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

NCT ID: NCT01913405

Last Updated: 2021-05-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-12-20
• Study Completion Date: 2016-09-23

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.

Conditions

Hemophilia A

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

BAX855

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.

Group Type: EXPERIMENTAL

PEGylated Recombinant factor VIII (rFVIII)

Intervention Type: BIOLOGICAL

Lyophilized powder and solvent for solution for injection

Interventions

PEGylated Recombinant factor VIII (rFVIII)

Lyophilized powder and solvent for solution for injection

Intervention Type: BIOLOGICAL

Other Intervention Names

BAX 855; ADYNOVATE

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.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

University of Florida College of Medicine

Gainesville, Florida, United States

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

University of Louisville

Louisville, Kentucky, United States

Children's Mercy Hospitals & Clinics

Kansas City, Missouri, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

University of Utah Health Sciences Center

Salt Lake City, Utah, United States

University of Washington

Seattle, Washington, United States

SHAT of Oncohaematology Diseases

Sofia, , Bulgaria

MHAT 'Sv. Marina', EAD

Varna, , Bulgaria

Vilnius University Hospital Santariskiu Clinics, Public Institution

Vilnius, , Lithuania

Academisch Medisch Centrum

Amsterdam, , Netherlands

FSHI "Kirov SR Institute of Hematology and Blood Transfusion FMBA"

Kirov, , Russia

Hospital Universitari Son Espases

Palma de Mallorca, Balearic Islands, Spain

Complejo Hospitalario Universitario A Coruña

A Coruña, La Coruña, Spain

Hospital Regional Universitario de Malaga

Málaga, Málaga, Spain

Hospital Universitari i Politecnic La Fe

Valencia, , Spain

Universitaetsspital Zuerich

Zurich, , Switzerland

SI Institute of Blood Pathology and Transfusion Medicine of AMSU

Lviv, , Ukraine

Royal Free Hospital

London, Greater London, United Kingdom

Great Ormond Street Hospital for Children

London, Greater London, United Kingdom

Royal Manchester Children's Hospital

Manchester, Greater Manchester, United Kingdom

Countries

United States; Bulgaria; Lithuania; Netherlands; Russia; Spain; Switzerland; Ukraine; United Kingdom

References

Brand B, Gruppo R, Wynn TT, Griskevicius L, Lopez Fernandez MF, Chapman M, Dvorak T, Pavlova BG, Abbuehl BE. Efficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patients. Haemophilia. 2016 Jul;22(4):e251-8. doi: 10.1111/hae.12963. Epub 2016 Jun 21.

Reference Type: RESULT

PMID: 27328112 (View on PubMed)

Other Identifiers

2013-001359-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

261204

Identifier Type: -

Identifier Source: org_study_id