Back to resultsBAY81-8973 Pediatric Safety and Efficacy Trial
Primary Purpose
Haemophilia A
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Sponsored by
About this trial
This is an interventional treatment trial for Haemophilia A focused on measuring Recombinant factor VIII, Pediatric use
Eligibility Criteria
Inclusion Criteria:
- Male
- PTPs (previously treated patients): aged <= 12 years
- PUPs (previously untreated patients) / MTPs (minimally treated patients): aged < 6 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
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
PTPs 0-12 years
PUPs/MTPs 0-<6 years
Arm Description
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).
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.
Outcomes
Primary Outcome Measures
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').
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').
Secondary Outcome Measures
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').
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').
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.
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).
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).
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.
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.
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.
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.
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.
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.
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01311648
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2021
Overall Recruitment Status
Completed
Study Start Date
June 9, 2011 (Actual)
Primary Completion Date
September 9, 2019 (Actual)
Study Completion Date
October 27, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bayer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Haemophilia A
Keywords
Recombinant factor VIII, Pediatric use
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
94 (Actual)
8. Arms, Groups, and Interventions
Arm Title
PTPs 0-12 years
Arm Type
Experimental
Arm Description
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).
Arm Title
PUPs/MTPs 0-<6 years
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Intervention Description
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.
Intervention Type
Biological
Intervention Name(s)
Recombinant Factor VIII (Kovaltry, BAY81-8973)
Intervention Description
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
Primary Outcome Measure Information:
Title
Annualized Number of Total Bleeds Within 48 h
Description
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').
Time Frame
Within 48 hours post infusion
Title
Annualized Number of Total Bleeds Within 48 h
Description
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').
Time Frame
Within 48 hours post infusion
Secondary Outcome Measure Information:
Title
Annualized Number of Total Bleeds During Prophylaxis Treatment
Description
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').
Time Frame
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)
Title
Annualized Number of Total Bleeds During Prophylaxis Treatment
Description
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').
Time Frame
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)
Title
Hemostatic Control During Major and Minor Surgeries
Description
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.
Time Frame
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)
Title
Number of Participants With Inhibitor Development in Main Study
Description
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).
Time Frame
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)
Title
Number of Participants With New Inhibitor Development in Extension Study
Description
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).
Time Frame
From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years)
Title
Factor VIII Recovery Values
Description
Incremental recovery of Factor VIII (FVIII) at 20-30 min after end of infusions was determined and mean recovery values were reported.
Time Frame
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)
Title
Consumption of Factor VIII in All Infusions
Description
Factor VIII (FVIII) usage/consumption was summarized for all infusions. Consumption per participant's body weight per year was calculated and reported.
Time Frame
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)
Title
Consumption of FVIII in Infusions for Prophylaxis
Description
Factor VIII (FVIII) usage/consumption was summarized for prophylaxis infusions. Consumption per participant's body weight per year was calculated and reported.
Time Frame
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)
Title
Consumption of FVIII in Infusions for the Treatment of Bleeds
Description
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.
Time Frame
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)
Title
Number of Infusions Per Bleed
Description
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.
Time Frame
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)
Title
Response to Treatment of Bleeds
Description
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.
Time Frame
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)
Title
Half-life (t1/2) of BAY81-8973 in Plasma
Description
Half-life (t1/2) of BAY81-8973 in plasma was measured. Geometric mean and percentage geometric coefficient of variation (%CV) were reported.
Time Frame
Pre-infusion and until 24 hours post infusion
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male
PTPs (previously treated patients): aged <= 12 years
PUPs (previously untreated patients) / MTPs (minimally treated patients): aged < 6 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bayer Study Director
Organizational Affiliation
Bayer
Official's Role
Study Director
Facility Information:
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205-2696
Country
United States
City
Bahía Blanca
State/Province
Buenos Aires
ZIP/Postal Code
B8001HXM
Country
Argentina
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
City
Varna
ZIP/Postal Code
9010
Country
Bulgaria
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2C8
Country
Canada
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
City
Århus N
ZIP/Postal Code
8200
Country
Denmark
City
Budapest
ZIP/Postal Code
1089
Country
Hungary
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
City
Mohacs
ZIP/Postal Code
7700
Country
Hungary
City
Crumlin
State/Province
Dublin
ZIP/Postal Code
12
Country
Ireland
City
Ramat Gan
ZIP/Postal Code
5262000
Country
Israel
City
Roma
State/Province
Lazio
ZIP/Postal Code
00165
Country
Italy
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20122
Country
Italy
City
Bari
State/Province
Puglia
ZIP/Postal Code
70126
Country
Italy
City
Catania
State/Province
Sicilia
ZIP/Postal Code
95123
Country
Italy
City
Padova
State/Province
Veneto
ZIP/Postal Code
35128
Country
Italy
City
Riga
ZIP/Postal Code
LV-1004
Country
Latvia
City
Vilnius
ZIP/Postal Code
08406
Country
Lithuania
City
Guadalajara
State/Province
Jalisco
ZIP/Postal Code
44280
Country
Mexico
City
San Juan del Río
State/Province
Querétaro
ZIP/Postal Code
76800
Country
Mexico
City
Oaxaca
ZIP/Postal Code
68000
Country
Mexico
City
Oslo
Country
Norway
City
Lodz
ZIP/Postal Code
91-738
Country
Poland
City
Warszawa
ZIP/Postal Code
00-576
Country
Poland
City
Wroclaw
ZIP/Postal Code
50-368
Country
Poland
City
Bucharest
ZIP/Postal Code
011026
Country
Romania
City
Bucharest
ZIP/Postal Code
022328
Country
Romania
City
Cluj-Napoca
ZIP/Postal Code
400177
Country
Romania
City
Timisoara
ZIP/Postal Code
300011
Country
Romania
City
Kazan
ZIP/Postal Code
139445
Country
Russian Federation
City
Kirov
ZIP/Postal Code
610027
Country
Russian Federation
City
Volgograd
ZIP/Postal Code
400138
Country
Russian Federation
City
Esplugues de LLobregat
State/Province
Barcelona
ZIP/Postal Code
08950
Country
Spain
City
A Coruña
ZIP/Postal Code
15006
Country
Spain
City
Alicante
ZIP/Postal Code
03010
Country
Spain
City
Cáceres
ZIP/Postal Code
10003
Country
Spain
City
Madrid
ZIP/Postal Code
28046
Country
Spain
City
Valencia
ZIP/Postal Code
46026
Country
Spain
12. IPD Sharing Statement
Citations:
PubMed Identifier
26931631
Citation
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.
Results Reference
result
PubMed Identifier
26663410
Citation
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.
Results Reference
result
PubMed Identifier
27577234
Citation
Keating GM. BAY 81-8973 (Octocog Alfa; Kovaltry(R)): A Review in Haemophilia A. BioDrugs. 2016 Oct;30(5):453-459. doi: 10.1007/s40259-016-0191-4.
Results Reference
result
PubMed Identifier
25952661
Citation
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.
Results Reference
result
PubMed Identifier
27436242
Citation
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.
Results Reference
result
PubMed Identifier
28220555
Citation
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.
Results Reference
result
PubMed Identifier
30013766
Citation
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.
Results Reference
result
PubMed Identifier
32197139
Citation
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.
Results Reference
result
Links:
URL
http://www.clinicaltrialsregister.eu/
Description
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URL
https://clinicaltrials.bayer.com/
Description
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Learn more about this trial
BAY81-8973 Pediatric Safety and Efficacy Trial
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