BAX 326 Surgery Study in Hemophilia B Patients

NCT ID: NCT01507896

Last Updated: 2021-05-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-12-19
• Study Completion Date: 2014-05-15

Brief Summary

The purpose of the study is to assess the hemostatic efficacy and safety of BAX 326 in subjects with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B undergoing major or minor elective or emergency surgical, dental or other invasive procedures.

Conditions

Hemophilia B

Study Design

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

Study Groups

BAX326 in Surgery

BAX 326 (recombinant factor IX) in Surgery

Group Type: EXPERIMENTAL

Recombinant factor IX

Intervention Type: BIOLOGICAL

Following a loading dose with BAX326, participants will receive BAX326 as a bolus infusion. The treatment regimen will be determined by the intensity and duration of the hemostatic challenge and the institution´s standard of care. The dose will be tailored to raise FIX concentration to at least 80%-100% of normal for major surgeries and to at least 30%-60% of normal for minor surgeries.

Interventions

Recombinant factor IX

Following a loading dose with BAX326, participants will receive BAX326 as a bolus infusion. The treatment regimen will be determined by the intensity and duration of the hemostatic challenge and the institution´s standard of care. The dose will be tailored to raise FIX concentration to at least 80%-100% of normal for major surgeries and to at least 30%-60% of normal for minor surgeries.

Intervention Type: BIOLOGICAL

Other Intervention Names

BAX326; RIXUBIS

Eligibility Criteria

Inclusion Criteria

• Participant and/or legal representative has/have voluntarily provided signed informed consent.
• Participant has severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory.
• Participant requires surgery
• Participant has previously been treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 exposure days
• Participant has no evidence of a history of FIX inhibitors
• Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm3.
• Participant is human immunodeficiency (HIV) negative or is HIV+ with a viral load < 200 particles/μL ~ < 400,000 copies/mL.

Exclusion Criteria

• Participant has a history of FIX inhibitors with a titer ≥ 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) at any time prior to screening.
• Participant has a detectable FIX inhibitor at screening, with a titer ≥0.6 Bethesda Units (BU) as determined by the Nijmegen modification of the Bethesda assay in the central laboratory.
• Participant has a history of allergic reaction or evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
• Known hypersensitivity to hamster proteins or recombinant furin.
• Evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
• Abnormal renal function
• Severe chronic liver disease
• Active hepatic disease with ALT or AST levels > 5 times the upper limit of normal.
• Diagnosis of an iherited or acquired hemostatic defect other than hemophilia B.
• Platelet count < 100,000/mL.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• 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

Instituto de Hematología y Medicina Clínica Rubén Dávoli

Rosario, , Argentina

Specialized Haematological Hospital "Joan Pavel"

Sofia, , Bulgaria

Hospital Dr. Sotero del Rio

Santiago, , Chile

Centro Medico Imbanaco

Cali, , Colombia

Klinika detska hematologie a onkologie, Fakultni Nemocnice Motol

Prague, , Czechia

Medical University Lodz, Copernicus Hospital, Department of Hematology

Lodz, , Poland

Independent Public Pediatric Teaching Hospital, Clinical Department of Hematology and Pediatrics

Warsaw, , Poland

Institute of Haematology and Transfusion Medicine

Warsaw, , Poland

Louis Turcanu Emergency Clinical Children´s Hospital

Timișoara, , Romania

Federal State Institution Kirov, Hematology and Blood Transfusion Research Institute under the Federal Agency for High-Tech Medical Care

Kirov, , Russia

Children's Territorial Clinical Hospital

Krasnodar, , Russia

Hematology Research Center RAMS

Moscow, , Russia

State Institution "Institute of Blood Pathology and Transfusion Medicine under the Academy of Medical Sciences of Ukraine"

Lviv, , Ukraine

Royal Manchester Children's Hospital, Department of Hematology

Manchester, , United Kingdom

Countries

Argentina; Bulgaria; Chile; Colombia; Czechia; Poland; Romania; Russia; Ukraine; United Kingdom

References

Windyga J, Lissitchkov T, Stasyshyn O, Mamonov V, Ghandehari H, Chapman M, Fritsch S, Wong WY, Pavlova BG, Abbuehl BE. Efficacy and safety of a recombinant factor IX (Bax326) in previously treated patients with severe or moderately severe haemophilia B undergoing surgical or other invasive procedures: a prospective, open-label, uncontrolled, multicentre, phase III study. Haemophilia. 2014 Sep;20(5):651-8. doi: 10.1111/hae.12419. Epub 2014 Apr 3.

Reference Type: RESULT

PMID: 24697870 (View on PubMed)

Windyga J, Timofeeva M, Stasyshyn O, Mamonov V, Lamas Castellanos JL, Lissitchkov T, Chojnowski K, Chapman M, Pavlova BG, Tangada S. Phase 3 Clinical Trial: Perioperative Use of Nonacog Gamma, a Recombinant Factor IX, in Previously Treated Patients With Moderate/Severe Hemophilia B. Clin Appl Thromb Hemost. 2020 Jan-Dec;26:1076029620946839. doi: 10.1177/1076029620946839.

Reference Type: DERIVED

PMID: 32816519 (View on PubMed)

Other Identifiers

2011-000413-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

251002

Identifier Type: -

Identifier Source: org_study_id