Back to resultsBAX 855 Dose-Escalation Safety Study
Primary Purpose
Hemophilia A
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
PEGylated Recombinant Factor VIII
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria:
- The subject is male and is 18 to 65 years of age at the time of screening
- The subject has severe hemophilia A (factor VIII level < 1%)
- The subject was previously treated with plasma-derived factor VIII (FVIII) concentrates or recombinant FVIII for at least 150 exposure days (EDs)
Exclusion Criteria:
- The subject has a detectable FVIII inhibitor at screening, with a titer >= 0.6 BU
- The subject has a documented history of FVIII inhibitors with a titer >= 0.4 BU at any time prior to screening
- The subject has a known hypersensitivity towards mouse or hamster proteins or to polyethylene glycol (PEG)
Sites / Locations
- Specialized Hospital for Active Treatment "Joan Pavel"
- Vivantes Klinikum im Friedrichshain, Hemophilia Care Center for Children and Adults
- UKGM Uniklinikum Giessen & Marburg
- Harrison Clinical Research Deutschland
- Nara Medical University Hospital
- Tokyo Medical University Hospital
- Quintiles Drug Research Unit at Guy´s Hospital
- Manchester Haemophilia Comprehensive Care Centre, Manchester Royal Infirmary
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Cohort 1
Cohort 2
Arm Description
Low dose of ADVATE followed by low dose of BAX 855
High dose of ADVATE followed by high dose of BAX 855
Outcomes
Primary Outcome Measures
Serious and non-serious AEs
Secondary Outcome Measures
Full Information
NCT ID
NCT01599819
First Posted
May 15, 2012
Last Updated
April 30, 2021
Sponsor
Baxalta now part of Shire
Collaborators
Baxter Innovations GmbH
1. Study Identification
Unique Protocol Identification Number
NCT01599819
Brief Title
BAX 855 Dose-Escalation Safety Study
Official Title
BAX 855 (PEGylated Recombinant Factor VIII): A Phase 1, Prospective, Open Label, Cross-Over, Dose-Escalation Study in Previously Treated Patients (PTPs) With Severe (FVIII < 1%) Hemophilia A
Study Type
Interventional
2. Study Status
Record Verification Date
April 2021
Overall Recruitment Status
Completed
Study Start Date
September 30, 2011 (Actual)
Primary Completion Date
July 27, 2012 (Actual)
Study Completion Date
July 27, 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baxalta now part of Shire
Collaborators
Baxter Innovations GmbH
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The objectives of this study are to assess the tolerability and safety after single dose treatments of BAX 855 in previously treated patients (PTPs) with severe hemophilia A, to determine the pharmacokinetic (PK) parameters of BAX 855 compared in crossover with ADVATE, and to evaluate the impact of anti-polyethylene glycol (PEG) antibodies on PK parameters.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
Low dose of ADVATE followed by low dose of BAX 855
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
High dose of ADVATE followed by high dose of BAX 855
Intervention Type
Biological
Intervention Name(s)
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Other Intervention Name(s)
ADVATE
Intervention Description
1 PK infusion of ADVATE (low dose in Cohort 1, high dose in Cohort 2)
Intervention Type
Biological
Intervention Name(s)
PEGylated Recombinant Factor VIII
Other Intervention Name(s)
BAX 855
Intervention Description
1 PK infusion of BAX 855 (low dose in Cohort 1, high dose in Cohort 2)
Primary Outcome Measure Information:
Title
Serious and non-serious AEs
Time Frame
4 weeks after infusion with BAX 855 and ADVATE
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
The subject is male and is 18 to 65 years of age at the time of screening
The subject has severe hemophilia A (factor VIII level < 1%)
The subject was previously treated with plasma-derived factor VIII (FVIII) concentrates or recombinant FVIII for at least 150 exposure days (EDs)
Exclusion Criteria:
The subject has a detectable FVIII inhibitor at screening, with a titer >= 0.6 BU
The subject has a documented history of FVIII inhibitors with a titer >= 0.4 BU at any time prior to screening
The subject has a known hypersensitivity towards mouse or hamster proteins or to polyethylene glycol (PEG)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Specialized Hospital for Active Treatment "Joan Pavel"
City
Sofia
ZIP/Postal Code
1233
Country
Bulgaria
Facility Name
Vivantes Klinikum im Friedrichshain, Hemophilia Care Center for Children and Adults
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
UKGM Uniklinikum Giessen & Marburg
City
Giessen
ZIP/Postal Code
35392
Country
Germany
Facility Name
Harrison Clinical Research Deutschland
City
Munich
ZIP/Postal Code
80636
Country
Germany
Facility Name
Nara Medical University Hospital
City
Nara
ZIP/Postal Code
630-8521
Country
Japan
Facility Name
Tokyo Medical University Hospital
City
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Quintiles Drug Research Unit at Guy´s Hospital
City
London
ZIP/Postal Code
SE1 1YR
Country
United Kingdom
Facility Name
Manchester Haemophilia Comprehensive Care Centre, Manchester Royal Infirmary
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
26157075
Citation
Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.
Results Reference
derived
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BAX 855 Dose-Escalation Safety Study
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