Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

Baxalta

Status and phase

Completed

Phase 3

Phase 2

Conditions

Hemophilia A

Treatments

Biological: PEGylated Recombinant Factor VIII

Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Study type

Interventional

Funder types

Industry

Identifiers

NCT01736475

2012-003599-38 (EudraCT Number)

261201

Details and patient eligibility

About

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.

Enrollment

159 patients

Sex

Male

Ages

12 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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).