BAX326 (recombinant Factor Nine): A Phase 3 Prospective, Multicenter Study Evaluating Efficacy and Safety in Previously Treated Patients with Severe or Moderately Severe Hemophilia B undergoing Surgical or Other Invasive Procedures.

• Conditions: Previously treated patients with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B undergoing surgical or other invasive procedures.; MedDRA version: 16.1Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2011-000413-39-SE
• Lead Sponsor: Baxter Innovations GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-11-08
• Last Update Posted: 17 November 2014

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

•Subject is participating in either the BAX 326 Pivotal Study (#250901) or the BAX 326 Continuation Study (#251001) or the BAX326 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 < 1%) or moderately severe (FIX level = 2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory.
e)Subject is previously treated with plasma-derived and/or recombinant FIX concentrate(s) for a minimum of 150 EDs (based and documented on the subject’s medical records).
f)Subject has no evidence of a history of FIX inhibitors (based on the subject’s medical records).
g)Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm3.
h)Subject is human immunodeficiency (HIV) negative or is HIV+ with a viral load
< 200 particles/µL ~ <400,000 copies/mL
i)If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
j)Subject is willing and able to comply with the requirements of the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 < 35 kg or > 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified