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 these patients can transition into the BAX 326 pivotal or pediatric study evaluating a new rFIX, provided all eligibility criteria are met. MedDRA
Conditions
Interventions
Trade Name: IMMUNINE Baxter 600 IU
Product Name: IMMUNINE Baxter 600 IU
Product Code: n.a.
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Human Coagulation Fac
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • - 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 = 6 years old or 20-50 ED if =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: E.2.1 Main objective: To prospectively document the exposure to IMMUNINE and to monitor FIX inhibitors while receiving prophylactic treatment over a period of approximately 20 to 50 EDs in up to 50 PTPs aged 12-64 years and approximately 20 pediatric subjects up to 11 years of age with severe (FIX level < 1%) or moderately severe (FIX level = 2%) hemophilia B who are planned to enter the BAX 326 pivotal or pediatric study evaluating a new rFIX, provided all eligibility criteria are met.;Secondary Objective: E.2.2 S econdary objectives: •To evaluate safety in terms of investigational product related adverse events, immunogenicity and thrombogenicity • To monitor hemostatic efficacy of IMMUNINE in the management and prevention of acute bleeding episodes, and surgical prophylaxis if necessary • To evaluate the subject’s compliance with the protocol specific treatment regimen • To monitor incremental recovery of IMMUNINE over time • To evaluate HR QoL at baseline;Primary end point(s): The primary objective of the study is to prospectively document exposure to IMMUNINE and to monitor FIX inhibitors for approximately 20 to 50 EDs in up to 50 PTPs with severe or moderately severe hemophilia B so that these patients can transition into BAX326 study 250901 provided all eligibility criteria are met.;Timepoint(s) of evaluation of this end point: Study End | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary study objectives are the evaluation of efficacy, safety, immunogenicity, thrombogenicity, HR QoL, and the subject’s compliance with the study specific treatment regimen.;Timepoint(s) of evaluation of this end point: Study End | — |
Countries
Argentina, Brazil, Bulgaria, Chile, Colombia, Czech Republic, Poland, Romania, Russian Federation, Ukraine
Contacts
Public ContactClinical Study Team Lead
Baxter Innovations GmbH
Outcome results
None listed