Previously treated patients with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B undergoing surgical or other invasive procedures MedDRA version: 14.1 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Recombinant Coagulation Factor IX
Product Code: BAX326
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Subject is participating in either the BAX 326 Pivotal Study (#250901), the BAX 326 Continuation Study (#251001), or the BAX 326 Pediatric Study (#251101) requiring emergency or elective major or minor surgical, dental, or other invasive procedures a) Subject and/or legal representative has/have provided signed informed consent b) Subject continues to meet eligibility criteria as outlined in the BAX 326 pivotal, continuation or pediatric study. • For newly entering subjects who do not participate in any other BAX 326 clinical study the following inclusion criteria apply: a) Subject is 12 to 65 years old at the time of screening. b) Subject requires elective major surgery. c) Subject and/or legal representative has/have provided signed informed consent d) Subject has severe (FIX level =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: For newly entering subjects who do not participate in any other clinical study with BAX 326 the following exclusion criteria apply: a) The subject 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. b) The subject has a detectable FIX inhibitor at screening, with a titer =0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory. c) The subject requires emergency surgery. d) The subject’s weight is 120 kg e) The subject has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s). f) The subject has a known hypersensitivity to hamster proteins or rFurin. g) The subject has evidence of an ongoing or recent thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC). h) The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal). The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices. i) The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels > 5 times the upper limit of normal. j) The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B. k) The subject’s platelet count is < 100,000/mL. l) The subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject’s safety or compliance. m) The subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or a-interferon) other than anti-retroviral chemotherapy. n) The subject has participated in another investigational study within 30 days of enrollment. o) The subject is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (i.e., children, partner/spouse, siblings, parents) as well as employees.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the hemostatic efficacy and safety of BAX 326 in the peri- and postoperative setting in subjects with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B undergoing major or minor elective or emergency surgical, dental or other invasive procedures. ;Secondary Objective: n.a.; Timepoint(s) of evaluation of this end point: Hemostatic efficacy: intraoperative and postoperative at drain removal or postoperative day 3, as well as at discharge. Safety:end of trial ; Primary end point(s): • Hemostatic efficacy a) Intraoperative hemostatic efficacy 1. Intraoperative Hemostatic Efficacy Assessment on a scale of “excellent”, “good”, “fair” and “none” 2. Actual intraoperative blood loss compared to average and maximum blood loss predicted preoperatively b) Postoperative hemostatic efficacy 1. Postoperative Hemostatic Efficacy Assessment at the time of drain removal, if applicable,or at postoperative day 3 (approximately 72 hours postoperatively) in case of major surgery and no drain employed on a scale of “excellent”, “good”, “fair” and “none” 2. Postoperative Hemostatic Efficacy Assessment at the time of discharge from the hospital on a scale of “excellent”, “good”, “fair” and “none” 3. Actual postoperative blood loss until drain removal, if applicable, compared to average and maximum blood loss predicted preoperatively c) BAX 326 consumption and blood product use 1. Daily and total weight-adjusted dose of BAX 326 per subject 2. Number of units and amount (in mL) of blood product transfused • Safety a) Development of inhibitory and total binding antibodies to FIX b) IP-related AEs • Determine the occurrence of thrombotic events | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): n.a.;Timepoint(s) of evaluation of this end point: n.a. | — |
Countries
Argentina, Brazil, Bulgaria, Chile, Colombia, Czech Republic, Japan, Poland, Russian Federation, Sweden, Ukraine, United Kingdom, United States
Contacts
Public ContactGuido Wuerth
Baxter Innovations GmbH
Outcome results
None listed