Phase 3 Efficacy and Safety Study of BAX 855 in Severe Hemophilia A Patients Undergoing Surgical Procedures

Baxalta

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Biological: PEGylated Recombinant factor VIII (rFVIII)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01913405

2013-001359-11 (EudraCT Number)

261204

Details and patient eligibility

About

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.

Enrollment: 30 patients

Sex: Male

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

BAX855

Experimental group

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.

Treatment:

Biological: PEGylated Recombinant factor VIII (rFVIII)