IMMUNINE - A Clinical Study with IMMUNINE (A Purified Factor IX Concentrate Virus-Inactivated) in Previously Treated Patients with Severe or Moderately Severe Hemophilia B Who are Planned to Enter the BAX 326 Study (250901, Investigating a new Factor IX Product)
- Conditions
- To expose up to 50 patients aged 12-64 years, and approximately 20 pediatric patients up to 11 years of age with severe or moderately severe hemophilia B to IMMUNINE for a period of approximately 20 to 50 exposure days (EDs), prospectively document the exposure days (EDs) and factor IX (FIX) inhibitors while receiving prophylactic treatment so that thesepatients can transition into the BAX 326 pivotal or pediatric study evaluating a new rFIX, provided all eligibility criteria are met.MedDRA version: 15.0Level: LLTClassification code 10060614Term: Hemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2009-016719-39-CZ
- Lead Sponsor
- Baxter Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 70
•- Subject and/or legal representative has/have provided signed informed consent.
•Subject is up to 64 years old at the time of screening.
•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.
•Subject is previously treated with plasma-derived or recombinant FIX concentrate(s), cryoprecipitate or fresh frozen plasma (FFP) for approximately 100 - 150 exposure days (EDs) if > = 6 years old or 20-50 ED if < 6 years old and is planned to enter either the study BAX326 pivotal or BAX 326 pediatric study 250901. The number of EDs is derived from the subject’s treatment regimen and his/her bleeding pattern.
•Subject/legal representative is willing/accepts to to receive prophylactic treatment for the duration of the study.
•Subject is immunocompetent as evidenced by a CD4 count = 200 cells/mm.
•Subject is human immunodeficiency virus (HIV) negative or is HIV+ with a viral load < 200 particles/µL, approximately <400,000 copies/mL.
•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.
Subject/legal representative 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: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 35
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
•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.
•The subject has a history of FIX inhibitors with a titer = 0.6 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.
•The subject has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s).The subject has a known hypersensitivity to hamster proteins.
•The subject has evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC).
•Subject is scheduled for elective surgery, unless the surgery is medically required within the anticipated study period
•The subject has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal [ULN]).
•The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) exceeding the upper limit of normal hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
•The subject has active hepatic disease with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels = 5 times the upper limit of normal. During the study, subjects with chronic hepatitis B or C may have fluctuations of up to 5 times the upper limit of normal (ULN) but will not require discontinuation.
•The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia B.
•The subject’s platelet count is < 100,000/mL.
•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.
•The subject is currently receiving, or is scheduled to receive during the course of the study, an immunomodulating drug other than anti-retroviral chemotherapy (eg, a-interferon, corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day).
•The subject is pregnant or lactating at the time of study enrollment.
•The subject is unwilling to consider further participation in the BAX 326 pivotal or pediatric study
•Subject has participated in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment.
•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 (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method