Brief Title
Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)
Official Title
A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A
Brief Summary
To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Annualized Bleeding Rate (ABR)
Secondary Outcome
Rate of Success of BAX 855 for Treatment of Bleeding Episodes
Condition
Hemophilia A
Intervention
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Study Arms / Comparison Groups
Prophylaxis
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
159
Start Date
January 31, 2013
Completion Date
July 17, 2014
Primary Completion Date
July 17, 2014
Eligibility Criteria
Main Inclusion Criteria: - Participant and/or legal representative has/have voluntarily provided signed informed consent - Participant is 12 to 65 years old at the time of screening - Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity < 1%) as confirmed by central laboratory at screening after the appropriate washout period or a documented FVIII clotting activity <1% - Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥150 documented exposure days (EDs) - Participant is currently receiving prophylaxis or on-demand therapy with FVIII - Participant is willing and able to comply with the requirements of the protocol Main Exclusion Criteria: - Participant has detectable FVIII inhibitory antibodies (≥ 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening - Participant has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening - Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
Gender
Male
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT01736475
Organization ID
261201
Secondary IDs
2012-003599-38
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Study Sponsor
Study Director, Study Director, Shire
Verification Date
April 2019