Brief Title
Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures
Official Title
A Phase 3, Multi-Center, Open Label Study of Efficacy and Safety of PEGylated rFVIII (BAX 855) in Previously Treated Patients With Severe Hemophilia A Undergoing Surgical or Other Invasive Procedures
Brief Summary
The purpose of the study is to evaluate the efficacy and safety of BAX 855 in severe
hemophilia A previously treated (PTP) males, 12 to 65 years of age who are undergoing
elective surgical or other invasive procedures.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Global Hemostatic Efficacy Assessment Score (GHEA) - Composed of 3 Individual Ratings
Secondary Outcome
Intraoperative Blood Loss
Condition
Hemophilia A
Intervention
PEGylated Recombinant factor VIII (rFVIII)
Study Arms / Comparison Groups
BAX855
Description: Pre-operative loading dose: Single loading dose, pre-surgery administered based on each study participant's individual PK results as well as target trough level for type and character of surgery, dental or invasive procedure being performed. In general, major surgery will target an 80-150% FVIII trough level, and minor surgery will target an initial 30-100% FVIII trough level. Intra-operative and post-operative dosing of BAX855 must be based on pre-dosage measurements of FVIII and the type and character of the surgery performed.
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
30
Start Date
December 20, 2013
Completion Date
September 23, 2016
Primary Completion Date
September 23, 2016
Eligibility Criteria
Inclusion Criteria:
- Participant requires an elective major or minor surgical, dental or other invasive
procedure (e.g. biopsy, endoscopy).
- Participant and/or legal representative has/have provided signed informed consent.
- Participant has severe hemophilia A (Factor VIII (FVIII) level <1%) as confirmed by
the central lab at screening or a documented FVIII activity level <1%.
- Participant was previously treated with FVIII concentrates with ≥150 documented
exposure days (EDs).
- Participant is currently receiving prophylaxis or on-demand therapy with FVIII
concentrate.
- Participant has a Karnofsky performance score of ≥60 at screening.
- Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable
disease and CD4+ count ≥200 cells/mm^3, as confirmed by central laboratory at
screening.
- Participant is Hepatitis C virus negative (HCV-) by antibody or PCR testing (if
positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory
at screening; or HCV+ with chronic stable hepatitis as assessed by the investigator.
- Participant is willing and able to comply with the requirements of the study protocol.
Exclusion Criteria:
- Participant has detectable FVIII inhibitory antibodies (≥0.4 Bethesda Unit (BU) using
the Nijmegen modification of the Bethesda assay) at screening as determined by the
central laboratory or at any timepoint prior to screening (≥0.4 BU using the Nijmegen
modification of the Bethesda assay or ≥0.6 BU using the Bethesda assay).
- History of ongoing or recent thrombotic disease, fibrinolysis or disseminated
intravascular coagulation (DIC).
- Participant has a platelet count <100 x 10^9/L, as confirmed by central laboratory at
screening.
- Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed
by central laboratory at screening.
- Participant has severe chronic hepatic dysfunction (eg ≥5 X upper limit of normal
alanine aminotransferase (ALT), as confirmed by the central laboratory at screening,
or a documented International Normalized Ratio (INR) > 1.5).
- Participant has a known hypersensitivity towards mouse or hamster proteins,
polysorbate 80 or to PEG.
- Participant is currently using or has recently (< 30 days) used pegylated drugs (other
than BAX 855) prior to study participation or is scheduled to use such drugs during
trial participation.
- Participant is currently participating in another clinical drug (other than BAX 855)
or device study or use of another investigational product or device within 30 days
prior to study entry.
- Participant has a diagnosis of an inherited or acquired hemostatic defect other than
hemophilia A.
- Participant is currently receiving, or scheduled to receive during the course of the
study, an immunomodulating drug (eg, systemic corticosteroid agent at a dose
equivalent to hydrocortisone >10 mg/day, or alpha interferon) other than
anti-retroviral chemotherapy.
- Participant has a clinically significant medical, psychiatric, or cognitive illness,
or recreational drug/alcohol use that, in the opinion of the investigator, would
affect participant safety or compliance.
Gender
Male
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
Bulgaria
Location Countries
Bulgaria
Administrative Informations
NCT ID
NCT01913405
Organization ID
261204
Responsible Party
Sponsor
Study Sponsor
Baxalta now part of Shire
Study Sponsor
Study Director, Study Director, Baxalta now part of Shire
Verification Date
April 2019