first multicenter publication. If there is no multicenter publication within 18 months after the trial completion the PI has the right to publish the results from the PI site
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Browse Leaves
Not provided
Browse Branches
Not provided
ID
Term
D005169
Factor VIII
C078147
F8 protein, human
Ancestor Terms
ID
Term
D001779
Blood Coagulation Factors
D001798
Blood Proteins
D011506
Proteins
D000602
Amino Acids, Peptides, and Proteins
D011498
Protein Precursors
D001685
Biological Factors
Browse Leaves
Not provided
Browse Branches
Not provided
1 subjects
Incorrect visit planning
FG0000 subjects
FG0011 subjects
Family's decision
FG0000 subjects
FG0011 subjects
Failure of ITI therapy
FG0000 subjects
FG0013 subjects
Long travel
FG0000 subjects
FG0011 subjects
Diagnosed with von Willebrand disease
FG0001 subjects
FG0010 subjects
Withdrawal by Subject
FG0000 subjects
FG0011 subjects
Inhibitor management
FG0000 subjects
FG0012 subjects
3.9
± 3.4
0
BG0030
Male
BG00025
BG00126
BG00243
BG00394
9
BG00310
Not Hispanic or Latino
BG00023
BG00125
BG00234
BG00382
Unknown or Not Reported
BG0001
BG0011
BG0020
BG0032
37
BG00385
Black
BG0001
BG0012
BG0021
BG0034
American Indian or Alaska native
BG0000
BG0010
BG0021
BG0031
White, American Indian or Alaska native
BG0000
BG0010
BG0021
BG0031
Not reported
BG0000
BG0010
BG0023
BG0033
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Units
Counts
Participants
OG00025
OG00126
OG00243
Title
Denominators
Categories
Title
Measurements
OG0001.88± 0.00(0.00 to 3.97)
OG0010.00± 0.00(0.00 to 1.96)
OG0020.0± 0.0(0.0 to 2.2)
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Units
Counts
Participants
OG00025
OG00126
OG00243
Title
Denominators
Categories
Title
Measurements
OG0004.16± 5.02(5.02 to )
OG0013.37± 5.01(5.01 to )
OG0027.1± 8.6(8.6 to )
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.
Units
Counts
Participants
OG00025
OG00126
OG00243
Title
Denominators
Categories
Title
Measurements
OG0002.03± 0.00(0.00 to 6.02)
OG0010.93± 0.00(0.00 to 5.77)
OG0024.7± 2.1(2.1 to 8.9)
Units
Counts
Participants
OG0000
OG0011
OG0026
Surgeries
OG0000
OG0011
OG0026
Title
Denominators
Categories
Minor surgery
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0025
SurgeriesOG0000
SurgeriesOG0010
SurgeriesOG0025
Title
Measurements
Excellent
OG0000
OG0010
OG0023
Good
OG000
Major surgery
ParticipantsOG0000
ParticipantsOG0011
ParticipantsOG0021
SurgeriesOG000
Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more then 3 EDs with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 exposure days (ED) or until inhibitor development in main study - Part B.